NCT06747273

Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United States

A Phase 1b, Multicenter, Single Dose Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Subjects in the United States

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: Sarepta Therapeutics, Inc. · Industry
• Sex: All
• Age: 4 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to evaluate the safety of SRP-9004.

Conditions

• Limb Girdle Muscular Dystrophy
• Limb Girdle Muscular Dystrophy Type 2D/R3

Interventions

Type	Name	Description
DRUG	SRP-9004	Intravenous (IV) infusion.

Timeline

• Start date: 2025-01-09
• Primary completion: 2025-06-18
• Completion: 2025-06-18
• First posted: 2024-12-24
• Last updated: 2025-09-04

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study