Ignite Creation Date:
2025-12-24 @ 4:47 PM
Ignite Modification Date:
2025-12-28 @ 1:08 AM
Study NCT ID:
NCT06241950
Status:
TERMINATED
Last Update Posted:
2025-11-20
First Post:
2024-01-26
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
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)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020388', 'term': 'Muscular Dystrophy, Duchenne'}], 'ancestors': [{'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C442815', 'term': 'Mac-1-like protein, Streptococcus'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 5}}, 'statusModule': {'whyStopped': 'Study is being terminated due to a business decision.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2024-01-29', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2025-10-10', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-11-17', 'studyFirstSubmitDate': '2024-01-26', 'studyFirstSubmitQcDate': '2024-01-26', 'lastUpdatePostDateStruct': {'date': '2025-11-20', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2024-02-05', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-10-10', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content', 'timeFrame': 'Baseline, Week 12'}, {'measure': 'Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by Immunofluorescence (IF) Fiber Intensity', 'timeFrame': 'Baseline, Week 12'}, {'measure': 'Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by IF Percent Dystrophin-positive Fibers (PDPF)', 'timeFrame': 'Baseline, Week 12'}, {'measure': 'Mean Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Muscle Tissue Biopsy, After Delandistrogene Moxeparvovec Administration', 'timeFrame': 'Week 12'}], 'secondaryOutcomes': [{'measure': 'Maximum Observed Plasma Concentration (Cmax) of Imlifidase', 'timeFrame': 'Up to Day 7'}, {'measure': 'Total IgG in Serum After Imlifidase Administration', 'timeFrame': 'Up to Week 12'}, {'measure': 'rAAVrh74 Antibody Titers After Imlifidase Administration', 'timeFrame': 'Up to Hour 120'}, {'measure': 'Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Serum, After Delandistrogene Moxeparvovec Administration', 'timeFrame': 'Up to Day 7'}, {'measure': 'Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE)', 'timeFrame': 'Up to Week 104'}]}, 'oversightModule': {'isUsExport': True, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Gene-Delivery', 'DMD', 'Ambulatory', 'Pediatric', 'Duchenne', 'Pre-existing antibodies', 'rAAVrh74 Antibody', 'Seropositive', 'Seropositivity', 'Titer'], 'conditions': ['Duchenne Muscular Dystrophy']}, 'descriptionModule': {'briefSummary': '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.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD'], 'maximumAge': '9 Years', 'minimumAge': '4 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Ambulatory per protocol specified criteria.\n* Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and confirmatory genetic testing.\n* Ability to cooperate with motor assessment testing.\n* Has elevated rAAVrh74 antibody titers per protocol-specified requirements.\n* 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.\n* 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).\n\nExclusion Criteria:\n\n* Previous treatment with imlifidase.\n* 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.\n* Exposure to gene therapy, investigational medication, or other protocol-specified treatment within the protocol specified time limits.\n* Abnormality in protocol-specified diagnostic evaluations or laboratory tests.\n\nNote: Other inclusion or exclusion criteria could apply."}, 'identificationModule': {'nctId': 'NCT06241950', 'briefTitle': '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)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Sarepta Therapeutics, Inc.'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'SRP-9001-104'}, 'secondaryIdInfos': [{'id': '2022-003407-15', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Delandistrogene Moxeparvovec after Imlifidase Infusion', 'description': '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.', 'interventionNames': ['Genetic: delandistrogene moxeparvovec', 'Biological: imlifidase']}], 'interventions': [{'name': 'delandistrogene moxeparvovec', 'type': 'GENETIC', 'otherNames': ['SRP-9001', 'delandistrogene moxeparvovec-rokl', 'ELEVIDYS'], 'description': 'Single IV infusion of delandistrogene moxeparvovec', 'armGroupLabels': ['Delandistrogene Moxeparvovec after Imlifidase Infusion']}, {'name': 'imlifidase', 'type': 'BIOLOGICAL', 'otherNames': ['Idefirix'], 'description': 'IV infusion of Imlifidase', 'armGroupLabels': ['Delandistrogene Moxeparvovec after Imlifidase Infusion']}]}, 'contactsLocationsModule': {'locations': [{'zip': '08950', 'city': 'Barcelona', 'country': 'Spain', 'facility': 'Hospital Sant Joan de Déu', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}], 'overallOfficials': [{'name': 'Medical Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Sarepta Therapeutics, Inc.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Sarepta Therapeutics, Inc.', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Hansa Biopharma AB', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}