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Spinraza in Adult Spinal Muscular Atrophy (SAS)

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ClinicalTrials.gov Identifier: NCT03709784
Recruitment Status : Recruiting
First Posted : October 17, 2018
Last Update Posted : August 11, 2020
Sponsor:
Information provided by (Responsible Party):
Washington University School of Medicine

Tracking Information
First Submitted Date October 15, 2018
First Posted Date October 17, 2018
Last Update Posted Date August 11, 2020
Actual Study Start Date August 16, 2018
Estimated Primary Completion Date January 30, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: October 15, 2018)
  • Change from baseline in the 6-Minute Walk Test (6MWT) for ambulatory SMA patients [ Time Frame: 30 months ]
    Assess effectiveness of SPINRAZA® (nusinersen) treatment on mobility and ambulation in ambulatory adult SMA patients, comparing changes in total distance walked in in six minutes from baseline until end of treatment at 30 months..
  • Change from baseline in Revised Upper Limb Module (RULM) for weak ambulatory and non-ambulatory SMA patients [ Time Frame: 30 months ]
    Assess the effectiveness of SPINRAZA® (nusinersen) treatment on upper extremity function in ambulatory and non-ambulatory adult SMA patients, comparing change in RULM score from baseline until end of treatment at 30 months.
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Spinraza in Adult Spinal Muscular Atrophy
Official Title A Prospective, Multi-center, Observational Study of the Safety, Tolerability and Effectiveness of SPINRAZA® (Nusinersen) in Adult Patients With Spinal Muscular Atrophy
Brief Summary This is a longitudinal, observational study of adult patients with genetically confirmed chromosome 5q SMA to examine the safety, tolerability, and effectiveness of SPINRAZA® (nusinersen) for up to 30 months.
Detailed Description This is a prospective, longitudinal, multi-center, observational study designed to evaluate the safety, tolerability, and effectiveness of SPINRAZA® (nusinersen) in ambulatory and non-ambulatory adult patients with SMA. Subjects with SMA II/III that are 18 years to 70 years of age who are planning to initiate treatment with SPINRAZA® (nusinersen) as part of their clinical care plan will be enrolled in this study. This study does not provide SPINRAZA® (nusinersen) or cover costs associated with standard clinical care.These patients will be treated by their respective physicians according to standard clinical practice. Study visits, some of which including standardized assessments of strength and function, will occur at baseline, day 15 after treatment initiation, day 30, day 60, and then 4-month intervals through month 30.
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
5cc of Cerebral Spinal Fluid (CSF) is collected and typically discarded as part of the typical clinical encounter when SPINRAZA® (nusinersen) is administered. This 5cc of CSF will be collected for research purposes at each dosing visit. Blood samples, including whole blood, serum, and plasma (3 teaspoons total) will be collected within seven days of standard of care SPINRAZA® (nusinersen) administration at dose #1, 2, 4, 5, 7, 9 and 11.
Sampling Method Non-Probability Sample
Study Population Subjects with SMA that are 18 years to 70 years of age who are planning to initiate treatment with SPINRAZA® (nusinersen) as part of their clinical care plan.
Condition
  • Spinal Muscular Atrophy
  • Spinal Muscular Atrophy Type II
  • Spinal Muscular Atrophy Type 3
Intervention Other: Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care
This is an observational study of adult patients with SMA to examine the safety, tolerability, and effectiveness of SPINRAZA® (nusinersen) for up to 30 months.
Study Groups/Cohorts Not Provided
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: October 15, 2018)
73
Original Estimated Enrollment Same as current
Estimated Study Completion Date January 30, 2022
Estimated Primary Completion Date January 30, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  1. Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations.
  2. Males and females with SMA type II or type III, aged 18 to 70 years at the time of enrollment.
  3. Genetic documentation of 5Q SMA homozygous gene deletion, mutation, or compound heterozygote.
  4. Are treatment naïve for SPINRAZA® (nusinersen).
  5. Have been prescribed SPINRAZA® (nusinersen) by the treating physician as part of their clinical care for SMA following the FDA approved prescribing information guidelines as follows: dose level (12 mg), dosing schedule (3 loading doses administered at 14-day intervals, and the fourth loading dose administered 30 days after the third dose and subsequent maintenance doses administered every 4 months) and safety lab monitoring (CBC, PT, INR, PTT, UA) done prior to each dose administration.
  6. Believed to be able to complete all study procedures, measurements and visits.
  7. Estimated life expectancy at least 30 months from first dosing, in the opinion of the Investigator.
  8. Revised upper limb module (RULM) score ≥ 4 (more than marginal upper extremity function/strength.
  9. Must meet either Group 1 or Group 2 criteria.

For Group 1 subjects:

  1. May be ambulatory or non-ambulatory (defined as being wheelchair reliant at least 75% of time and unable to walk at least 10 meters without assistance).
  2. RULM score of 4-34, inclusive.

For Group 2 subjects:

  1. Ability to walk at least 10 meters without assistance (i.e., four point walking aid).
  2. Be free of major orthopedic deformities that limit ambulation.
  3. An ambulatory subject can qualify for both Group 1 and Group 2 if the RULM score is ≤ 34.

Exclusion Criteria:

  1. Revised upper limb score ≤ 3.
  2. Respiratory insufficiency, defined by the medical necessity for invasive or noninvasive ventilation for >16 hours during a 24-hour period, at screening.
  3. Hospitalization for major medical event including: surgery (i.e., scoliosis surgery, other surgery), cardiac event, pulmonary event, or other major medical problem within 2 months of screening or planned major surgical procedure likely to impact the clinical assessments during the duration of the study. Outpatient surgical procedure (i.e., placement of feeding tube) is not considered an exclusionary major medical event.
  4. Presence of a symptomatic severe active infection or illness during the screening period that is likely to impact the performance on the clinical assessments.
  5. Prior exposure to SPINRAZA® (nusinersen).
  6. Prior disorder, injury (e.g., upper or lower limb fracture) or surgical procedure which impacts the subject's ability to perform any of the outcome measure testing required in the protocol and from which the subject has not fully recovered or achieved a stable baseline.
  7. Treatment with an investigational drug (e.g., oral albuterol/salbutamol, riluzole, carnitine, creatine, sodium phenylbutyrate, etc.), biological agent, or device within 1-month of Screening or 5 half-lives of study agent, whichever is longer.Patients using intrathecal drug delivery devices, including investigational devices with an active IDE designation in the United States, may be eligible but require Study PI approval prior to enrollment.
  8. Any history of exposure to gene therapy, antisense oligonucleotide therapy, or cell transplantation that was intended for the treatment of SMA.
  9. Ongoing medical condition that according to the Clinical Center Investigator would interfere with the conduct and assessments of the study. Examples are medical disability (e.g., wasting or cachexia, severe anemia, etc.) that would interfere with the assessment of safety or would compromise the ability of the subject to undergo study procedures.
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years to 70 Years   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Craig Zaidman, MD 314-273-2630 SAStrial@wustl.edu
Listed Location Countries Canada,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03709784
Other Study ID Numbers 201805187
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Product Manufactured in and Exported from the U.S.: Yes
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Washington University School of Medicine
Study Sponsor Washington University School of Medicine
Collaborators Not Provided
Investigators
Principal Investigator: Craig Zaidman, MD Washington University School of Medicine
PRS Account Washington University School of Medicine
Verification Date August 2020