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

NCT06747273 | Terminated Phase 1 DMC FDA Drug

• 1 other identifier: SRP-9004-102
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 2

Brief 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

Keywords

Limb Girdle Muscular Dystrophy Type 2D/R3; SRP-9004

Outcome Measures

Primary Outcomes (1)

• Number of Participants with Treatment-emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESI) and Treatment-emergent Serious Adverse Events (SAEs)

  Up to 60 months

Secondary Outcomes (19)

• Change from Baseline Through Day 60 in Quantity of Alpha-sarcoglycan (α-SG) Protein Expression

  Baseline, Day 60

• Change from Baseline Through Month 24 in Quantity of α-SG Protein Expression

  Baseline, Month 24

• Change from Baseline Through Month 60 in North Star Assessment for Dysferlinopathy (NSAD) Total Score

  Baseline, Month 60

• Change from Baseline Through Month 60 in Performance of the Upper Limb (PUL) Version 2.0 Total Score

  Baseline, Month 60

• Change from Baseline Through Month 60 in Time to Rise From Floor

  Baseline, Month 60

Study Arms (1)

SRP-9004

EXPERIMENTAL

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

Drug: SRP-9004

Interventions

SRP-9004 DRUG

Intravenous (IV) infusion.

SRP-9004

Eligibility Criteria

• Age: 4 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Ambulatory participants, defined as able to walk without assistive aid, 10MWR \<30 seconds, and NSAD total score ≥25; non-ambulatory participant, defined as 10MWR ≥30 seconds or unable to perform, and PUL 2.0 entry scale score ≥3.
• Ambulatory participants must be 4 to 20 years of age and the non-ambulatory participant must be ≥4 years of age.
• All participants must be ≤70 kilograms
• Possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic α-SG deoxyribonucleic acid (DNA) gene mutations as documented prior to screening.
• Able to cooperate with muscle testing.
• Participants must have adeno-associated virus (AAV) serotype Rh74 (rh74) antibody titers \<1:400 (that is, not elevated) as determined by an enzyme-linked immunosorbent assay (ELISA).

You may not qualify if:

• Left ventricular ejection fraction \<40% or clinical signs and/or symptoms of cardiomyopathy
• FVC ≤40% of predicted value and/or requirement for nocturnal ventilation
• 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.

Sponsors & Collaborators

• Sarepta Therapeutics, Inc.: lead

Study Sites (2)

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Children's Hospital of the King's Daughters

Norfolk, Virginia, 23510, United States

Location

MeSH Terms

Conditions

Muscular Dystrophies, Limb-Girdle

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 18, 2024
• First Posted: December 24, 2024
• Study Start: January 9, 2025
• Primary Completion: June 18, 2025
• Study Completion: June 18, 2025
• Last Updated: September 4, 2025

Record last verified: 2025-08

Locations

US (2)