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)
Phase 1
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Genetic: delandistrogene moxeparvovec
- Registration Number
- NCT06241950
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Brief Summary
This is a gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec and delandistrogene moxeparvovec dystrophin expression in association with imlifidase, in participants with DMD with pre-existing antibodies to rAAVrh74 over a period of 104 weeks.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- Male
- Target Recruitment
- 6
Inclusion Criteria
- 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).
Exclusion Criteria
- 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.
- Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
Note: Other inclusion or exclusion criteria could apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Delandistrogene Moxeparvovec after Imlifidase Infusion imlifidase Participants may receive 1 or 2 doses of imlifidase, depending on rAAVrh74 antibody titer results. Then, based on their rAAVrh74 antibody titer, eligible participants may receive 1 dose of delandistrogene moxeparvovec infusion. Delandistrogene Moxeparvovec after Imlifidase Infusion delandistrogene moxeparvovec Participants may receive 1 or 2 doses of imlifidase, depending on rAAVrh74 antibody titer results. Then, based on their rAAVrh74 antibody titer, eligible participants may receive 1 dose of delandistrogene moxeparvovec infusion.
- Primary Outcome Measures
Name Time Method Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by IF Percent Dystrophin-positive Fibers (PDPF) Baseline, Week 12 Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content Baseline, Week 12 Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by Immunofluorescence (IF) Fiber Intensity Baseline, Week 12 Mean Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Muscle Tissue Biopsy, After Delandistrogene Moxeparvovec Administration Week 12
- Secondary Outcome Measures
Name Time Method Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE) Up to Week 104 Maximum Observed Plasma Concentration (Cmax) of Imlifidase Up to Day 7 Total IgG in Serum After Imlifidase Administration Up to Week 12 rAAVrh74 Antibody Titers After Imlifidase Administration Up to Hour 120 Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Serum, After Delandistrogene Moxeparvovec Administration Up to Day 7
Trial Locations
- Locations (1)
Hospital Sant Joan de Déu
🇪🇸Barcelona, Spain