A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 (Delandistrogene Moxeparvovec) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

JRCT ID: jRCT2033230274

Registered date:02/08/2023

Basic Information

• Recruitment status: Pending
• Health condition(s) or Problem(s) studied: Duchenne Muscular Dystrophy
• Date of first enrollment: 06/09/2023
• Target sample size: 4
• Countries of recruitment: US,Japan,UK,Japan,Italy,Japan,Germany,Japan,Belgium,Japan,France,Japan,Spain,Japan,Australia,Japan,Israel,Japan,Sweden,Japan
• Study type: Interventional
• Intervention(s): Participants will receive single IV infusion of SRP-9001 or a matching placebo on Day 1. Then, participants will receive a single IV infusion of matching placebo or SRP-9001 at approximately 72 weeks.

Outcome(s)

• Primary Outcome: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72

Secondary Outcome

- Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72
- Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72
- Quantity of SRP-9001 Protein Expression at Week 12 as Measured by Western Blot
- Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72
- (For Cohort 2 Only) Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72
- Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72
- Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE)

Key inclusion & exclusion criteria

• Age minimum: >= 8age old
• Age maximum: < 18age old
• Gender: Male

Include criteria

1.Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
2.Cohort 1 only: Non-ambulatory per protocol specified criteria.
3.Cohort 2 only: Ambulatory per protocol specified criteria and >=8 to <18 years of age at the time of Screening.
4.Ability to cooperate with motor assessment testing.
5.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).
6.Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) antibody titers are not elevated as per protocol-specified requirements.
7.A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive).

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.

Other exclusion criteria could apply.

Related Information

• Primary Sponsor: Patrick O Malley
• Secondary ID(s): NCT05881408,2020-002372-13