A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Patients With Duchenne Muscular Dystrophy (EMBARK)

JRCT ID: jRCT2053210141

Registered date:25/12/2021

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Duchenne Muscular Dystrophy
• Date of first enrollment: 20/07/2022
• Target sample size: 6
• Countries of recruitment: US,Japan,UK,Japan,Italy,Japan,Germany,Japan,Belgium,Japan,France,Japan,Spain,Japan,Hong Kong,Japan,Taiwan,Japan
• Study type: Interventional
• Intervention(s): 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 Year 2. Participants randomized to placebo will have the opportunity to receive a single IV infusion of SRP-9001 at Year 2.

Outcome(s)

• Primary Outcome: Part 1: Change From Baseline in NSAA Total Score at Week 52

Secondary Outcome

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)

Key inclusion & exclusion criteria

• Age minimum: >= 4age old
• Age maximum: < 8age old
• Gender: Male

Include 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.

Exclude 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.

Related Information

• Primary Sponsor: O Malley Patrick
• Secondary ID(s): NCT05096221