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)

Duchenne Muscular Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT06241950
• Other study ID #: SRP-9001-104
• Secondary ID: 2022-003407-15
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: January 29, 2024
• Est. completion date: September 30, 2026

Study information

• Verified date: March 2024
• Source: Sarepta Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 6
• Est. completion date: September 30, 2026
• Est. primary completion date: January 31, 2025
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 4 Years to 9 Years

Eligibility

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.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Genetic:
• delandistrogene moxeparvovec
Single IV infusion of delandistrogene moxeparvovec

Biological:
• imlifidase
IV infusion of Imlifidase

Locations

Country	Name	City	State
Spain	Hospital Sant Joan de Déu	Barcelona

Sponsors (2)

Lead Sponsor	Collaborator
Sarepta Therapeutics, Inc.	Hansa Biopharma AB

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content		Baseline, Week 12
Primary	Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by Immunofluorescence (IF) Fiber Intensity		Baseline, Week 12
Primary	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
Primary	Mean Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Muscle Tissue Biopsy, After Delandistrogene Moxeparvovec Administration		Week 12
Secondary	Maximum Observed Plasma Concentration (Cmax) of Imlifidase		Up to Day 7
Secondary	Total IgG in Serum After Imlifidase Administration		Up to Week 12
Secondary	rAAVrh74 Antibody Titers After Imlifidase Administration		Up to Hour 120
Secondary	Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Serum, After Delandistrogene Moxeparvovec Administration		Up to Day 7
Secondary	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