NCT06747273 (DISCOVERY) is a Phase 1 clinical trial of SRP-9004 in Limb Girdle Muscular Dystrophy, sponsored by Sarepta Therapeutics, Inc..

Who is sponsoring NCT06747273?

NCT06747273 is sponsored by Sarepta Therapeutics, Inc..

What drugs are being tested in NCT06747273?

Interventions in NCT06747273: SRP-9004.

What condition does NCT06747273 study?

NCT06747273 studies Limb Girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type 2D/R3.

Is NCT06747273 still recruiting?

NCT06747273 is currently terminated. Last updated 4 September 2025.

Last reviewed 2025-09-04 · How we verify

NCT06747273: DISCOVERY

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

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

Terminated Phase 1 Last updated 4 September 2025

What this trial tests

Phase 1 trial testing SRP-9004 in Limb Girdle Muscular Dystrophy in 4 participants. Terminated before completion.

Timeline

9 January 2025

Primary endpoint
18 June 2025

18 June 2025

Quick facts

Lead sponsor	Sarepta Therapeutics, Inc.
Phase	Phase 1
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	4
Start date	9 January 2025
Primary completion	18 June 2025
Estimated completion	18 June 2025
Sites	2 locations across United States

Drugs / interventions tested

SRP-9004 — full drug profile →

Conditions studied

Limb Girdle Muscular Dystrophy — all drugs for Limb Girdle Muscular Dystrophy →
Limb Girdle Muscular Dystrophy Type 2D/R3 — all drugs for Limb Girdle Muscular Dystrophy Type 2D/R3 →

Sponsor

Sarepta Therapeutics, Inc. — full company profile →

Who can join

4 and older, any sex, with Limb Girdle Muscular Dystrophy or Limb Girdle Muscular Dystrophy Type 2D/R3. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

Bridging science and hope: the evolving story of gene therapy for neuromuscular diseases.
Wein N, Barthélémy F. · · 2026 · PMID 42058150 · DOI 10.3389/fcell.2026.1765367
Deaths in gene therapy of Duchenne muscular dystrophy and other diseases: Underlying mechanisms and mitigating strategies.
Duan D, Herzog RW. · · 2026 · PMID 41502088 · DOI 10.1016/j.ymthe.2025.12.067
CRISPR-Cas editing technologies for viral-mediated gene therapies of human diseases: Mechanisms, progress, and challenges.
Kantor B, Duke L, Bhide PG. · · 2026 · PMID 41496894 · DOI 10.1016/j.omtn.2025.102786
Sarcoglycanopathies: From clinical diagnosis to new promising therapies.
Borland H, Diaz-Manera J. · · 2025 · PMID 40129306 · DOI 10.1177/22143602251324855

Verify or expand the search:

Other trials of SRP-9004

Trials testing the same drug.

NCT01976091 — A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb · Phase 1, PHASE2 · completed

Other recruiting trials for Limb Girdle Muscular Dystrophy

Currently open trials in the same condition.

NCT06390566 — Evolution of the Functional and Muscular State of Patients With Muscular Dystrophy 2A Belts · active not recruiting
NCT06378203 — Rehabilitation in Muscular Dystrophies From the Hospital Facility to the Home: Pilot Project [RIMUDI] · NA · recruiting
NCT05876780 — A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Am · Phase 1 · active not recruiting
NCT05102799 — MRI-phenotyping of Patients With Pathogenic Anoctamin 5 Variants · recruiting
NCT05206617 — 3 Year Follow up on ANO5 Patients · active not recruiting

Other Sarepta Therapeutics, Inc. trials

Trials by the same sponsor.

NCT07536061 — A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease · Phase 1 · not yet recruiting
NCT07542314 — Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a · Phase 4 · not yet recruiting
NCT06952686 — A Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult Participants · Phase 3 · withdrawn
NCT06597656 — A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therape · Phase 1 · terminated
NCT06270719 — An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscul · enrolling by invitation

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT06747273 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Sarepta Therapeutics, Inc.
Last refreshed: 4 September 2025

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT06747273.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing