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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-003407-15 | EudraCT Number |
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Study is being terminated due to a business decision.
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| Name | Class |
|---|---|
| Hansa Biopharma AB | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Delandistrogene Moxeparvovec after Imlifidase Infusion | Experimental | 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| delandistrogene moxeparvovec | Genetic | Single IV infusion of delandistrogene moxeparvovec |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 | |
| 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 | |
| Mean Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Muscle Tissue Biopsy, After Delandistrogene Moxeparvovec Administration | Week 12 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
Note: Other inclusion or exclusion criteria could apply.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Sarepta Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hospital Sant Joan de Déu | Barcelona | 08950 | Spain |
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| imlifidase | Biological | IV infusion of Imlifidase |
|
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| Up to Hour 120 |
| Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Serum, After Delandistrogene Moxeparvovec Administration | 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) | Up to Week 104 |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C442815 | Mac-1-like protein, Streptococcus |
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