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TerminatedNCT06597656HORIZONUpdated Sep 4, 2025

A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74

A Phase 1 interventional study of delandistrogene moxeparvovec and Plasmapheresis in Duchenne Muscular Dystrophy, sponsored by Sarepta Therapeutics, Inc.. Terminated at 3 sites in United States. Open to male participants aged 4 Years to 8 Years. Per ClinicalTrials.gov, last updated 2025-09-04.

Sponsored by Sarepta Therapeutics, Inc. · Phase 1, Interventional, and Treatment

Why this study was terminated
Study is being terminated due to a business decision.

From the registry’s dates

  • Primary completion was Aug 2025, 1 year 2 months ago, and no results have been posted to the registry.
Phase
Phase 1
Study type
Interventional
Enrollment
3
Allocation
Not applicable
Ages
4 Years to 8 Years
Sex
Male
01

Study summary

This is a gene transfer therapy study evaluating the safety of and delandistrogene moxeparvovec dystrophin protein expression from delandistrogene moxeparvovec following therapeutic plasma exchange (plasmapheresis) in ambulatory male participants with DMD and pre-existing antibodies to AAVrh74 over a period of 59 weeks.

02

Conditions studied

  • Duchenne Muscular Dystrophy

Keywords

  • Gene-Delivery
  • DMD
  • Ambulatory
  • Pediatric
  • Duchenne
  • Seropositive
  • Pre-existing antibodies
  • AAVrh74 antibody
  • Plasmapheresis
  • Titer
  • Seropositivity
03

In context

Muscular Dystrophy, Duchenne

473 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 107 are open to participants now.

This study's enrollment of 3 is below the median of 26 across 326 interventional studies indexed under Muscular Dystrophy, Duchenne.

Browse Muscular Dystrophy, Duchenne studies →

Lead sponsor

Sarepta Therapeutics, Inc. is the lead sponsor of 52 studies on the registry; 8 are open to participants now.

Of its 20 completed or terminated interventional studies of FDA-regulated products, 12 (60%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
4 Years to 8 Years
Sexes eligible
Male
Accepts healthy volunteers
No

Inclusion criteria

  • Ambulatory per protocol specified criteria.
  • Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and prior confirmatory genetic testing.
  • Ability to cooperate with motor assessment testing.
  • Has elevated AAVrh74 antibody titers per protocol-specified requirements.
  • A pathogenic frameshift mutation, nonsense mutation or premature stop codon or pathogenic variant in the DMD gene that is expected to lead to absence of dystrophin protein with exception of a mutation in exon 8 and/or 9.
  • Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).

Exclusion criteria

Exclusion Criteria:

  • Has reduced left ventricular ejection fraction on the screening ECHO or clinical signs and/or symptoms of cardiomyopathy.
  • Presence of any other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or concomitant illness or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risks for gene transfer or a medical condition or extenuating circumstance 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.
  • Exposure to gene therapy, investigational medication, or other protocol-specified treatment within the protocol specified time limits.
  • Abnormality in protocol-specified diagnostic evaluations or laboratory tests. .

Note: Other inclusion or exclusion criteria could apply.

05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
3 participants (actual)

Study arms

  • Experimental
    Delandistrogene Moxeparvovec After Plasmapheresis Procedure

    Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1 after plasmapheresis procedure if AAVrh74 antibodies are sufficiently low.

    Genetic: delandistrogene moxeparvovec · Procedure: Plasmapheresis

Interventions

  • Geneticdelandistrogene moxeparvovec

    Single IV infusion of delandistrogene moxeparvovec

    Also known as: SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS

  • ProcedurePlasmapheresis

    Therapeutic plasma exchange procedure

06

What researchers measure

Primary outcomes

  1. Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression Adjusted by Muscle Content Biopsied Muscle as Measured by Western Blot

    Time frame: Baseline, Week 12

  2. Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by Immunofluorescence (IF) Fiber Intensity

    Time frame: Baseline, Week 12

  3. Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by IF Percent Dystrophin-positive Fibers (PDPF)

    Time frame: Baseline, Week 12

  4. Mean Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Muscle Tissue Biopsy, After Delandistrogene Moxeparvovec Administration

    Time frame: Week 12

Secondary outcomes

  1. Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE)

    Time frame: Baseline up to End of Study (Up to Week 59)

  2. Change from Baseline in rAAVrh74 Antibody Titers

    Time frame: Baseline, Week 1

07

Study locations

3 sites
  • University of Florida, College of Medicine
    Gainesville, Florida 32610, United States
  • Washington University School of Medicine in St. Louis
    St Louis, Missouri 63110, United States
  • Nationwide Children's Hospital
    Columbus, Ohio 43205, United States
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Sep 4, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT06597656
Lead sponsor
Sarepta Therapeutics, Inc.
Responsible party
Sponsor
First posted
Sep 19, 2024
Start date
Sep 18, 2024
Primary completion
Aug 5, 2025
Completion
Aug 5, 2025
Last update
Sep 4, 2025

Study contacts

Medical Director
study director · Sarepta Therapeutics, Inc.

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is terminated, as verified in Aug 2025. You cannot join it, but the record below documents what was studied.

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