Ignite Creation Date:
2025-12-25 @ 2:38 AM
Ignite Modification Date:
2025-12-26 @ 1:16 AM
Study NCT ID:
NCT07160634
Status:
RECRUITING
Last Update Posted:
2025-12-12
First Post:
2025-08-29
Is Possible Gene Therapy:
True
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
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'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 80}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2025-10-22', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2034-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-05', 'studyFirstSubmitDate': '2025-08-29', 'studyFirstSubmitQcDate': '2025-08-29', 'lastUpdatePostDateStruct': {'date': '2025-12-12', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-09-08', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2029-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540', 'timeFrame': 'Baseline, Day 540'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in Stride Velocity 95th Centile (SV95C) (m/s) at Day 540', 'timeFrame': 'Baseline, Day 540'}, {'measure': 'Change From Baseline in 4-Stair Climb (4SC) Velocity (tasks/s) at Day 540', 'timeFrame': 'Baseline, Day 540'}, {'measure': 'Change From Baseline in 10-meter Walk/Run (10MWR) Velocity (m/s) at Day 540', 'timeFrame': 'Baseline, Day 540'}, {'measure': 'Change From Baseline in North Star Ambulatory Assessment (NSAA) total score at Day 540', 'timeFrame': 'Baseline, Day 540'}, {'measure': 'Cumulative Loss of Function in NSAA Items at Day 540', 'timeFrame': 'At Day 540'}, {'measure': 'Change From Baseline in Microdystrophin Protein Levels by western blot (% of normal dystrophin) at Day 90', 'timeFrame': 'Baseline, Day 90'}, {'measure': 'Change From Baseline in Microdystrophin Tissue Distribution by Immunofluorescence (% positive fibers) at Day 90', 'timeFrame': 'Baseline, Day 90'}, {'measure': 'Change from baseline in Percent Predicted Forced Vital Capacity (FVC) at Day 540', 'timeFrame': 'Baseline, Day 540'}, {'measure': 'Change from baseline in Percent Predicted Peak Expiratory Flow (PEF) at Day 540', 'timeFrame': 'Baseline, Day 540'}, {'measure': 'Change from baseline in Percent Predicted Forced Expiratory Volume in 1 second (FEV1) at Day 540', 'timeFrame': 'Baseline, Day 540'}, {'measure': 'Change from baseline in the Pediatric Outcomes Data Collection Instrument (PODCI) Global score at Day 540', 'timeFrame': 'Baseline, Day 540'}, {'measure': 'Number of Participants with Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Adverse Events of special interest (AESIs), and clinically significant changes in Electrocardiogram (ECG) and Echocardiography (ECHO)', 'timeFrame': 'From first dose up to Day 540'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['SGT-003', 'Duchenne Muscular Dystrophy (DMD)', 'adeno-associated virus (AAV)', 'IMPACT DUCHENNE'], 'conditions': ['Duchenne Muscular Dystrophy']}, 'descriptionModule': {'briefSummary': 'This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ambulant male participants with DMD. The secondary objectives include the evaluation of additional efficacy and safety outcomes. The study will be divided into 2 parts. Participants will be randomized 1:1 to either SGT-003 in Part 1 followed by placebo in Part 2 or to placebo in Part 1 followed by SGT-003 in Part 2. Participants will continue to be monitored in long term follow up (LTFU) for at least 5 years from their SGT-003 dosing date.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD'], 'maximumAge': '11 Years', 'minimumAge': '7 Years', 'genderBased': True, 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Participant is ambulatory.\n* Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype.\n* Negative for antibodies against adeno-associated virus.\n* On a stable daily oral regimen of at least 0.5 mg/kg/day prednisone or 0.75 milligrams per kilogram per day (mg/kg/day) deflazacort for at least 6 months prior to entering the study, allowing for weight-based dose modifications in accordance with clinical practice.\n* Meet 10-meter walk/run time criteria.\n* Meet time to rise from supine criteria.\n* Participant has bodyweight ≤50 kg.\n\nExclusion Criteria:\n\n* Current or prior treatment with an approved or investigational gene transfer drug or gene editing therapy.\n* Exposure to vamorolone, givinostat, approved or investigational dystrophin- or disease-modifying drugs (such as eteplirsen, golodirsen, casimersen, viltolarsen, and ataluren), or another investigational drug for any indication within 6 months or 5 half-lives, whichever is longer, prior to enrollment.\n* Established clinical diagnosis of DMD that is associated with any deletion variant or variant predicted not to express exons 1 to 11, exons 42 to 45, or exons 57 to 69, inclusive of the DMD gene as documented by a genetic report.\n\nOther Inclusion/Exclusion criteria to be applied as per protocol.'}, 'identificationModule': {'nctId': 'NCT07160634', 'briefTitle': 'A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Solid Biosciences Inc.'}, 'officialTitle': 'A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy', 'orgStudyIdInfo': {'id': 'SGT-003-301'}, 'secondaryIdInfos': [{'id': '2025-522949-22', 'type': 'EUDRACT_NUMBER'}, {'id': '1013075', 'type': 'OTHER', 'domain': 'IRAS'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'SGT-003 followed by Placebo', 'description': 'Enrolled participants will receive a single intravenous (IV) infusion of SGT-003 in Part 1 and a single IV infusion of matching Placebo in Part 2.', 'interventionNames': ['Drug: SGT-003', 'Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Placebo followed by SGT-003', 'description': 'Enrolled participants will receive a single intravenous (IV) infusion of matching Placebo in Part 1 and a single IV infusion of SGT-003 in Part 2.', 'interventionNames': ['Drug: SGT-003', 'Drug: Placebo']}], 'interventions': [{'name': 'SGT-003', 'type': 'DRUG', 'description': 'Adeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5)', 'armGroupLabels': ['Placebo followed by SGT-003', 'SGT-003 followed by Placebo']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'IV infusion', 'armGroupLabels': ['Placebo followed by SGT-003', 'SGT-003 followed by Placebo']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Vancouver', 'state': 'British Columbia', 'status': 'RECRUITING', 'country': 'Canada', 'contacts': [{'name': 'Nela Martic', 'role': 'CONTACT', 'email': 'nmartic3@cw.bc.ca', 'phone': '604-875-2345', 'phoneExt': '6549'}, {'name': 'Kathryn Selby, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "BC Children's Hospital", 'geoPoint': {'lat': 49.24966, 'lon': -123.11934}}], 'centralContacts': [{'name': 'Solid Bio Clinical Trials', 'role': 'CONTACT', 'email': 'clinicaltrials@solidbio.com', 'phone': '6173374680'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Solid Biosciences Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}