SRP-9004 for Limb-Girdle Muscular Dystrophy
(DISCOVERY Trial)

Recruiting in Palo Alto (17 mi)

+1 other location

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: Sarepta Therapeutics, Inc.

No Placebo Group

Trial Summary

What is the purpose of this trial?The primary objective of this study is to evaluate the safety of SRP-9004.

Eligibility Criteria

This trial is for individuals aged 4 to 20 with Limb Girdle Muscular Dystrophy Type 2D/R3 who weigh less than 70 kg and have specific genetic mutations. Participants must be able to perform muscle testing, with ambulatory participants being able to walk unaided and non-ambulatory ones having a certain level of upper limb function.

Inclusion Criteria

Participants must be able to cooperate with muscle testing

I weigh 70 kg or less.

Ambulatory participants must be able to walk without assistive aid, have a 10MWR time of less than 30 seconds, and NSAD total score of at least 25. Non-ambulatory participants must have a 10MWR time of 30 seconds or more or be unable to perform, and a PUL 2.0 entry scale score of at least 3

+3 more

Exclusion Criteria

Any other clinically significant illness, including neuromuscular (other than limb girdle muscular dystrophy type 2D/R3 [LGMD2D/R3]), that in the opinion of the Investigator might compromise the participant's ability to comply with the protocol required testing or procedures or compromise the participant's wellbeing, safety, or clinical interpretability

My lung function is very low or I need help breathing at night.

My heart's pumping ability is weak.

Participant Groups

The study is focused on the safety and tolerability of SRP-9004, which is administered through systemic infusion. The main goal is to see how well patients with LGMD2D/R3 handle this treatment over time.

1Treatment groups

Experimental Treatment

Group I: SRP-9004Experimental Treatment1 Intervention

Participants will receive a single dose of SRP-9004 on Day 1.