Duchenne Muscular Dystrophy
Conditions
Interventions
Subjects will receive a single intravenous (IV) infusion of SRP-9001 or matching placebo on Day 1. Then, participants randomized to SRP-9001 will receive a single IV infusion of matching placebo at Y
Sponsors
O Malley Patrick
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1.Is ambulatory and from 4 to under 8 years of age at time of randomization. 2.Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing. 3.Ability to cooperate with motor assessment testing. 4.Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight). 5.rAAVrh74 antibody titers are not elevated as per protocol-specified requirements. 6.Other inclusion criteria could apply.
Exclusion criteria
Exclusion criteria: 1.Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits. 2.Abnormality in protocol-specified diagnostic evaluations or laboratory tests. 3.Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer. 4.Other inclusion or exclusion criteria could apply.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part 1: Change From Baseline in NSAA Total Score at Week 52 | — |
Secondary
| Measure | Time frame |
|---|---|
| Part 1: - Quantity of Micro-Dystrophin Protein Expression at Week 12 as Measured by Western Blot, in a Subset of Participants - Change From Baseline in Time to Rise From the Floor, Time to Complete 100 and 10 meter Walk/Run, and the Timed Stair Ascend 4 Steps Test at Week 52 - Change From Baseline in Stride Velocity 95th Centile (SV95C) Measured by a Wearable Device - Change from Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score per Domain at Week 52 - Number of Skills Gained or Improved at Week 52 as Measured by the NSAA (Baseline, up to Week 52) - Number of Participants with a Treatment Emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI) (Baseline up to Week 52) | — |
Countries
Belgium, France, Germany, Hong Kong, Italy, Japan, Spain, Taiwan, UK, US
Contacts
Public ContactChikako Rosario
Parexel International
Outcome results
None listed