Phase 1/2 study of SRP-9001 Micro-Dystrophin Gene Transfer in people with Duchenne

A 48-Week, Randomized, Double-Blind, Placebo-Controlled, Systemic, Gene-Delivery Clinical Trial for Duchenne Muscular Dystrophy Using SRP-9001 With a 96-Week Extension

Questions and Answers

What stage is this research?

This trial is a Phase l/ll study that is currently recruiting participants in the United States.

What is the goal or purpose of this study?

The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD patients by measuring biological and clinical endpoints in two parts: a 48-week randomized, double-blinded, placebo-controlled period (Part 1), and a 96-week, double-blinded extension period (Part 2).

Who is funding this study?

The study is sponsored by Sarepta Therapeutics, Inc.

Who is eligible to participate in this study?

Key inclusion criteria include but are not limited to:

  • Established clinical diagnosis of DMD and documented dystrophin gene mutation of DMD phenotype.
  • Indication of symptomatic muscular dystrophy by protocol-specified criteria.
  • Ability to cooperate with motor assessment testing.
  • Stable dose equivalent of oral corticosteroids for at least 12 weeks.
  • Male, 4-7 years of age, inclusive

What do I have to do if I decide to participate in this study?

After providing informed consent, patients will be evaluated for eligibility. Enrolled patients will perform functional tests, undergo laboratory work, and muscle biopsies throughout the course of the study.

Where does this study take place?

This study currently takes place at 1 clinical site in the United States. To view the participating study site, visit www.clinicaltrials.gov (Identifier NCT03769116).

How many visits to the study site are necessary?

25

Can any visits be done locally?

No

Is there any funding to help pay for travel?

Yes, costs associated with travel and parking are reimbursed.

Will I get paid for participating in this study?

No

Why should I consider participating in this study?

While no personal benefit can ever be guaranteed by participation in a clinical trial, there are other benefits, including allowing you to play an active role in your own health care (or that of your child), gaining access to new research treatments before they are widely available and having access to medical specialists that are normally not available to you or your child, and helping others by contributing to the better understanding of DMD.

Where can I learn more about this study?

You can learn more about this study at www.ClinicalTrials.gov (NCT# 03769116).

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