Status:
COMPLETED
Phase I/II Study of SRP-4053 in DMD Patients
Lead Sponsor:
Sarepta Therapeutics, Inc.
Collaborating Sponsors:
Institut de Myologie, France
Consultants for Research in Imaging and Spectroscopy
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
6-15 years
Phase:
PHASE1
PHASE2
Brief Summary
This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions amenabl...
Detailed Description
Part 1: Randomized, placebo-controlled dose-titration to assess safety, tolerability and pharmacokinetics of 4 dose levels of SRP-4053 in genotypically-confirmed DMD patients with deletions amenable t...
Eligibility Criteria
Inclusion
- Diagnosed with DMD, genotypically confirmed.
- Intact right and left biceps muscles or an alternative upper arm muscle group.
- Stable pulmonary and cardiac function.
- Minimum performance on 6MWT, North Star Ambulatory Assessment, and rise (Gowers) test as specified in the study protocol.
- On a stable dose of corticosteroids for at least 6 months.
Exclusion
- Previous treatment with the experimental agents BMN-195 (SMT C1100) or PRO053.
- Current or previous treatment with any other experimental treatments within 12 weeks prior to study entry.
- Major surgery within the last 3 months.
- Presence of other clinically significant illness.
- Major change in physical therapy regime within the last 3 months.
- Other inclusion and exclusion criteria may apply.
Key Trial Info
Start Date :
January 13 2015
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 25 2019
Estimated Enrollment :
39 Patients enrolled
Trial Details
Trial ID
NCT02310906
Start Date
January 13 2015
End Date
March 25 2019
Last Update
October 19 2020
Active Locations (5)
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1
Boston Children's Hospital
Boston, Massachusetts, United States, 02116
2
Institute de Myologie
Paris, France, 75013
3
Policlinico Universitario A Gemelli
Rome, Italy, 00168
4
Great Ormond Street Hospital for Children NHS Foundation Trust
London, United Kingdom, WC1N 3JH