NCT06241950CompletedPhase 1
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)
An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in Association with Imlifidase in Subjects with Duchenne Muscular Dystrophy with Pre-existing Antibodies to rAAVrh74
Source: EU CTIS (euclinicaltrials.eu) · Last updated 18 Mar 2025
Brief summary (registry, in English)
• Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content [Time Frame: Baseline, Week 12] • 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] • 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] • Mean Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Muscle Tissue Biopsy, After Delandistrogene Moxeparvovec Administration [Time Frame: Week 12]
- Status (registry)
- Completed
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 7 (actual)
- First submitted
- 23 Aug 2024
- Start date
- Dec 2023
- Lead sponsor
- Sarepta Therapeutics Inc.
- Diseases
- Duchenne muscular dystrophy
- NCT number
- NCT06241950
- EU CT number
- 2024-512624-11-00
- EudraCT number
- 2022-003407-15
What you commit to
- Study type: Interventional · Phase 1
- Participants enrolled: 7
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of EU CTIS (euclinicaltrials.eu). It describes the study, not your situation.
Who cannot take part
Copied verbatim from EU CTIS (euclinicaltrials.eu) and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Previous treatment with imlifidase.
- 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 receiving the study drugs 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.
Lab values
- Abnormality in protocol-specified diagnostic evaluations or laboratory tests. Note: Other inclusion or exclusion criteria could apply.
Who the study is looking for
- Ambulatory per protocol specified criteria.
- Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and confirmatory genetic testing.
- Ability to cooperate with motor assessment testing.
- Has elevated rAAVrh74 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.
- 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).
Age in the registry: - to 17 years · Sex: Male · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Hospital Sant Joan De Deu Barcelona Coordinator of this site? Confirm its status | Esplugues de Llobregat | Spain | Completed |
This page is information, not medical advice. Discuss any trial with your doctor.