Who is sponsoring NCT04626674?

NCT04626674 is sponsored by Sarepta Therapeutics, Inc..

What drugs are being tested in NCT04626674?

Interventions in NCT04626674: delandistrogene moxeparvovec.

What condition does NCT04626674 study?

NCT04626674 studies Muscular Dystrophy, Duchenne.

Is NCT04626674 still recruiting?

NCT04626674 is currently recruiting now. Last updated 6 April 2026.

Last reviewed 2026-04-06 · How we verify

NCT04626674: ENDEAVOR

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

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

Recruiting now Phase 1 Last updated 6 April 2026

What this trial tests

Phase 1 trial testing delandistrogene moxeparvovec in Muscular Dystrophy, Duchenne in 83 participants. Currently enrolling.

Timeline

23 November 2020

Primary endpoint
31 December 2027

29 February 2028

Quick facts

Lead sponsor	Sarepta Therapeutics, Inc.
Phase	Phase 1
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	83
Start date	23 November 2020
Primary completion	31 December 2027
Estimated completion	29 February 2028
Sites	7 locations across United States

Drugs / interventions tested

delandistrogene moxeparvovec — full drug profile →

Conditions studied

Muscular Dystrophy, Duchenne — all drugs for Muscular Dystrophy, Duchenne →

Sponsor

Sarepta Therapeutics, Inc. — full company profile →

Who can join

2 and older, male only, with Muscular Dystrophy, Duchenne. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with DMD. The maximum participant duration for this study is 156 weeks.

Publications & conference data

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

Duchenne muscular dystrophy: disease mechanism and therapeutic strategies.
Bez Batti Angulski A, Hosny N, Cohen H, Martin AA, et al · · 2023 · cited 109× · PMID 37435300 · DOI 10.3389/fphys.2023.1183101
Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR).
Zaidman CM, Proud CM, McDonald CM, Lehman KJ, et al · · 2023 · cited 62× · PMID 37539981 · DOI 10.1002/ana.26755
Drug development progress in duchenne muscular dystrophy.
Deng J, Zhang J, Shi K, Liu Z. · · 2022 · cited 57× · PMID 35935842 · DOI 10.3389/fphar.2022.950651
Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy.
Mendell JR, Shieh PB, McDonald CM, Sahenk Z, et al · · 2023 · cited 51× · PMID 37497476 · DOI 10.3389/fcell.2023.1167762
A systematic review of immunosuppressive protocols used in AAV gene therapy for monogenic disorders.
Vrellaku B, Sethw Hassan I, Howitt R, Webster CP, et al · · 2024 · cited 39× · PMID 39044426 · DOI 10.1016/j.ymthe.2024.07.016
A versatile toolkit for overcoming AAV immunity.
Li X, Wei X, Lin J, Ou L. · · 2022 · cited 31× · PMID 36119036 · DOI 10.3389/fimmu.2022.991832
Therapeutic Strategies for Dystrophin Replacement in Duchenne Muscular Dystrophy.
Happi Mbakam C, Lamothe G, Tremblay JP. · · 2022 · cited 28× · PMID 35419381 · DOI 10.3389/fmed.2022.859930
Antisense and Gene Therapy Options for Duchenne Muscular Dystrophy Arising from Mutations in the N-Terminal Hotspot.
Wilton-Clark H, Yokota T, Yokota T. · · 2022 · cited 25× · PMID 35205302 · DOI 10.3390/genes13020257

Verify or expand the search:

Other trials of delandistrogene moxeparvovec

Trials testing the same drug.

NCT06597656 — A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therape · Phase 1 · terminated
NCT06241950 — A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following I · Phase 1 · terminated
NCT06128564 — A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Ag · Phase 2 · active not recruiting
NCT05967351 — A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical · Phase 3 · enrolling by invitation
NCT05881408 — A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambu · Phase 3 · active not recruiting

Other recruiting trials for Muscular Dystrophy, Duchenne

Currently open trials in the same condition.

NCT06833489 — Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases · NA · recruiting
NCT06363357 — The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients Wi · NA · recruiting
NCT05066633 — The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in Patients W · Phase 3 · recruiting
NCT03992430 — A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD · Phase 3 · 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
NCT06747273 — Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Mu · Phase 1 · terminated
NCT06597656 — A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therape · Phase 1 · terminated

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 NCT04626674 (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: 6 April 2026

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/NCT04626674.

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