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)

NCT ID: NCT06241950

Last Updated: 2025-11-20

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE1
• Total Enrollment: 5 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-01-29
• Study Completion Date: 2025-10-10

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.

Conditions

Duchenne Muscular Dystrophy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Delandistrogene Moxeparvovec after Imlifidase Infusion

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.

Group Type: EXPERIMENTAL

delandistrogene moxeparvovec

Intervention Type: GENETIC

Single IV infusion of delandistrogene moxeparvovec

imlifidase

Intervention Type: BIOLOGICAL

IV infusion of Imlifidase

Interventions

delandistrogene moxeparvovec

Single IV infusion of delandistrogene moxeparvovec

Intervention Type: GENETIC

imlifidase

IV infusion of Imlifidase

Intervention Type: BIOLOGICAL

Other Intervention Names

SRP-9001; delandistrogene moxeparvovec-rokl; ELEVIDYS; Idefirix

Eligibility Criteria

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.

• Minimum Eligible Age: 4 Years
• Maximum Eligible Age: 9 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Hansa Biopharma AB

INDUSTRY

Sponsor Role: collaborator

Sarepta Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Director

Role: STUDY_DIRECTOR

Sarepta Therapeutics, Inc.

Locations

Hospital Sant Joan de Déu

Barcelona, , Spain

Countries

Spain

Other Identifiers

2022-003407-15

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

SRP-9001-104

Identifier Type: -

Identifier Source: org_study_id