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Trial record 1 of 2 for:    SRP-5051 | Phase 2
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Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM)

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ClinicalTrials.gov Identifier: NCT04004065
Recruitment Status : Recruiting
First Posted : July 1, 2019
Last Update Posted : January 7, 2021
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Study Description
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Brief Summary:
This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose [MAD]) will be conducted to evaluate the safety and tolerability of SRP-5051 at multiple ascending dose levels to determine the maximum tolerated dose (MTD), and 2) Part B (Dose Expansion) will be conducted to evaluate SRP-5051 administered at the MTD determined in Part A or, if applicable, at a second dose introduced in Part B. Patients enrolling in Part B will be those who completed Part A as well as additional expansion cohort patients who will enroll at the beginning of Part B.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: SRP-5051 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 70 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, Then Dose Expansion, in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
Actual Study Start Date : June 26, 2019
Estimated Primary Completion Date : May 31, 2022
Estimated Study Completion Date : May 31, 2022

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Part A: SRP-5051
Patients will be sequentially assigned to receive 1 of the 5 escalating dose levels of SRP-5051, monthly, via intravenous (IV) infusion for at least 12 weeks during Part A. Once the maximum tolerated dose (MTD) has been determined in Part A, all patients who have completed Part A will transition to Part B.
 Drug: SRP-5051
SRP-5051 injection, for IV use
Experimental: Part B: SRP-5051
Patients will receive SRP-5051 at the MTD determined in Part A, or if applicable, at a second dose introduced in Part B, monthly, via IV infusion, for 24 weeks. This includes the patients who roll over from Part A, as well as expansion cohort patients who will enroll in the study at the beginning of Part B.
 Drug: SRP-5051
SRP-5051 injection, for IV use


Outcome Measures
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Primary Outcome Measures :
  1. Part A: Incidence of Adverse Events (AEs) [ Time Frame: Part A: approximately 68 weeks ]
    Incidence of adverse events includes clinically significant laboratory abnormalities.

  2. Part B: Change From Baseline in Dystrophin Protein Level [ Time Frame: Baseline, Week 12 or Week 24 ]

Secondary Outcome Measures :
  1. Part A: Pharmacokinetics (PK): Plasma Concentration of SRP-5051 [ Time Frame: Pre-dose and at multiple time points (up to 32 hours) after end of infusion ]
  2. Part A: PK: Urine Concentration of SRP-5051 [ Time Frame: Pre-dose and at multiple time periods (up to 48 hours) after end of infusion ]
  3. Part B: Change From Baseline in Exon-Skipping Levels [ Time Frame: Baseline, Week 12 or Week 24 ]
  4. Part B: Incidence of Adverse Events (AEs) [ Time Frame: Part B: approximately 44 weeks ]
    Incidence of adverse events includes clinically significant laboratory abnormalities.

  5. Part B: Change From Baseline in Forced Vital Capacity (FVC) (percent predicted) [ Time Frame: Baseline, Week 12 or Week 24 ]
  6. Part B: Change From Baseline in the North Star Ambulatory Assessment (NSAA) [ Time Frame: Baseline, Week 12 or Week 24 ]
  7. Part B: Change From Baseline in the Performance of Upper Limb (PUL) Scores [ Time Frame: Baseline, Week 12 or Week 24 ]
  8. Part B: Change From Baseline in the Brooke Upper Extremity Scale score (Brooke score) [ Time Frame: Baseline, Week 12 or Week 24 ]
  9. Part B: PK: Plasma Concentration of SRP-5051 [ Time Frame: Part B predose and at multiple timepoints (up to 24 hours) after end of infusion ]

Eligibility Criteria
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Ages Eligible for Study:   4 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Has a genetic diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51-skipping treatment.
  • Has been on a stable dose of oral corticosteroids for at least 12 weeks prior to study drug administration, or has not received corticosteroids for at least 12 weeks prior to study drug administration.
  • Part A age group (ages 7 to 21)
  • Part B age group (ages 4 to 21)

Exclusion Criteria:

  • Has a left ventricular ejection fraction (LVEF) less than (<) 40.0 percent (%) based on an echocardiogram (ECHO) performed within 12 weeks prior to Screening or at the Screening visit.
  • Has a FVC < 40.0% of predicted value within 12 weeks prior to Screening or at Screening.
  • Initiation or change of dosing (except for modifications to accommodate changes in weight or changes in standard of care) within 12 weeks prior to Screening for any of the following: angiotensin-converting enzyme inhibitors, angiotensin receptor-blocking agents, β-blockers, or potassium.
  • Initiation or change of dosing within 12 weeks prior to Screening for over-the-counter preparations, such as herbal/nonherbal supplements, vitamins, minerals, and homeopathic preparations.
  • Treatment with any exon 51-skipping therapy within 12 weeks prior to Screening, or with any experimental gene therapy for the treatment of DMD at any time.

Other inclusion/exclusion criteria apply

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04004065


Contacts
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Contact: Medical Information +1 800-690-2003 clinicaltrials@sarepta.com

Locations
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United States, Florida
Northwest Florida Clinical Research Group, LLC Recruiting
Gulf Breeze, Florida, United States, 32561
Contact: Genei Bougher, APRN    850-934-1299    info@nwflcrg.com   
Principal Investigator: Weldon Mauney, MD         
United States, Georgia
Rare Disease Research, LLC Recruiting
Atlanta, Georgia, United States, 30318
Contact    678-883-6897    Info@rarediseaseresearch.com   
Principal Investigator: Han Phan, MD         
United States, Iowa
University of Iowa Hospitals and Clinics Recruiting
Iowa City, Iowa, United States, 52242
Contact: Bryn Miller    319-335-5836    bryn-miller@uiowa.edu   
Principal Investigator: Katherine Mathews, MD         
United States, Pennsylvania
Children's Hospital of Pittsburgh of UPMC Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Jennifer Monahan    412-692-5176    jennifer.monahan@chp.edu   
Principal Investigator: Hoda Abdel-Hamid, MD         
United States, Texas
Austin Neuromuscular Center Active, not recruiting
Austin, Texas, United States, 78756
Belgium
Universitair Ziekenhuis Gent Recruiting
Gent, Belgium, 9000
Contact: Elke De Vos       elke.devos@uzgent.be   
Principal Investigator: Nicolas Deconinck, MD         
Canada, Ontario
Children's Hospital - London Health Sciences Centre (LHSC) Recruiting
London, Ontario, Canada, N6A 5W9
Contact: Chelsea Huisman       Chelsea.Huisman@lhsc.on.ca   
Principal Investigator: Craig Campbell, MD         
Spain
Hospital Sant Joan de Déu. U.B. Recruiting
Barcelona, Spain, 08950
Contact: Andres Nascimento Osorio       anascimento@hsjdbcn.org   
Principal Investigator: Andres Nascimento Osorio, MD         
Hospital Universitari I Politecnic La Fe de Valencia Recruiting
Valencia, Spain, 46026
Contact: Juan Jesús Vilchez Padilla       vilchez_jje@gva.es   
Principal Investigator: Juan Jesús Vilchez Padilla, MD         
United Kingdom
Alder Hey Children's NHS Foundation Trust Recruiting
Liverpool, United Kingdom, L12 2AP
Contact: Clinical Research Facility    +44 151-252-5115    neuromuscularresearch@alderhey.nhs.uk   
Principal Investigator: Stephan Spinty, MD         
Sponsors and Collaborators
Sarepta Therapeutics, Inc.
Investigators
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Study Director: Medical Director Sarepta Therapeutics, Inc.
More Information
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Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04004065    
Other Study ID Numbers: 5051-201
2019-000601-77 ( EudraCT Number )
First Posted: July 1, 2019    Key Record Dates
Last Update Posted: January 7, 2021
Last Verified: January 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sarepta Therapeutics, Inc.:
DMD
Duchenne
Dystrophy
Dystrophin
Exon Skipping
Ambulatory
 Duchenne Muscular Dystrophy
Exon 51
Nonambulatory
Pediatric
Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO)
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked