Phase I/II Study of SRP-4053 in DMD Patients
This trial is active, not recruiting.
|Condition||duchenne muscular dystrophy|
|Phase||phase 1/phase 2|
|Collaborator||Institut de Myologie, France|
|Start date||December 2014|
|End date||May 2019|
|Trial size||39 participants|
|Trial identifier||NCT02310906, 4053-101|
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 amenable to exon 53 skipping.
|United States||No locations recruiting|
|Other countries||No locations recruiting|
|Paris, France||Institute de Myologie||no longer recruiting|
|Rome, Italy||Policlinico Universitario A Gemelli||no longer recruiting|
|London, United Kingdom||Great Ormond Street Hospital for Children NHS Foundation Trust||no longer recruiting|
|Newcastle, United Kingdom||Newcastle University Hospital||no longer recruiting|
|Endpoint classification||safety/efficacy study|
|Intervention model||parallel assignment|
|Masking||double blind (subject, caregiver, investigator, outcomes assessor)|
Incidence of Adverse Events
time frame: approximately 12 weeks (Part 1)
Change in 6-Minute Walk Test (6MWT) from Baseline
time frame: 144 weeks (Part 2)
Percentage of dystrophin-positive fibers
time frame: 48 weeks (Part 2)
Drug concentration in plasma
time frame: Approximately 12 weeks (Part 1)
Maximum inspiratory pressure (MIP) % predicted, maximum expiratory pressure (MEP) % predicted
time frame: 144 weeks (Part 2)
Male participants from 6 years up to 15 years old.
- 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.
- 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.
|Official title||A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping|
|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 to exon 53 skipping. Part 2: Open-label evaluation of SRP-4053 in patients from Part 1, along with newly enrolled DMD patients with deletions amenable to exon 53 skipping, compared to untreated control DMD patients with deletions not amenable to exon 53 skipping. Safety, including adverse event monitoring and routine laboratory assessments, will be followed on an ongoing basis for all patients. Clinical efficacy, including functional tests such as the six-minute walk test (6MWT), will be assessed at regularly scheduled study visits. Patients in the treated groups will undergo one baseline and one follow-up muscle biopsy. Patients in the untreated control group will not undergo biopsies and will follow an abbreviated schedule of study assessments.|
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