A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)
| Status: | Recruiting |
|---|---|
| Conditions: | Neurology |
| Therapuetic Areas: | Neurology |
| Healthy: | No |
| Age Range: | 4 - 7 |
| Updated: | 1/17/2019 |
| Start Date: | December 22, 2018 |
| End Date: | December 31, 2021 |
| Contact: | Medical Information |
| Email: | clinicaltrials@sarepta.com |
| Phone: | +1-888-727-3782 |
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
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).
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).
Inclusion Criteria:
- 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.
Exclusion Criteria:
- Impaired cardiovascular function on ECHO.
- Prior or ongoing medical condition on physical examination, ECG, or laboratory
findings that could adversely affect subject safety, compromise completion of
follow-up, or impair assessment of study results.
- Exposure to another investigational drug or exon skipping medication within months.
- Exposure to an investigational or commercial gene therapy product.
- Abnormal liver or renal function by protocol-specified criteria
Other inclusion/exclusion criteria apply.
We found this trial at
1
site
700 Childrens Drive
Columbus, Ohio 43205
Columbus, Ohio 43205
(616) 722-2000
Principal Investigator: Jerry Mendell, MD
Phone: 614-722-2650
Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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