Overview

This trial is active, not recruiting.

Condition duchenne muscular dystrophy
Treatments placebo, srp-4053
Phase phase 1/phase 2
Sponsor Sarepta Therapeutics
Collaborator Institut de Myologie, France
Start date December 2014
End date May 2019
Trial size 39 participants
Trial identifier NCT02310906, 4053-101

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 amenable to exon 53 skipping.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator, outcomes assessor)
Primary purpose treatment
Arm
(Experimental)
Approximately 8 genotypically confirmed DMD patients amenable to exon 53 skipping
srp-4053
(Placebo Comparator)
Approximately 4 genotypically confirmed DMD patients amenable to exon 53 skipping
placebo
srp-4053
(Experimental)
Approximately 12 genotypically confirmed DMD patients amenable to exon 53 skipping
srp-4053
(No Intervention)
Approximately 24 genotypically confirmed DMD patients with deletions not amenable to exon 53 skipping

Primary Outcomes

Measure
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)

Secondary Outcomes

Measure
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)

Eligibility Criteria

Male participants from 6 years up to 15 years old.

Inclusion Criteria: - 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 Criteria: - 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.

Additional Information

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.
Trial information was received from ClinicalTrials.gov and was last updated in July 2016.
Information provided to ClinicalTrials.gov by Sarepta Therapeutics.