| 1 |
NCT00774423 |
Completed |
Study to Evaluate the Efficacy of Riluzole in Children and Young Adults With Spinal Muscular Atrophy (SMA) |
|
|
Interventional
|
Phase 2
Phase 3 |
- Assistance Publique - Hôpitaux de Paris
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- The drug could stabilize patients condition during 24 months, and especially interrupt paralysis progression: Motor function (MFM scale)
- Forced vital capacity (spirometry)
- Quality of life (OKado questionnaire)
- (and 2 more...)
|
141 |
All |
6 Years to 20 Years (Child, Adult) |
NCT00774423 |
P040904 |
ASIRI |
January 2006 |
September 2011 |
December 2011 |
October 17, 2008 |
February 15, 2013 |
|
- Hopital Raymond Poincare
Garches, France
|
|
| 2 |
NCT01736553 |
Completed
Has Results |
Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development |
- Spinal Muscular Atrophy (SMA)
|
|
Observational
|
|
- Ohio State University
- National Institute of Neurological Disorders and Stroke (NINDS)
- Cure SMA
- (and 2 more...)
|
Other / NIH |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Motor Function Assessments- Test for Infant Motor Performance Screening Items (TIMPSI)
- Motor Function Assessments- The Children's Hospital of Philadelphia Infant Test for Neuromuscular Disorders (CHOP-INTEND)
- Motor Function Assessments-Alberta Infant Motor Scale (AIMS)
- (and 22 more...)
|
53 |
All |
up to 6 Months (Child) |
NCT01736553 |
NN101
U01NS079163 |
|
December 2012 |
September 2015 |
September 2015 |
November 29, 2012 |
May 4, 2018 |
May 4, 2018 |
- University of California - Davis
Davis, California, United States - University of California - Los Angeles
Los Angeles, California, United States - Children's Hospital Colorado
Aurora, Colorado, United States - (and 12 more...)
|
|
| 3 |
NCT03461289 |
Active, not recruiting |
Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 |
|
- Biological: Onasemnogene Abeparvovec-xioi
|
Interventional
|
Phase 3 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Sitting without support
- Survival
|
33 |
All |
up to 6 Months (Child) |
NCT03461289 |
AVXS-101-CL-302
2017-000266-29 |
STRIVE-EU |
August 29, 2018 |
November 2020 |
November 2020 |
March 12, 2018 |
August 26, 2019 |
|
- University Hospital Ghent Neuromuscular reference center
Ghent, Belgium - Istituto Gianninia Gaslini
Genova, Italy - Policlinico "G. Martino"
Messina, Italy - (and 5 more...)
|
|
| 4 |
NCT02941328 |
Completed |
SPACE Trial: Pyridostigmine vs Placebo in SMA Types 2, 3 and 4 |
- Spinal Muscular Atrophy
- SMA
- Kugelberg-Welander Disease
|
- Drug: Pyridostigmine
- Drug: Placebo
|
Interventional
|
Phase 2 |
|
Other |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- MFM (Motor Function Measurement).
- repeated nine-hole peg test performance
- endurance nine hole peg test
- (and 2 more...)
|
39 |
All |
12 Years and older (Child, Adult, Older Adult) |
NCT02941328 |
NL38048.041.14 |
SPACE |
December 2015 |
January 2018 |
January 2018 |
October 21, 2016 |
February 2, 2018 |
|
- University Medical Center of Utrecht (UMCU)
Utrecht, Netherlands
|
|
| 5 |
NCT02876094 |
Recruiting |
Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy |
- Spinal Muscular Atrophy (SMA)
|
|
Interventional
|
Phase 2 |
- Hugh McMillan
- Families of Spinal Muscular Atrophy
- Gwendolyn Strong Foundation
- Children's Hospital of Eastern Ontario
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- low-dose oral celecoxib administered to patients with SMA type II and III is associated with an increase in the levels of peripheral leukocyte SMN protein compared to baseline
- Safety Profile Measured by Adverse Event Frequency,Type and Severity
- Recruitment Plan Measured by Number of Potentially Eligible Subjects
- (and 3 more...)
|
12 |
All |
2 Years to 80 Years (Child, Adult, Older Adult) |
NCT02876094 |
15/22E |
SMA |
January 29, 2019 |
July 2020 |
July 2020 |
August 23, 2016 |
July 30, 2019 |
|
- Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada
|
|
| 6 |
NCT02466529 |
Completed |
Natural History of Spinal Muscular Atrophy Type 1 in Taiwan |
- Natural History of Type 1 Spinal Muscular Atrophy (SMA)
|
|
Observational
|
|
- Kaohsiung Medical University Chung-Ho Memorial Hospital
- Biogen
- Taipei Medical University WanFang Hospital
- (and 17 more...)
|
Other / Industry |
- Observational Model: Case-Only
- Time Perspective: Retrospective
|
- Age of death
- Age of permanent ventilation
|
111 |
All |
up to 36 Years (Child, Adult) |
NCT02466529 |
SMA-NHR10311 |
|
June 2015 |
December 2015 |
December 2015 |
June 9, 2015 |
June 22, 2017 |
|
|
|
| 7 |
NCT01862042 |
Completed |
Palliative Care in Spinal Muscular Atrophy (SMA) 1 |
- Spinal Muscular Atrophy 1
|
- Other: Follow-up diary and questionnaire
|
Interventional
|
Not Applicable |
- Assistance Publique - Hôpitaux de Paris
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Supportive Care
|
- %O2
- Qualitative evaluation of the practices of care
- Evaluation of nutritional status
- (and 2 more...)
|
39 |
All |
up to 1 Year (Child) |
NCT01862042 |
P110135
2012-A00024-39 |
ASI1 |
June 2012 |
June 11, 2018 |
June 11, 2018 |
May 24, 2013 |
July 2, 2019 |
|
- Necker Hospital
Paris, France
|
|
| 8 |
NCT03554343 |
Recruiting |
Sun May Arise on SMA : Newborn Screening of SMA in Belgium |
|
- Diagnostic Test: test for SMN1 exon 7 deletion
|
Observational
|
|
- Centre Hospitalier Régional de la Citadelle
- AveXis, Inc.
- Biogen
- (and 2 more...)
|
Other / Industry |
- Observational Model: Ecologic or Community
- Time Perspective: Prospective
|
- Cases detected
- Incidence of SMA
|
180000 |
All |
up to 7 Days (Child) |
NCT03554343 |
B412201734396 |
|
March 5, 2018 |
March 4, 2021 |
July 31, 2021 |
June 13, 2018 |
November 20, 2018 |
|
- CRMN, Hôpital La Citadelle
Liege, Wallonia, Belgium
|
|
| 9 |
NCT01645787 |
Completed |
Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients |
|
- Drug: 4-aminopyridine
- Drug: Placebo
|
Interventional
|
Phase 2
Phase 3 |
|
Other |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Triple (Participant, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Six Minute Walk Test (6MWT) with Kinematic Evaluation of Gait
- Hammersmith Functional Motor Scale, Expanded (HFMSE)
- Manual Muscle Testing (MMT)/Hand Held Dynamometer (HHD)
- Change in Motor Unit Number Estimation (MUNE)/Nerve Conduction Study (NCS)
|
11 |
All |
18 Years to 50 Years (Adult) |
NCT01645787 |
AAAI7400 |
|
June 2012 |
September 2015 |
September 2015 |
July 20, 2012 |
May 18, 2016 |
|
- Columbia University Medical Center
New York, New York, United States
|
|
| 10 |
NCT00961103 |
Unknown † |
Motor Development and Orthoses in Spinal Muscular Atrophy (SMA) |
|
|
Observational
|
|
- Arcispedale Santa Maria Nuova-IRCCS
|
Other |
- Observational Model: Cohort
- Time Perspective: Retrospective
|
- time and percentage of sitting, standing, walking achievement in SMA II and SMA III
- characteristics and percentage of orthoses for postural sitting control , standing and walking in SMA II and SMA III
|
70 |
All |
Child, Adult, Older Adult |
NCT00961103 |
UDGEE-SMA |
|
September 2009 |
November 2009 |
December 2009 |
August 18, 2009 |
August 18, 2009 |
|
- UDGEE Hospital S.Maria Nuova
Reggio Emilia, RE, Italy
|
|
| 11 |
NCT03520179 |
Recruiting |
Improving Standards of Care and Translational Research in Spinal Muscular Atrophy (SMA) |
|
|
Observational
|
|
- Great Ormond Street Hospital for Children NHS Foundation Trust
|
Other |
- Observational Model: Cohort
- Time Perspective: Cross-Sectional
|
- Physiotherapy assessment using The Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND)
- Physiotherapy assessment using the Test of Infant Motor Performance Screening Items (TIMPSI)
- Patient´s perception about the condition, interventions performed and Standards of Care assessed by patient interviews
- (and 2 more...)
|
70 |
All |
3 Months to 99 Years (Child, Adult, Older Adult) |
NCT03520179 |
11DN15 |
SMA-REACH |
December 17, 2013 |
January 31, 2019 |
January 31, 2019 |
May 9, 2018 |
May 9, 2018 |
|
- Dubowitz Neuromuscular Centre
London, United Kingdom
|
|
| 12 |
NCT01302600 |
Completed |
Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients. |
- Spinal Muscular Atrophy Type II
- Spinal Muscular Atrophy Type III Non Ambulant
|
- Drug: Olesoxime
- Drug: Placebo
|
Interventional
|
Phase 2 |
- Hoffmann-La Roche
- Association Française contre les Myopathies (AFM), Paris
|
Industry / Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Motor Function Measure
- responder analyses on MFM and HFMS, time to 4 point decrease on HFMS, CMAP/MUNE, PedsQL, FVC, CGI and safety
|
165 |
All |
3 Years to 25 Years (Child, Adult) |
NCT01302600 |
WN29836
TRO19622 CL E Q 1275-1 |
|
November 2010 |
October 2013 |
October 2013 |
February 24, 2011 |
November 22, 2016 |
|
- University Hospitals
Ghent, Belgium - University Hospitals
Leuven, Belgium - Hôpital Femme-Mère-Enfant
Bron, France - (and 19 more...)
|
|
| 13 |
NCT01611610 |
Completed |
Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe) |
|
|
Interventional
|
Not Applicable |
- Institut de Myologie, France
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
|
- Change from baseline in total score of Motor Function Measure at one year
- Change from baseline of Pediatric Quality of Life Inventory Score at one year
|
4 |
All |
30 Months to 18 Years (Child, Adult) |
NCT01611610 |
SMA Europe |
|
May 2011 |
May 2013 |
May 2013 |
June 5, 2012 |
March 5, 2014 |
|
|
|
| 14 |
NCT02865109 |
Available |
Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA) |
- Infantile-onset Spinal Muscular Atrophy
|
|
Expanded Access
|
|
|
Industry |
|
|
|
All |
Child, Adult, Older Adult |
NCT02865109 |
232-SM-901 |
|
|
|
|
August 12, 2016 |
June 21, 2019 |
|
- Kaohsiung Medical University Hospital
Kaohsiung, Taiwan, Province OF China, China - National Taiwan University Hospital
Taipei, Taiwan, Province OF China, China - Hospital Pablo Tobon Uribe
Medellín, Antioquia, Colombia - (and 24 more...)
|
|
| 15 |
NCT02462759 |
Completed |
A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA). |
|
- Drug: Nusinersen
- Procedure: Sham Procedure
|
Interventional
|
Phase 2 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Number of participants with adverse events and serious adverse events
- Change from Baseline in clinical laboratory parameters
- Change from Baseline in electrocardiograms (ECGs)
- (and 16 more...)
|
21 |
All |
Child, Adult, Older Adult |
NCT02462759 |
232SM202
2014-003657-33 |
EMBRACE |
August 19, 2015 |
September 24, 2018 |
September 24, 2018 |
June 4, 2015 |
January 16, 2019 |
|
- David Geffen School of Medicine at UCLA
Los Angeles, California, United States - Connecticut Childrens Medical
Hartford, Connecticut, United States - The Johns Hopkins Hospital
Baltimore, Maryland, United States - (and 4 more...)
|
|
| 16 |
NCT00061607 |
Completed |
Measuring Levels of SMN in Blood Samples of SMA Patients |
|
|
Observational
|
|
- National Institute of Neurological Disorders and Stroke (NINDS)
- National Institutes of Health Clinical Center (CC)
|
NIH |
|
|
73 |
All |
2 Years and older (Child, Adult, Older Adult) |
NCT00061607 |
030203
03-N-0203 |
|
May 19, 2003 |
|
April 4, 2017 |
May 30, 2003 |
October 6, 2017 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 17 |
NCT00661453 |
Completed
Has Results |
CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I |
- Spinal Muscular Atrophy Type I
|
- Drug: Valproic Acid and Levocarnitine
|
Interventional
|
Phase 1
Phase 2 |
- University of Utah
- Families of Spinal Muscular Atrophy
- Leadiant Biosciences, Inc.
|
Other / Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Laboratory Safety Data
- Anthropometric Measures of Nutritional Status (Body Mass Index [BMI] Z-scores, Weight for Length Ratios, Lean/Fat Mass Via DEXA, Growth Parameters, and Triceps Skinfold Measures)
- Time to Death or Ventilator Dependence (Defined as >16 Hours/Day)
- (and 5 more...)
|
40 |
All |
up to 12 Months (Child) |
NCT00661453 |
25409
IND 79276 |
|
April 2008 |
May 2012 |
June 2012 |
April 18, 2008 |
June 15, 2015 |
June 15, 2015 |
- Johns Hopkins University
Baltimore, Maryland, United States - Children's Hospital of Michigan
Detroit, Michigan, United States - Duke University Medical Center
Durham, North Carolina, United States - (and 5 more...)
|
|
| 18 |
NCT03300869 |
Recruiting |
Natural History of Types 2 and 3 SMA in Taiwan |
|
|
Observational
|
|
- Kaohsiung Medical University Chung-Ho Memorial Hospital
- Biogen
- National Taiwan University Hospital
- (and 4 more...)
|
Other / Industry |
- Observational Model: Other
- Time Perspective: Retrospective
|
- Describe the correlation of genotype and phenotype in SMA types 2 and 3
- Disease onset in patients with SMA types 2 and 3
- Disease mortality in patients with SMA types 2 and 3
- (and 2 more...)
|
300 |
All |
6 Months to 70 Years (Child, Adult, Older Adult) |
NCT03300869 |
SMA_RV |
|
September 1, 2017 |
December 2019 |
December 2019 |
October 4, 2017 |
July 26, 2019 |
|
- Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung, Taiwan
|
|
| 19 |
NCT03217578 |
Recruiting |
Neonatal Spinal Muscular Atrophy (SMA) Screening |
|
- Other: SMA Newborn Screening
|
Observational
|
|
- Kaohsiung Medical University Chung-Ho Memorial Hospital
- The Chinese Foundation of Health
- Taipei Institute of Pathology
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Number of neonates with confirmed the deletion of 2 copies of SMN1 gene
- Number of neonates with confirmed SMA
|
250000 |
All |
up to 2 Weeks (Child) |
NCT03217578 |
SMA-NBS001 |
SMA |
September 1, 2017 |
December 31, 2019 |
December 31, 2019 |
July 14, 2017 |
July 26, 2019 |
|
- Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung, Taiwan
|
|
| 20 |
NCT02831296 |
Recruiting |
Prospective Evaluation of Infants With Spinal Muscular Atrophy: |
|
|
Observational
|
|
- Massachusetts General Hospital
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- University of Utah
- (and 5 more...)
|
Other / NIH |
- Observational Model: Case-Control
- Time Perspective: Prospective
|
- Time to death and/or full time invasive ventilation or need for > 16 hours/day of bilevel respiratory support
- Maximum Ulnar CMAP Amplitude
- CHOP-INTEND
- (and 7 more...)
|
200 |
All |
Child, Adult, Older Adult |
NCT02831296 |
2015P001934
R01HD069045 |
SPOTSMA |
February 2016 |
March 2022 |
|
July 13, 2016 |
March 6, 2019 |
|
- Massachusetts General Hospital
Boston, Massachusetts, United States
|
|
| 21 |
NCT02268552 |
Recruiting |
An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA) |
|
|
Interventional
|
Phase 1
Phase 2 |
- Novartis Pharmaceuticals
- Novartis
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants with adverse events (AEs), serious adverse events (SAEs)
- Change from baseline in length
- Change from baseline in pulse oximetry
- (and 13 more...)
|
44 |
All |
up to 182 Days (Child) |
NCT02268552 |
CLMI070X2201 |
|
April 2, 2015 |
July 23, 2020 |
July 23, 2020 |
October 20, 2014 |
July 26, 2019 |
|
- Novartis Investigative Site
Gent, Belgium - Novartis Investigative Site
Leuven, Belgium - Novartis Investigative Site
Sofia, Bulgaria - (and 15 more...)
|
|
| 22 |
NCT02895789 |
Recruiting |
Oxidative Capacity and Exercise Tolerance in Ambulatory SMA |
- Spinal Muscular Atrophy Type 3
- Mitochondrial Myopathy
|
|
Observational
|
|
- Columbia University
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
|
Other / NIH |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Change in NIRS derived index of muscle oxygen extraction
- Change in Peak oxygen uptake (V02 max)
- Change in Distance walked during the Six Minute Walk Test (6MWT)
- Change in Lean body mass assessed with Dual Energy X-ray Absorptiometry (DEXA)
|
42 |
All |
8 Years to 55 Years (Child, Adult) |
NCT02895789 |
AAAQ9447
1K01HD084690-01A1 |
|
November 2016 |
July 2019 |
May 2020 |
September 12, 2016 |
May 1, 2018 |
|
- Columbia University Medical Center
New York, New York, United States
|
|
| 23 |
NCT01547871 |
Terminated |
Infants With Spinal Muscular Atrophy Type I |
|
|
Observational
|
|
- National Institute of Neurological Disorders and Stroke (NINDS)
- National Institutes of Health Clinical Center (CC)
|
NIH |
|
- The primary outcome of this pilot study is to demonstrate the feasibility of the study design and test the outcome measures in patients with SMA 1.
- Secondary outcomes include evaluation for time of events, i.e., time of ventilator support for greater than or equal to 16 hours a day continuously for more than 2 weeks and time of death.
|
4 |
All |
1 Year to 6 Years (Child) |
NCT01547871 |
999912067
12-N-N067 |
|
February 7, 2012 |
|
April 14, 2016 |
March 8, 2012 |
September 18, 2019 |
|
- National Institute of Neurological Disorders and Stroke (NINDS), 9000 Rockville
Bethesda, Maryland, United States
|
|
| 24 |
NCT01166022 |
Completed |
Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA) |
- Spinal Muscular Atrophy
- Neuromuscular Disease
|
|
Interventional
|
Not Applicable |
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Single (Outcomes Assessor)
- Primary Purpose: Treatment
|
- Distance walked in the Six Minute Walk Test (6MWT).
|
14 |
All |
8 Years to 50 Years (Child, Adult) |
NCT01166022 |
AAAE8200
A-15886 |
|
December 2010 |
August 2014 |
August 2014 |
July 20, 2010 |
August 12, 2016 |
|
- Columbia University SMA Clinical Research Center
New York, New York, United States
|
|
| 25 |
NCT04010604 |
Not yet recruiting |
A Registered Cohort Study on SMA |
|
|
Observational
|
|
- Wan-Jin Chen
- First Affiliated Hospital of Fujian Medical University
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- The time to death
- The correlation of genotype and phenotype
|
2000 |
All |
up to 70 Years (Child, Adult, Older Adult) |
NCT04010604 |
MRCTA,ECFAHOFFMU[2019]196 |
|
July 2019 |
December 31, 2039 |
December 31, 2049 |
July 8, 2019 |
July 8, 2019 |
|
- Department of Neurology, First Affiliated Hospital Fujian Medical University
Fuzhou, Fujian, China
|
|
| 26 |
NCT00227266 |
Completed
Has Results |
Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy |
|
- Drug: Valproic Acid and Levocarnitine
- Drug: Placebo
|
Interventional
|
Phase 2 |
- University of Utah
- Families of Spinal Muscular Atrophy
- Leadiant Biosciences, Inc.
- Abbott
|
Other / Industry |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Safety Labs
- Efficacy, Measured Through Motor Function Assessments
- Modified Hammersmith Change From Baseline to 6 Months
- (and 10 more...)
|
94 |
All |
2 Years to 17 Years (Child) |
NCT00227266 |
13698 |
|
September 2005 |
November 2007 |
November 2007 |
September 27, 2005 |
September 26, 2011 |
May 3, 2011 |
- Johns Hopkins University
Baltimore, Maryland, United States - Children's Hospital of Michigan
Detroit, Michigan, United States - Ohio State University
Columbus, Ohio, United States - (and 3 more...)
|
|
| 27 |
NCT02061189 |
Active, not recruiting |
Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy (SMA) |
- Infantile Spinal Muscular Atrophy of Type 2 or 3
|
- Other: Physical exercise in a swimming pool during 6 months
|
Interventional
|
Not Applicable |
- Assistance Publique - Hôpitaux de Paris
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Motor function measurement (MFM)
- Hammersmith scale
- Questionnaire on post-exercise physical well-being
- Non invasive motor capacity assessment
|
30 |
All |
5 Years to 10 Years (Child) |
NCT02061189 |
P120124
2013-A01331-44 |
ExerASI |
March 2014 |
September 2019 |
March 2020 |
February 12, 2014 |
October 12, 2018 |
|
- Raymond Poincaré Hospital
Garches, Hauts-de-Seine, France
|
|
| 28 |
NCT02594124 |
Active, not recruiting |
A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies. |
|
|
Interventional
|
Phase 3 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: Triple (Participant, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Number of participants experiencing Adverse events (AEs) and/or Serious Adverse Events (SAEs)
- Number of participants with clinically significant vital sign abnormalities
- Number of participants with clinically significant weight abnormalities
- (and 29 more...)
|
292 |
All |
Child, Adult, Older Adult |
NCT02594124 |
ISIS 396443-CS11
2015-001870-16 |
SHINE |
November 4, 2015 |
August 29, 2023 |
August 29, 2023 |
November 2, 2015 |
January 30, 2019 |
|
- UCLA Medical Center
Los Angeles, California, United States - Stanford University School of Medicine
Palo Alto, California, United States - Children's Hospital Colorado
Aurora, Colorado, United States - (and 39 more...)
|
|
| 29 |
NCT02292537 |
Completed
Has Results |
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA) |
|
- Drug: Nusinersen
- Procedure: Sham procedure
|
Interventional
|
Phase 3 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score at Month 15
- Proportion of Participants Who Achieved a 3-Point Increase From Baseline in HFMSE Score at Month 15
- Proportion of Participants That Achieved Any New Motor Milestone at Month 15
- (and 12 more...)
|
126 |
All |
2 Years to 12 Years (Child) |
NCT02292537 |
ISIS 396443-CS4
2014-001947-18 |
CHERISH |
November 24, 2014 |
February 20, 2017 |
February 20, 2017 |
November 17, 2014 |
February 21, 2018 |
February 21, 2018 |
- UCLA Clinical and Translational Research Center
Los Angeles, California, United States - Lucile Packard Children's Hospital at Stanford
Palo Alto, California, United States - Children's Hospital Colorado
Aurora, Colorado, United States - (and 21 more...)
|
|
| 30 |
NCT01839656 |
Completed
Has Results |
A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA) |
|
|
Interventional
|
Phase 2 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Percent of Participants Who Achieved Improvement in Motor Milestones as Assessed by Section 2 of the HINE at the Last Visit
- Event-free Survival at the End of Study
- Percent of Participants With Improved Motor Function at the Last Visit as Assessed by the CHOP-INTEND Motor Function Scale
- (and 6 more...)
|
21 |
All |
up to 210 Days (Child) |
NCT01839656 |
ISIS 396443-CS3A
2017-000621-12 |
|
May 8, 2013 |
August 21, 2017 |
August 21, 2017 |
April 25, 2013 |
October 30, 2018 |
October 30, 2018 |
- Stanford University Medical Center
Stanford, California, United States - Nemours Children's Hospital
Orlando, Florida, United States - Columbia University Medical Center
New York, New York, United States - The Hospital for Sick Children (SickKids)
Toronto, Ontario, Canada
|
|
| 31 |
NCT02052791 |
Completed |
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246) |
|
|
Interventional
|
Phase 1 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants that experience Adverse Events (AEs) and Serious Adverse Events
- Number of participants with clinically significant neurological examination abnormalities
- Number of participants with clinically significant vital sign abnormalities
- (and 9 more...)
|
47 |
All |
Child, Adult, Older Adult |
NCT02052791 |
ISIS 396443-CS12 |
|
January 2014 |
January 2017 |
January 2017 |
February 3, 2014 |
March 24, 2017 |
|
- Boston Children's Hospital
Boston, Massachusetts, United States - Columbia University Medical Center
New York, New York, United States - UT Southwestern Medical Center - Children's Medical Center Dallas
Dallas, Texas, United States - University of Utah School of Medicine
Salt Lake City, Utah, United States
|
|
| 32 |
NCT01494701 |
Completed |
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) |
|
|
Interventional
|
Phase 1 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants that experience Adverse Events (AEs) and Serious Adverse Events
- Number of participants with clinically significant neurological examination abnormalities
- Number of participants with clinically significant vital sign abnormalities
- (and 10 more...)
|
28 |
All |
2 Years to 14 Years (Child) |
NCT01494701 |
ISIS 396443 - CS1 |
SMNRx |
November 30, 2011 |
January 31, 2013 |
January 31, 2013 |
December 19, 2011 |
April 6, 2017 |
|
- Children's Hospital Boston
Boston, Massachusetts, United States - Columbia University Medical Center
New York, New York, United States - UT Southwestern Medical Center - Children's Medical Center Dallas
Dallas, Texas, United States - University of Utah School of Medicine
Salt Lake City, Utah, United States
|
|
| 33 |
NCT01033331 |
Completed |
Evaluation of the Muscle Strength and Motor Ability in Children With Spinal Muscle Atrophy(SMA) Treated With Valproic Acid |
|
|
Observational
|
|
- University of Sao Paulo General Hospital
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Manual Muscle Test (Medical Research Council scale-MRC), the Hammersmith Motor Ability Score
- Barthel Index
|
22 |
All |
2 Years to 18 Years (Child, Adult) |
NCT01033331 |
290/06
CAPES9030 |
|
July 2006 |
August 2008 |
December 2008 |
December 16, 2009 |
December 16, 2009 |
|
- Section of Neuromuscular Disorders and Service of Child Neurology, Clinics Hospital of the School of Medicine at São Paulo University
Sao PAulo, Brazil
|
|
| 34 |
NCT03505099 |
Recruiting |
Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 |
|
- Biological: onasemnogene abeparvovec-xioi
|
Interventional
|
Phase 3 |
- AveXis, Inc.
- PRA Health Sciences
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Percentage of participants achieving functional independent sitting for at least 30 seconds at any visit
- Percentage of participants achieving the ability to stand without support for at least 3 seconds at any visit
- Percentage of participants surviving without permanent ventilation in the absence of acute illness and perioperatively
- (and 2 more...)
|
27 |
All |
up to 42 Days (Child) |
NCT03505099 |
AVXS-101-CL-304
2017-004087-35
JapicCTI-184203 |
SPR1NT |
April 10, 2018 |
October 2020 |
June 2023 |
April 23, 2018 |
August 8, 2019 |
|
- David Geffen School of Medicine at UCLA
Los Angeles, California, United States - Stanford University
Stanford, California, United States - Children's Hospital Colorado
Aurora, Colorado, United States - (and 26 more...)
|
|
| 35 |
NCT03306277 |
Active, not recruiting |
Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 |
- SMA - Spinal Muscular Atrophy
- Gene Therapy
|
- Biological: Onasemnogene Abeparvovec-xioi
|
Interventional
|
Phase 3 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Achievement of independent sitting for at least 30 seconds
- Event-free survival
- Ability to thrive
- Ventilatory support independence
|
20 |
All |
up to 180 Days (Child) |
NCT03306277 |
AVXS-101-CL-303 |
STR1VE |
October 24, 2017 |
November 12, 2019 |
November 12, 2019 |
October 11, 2017 |
August 15, 2019 |
|
- David Geffen School of Medicine at UCLA
Los Angeles, California, United States - Stanford University
Stanford, California, United States - Children's Hospital Colorado
Aurora, Colorado, United States - (and 13 more...)
|
|
| 36 |
NCT02644668 |
Completed |
A Study of CK-2127107 in Patients With Spinal Muscular Atrophy |
|
- Drug: CK-2127107 150 mg
- Drug: Placebo
- Drug: CK-2127107 450 mg
|
Interventional
|
Phase 2 |
- Cytokinetics
- Astellas Pharma Global Development, Inc.
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Triple (Participant, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Change from baseline and slope of change from baseline in Forced Vital Capacity (FVC)
- Change from baseline and slope of change from baseline in Maximum Inspiratory Pressure (MIP)/Maximum Expiratory Pressure (MEP)
- Change from baseline and slope of change from baseline in Hand-Held Dynamometry (HHD)
- (and 10 more...)
|
70 |
All |
12 Years and older (Child, Adult, Older Adult) |
NCT02644668 |
CY 5021 |
|
December 2015 |
May 31, 2018 |
May 31, 2018 |
January 1, 2016 |
March 6, 2019 |
|
- UCLA
Los Angeles, California, United States - University of California Irvine
Orange, California, United States - Pediatric Neuromuscular Clinic Stanford University
Palo Alto, California, United States - (and 16 more...)
|
|
| 37 |
NCT03395795 |
Recruiting |
Trial Evaluating the Interest of Noninvasive Ventilation in NAVA Mode in Respiratory Decompensations Children With Infantile Spinal Muscular Atrophy Type II |
- Infantile Spinal Muscular Atrophy
|
|
Interventional
|
Not Applicable |
- Assistance Publique - Hôpitaux de Paris
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Asynchrony percentage
- Feasibility
- Efficacy and tolerance
- Rate of recourse to intubation
|
20 |
All |
1 Year to 18 Years (Child, Adult) |
NCT03395795 |
P160941J
IDRCB 2017-A02785-48 |
NAVASI |
February 13, 2018 |
February 2020 |
February 2020 |
January 10, 2018 |
May 13, 2019 |
|
- Service de pédiatrie, Hôpital Raymond Poincaré
Garches, Hauts-de-Seine, France
|
|
| 38 |
NCT01754441 |
Unknown † |
Mechanisms of Cell Death in Spinal Muscular Atrophy |
|
|
Observational
|
|
- Nemours Children's Clinic
|
Other |
- Time Perspective: Prospective
|
- SMN localization in SMA fibroblasts
- SMN isoform mRNA levels
- Protein levels of putative SMA phenotypic modifiers
- (and 4 more...)
|
50 |
All |
up to 21 Years (Child, Adult) |
NCT01754441 |
82008 |
|
May 2008 |
December 2017 |
December 2017 |
December 21, 2012 |
August 24, 2015 |
|
- Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States - Nemours Children's Specialty Care, Jacksonville
Jacksonville, Florida, United States - Nemours Children's Hospital
Orlando, Florida, United States
|
|
| 39 |
NCT02123186 |
Completed |
Newborn Screening for Spinal Muscular Atrophy |
|
- Other: newborn screening test for SMA
|
Observational
|
|
- National Taiwan University Hospital
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- numbers of newborn with spinal muscular atrophy
|
120267 |
All |
up to 3 Months (Child) |
NCT02123186 |
201308058RIND |
|
October 2013 |
October 2016 |
October 2016 |
April 25, 2014 |
November 2, 2016 |
|
- National Taiwan University Hospital
Taipei, Taiwan,, Taiwan
|
|
| 40 |
NCT03339830 |
Recruiting |
European Registry of Patients With Infantile-onset Spinal Muscular Atrophy |
|
|
Observational
|
|
- Institut de Myologie, France
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Change from Baseline in survival
- Change from Baseline in psychomotor development
- Change from Baseline in the number of lower track infections
- (and 22 more...)
|
100 |
All |
Child, Adult, Older Adult |
NCT03339830 |
IO-SMA-Registry
2017-A02291-52 |
|
October 10, 2017 |
December 1, 2022 |
December 1, 2022 |
November 13, 2017 |
January 24, 2018 |
|
- Hopital Morvan - CHU de Brest
Brest, France - Service de Rééducation Pédiatrique Infantile " L'Escale " - Hôpital Femme Mère Enfant
Bron, France - Hôpital le Bocage - CHU Dijon
Dijon, France - (and 3 more...)
|
|
| 41 |
NCT03837184 |
Recruiting |
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies |
- Spinal Muscular Atrophy Type I
|
- Biological: Onasemnogene Abeparvovec-xioi
|
Interventional
|
Phase 3 |
- AveXis, Inc.
- PRA Health Sciences
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Proportion of Participants Sitting Without Support
- Proportion of Participants Surviving
|
6 |
All |
up to 6 Months (Child) |
NCT03837184 |
AVXS-101-CL-306 |
|
May 15, 2019 |
April 1, 2021 |
April 1, 2021 |
February 12, 2019 |
August 14, 2019 |
|
- Tokyo Women's Medical University
Tokyo, Japan - Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam-do, Korea, Republic of - National Taiwan University Hospital
Taipei, Taiwan
|
|
| 42 |
NCT02391831 |
Completed |
Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy |
- Type 2 Spinal Muscular Atrophy
- Type 3 Spinal Muscular Atrophy
|
- Other: Strength, function and activity measurements
- Other: Muscle MRI
- Other: Electrophysiology measurements
- Other: Blood sampling for biomarker analysis
|
Interventional
|
Not Applicable |
- Institut de Myologie, France
- Institut Roche
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Basic Science
|
- Change from baseline of muscle strength
- Change from baseline of motor function
- Change from baseline of respiratory function
- (and 4 more...)
|
81 |
All |
2 Years to 30 Years (Child, Adult) |
NCT02391831 |
NatHis-SMA
IDRCB-2014-A01263-44 |
NatHis-SMA |
May 2015 |
June 2018 |
June 2018 |
March 18, 2015 |
July 19, 2018 |
|
- Reference centre for neuromuscular diseases - UZ Leuven - Department of Pediatrics - University Hospitals Leuven
Leuven, Belgium - Centre de Référence neuromusculaire - CHR La Citadelle
Liege, Belgium - Service de Rééducation Pédiatrique Infantile " L'Escale " - Hôpital Femme Mère Enfant
Bron, France - (and 6 more...)
|
|
| 43 |
NCT00528268 |
Completed
Has Results |
Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy |
|
- Drug: Sodium phenylbutyrate (NaPB)
|
Interventional
|
Phase 1
Phase 2 |
- University of Utah
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
|
Other / NIH |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- The Study Will Assess the Safety, Tolerability and Potential Efficacy of Sodium Phenylbutyrate (NaPB) in Presymptomatic Infants Genetically Confirmed to Have SMA. It Will Also Determine Selected Pharmacokinetic Parameters.
- The Study Will Determine Potential Benefit of NaPB on Lean Body Mass; Overall Motor Function; Potential Cellular Response to NaPB; and Drug Compliance.
|
14 |
All |
up to 6 Months (Child) |
NCT00528268 |
22183
1R01HD054599-01 |
STOPSMA |
July 2007 |
December 2013 |
December 2013 |
September 12, 2007 |
July 3, 2015 |
July 3, 2015 |
- University of Utah
Salt Lake City, Utah, United States
|
|
| 44 |
NCT02227823 |
Unknown † |
Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3 |
- Spinal Muscular Atrophy Type 3
|
- Drug: Pyridostigmine Bromide
|
Interventional
|
Phase 2 |
- Centre Hospitalier Régional de la Citadelle
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Factorial Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change from Baseline in the distance walked at 6-minute walk test at 6 months
- Change from baseline of decrement at 6 months
- Change from baseline of MFM-D1
- (and 2 more...)
|
20 |
All |
6 Years and older (Child, Adult, Older Adult) |
NCT02227823 |
1376 |
EMOTAS |
July 2014 |
July 2017 |
July 2017 |
August 28, 2014 |
August 28, 2014 |
|
- Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle
Liège, Belgium
|
|
| 45 |
NCT00374075 |
Completed |
Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy |
|
|
Interventional
|
Phase 1 |
- University of Utah
- Families of Spinal Muscular Atrophy
- Leadiant Biosciences, Inc.
- Abbott
|
Other / Industry |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- To assess safety and tolerability of VPA in SMA patients greater than 2 years of age
- To look for a potential in vivo effect of VPA on SMN mRNA in patient blood cells at routinely used clinical doses
- Measures of gross motor function
- (and 3 more...)
|
42 |
All |
2 Years and older (Child, Adult, Older Adult) |
NCT00374075 |
11893 |
|
September 2003 |
|
February 2006 |
September 8, 2006 |
August 23, 2016 |
|
- University of Utah/Primary Children's Medical Center
Salt Lake City, Utah, United States
|
|
| 46 |
NCT01522079 |
Completed |
Spinal Muscular Atrophy and Cardiac Autonomic Function |
|
- Procedure: Air stacking manuever
|
Interventional
|
Not Applicable |
- Centro Universitário Augusto Motta
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Cardiac autonomic function
|
9 |
All |
5 Years to 25 Years (Child, Adult) |
NCT01522079 |
Dias |
|
January 2011 |
August 2011 |
October 2011 |
January 31, 2012 |
January 31, 2012 |
|
|
|
| 47 |
NCT02122952 |
Completed
Has Results |
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 |
- Spinal Muscular Atrophy 1
|
|
Interventional
|
Phase 1 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of Participants That Experienced One Grade III or Higher Unanticipated, Treatment-related Toxicity That Presents With Clinical Symptoms and Requires Medical Treatment
- Number of Participants Who Experienced (a) Requirement of ≥16-hour Respiratory Assistance Per Day Continuously for ≥2 Weeks in the Absence of an Acute Reversible Illness, Excluding Perioperative Ventilation, or (b) Death
- Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND)
|
15 |
All |
up to 6 Months (Child) |
NCT02122952 |
AVXS-101-CL-101 |
|
May 5, 2014 |
December 15, 2017 |
December 15, 2017 |
April 25, 2014 |
May 10, 2019 |
May 10, 2019 |
- Nationwide Children's Hospital
Columbus, Ohio, United States
|
|
| 48 |
NCT03648658 |
Recruiting |
Paracetamol Study in Patients With Low Muscle Mass |
|
- Drug: Paracetamol 120Mg/5mL Oral Suspension
|
Interventional
|
Phase 4 |
- Mette Cathrine Oerngreen
- Elsass Foundation
- Rigshospitalet, Denmark
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Basic Science
|
- Clearance paracetamol
- Volume of distribution of paracetamol
- Liver function tests: ALT, AST, LDH, Alkaline Phosphatase, Bilirubin (U/L)
- Concentration-time data on liver function and paracetamolparametres
|
48 |
All |
6 Years to 45 Years (Child, Adult) |
NCT03648658 |
08-06-2018-paracet
2018-002295-40 |
|
February 18, 2019 |
July 2021 |
July 2021 |
August 27, 2018 |
February 27, 2019 |
|
- Copenhagen Neuromuscular Center
Copenhagen, Denmark
|
|
| 49 |
NCT00439218 |
Terminated
Has Results |
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I |
- Spinal Muscular Atrophy Type I
|
- Drug: sodium phenylbutyrate
|
Interventional
|
Phase 1
Phase 2 |
- Westat
- National Institute of Neurological Disorders and Stroke (NINDS)
|
Other / NIH |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Dose Limiting Toxicities (DLT)
- Survival Motor Neuron (SMN) Messenger Ribonucleic Acid (mRNA)
- Survival Motor Neuron (SMN) Protein
- (and 5 more...)
|
5 |
All |
2 Months to 48 Months (Child) |
NCT00439218 |
N01NS42361_NPTUNE02
HHSN265200423611C |
NPTUNE 02 |
January 2008 |
May 2009 |
May 2009 |
February 23, 2007 |
November 9, 2010 |
July 20, 2010 |
- Stanford University Medical Center, 300 Pasteur Drive, Room A343
Stanford, California, United States - Children's Hospital, Boston, 300 Longwood Avenue, Fegan 11
Boston, Massachusetts, United States - Columbia University, 180 Fort Washington Avenue, 5th Floor
New York, New York, United States - (and 2 more...)
|
|
| 50 |
NCT00439569 |
Terminated
Has Results |
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III |
- Spinal Muscular Atrophy Type II
- Spinal Muscular Atrophy Type III
|
- Drug: sodium phenylbutyrate
|
Interventional
|
Phase 1
Phase 2 |
- Westat
- National Institute of Neurological Disorders and Stroke (NINDS)
|
Other / NIH |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Dose Limiting Toxicities (DLT)
- Survival Motor Neuron (SMN) Messenger Ribonucleic Acid (mRNA)
- Survival Motor Neuron (SMN) Protein
- (and 5 more...)
|
9 |
All |
2 Years to 11 Years (Child) |
NCT00439569 |
N01NS42361_NPTUNE01
HHSN265200423611C |
NPTUNE01 |
January 2008 |
August 2008 |
August 2008 |
February 23, 2007 |
September 8, 2010 |
February 12, 2010 |
- Stanford University Medical Center, 300 Pasteur Drive, Room A343
Stanford, California, United States - Washington University Medical School, Washington University, 660 S. Euclid Avenue, Box 8111
St. Louis, Missouri, United States - Columbia University, 180 Fort Washington Avenue, 5th Floor
New York, New York, United States - (and 2 more...)
|
|
| 51 |
NCT00481013 |
Completed |
Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy |
|
- Drug: Valproic Acid (VPA)
- Drug: Placebo
|
Interventional
|
Phase 2 |
- University of Utah
- Families of Spinal Muscular Atrophy
- Abbott
|
Other / Industry |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- The primary outcome for the study is change in muscle strength from baseline to six months in muscle strength as assessed by MVICT using a fixed testing system.
- Change in SMAFRS
- Change in strength assessed by hand-held dynamometer
- (and 7 more...)
|
33 |
All |
18 Years to 60 Years (Adult) |
NCT00481013 |
2006H0249 |
VALIANTSMA |
July 2007 |
December 2009 |
November 2010 |
June 1, 2007 |
December 6, 2016 |
|
- Ohio State University Medical Center, Dept. of Neurology
Columbus, Ohio, United States
|
|
| 52 |
NCT01671384 |
Unknown † |
Valproate and Levocarnitine in Children With Spinal Muscular Atrophy |
|
- Drug: Valproate, Levocarnitine
- Drug: Placebo
|
Interventional
|
Phase 3 |
- All India Institute of Medical Sciences, New Delhi
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Change in muscle power on a 5 point scale as per the principles of manual muscle testing at 12, 24, 36 and 52 weeks
- a) Change in functional measure using modified Hammersmith Functional Motor Scale (MHFMS) score at 12, 24, 36 and 52 weeks 12, 24, 36 and 52 weeks.
|
60 |
All |
2 Years to 15 Years (Child) |
NCT01671384 |
Valproate and SMA |
|
August 2013 |
December 2016 |
December 2016 |
August 23, 2012 |
January 1, 2016 |
|
- All India Institute of Medical Sciences
New Delhi, Delhi, India
|
|
| 53 |
NCT03056144 |
Terminated
Has Results |
Whole Body Vibration Therapy in Children With Spinal Muscular Atrophy |
- Spinal Muscular Atrophy Type 3
|
- Device: whole body vibration therapy
|
Interventional
|
Not Applicable |
- The Hong Kong Polytechnic University
- Manchester Metropolitan University
- The University of Hong Kong
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- North Star Ambulatory Assessment
- 2-minute Walk Test
- Segmental Assessment of Trunk Control-static
- (and 41 more...)
|
1 |
All |
6 Years to 18 Years (Child, Adult) |
NCT03056144 |
YBPA |
|
August 1, 2017 |
July 12, 2018 |
July 12, 2018 |
February 17, 2017 |
July 26, 2019 |
July 26, 2019 |
- The Hong Kong Polytechnic University
Hung Hom, Hong Kong
|
|
| 54 |
NCT02193074 |
Terminated
Has Results |
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy |
|
- Drug: nusinersen
- Procedure: Sham procedure
|
Interventional
|
Phase 3 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Percentage of Motor Milestones Responders
- Time to Death or Permanent Ventilation
- Percentage of Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Responders
- (and 11 more...)
|
122 |
All |
up to 210 Days (Child) |
NCT02193074 |
ISIS 396443-CS3B
2013-004422-29 |
ENDEAR |
August 19, 2014 |
November 21, 2016 |
November 21, 2016 |
July 17, 2014 |
July 28, 2017 |
July 28, 2017 |
- UCLA Medical Center
Los Angeles, California, United States - Children's Hospital Colorado
Aurora, Colorado, United States - Nemours Children's Hospital
Orlando, Florida, United States - (and 28 more...)
|
|
| 55 |
NCT01780246 |
Completed |
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701) |
|
|
Interventional
|
Phase 1 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants that experience Adverse Events (AEs) and Serious Adverse Events
- Number of participants with clinically significant neurological examination abnormalities
- Number of participants with clinically significant vital sign abnormalities
- (and 9 more...)
|
18 |
All |
2 Years to 15 Years (Child) |
NCT01780246 |
ISIS 396443-CS10 |
|
January 2013 |
February 2014 |
February 2014 |
January 31, 2013 |
April 4, 2017 |
|
- Boston Children's Hospital
Boston, Massachusetts, United States - Columbia University Medical Center
New York, New York, United States - UT Southwestern Medical Center - Children's Medical Center Dallas
Dallas, Texas, United States - University of Utah School of Medicine
Salt Lake City, Utah, United States
|
|
| 56 |
NCT01703988 |
Completed
Has Results |
An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy |
|
|
Interventional
|
Phase 1
Phase 2 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Discontinuations Due to AEs, and Highest Severity of AEs
- Plasma Pharmacokinetics: Maximal Observed Plasma Drug Concentration (Cmax)
- Plasma Pharmacokinetics: Time to Reach Cmax in Plasma
- (and 3 more...)
|
34 |
All |
2 Years to 15 Years (Child) |
NCT01703988 |
ISIS 396443 - CS2
2017-000327-27 |
|
October 2012 |
January 2015 |
January 2015 |
October 11, 2012 |
March 13, 2017 |
March 13, 2017 |
- Boston Children's Hospital
Boston, Massachusetts, United States - Columbia University Medical Center
New York, New York, United States - UT Southwestern Medical Center - Children's Medical Center Dallas
Dallas, Texas, United States - University of Utah School of Medicine
Salt Lake City, Utah, United States
|
|
| 57 |
NCT00756821 |
Completed |
A Pilot Study of Biomarkers for Spinal Muscular Atrophy |
|
|
Observational
|
|
- New England Research Institutes
- The Spinal Muscular Atrophy Foundation
|
Other |
- Observational Model: Cohort
- Time Perspective: Cross-Sectional
|
- To identify candidate blood and urine biochemical markers that correlate with disease severity as determined by the Modified Hammersmith Functional Motor Scale across a range of type I, type II and type III children with Spinal Muscular Atrophy (SMA)
- To determine if there are biomarkers from types I-III SMA patients that correlate with SMA type, age at disease onset, 10-meter Timed Walk Test, pulmonary function, nutritional assessment, SMN protein level, SMN transcript level or SMN2 copy number.
- To determine if identified candidate biomarkers are associated with the disease state through comparison of SMA specimens with control volunteer specimens.
|
130 |
All |
2 Years to 12 Years (Child) |
NCT00756821 |
BforSMA |
BforSMA |
October 2008 |
March 2009 |
March 2009 |
September 22, 2008 |
October 24, 2012 |
|
- University of Alabama at Birmingham
Birmingham, Alabama, United States - Stanford University
Stanford, California, United States - The Children's Hospital
Aurora, Colorado, United States - (and 15 more...)
|
|
| 58 |
NCT03808233 |
Completed |
Reliability and Validity of the ACTIVE-mini for Quantifying Movement in Infants With Spinal Muscular Atrophy |
|
|
Observational
|
|
- University of Texas Southwestern Medical Center
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
|
25 |
All |
Child, Adult, Older Adult |
NCT03808233 |
STU 052016-109 |
|
November 5, 2016 |
June 1, 2018 |
August 31, 2018 |
January 17, 2019 |
January 17, 2019 |
|
|
|
| 59 |
NCT04050852 |
Recruiting |
Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments |
|
- Drug: Nusinersen Treatments
|
Interventional
|
Early Phase 1 |
- NYU Langone Health
- Winthrop University Hospital
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- percent improvement Maximum inspiratory pressure (MIP)
- percent improvement Maximum expiratory pressure (/MEP)
|
10 |
All |
5 Years to 21 Years (Child, Adult) |
NCT04050852 |
19-00242 |
|
July 17, 2019 |
July 17, 2020 |
July 17, 2020 |
August 8, 2019 |
August 8, 2019 |
|
- NYU Langone Health
New York, New York, United States
|
|
| 60 |
NCT03381729 |
Recruiting |
Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy |
|
- Biological: Onasemnogene Abeparvovec-xioi
|
Interventional
|
Phase 1 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Incidence of Adverse Events
- Determine Optimal Dose
- Patients ≥ 6 months and < 24 months: Standing Milestone
- (and 4 more...)
|
51 |
All |
6 Months to 60 Months (Child) |
NCT03381729 |
AVXS-101-CL-102 |
STRONG |
December 14, 2017 |
June 1, 2021 |
June 1, 2021 |
December 22, 2017 |
August 15, 2019 |
|
- UCLA
Los Angeles, California, United States - Stanford University
Stanford, California, United States - Nemours Children's Hospital
Orlando, Florida, United States - (and 8 more...)
|
|
| 61 |
NCT03032172 |
Recruiting |
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy |
|
|
Interventional
|
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Percentage of Participants With Adverse Events (AEs) and Serious AEs (SAEs) with Severity Determined According to National Cancer Institute Common Terminology Criteria for Adverse Events Scale, V 4.0
- Percentage of Participants With Emergence or Worsening of Symptoms As Assessed Using Columbia Suicide Severity Rating Scale (C-SSRS) (Adult Version for Adults and Adolescents, Pediatric Version for Patients Aged 6-11 Years)
- Percentage of Participants With Protocol Defined Clinically Significant Changes in Ophthalmological Assessments
- (and 12 more...)
|
180 |
All |
6 Months to 60 Years (Child, Adult) |
NCT03032172 |
BP39054
2016-004184-39 |
Jewelfish |
March 3, 2017 |
December 31, 2021 |
December 27, 2024 |
January 26, 2017 |
August 29, 2019 |
|
- Stanford University Medical Center
Palo Alto, California, United States - Columbia University Medical Center; The Neurological Institute of New York
New York, New York, United States - UZ Gent
Gent, Belgium - (and 15 more...)
|
|
| 62 |
NCT03955679 |
Approved for marketing |
AveXis Managed Access Program Cohort for Access to AVXS-101 |
|
|
Expanded Access
|
|
- United BioSource Corporation
- AveXis, Inc.
|
Industry |
|
|
|
All |
Child, Adult, Older Adult |
NCT03955679 |
AVXS-101-MAP-001 |
|
|
|
|
May 20, 2019 |
June 5, 2019 |
|
- Nationwide Children's Hospital
Columbus, Ohio, United States
|
|
| 63 |
NCT03709784 |
Recruiting |
Spinraza in Adult Spinal Muscular Atrophy |
- Spinal Muscular Atrophy
- Spinal Muscular Atrophy Type II
- Spinal Muscular Atrophy Type 3
|
- Other: Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care
|
Observational
|
|
- Washington University School of Medicine
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Change from baseline in the 6-Minute Walk Test (6MWT) for ambulatory SMA patients
- Change from baseline in Revised Upper Limb Module (RULM) for weak ambulatory and non-ambulatory SMA patients
|
73 |
All |
18 Years to 70 Years (Adult, Older Adult) |
NCT03709784 |
201805187 |
SAS |
August 16, 2018 |
January 30, 2022 |
January 30, 2022 |
October 17, 2018 |
May 22, 2019 |
|
- Barrow Neurological Institute
Phoenix, Arizona, United States - Washington University School of Medicine
Saint Louis, Missouri, United States - Houston Methodist Neurological Institute
Houston, Texas, United States - (and 2 more...)
|
|
| 64 |
NCT02003937 |
Completed |
Aerobic Training in Patients With Spinal Muscular Atrophy Type III |
|
- Behavioral: Aerobic conditioning
|
Interventional
|
Not Applicable |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Changes in oxidative capacity with aerobic conditioning in SMAIII patients
- Changes in maximal workload capacity with aerobic conditioning in SMAIII patients
- Changes in isometric muscle strength with aerobic conditioning in SMAIII patients
- Changes in daily function with aerobic conditioning in SMAIII patients
|
15 |
All |
18 Years to 65 Years (Adult, Older Adult) |
NCT02003937 |
H-KF297836 |
|
September 2009 |
September 2010 |
September 2010 |
December 6, 2013 |
October 29, 2015 |
|
- Rigshospitalet, Neuromuscular Research Unit, 3342
Copenhagen E, Denmark
|
|
| 65 |
NCT00443066 |
Active, not recruiting |
Clinical Study of Spinal Muscular Atrophy |
|
|
Observational
|
|
- Columbia University
- The Spinal Muscular Atrophy Foundation
|
Other |
- Observational Model: Case-Only
- Time Perspective: Prospective
|
- Hammersmith Functional Motor Scale Expanded (HFMSE)
- Gross Motor Function Scale (GMFM)
|
120 |
All |
Child, Adult, Older Adult |
NCT00443066 |
AAAB1635
CU52029001 |
|
May 2005 |
October 2020 |
December 2022 |
March 5, 2007 |
April 9, 2019 |
|
- Columbia University SMA Clinical Research Center
New York, New York, United States
|
|
| 66 |
NCT03878030 |
Recruiting |
Effect of Nusinersen on Adults With Spinal Muscular Atrophy |
- Adult Spinal Muscular Atrophy
|
|
Observational
|
|
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Change in quantitative motor function
- Change in upper limb motor function
- Change in 6 minute walk test in ambulatory patients
- (and 4 more...)
|
40 |
All |
18 Years to 60 Years (Adult) |
NCT03878030 |
18-0149 |
|
April 1, 2019 |
January 1, 2023 |
January 1, 2023 |
March 18, 2019 |
May 30, 2019 |
|
- Northwell Health Neuroscience
Great Neck, New York, United States
|
|
| 67 |
NCT02386553 |
Active, not recruiting |
A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy |
|
|
Interventional
|
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Prevention
|
- Time to death or respiratory intervention
- Percentage of participants developing clinically manifested spinal muscular atrophy (SMA).
- Percentage of participants alive
- (and 15 more...)
|
25 |
All |
up to 6 Weeks (Child) |
NCT02386553 |
232SM201
2014-002098-12 |
NURTURE |
May 20, 2015 |
January 25, 2022 |
January 25, 2022 |
March 12, 2015 |
August 27, 2018 |
|
- Children's Hospital Colorado
Aurora, Colorado, United States - Nemours Children's Clinic
Orlando, Florida, United States - Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States - (and 12 more...)
|
|
| 68 |
NCT00485511 |
Completed |
A Trial of Hydroxyurea in Spinal Muscular Atrophy |
|
|
Interventional
|
Phase 2
Phase 3 |
- Kaohsiung Medical University Chung-Ho Memorial Hospital
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double
|
|
|
All |
4 Years and older (Child, Adult, Older Adult) |
NCT00485511 |
DOH96-TD-I-111-TM103 |
|
June 2007 |
June 2009 |
June 2009 |
June 13, 2007 |
February 15, 2010 |
|
- Kaohsiung Medical University Hospital
Kaohsiung, Taiwan
|
|
| 69 |
NCT00155168 |
Unknown † |
Quantitative Analysis of SMN1 and SMN2 Gene Based on DHPLC System |
|
|
Observational
|
|
- National Taiwan University Hospital
|
Other |
- Observational Model: Case Control
- Primary Purpose: Screening
- Time Perspective: Longitudinal
- Time Perspective: Prospective
|
|
500 |
All |
Child, Adult, Older Adult |
NCT00155168 |
9361700452 |
|
April 2004 |
|
|
September 12, 2005 |
September 12, 2005 |
|
- Dept of Medical Genetics;National Taiwan University Hospital
Taipei, Taiwan
|
|
| 70 |
NCT02073604 |
Completed |
Role of the SMA During Unimanual and Bimanual Movements Preparation: the Mirror Movements Paradigm |
- Congenital Mirror Movements
|
- Other: Healthy volunteers
- Other: Congenital mirror movements
|
Interventional
|
Not Applicable |
- Institut National de la Santé Et de la Recherche Médicale, France
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Basic Science
|
- SMA activation level and connectivity during preparation of unimanual/bimanual movements
- SMA activation level and connectivity during preparation of unimanual/bimanual movements in congenital mirror movements patients
|
47 |
All |
15 Years to 82 Years (Child, Adult, Older Adult) |
NCT02073604 |
C13-16
2013-A00616-39 |
MOMIC2 |
March 2014 |
February 2016 |
February 2016 |
February 27, 2014 |
August 31, 2016 |
|
- Fédération des Maladies du Système Nerveux, Hôpital Pitié Salpétrière Paris, France 75013
Paris, France
|
|
| 71 |
NCT03223051 |
Recruiting |
Development of a Space Exploration Assessment for Children With Spinal Muscular Atrophy |
- Spinal Muscular Atrophy
- Assessment, Self
|
|
Interventional
|
Not Applicable |
- Centre d'Investigation Clinique et Technologique 805
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Other
|
- Motor Function Measure (MFM)
- Tiredness
- Motivation
- Satisfaction
|
30 |
All |
3 Years to 16 Years (Child) |
NCT03223051 |
2017-A01017-46 |
ExplorASI |
September 7, 2017 |
September 7, 2017 |
September 2020 |
July 19, 2017 |
January 25, 2018 |
|
- Hopital Raymond Poincare
Garches, France - Hopital Raymond Poincare
Garches, France - Nicolas ROCHE, Md PhD
Garches, France
|
|
| 72 |
NCT02763865 |
Completed
Has Results |
A Pilot Study to Assess the Effects of Pre-SMA LF-rTMS for the Treatment of Essential Tremor |
|
- Device: MagVenture MagProx100 Stimulator with a Cool-B65 A/P coil
- Device: eSHAM system
|
Interventional
|
Not Applicable |
- Medical University of South Carolina
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Single (Participant)
- Primary Purpose: Treatment
|
- Mean TRS (Tremor Rating Score)
- Mean TRS (Tremor Rating Score) at 4 Week Followup
- Mean TRS (Tremor Rating Score) at 8 Week Followup
- Mean TRS (Tremor Rating Score) at 12 Week Followup
|
10 |
All |
18 Years and older (Adult, Older Adult) |
NCT02763865 |
Pro00033680 |
|
July 2014 |
December 2015 |
March 21, 2017 |
May 5, 2016 |
February 15, 2019 |
February 15, 2019 |
|
|
| 73 |
NCT03421977 |
Enrolling by invitation |
Long-Term Follow-up Study for Patients From AVXS-101-CL-101 |
- Spinal Muscular Atrophy 1
|
- Biological: Onasemnogene Abeparvovec-xioi
|
Observational
|
|
|
Industry |
- Observational Model: Other
- Time Perspective: Prospective
|
|
15 |
All |
Child, Adult, Older Adult |
NCT03421977 |
AVXS-101-LT-001 |
START |
August 15, 2017 |
December 2033 |
December 2033 |
February 5, 2018 |
August 15, 2019 |
|
- Nationwide Children's Hospital
Columbus, Ohio, United States
|
|
| 74 |
NCT02514928 |
Unknown † |
Resection of the Nerve Plexus on the Right Half of Celiac and SMA Associated With Extended Pancreatoduodenectomy in the Surgical Treatment for Adenocarcinoma of the Head of Pancreas |
|
- Procedure: resection of the nerve plexus on the right half of celiac and SMA associated with extended pancreatoduodenectomy
- Procedure: standard pancreatoduodenectomy
|
Interventional
|
Phase 3 |
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Single (Participant)
- Primary Purpose: Treatment
|
- Overall survival
- Pain Control
|
430 |
All |
18 Years to 80 Years (Adult, Older Adult) |
NCT02514928 |
PCI003 |
|
June 2015 |
June 2017 |
June 2019 |
August 4, 2015 |
August 11, 2016 |
|
- Pancreatic Cancer Institute
Shanghai, Shanghai, China
|
|
| 75 |
NCT02347475 |
Completed |
SMA Flow Patterns in Neonatal Transfusion |
- Necrotizing Enterocolitis
|
- Other: Observational Study
|
Observational
|
|
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Post-prandial SMA flows changes before and after PRBC transfusion
|
7 |
All |
up to 6 Months (Child) |
NCT02347475 |
14-0312 |
SPARTAN |
January 2015 |
December 2017 |
December 2017 |
January 27, 2015 |
May 3, 2018 |
|
- Foothills Medical Center
Calgary, Alberta, Canada
|
|
| 76 |
NCT00466349 |
Completed |
International SMA Patient Registry |
|
|
Observational
|
|
- Indiana University School of Medicine
- Cure SMA
- Indiana University
|
Other |
- Observational Model: Family-Based
- Time Perspective: Other
|
- Study is a patient registry
|
3000 |
All |
Child, Adult, Older Adult |
NCT00466349 |
0202-03 |
|
May 1986 |
January 2018 |
January 2018 |
April 27, 2007 |
August 7, 2018 |
|
- Indiana University School of Medicine
Indianapolis, Indiana, United States
|
|
| 77 |
NCT03329495 |
Not yet recruiting |
Laparoscopic Right Hemicoloectomy for Right Colon Cancer With a Medial-to-lateral Approach Orientated by SMA or SMV |
- Right Colic Lymphadenopathy
|
- Procedure: SMA-orientated right hemicoloectomy
- Procedure: SMV-orientated right hemicoloectomy
|
Interventional
|
Phase 2
Phase 3 |
- The First Affiliated Hospital with Nanjing Medical University
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- number of lymph nodes retrieved
- disease-free survival
- overall survival
- (and 3 more...)
|
200 |
All |
18 Years to 85 Years (Adult, Older Adult) |
NCT03329495 |
CRSYM201710 |
|
January 2018 |
January 2018 |
January 2023 |
November 6, 2017 |
November 6, 2017 |
|
- Jiangsu province hospital
Nanjing, Jiangsu, China
|
|
| 78 |
NCT02550691 |
Terminated |
Identification of a Biomarker Associated With Cis-duplication of the SMN1 Gene |
|
- Procedure: blood sampling
|
Interventional
|
Not Applicable |
- University Hospital, Rouen
- Society GENOMIC VISION
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Diagnostic
|
- Identification of a genetic signature indicating the presence of one allele with 2 copies in cis of the SMN1 gene.
- Specificity of a genetic signature
|
48 |
All |
18 Years and older (Adult, Older Adult) |
NCT02550691 |
2015/092/HP |
BADGES |
December 15, 2015 |
July 4, 2016 |
July 4, 2016 |
September 15, 2015 |
July 24, 2019 |
|
- Nantes University Hospital
Nantes, France - Rouen University Hospital
Rouen, France
|
|
| 79 |
NCT00533221 |
Completed |
Pilot Study of Growth Hormon to Treat SMA Typ II and III |
|
- Drug: somatotropin
- Drug: Placebo
|
Interventional
|
Phase 2 |
- University Hospital Freiburg
- Novo Nordisk A/S
|
Other / Industry |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Primary: sum of strength (hand held myometry)
- Functional (time) tests, lung function, quality of life,
|
20 |
All |
6 Years to 35 Years (Child, Adult) |
NCT00533221 |
SMA-GH
2005-002822-78 |
|
October 2007 |
February 2010 |
August 2011 |
September 21, 2007 |
December 18, 2013 |
|
- von Haunersches Kinderspital
Munich, Bavaria, Germany - University Children'S Hospital
Göttingen, Niedersachsen, Germany - University Children'S Hospital
Essen, Nordrhein-Westfalen, Germany - (and 3 more...)
|
|
| 80 |
NCT04042025 |
Not yet recruiting |
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi |
- Spinal Muscular Atrophy Type I
- Spinal Muscular Atrophy Type II
- Spinal Muscular Atrophy Type III
|
- Biological: Onasemnogene Abeparvovec-xioi
|
Interventional
|
Phase 4 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Prevention
|
- Number of participants who reach developmental milestones
- Change from baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) score
- Number of participants who experience a clinically significant change from baseline in pulmonary assessment results
- (and 10 more...)
|
308 |
All |
Child, Adult, Older Adult |
NCT04042025 |
AVXS-101-LT-002
2019-002611-26 |
|
September 1, 2019 |
December 31, 2034 |
December 31, 2034 |
August 1, 2019 |
September 2, 2019 |
|
- David Geffen School of Medicine at UCLA
Los Angeles, California, United States - Stanford University Medical Center
Palo Alto, California, United States - Children's Hospital Colorado
Aurora, Colorado, United States - (and 34 more...)
|
|
| 81 |
NCT03921528 |
Recruiting |
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy |
- Spinal Muscular Atrophy
- Spinal Muscular Atrophy Type 3
- Spinal Muscular Atrophy Type 2
- (and 6 more...)
|
|
Interventional
|
Phase 2 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Cohort 1: Change from Baseline in the Revised Hammersmith Scale (RHS)
- Cohort 2 and Cohort 3: Change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE)
|
55 |
All |
2 Years to 21 Years (Child, Adult) |
NCT03921528 |
SRK-015-002 |
TOPAZ |
April 22, 2019 |
January 2021 |
April 2021 |
April 19, 2019 |
August 22, 2019 |
|
- Stanford University
Palo Alto, California, United States - Children's Hospital Colorado
Aurora, Colorado, United States - Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States - (and 8 more...)
|
|
| 82 |
NCT02341053 |
Active, not recruiting |
Effects of Standing on Non-Ambulatory Children With Spinal Muscular Atrophy |
- Spinal Muscular Dystrophy
- Neuromuscular Disability
|
- Other: Assisted Standing Treatment Program
|
Interventional
|
Not Applicable |
- Gillette Children's Specialty Healthcare
- University of Minnesota, MN
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Collection of load magnitude and duration data (Use load-sensing platforms to collect load magnitude and duration during standing treatment program)
- Bone Mineral Density
|
6 |
All |
3 Years to 14 Years (Child) |
NCT02341053 |
1411M55281 |
|
September 2015 |
December 2019 |
December 2019 |
January 19, 2015 |
July 10, 2018 |
|
- Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, United States
|
|
| 83 |
NCT02235090 |
Withdrawn |
Study of Feasibility to Reliably Measure Functional Abilities' Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting |
- Spinal Muscular Atrophy
- Neuromuscular Disorders
|
- Other: Direct current stimulation of cervical spinal cord
|
Interventional
|
Not Applicable |
- Charitable Foundation Children with Spinal Muscular Atrophy
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Other
|
- Strength Changes from baseline measured by handheld myometry after spinal cord direct current stimulation of different intensity
- Short time fluctuations of Hammersmith Functional Motor Scale indexes
|
0 |
All |
5 Years and older (Child, Adult, Older Adult) |
NCT02235090 |
CSMA-INPN-1 |
|
October 2016 |
September 30, 2017 |
October 30, 2017 |
September 9, 2014 |
February 15, 2019 |
|
- SI "Institute of Neurology, Psychiatry and Narcology of NAMS of Ukraine"
Kharkiv, Ukraine
|
|
| 84 |
NCT02628743 |
Completed
Has Results |
A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA) |
|
|
Interventional
|
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Percentage of Participants With Adverse Events (AEs) or Serious Adverse Events (SAEs)
- Change From Baseline in Motor Function Measure (MFM) Dimension 1 (D1) + Dimension 2 (D2) Score
- Change From Baseline in MFM Total Score (D1+ D2 + Dimension 3 [D3]) Score
- (and 18 more...)
|
131 |
All |
Child, Adult, Older Adult |
NCT02628743 |
BN29854
2015-001589-25 |
|
January 20, 2016 |
December 18, 2018 |
December 18, 2018 |
December 11, 2015 |
August 9, 2019 |
August 9, 2019 |
- UZ Gent
Gent, Belgium - UZ Leuven Gasthuisberg
Leuven, Belgium - Hopital Femme Mere Enfant; Medecine Physique et Readaptation Pediatrique - L'ESCALE
Bron, France - (and 21 more...)
|
|
| 85 |
NCT03819660 |
Enrolling by invitation |
Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3 |
|
- Drug: Amifampridine Phosphate 10 MG Oral Tablet
|
Interventional
|
Phase 2 |
- Catalyst Pharmaceuticals, Inc.
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- The number and severity of treatment related adverse events and serious adverse events as assessed by CTCAE v4.0
|
12 |
All |
6 Years to 50 Years (Child, Adult) |
NCT03819660 |
SMA-002 |
SMA3 |
March 7, 2019 |
December 31, 2020 |
April 1, 2021 |
January 28, 2019 |
March 29, 2019 |
|
- Neurological Institute Carlo Besta
Milano, Lombardy, Italy
|
|
| 86 |
NCT02908685 |
Active, not recruiting |
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants |
|
- Drug: Placebo
- Drug: Risdiplam
|
Interventional
|
Phase 2
Phase 3 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Sequential Assignment
- Masking: Double (Participant, Investigator)
- Primary Purpose: Treatment
|
- Part 2: Change from Baseline in the Total Motor Function Measure 32 (MFM-32) Score at Month 12
- Part 1: Recommended Part 2 Dose of Risdiplam
- Survival of Motor Neuron 2 (SMN2) Messenger Ribonucleic Acid (mRNA) Levels in Blood
- (and 23 more...)
|
231 |
All |
2 Years to 25 Years (Child, Adult) |
NCT02908685 |
BP39055
2016-000750-35 |
SUNFISH |
October 20, 2016 |
September 30, 2019 |
September 2, 2023 |
September 21, 2016 |
September 4, 2019 |
|
- Stanford University Medical Center
Palo Alto, California, United States - Columbia University Medical Center; The Neurological Institute of New York
New York, New York, United States - UZ Gent
Gent, Belgium - (and 42 more...)
|
|
| 87 |
NCT02053831 |
Completed |
GalaFlex Mesh in Facelift |
- Superficial Musculoaponeurotic System (SMAS) Procedure
|
|
Observational
|
|
|
Industry |
- Observational Model: Case-Only
- Time Perspective: Prospective
|
- Physician Satisfaction
- Patient Satisfaction
|
15 |
All |
21 Years and older (Adult, Older Adult) |
NCT02053831 |
CP-1040 |
|
November 2011 |
March 2014 |
June 2014 |
February 4, 2014 |
March 10, 2015 |
|
- Ponte Vedra Plastic Surgery
Ponte Vedra Beach, Florida, United States - The Few Institute for Aesthetic Plastic Surgery
Chicago, Illinois, United States - Eye and Ear Institute
Chicago, Illinois, United States
|
|
| 88 |
NCT01910168 |
Completed |
A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis |
|
|
Observational
|
|
- Hoffmann-La Roche
- PTC Therapeutics
|
Industry |
- Observational Model: Case Control
- Time Perspective: Prospective
|
- SMN1/SMN2 detection in blood by mRNA assay
- SMN protein level in blood/lymphocytes
|
36 |
All |
Child, Adult, Older Adult |
NCT01910168 |
BE29002 |
|
August 2013 |
February 2014 |
February 2014 |
July 29, 2013 |
November 2, 2016 |
|
- Kalamazoo, Michigan, United States
- Salt Lake, Utah, United States
|
|
| 89 |
NCT03781479 |
Recruiting |
Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients |
|
- Drug: Amifampridine Phosphate
- Drug: Placebo Oral Tablet
|
Interventional
|
Phase 2 |
- Catalyst Pharmaceuticals, Inc.
|
Industry |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Change from baseline in the Hammersmith Functional Motor Scale Expanded (HFMSE)
|
12 |
All |
6 Years to 50 Years (Child, Adult) |
NCT03781479 |
SMA-001 |
|
January 21, 2019 |
December 15, 2019 |
March 15, 2020 |
December 20, 2018 |
January 25, 2019 |
|
- Neurological Institute Carlo Besta
Milano, Lombardy, Italy
|
|
| 90 |
NCT02044029 |
Completed |
Ability of Muscle Imaging and Motor Function Measure (MFM) to Detect Changes in Disease Progression in Ambulant Spinal Muscular Atrophy Patients Compared to Healthy Volunteers. |
- Healthy Volunteer, Muscular Atrophy, Spinal
|
|
Observational
|
|
|
Industry |
- Observational Model: Case Control
- Time Perspective: Prospective
|
- Differences in quantitative muscle MRI based on fat content and T2 values
- Disease progression, assessed through the Motor Function Measure test
- Disease progression, assessed with the 6-minute Walk Test.
- (and 2 more...)
|
38 |
All |
10 Years and older (Child, Adult, Older Adult) |
NCT02044029 |
BE29037 |
|
January 2014 |
July 2015 |
July 2015 |
January 23, 2014 |
November 2, 2016 |
|
|
|
| 91 |
NCT01059240 |
Completed |
SMN Copy Number Distribution in Mali, West Africa |
|
|
Observational
|
|
- National Institute of Neurological Disorders and Stroke (NINDS)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Time Perspective: Prospective
|
- Primary outcome measure for phase 1 is DNA extraction yield of sufficient quantity and quality for SMN genotyping in at least 90% of samples
- Secondary outcome measures for phase 2 are the frequency of SMA carriers (SMN1 deletion heterozygotes) and the SMN1 and SMN2 copy number distribution in Mali.
|
701 |
All |
18 Years and older (Adult, Older Adult) |
NCT01059240 |
999910033
10-N-N033 |
|
January 21, 2010 |
|
May 18, 2017 |
January 29, 2010 |
September 13, 2019 |
|
- University of Bamako, Faculty of Medicine, Pharmacy and Dentistry (FMPOS)
Bamako, Mali
|
|
| 92 |
NCT01663584 |
Withdrawn |
Multi-disease Carrier Screening Test Validation |
- Spinal Muscular Atrophy (SMA)
- Carrier Screening
- Genetic Testing
|
|
Observational
|
|
|
Industry |
|
|
0 |
All |
18 Years and older (Adult, Older Adult) |
NCT01663584 |
Natera014 |
|
August 2012 |
June 2013 |
|
August 13, 2012 |
July 16, 2013 |
|
- Natera, Inc
San Carlos, California, United States
|
|
| 93 |
NCT01984957 |
Completed |
Differential Study of Muscle Transcriptome |
|
|
Interventional
|
Not Applicable |
- Assistance Publique - Hôpitaux de Paris
- Direction Générale de l'Offre de Soins
- Institut National de la Santé Et de la Recherche Médicale, France
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Diagnostic
|
- Gene expression levels
- muscle denervation
- Functional evaluation
|
42 |
All |
18 Years to 75 Years (Adult, Older Adult) |
NCT01984957 |
P110401 |
TRANE |
January 2013 |
January 2015 |
January 2015 |
November 15, 2013 |
March 23, 2015 |
|
- Centre d'évaluation Thérapeutique, Dr LACOMBLEZ
Paris, France
|
|
| 94 |
NCT02855112 |
Unknown † |
Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients |
- Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman]
|
- Biological: Adipose derived mesenchymal stem cell
|
Interventional
|
Phase 1
Phase 2 |
- Tehran University of Medical Sciences
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Changes in action potential of muscles on ElectroMyoGram (EMG) test
- Changes in Motility on Modified Barthel Index Score
|
10 |
All |
5 Months to 12 Months (Child) |
NCT02855112 |
94-01-87-28524 |
|
June 2015 |
December 2016 |
July 2017 |
August 4, 2016 |
August 4, 2016 |
|
- Children's Medical Center
Tehran, Iran, Islamic Republic of
|
|
| 95 |
NCT03988907 |
Recruiting |
A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants |
|
- Drug: Risdiplam
- Drug: Midazolam
|
Interventional
|
Phase 1 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Sequential Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Plasma Concentration of Midazolam Alone and in Combination with Risdiplam
- Plasma Concentration of Midazolam Metabolite Alone and in Combination with Risdiplam
- Percentage of Participants with Adverse Events After Midazolam Administration Alone and in Combination with Risdiplam
- (and 3 more...)
|
40 |
All |
18 Years to 55 Years (Adult) |
NCT03988907 |
BP41361 |
|
June 18, 2019 |
October 18, 2019 |
October 18, 2019 |
June 18, 2019 |
September 19, 2019 |
|
- Covance Research Unit - Dallas
Dallas, Texas, United States
|
|
| 96 |
NCT00568698 |
Completed |
A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients |
|
|
Interventional
|
Phase 1
Phase 2 |
|
Other |
- Allocation: Randomized
- Intervention Model: Single Group Assignment
- Masking: Double
- Primary Purpose: Treatment
|
- Safety: Frequency of Adverse Events/Lab Abnormalities
- Efficacy: Length of survival (LOS) and age of ventilator dependence (AVD)
- Motor Unit Number Estimation (MUNE)
- Biomarker Assays: SMN Protein and SMN mRNA
|
18 |
All |
up to 2 Years (Child) |
NCT00568698 |
SU-11012007-783
78811 |
|
January 2004 |
November 2008 |
November 2008 |
December 6, 2007 |
July 8, 2009 |
|
- Stanford University School of Medicine
Stanford, California, United States
|
|
| 97 |
NCT00568802 |
Completed |
A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients |
|
- Drug: Hydroxyurea
- Drug: Placebo to match hydroxyurea
|
Interventional
|
Phase 1
Phase 2 |
|
Other |
- Allocation: Randomized
- Intervention Model: Single Group Assignment
- Masking: Double
- Primary Purpose: Treatment
|
- Efficacy: Functional Motor Testing, including Gross Motor Function Measure (GMFM) and Timed Motor Tests
- Safety: Frequency of Adverse Events/Lab Abnormalities
- Pulmonary Function Testing
- (and 2 more...)
|
27 |
All |
1 Year to 10 Years (Child) |
NCT00568802 |
SU-11012007-781
79233 |
|
January 2004 |
March 2010 |
March 2010 |
December 6, 2007 |
September 4, 2019 |
|
- Stanford University School of Medicine
Stanford, California, United States
|
|
| 98 |
NCT02633709 |
Completed |
A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers |
|
- Drug: Itraconazole
- Other: Placebo
- Drug: Risdiplam
|
Interventional
|
Phase 1 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double (Participant, Investigator)
- Primary Purpose: Treatment
|
- Percentage of Participants with Adverse Events (AEs)
- Percentage of Participants with Laboratory Test Abnormalities
- Percentage of Participants with Clinically Significant Changes in Safety Measurements, Including Vital Signs and Electrocardiograms (ECGs)
- (and 18 more...)
|
33 |
Male |
18 Years to 45 Years (Adult) |
NCT02633709 |
BP29840
2015-004605-16 |
|
January 7, 2016 |
August 4, 2016 |
August 4, 2016 |
December 17, 2015 |
October 4, 2018 |
|
- Pra International Group B.V
Groningen, Netherlands
|
|
| 99 |
NCT01233817 |
Completed
Has Results |
Progressive Strength Training in Spinal Muscular Atrophy |
|
- Other: Progressive strength training
|
Interventional
|
Not Applicable |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
|
12 |
All |
5 Years to 21 Years (Child, Adult) |
NCT01233817 |
39995 |
|
June 2010 |
June 2013 |
June 2013 |
November 3, 2010 |
June 14, 2017 |
March 12, 2013 |
- University of Utah
Salt Lake City, Utah, United States
|
|
| 100 |
NCT04089566 |
Not yet recruiting
New |
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy |
|
|
Interventional
|
Phase 2
Phase 3 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Sequential Assignment
- Masking: Double (Participant, Care Provider)
- Primary Purpose: Treatment
|
- Part B Infantile-onset SMA: Change from Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Total Score
- Part B Infantile-onset SMA: Percentage of Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestone Responders
- Part B Infantile-onset SMA: Change from Baseline in HINE Section 2 Motor Milestones Total Score
- (and 38 more...)
|
125 |
All |
Child, Adult, Older Adult |
NCT04089566 |
232SM203
2019-002663-10 |
|
February 26, 2020 |
June 13, 2022 |
December 18, 2022 |
September 13, 2019 |
September 13, 2019 |
|
|
|
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