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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

Status
Completed
Phases
Phase 1
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2024-512624-11-00
Acronym
SRP-9001-104
Enrollment
7
Registered
2024-08-29
Start date
2023-12-15
Completion date
2025-10-10
Last updated
2025-03-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne muscular dystrophy (DMD)

Brief summary

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]

Detailed description

Maximum Observed Plasma Concentration (Cmax) of Imlifidase [Time Frame: Up to Day 7], Total IgG in Serum After Imlifidase Administration [Time Frame: Up to Week 12], rAAVrh74 Antibody Titers After Imlifidase Administration [Time Frame: Up to Hour 120], Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Serum, After Delandistrogene Moxeparvovec Administration [Time Frame: Up to Day 7], Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE) [Time Frame: Up to Week 104]

Interventions

Sponsors

Sarepta Therapeutics Inc.
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
Male
Age
0 Years to 17 Years

Design outcomes

Primary

MeasureTime frame
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]

Secondary

MeasureTime frame
Maximum Observed Plasma Concentration (Cmax) of Imlifidase [Time Frame: Up to Day 7], Total IgG in Serum After Imlifidase Administration [Time Frame: Up to Week 12], rAAVrh74 Antibody Titers After Imlifidase Administration [Time Frame: Up to Hour 120], Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Serum, After Delandistrogene Moxeparvovec Administration [Time Frame: Up to Day 7], Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE) [Time Frame: Up to Week 104]

Countries

Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026