Overview

This trial is active, not recruiting.

Condition duchenne muscular dystrophy
Treatments srp-4045, placebo
Phase phase 1/phase 2
Sponsor Sarepta Therapeutics
Start date October 2015
End date March 2016
Trial size 12 participants
Trial identifier NCT02530905, 4045-101

Summary

This is a first-in-human dose-titration and open-label extension study to assess safety, tolerability, and pharmacokinetics of SRP-4045 in advanced-stage Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 45 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 45 skipping
srp-4045
(Placebo Comparator)
Approximately 4 genotypically confirmed DMD patients amenable to exon 45 skipping.
placebo
(Experimental)
Approximately 12 DMD patients who completed the double-blind dose titration part of the study.
srp-4045

Primary Outcomes

Measure
Incidence of adverse events
time frame: Approximately 12 Weeks (double-blind dose titration)

Secondary Outcomes

Measure
Drug concentration in plasma
time frame: Approximately 12 Weeks (double-blind dose titration)

Eligibility Criteria

Male participants from 7 years up to 21 years old.

Inclusion Criteria: - Genotypically confirmed DMD (amenable to exon 45 skipping). - Stable cardiac and pulmonary function. - Limited or no ambulation. - On a stable dose of oral corticosteroids for at least 24 weeks OR has not received corticosteroids for at least 24 weeks. Exclusion Criteria: - Current or previous treatment with the experimental agents SMT C1100 (BMN-195) or PRO045. - Other experimental treatment in the past 12 weeks. - If on cardiac medication, must be on a stable dose for the past 12 weeks. - Major surgery within the past 3 months. Other inclusion/exclusion criteria apply.

Additional Information

Official title A Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study Followed by an Open-Label Safety and Efficacy Evaluation of SRP-4045 in Advanced-Stage Patients With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping
Description This is a randomized, placebo-controlled dose-titration study to assess safety, tolerability, and pharmacokinetics of 4 dose levels of SRP-4045 in genotypically confirmed advanced-stage DMD patients with deletions amenable to exon 45 skipping. After completion of the dose-titration portion of the study and SRP-4045 is determined to be safe, all patients will be evaluated on open-label SRP-4045 for the duration of the study. Safety, including adverse event monitoring, routine laboratory assessments, and cardiac testing will be monitored through the duration of the dose-titration and open-label portions of the study. Clinical efficacy will be assessed at regularly scheduled study visits via quality of life questionnaires and tests of pulmonary and upper extremity function through the duration of the dose-titration and open-label portions of the trial.
Trial information was received from ClinicalTrials.gov and was last updated in April 2016.
Information provided to ClinicalTrials.gov by Sarepta Therapeutics.