Ignite Creation Date:
2025-12-25 @ 2:09 AM
Ignite Modification Date:
2026-01-07 @ 7:57 AM
Study NCT ID:
NCT05540860
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-11-12
First Post:
2022-09-06
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
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': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 76}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2022-10-24', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2027-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-10', 'studyFirstSubmitDate': '2022-09-06', 'studyFirstSubmitQcDate': '2022-09-12', 'lastUpdatePostDateStruct': {'date': '2025-11-12', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2022-09-15', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of adverse events during treatment with sevasemten or placebo', 'timeFrame': '48 months', 'description': 'All participants'}, {'measure': 'Severity of adverse events during treatment with sevasemten or placebo', 'timeFrame': '48 months', 'description': 'All participants'}], 'secondaryOutcomes': [{'measure': 'Incidence of laboratory test-related treatment emergent adverse events', 'timeFrame': '48 months', 'description': 'All participants'}, {'measure': 'Pharmacokinetics as measured by steady state plasma concentration', 'timeFrame': '48 months', 'description': 'All participants'}, {'measure': 'Change from Baseline in serum creatinine kinase', 'timeFrame': '12 weeks', 'description': 'All participants'}, {'measure': 'Change from Baseline in fast skeletal muscle troponin I', 'timeFrame': '12 weeks', 'description': 'All participants'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Duchenne Muscular Dystrophy'], 'conditions': ['Duchenne Muscular Dystrophy']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'http://edgewisetx.com', 'label': 'Sponsor Website'}]}, 'descriptionModule': {'briefSummary': 'The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an open-label part B.', 'detailedDescription': 'This is a 2-part, multi-center, Phase 2 study to evaluate the effect of sevasemten (EDG-5506) on safety, pharmacokinetics and biomarkers of muscle damage in approximately 72 children with DMD treated with oral, once-daily sevasemten for up to 48 months. This study will have up to a 4-week Screening period, a 12-week randomized, double-blind, placebo controlled treatment period (Part A), up to a 196-week open-label extension period (Part B), and a 2-week follow up period.\n\nApproximately 72 participants aged 4 to 9 years inclusive will be randomized to sevasemten or placebo in a 2:1 ratio. Five dose cohorts (C1, C2, C3, C4 and C5) of approximately 9 participants each will be enrolled sequentially. Approximately 18 total additional participants may be added across Cohorts 2, 3, or 4.\n\nAn additional cohort, Cohort 2NS, to include participants (aged 4 to 7 years inclusive) not currently treated with corticosteroids, will enroll approximately 9 participants after Cohort 2 safety review and in parallel with the additional cohorts.\n\nAfter review of emerging data, the protocol was amended so all dose cohorts receive the same dose in Part B.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD'], 'maximumAge': '9 Years', 'minimumAge': '4 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Common Inclusion Criteria:\n\n1. A documented mutation on the DMD gene and phenotype consistent with Duchenne muscular dystrophy.\n2. Able to complete the stand from supine in ≤ 10 seconds and able to perform the 4-stair climb in \\< 10 seconds at the Screening visit.\n3. Body weight greater than or equal to 15 kg at the Screening visit.\n\nFor Cohorts 1, 2, 3, 4 and 5:\n\nAged 4-9 years on a stable dose of corticosteroids for a minimum of 6 months prior to the Baseline visit.\n\nFor Cohort 2 Non-Steroid (Cohort 2NS):\n\nAged 4-7 years not on corticosteroids within 6 months prior to the Baseline visit.\n\nKey Common Exclusion Criteria:\n\n1. Medical history or clinically significant physical exam/laboratory result that, in the opinion of the investigator, would render the participant unsuitable for the study. This includes venous access that would be too difficult to facilitate repeated blood testing.\n2. A forced vital capacity \\< 60% predicted at the Screening visit for those participants who are \\> 8 years old at Screening.\n3. A cardiac echocardiography showing left ventricular ejection \\< 45% at the Screening visit.\n4. Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the Screening visit in the present study.\n5. Receipt of a stable dose of an approved exon-skipping therapy with a treatment duration of less than 1 year prior to the Screening visit.\n\nFor Cohort 2 Non-Steroid (Cohort 2NS):\n\nReceipt of oral corticosteroids for the treatment of Duchenne muscular dystrophy in the previous 6 months. Participants will not be tapered off steroids for the purpose of this study and oral corticosteroids for the treatment of Duchenne muscular dystrophy may be initiated after the Week 16 visit.'}, 'identificationModule': {'nctId': 'NCT05540860', 'briefTitle': 'A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Edgewise Therapeutics, Inc.'}, 'officialTitle': 'A 2-part Phase 2 Study of Safety, Pharmacokinetics and Biomarkers in Children With Duchenne Muscular Dystrophy Including a Randomized, Double-Blind, Placebo-Controlled Part A, Followed by an Open-Label Part B', 'orgStudyIdInfo': {'id': 'EDG-5506-210'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1', 'description': 'Drug: Sevasemten Drug: Placebo', 'interventionNames': ['Drug: Sevasemten Dose 1', 'Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2', 'description': 'Drug: Sevasemten Drug: Placebo', 'interventionNames': ['Drug: Sevasemten Dose 2', 'Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 3', 'description': 'Drug: Sevasemten Drug: Placebo', 'interventionNames': ['Drug: Sevasemten Dose 3', 'Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 4', 'description': 'Drug: Sevasemten Drug: Placebo', 'interventionNames': ['Drug: Sevasemten Dose 4', 'Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 5', 'description': 'Drug: Sevasemten Drug: Placebo', 'interventionNames': ['Drug: Sevasemten Dose 5', 'Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2NS', 'description': 'Drug: Sevasemten Drug: Placebo', 'interventionNames': ['Drug: Sevasemten Dose 2', 'Drug: Placebo']}], 'interventions': [{'name': 'Sevasemten Dose 1', 'type': 'DRUG', 'description': 'Sevasemten is administered orally once per day', 'armGroupLabels': ['Cohort 1']}, {'name': 'Sevasemten Dose 2', 'type': 'DRUG', 'description': 'Sevasemten is administered orally once per day', 'armGroupLabels': ['Cohort 2', 'Cohort 2NS']}, {'name': 'Sevasemten Dose 3', 'type': 'DRUG', 'description': 'Sevasemten is administered orally once per day', 'armGroupLabels': ['Cohort 3']}, {'name': 'Sevasemten Dose 4', 'type': 'DRUG', 'description': 'Sevasemten is administered orally once per day', 'armGroupLabels': ['Cohort 4']}, {'name': 'Sevasemten Dose 5', 'type': 'DRUG', 'description': 'Sevasemten is administered orally once per day', 'armGroupLabels': ['Cohort 5']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo is administered orally once per day', 'armGroupLabels': ['Cohort 1', 'Cohort 2', 'Cohort 2NS', 'Cohort 3', 'Cohort 4', 'Cohort 5']}]}, 'contactsLocationsModule': {'locations': [{'zip': '72202', 'city': 'Little Rock', 'state': 'Arkansas', 'country': 'United States', 'facility': "Arkansas Children's Hospital", 'geoPoint': {'lat': 34.74648, 'lon': -92.28959}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'UCLA Medical Center', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '95817', 'city': 'Sacramento', 'state': 'California', 'country': 'United States', 'facility': 'UC Davis Medical Center', 'geoPoint': {'lat': 38.58157, 'lon': -121.4944}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Children's Hospital Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '32610', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Florida', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '30329', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Rare Disease Research', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '52242', 'city': 'Iowa City', 'state': 'Iowa', 'country': 'United States', 'facility': 'University of Iowa', 'geoPoint': {'lat': 41.66113, 'lon': -91.53017}}, {'zip': '66160', 'city': 'Kansas City', 'state': 'Kansas', 'country': 'United States', 'facility': 'University of Kansas Medical Center', 'geoPoint': {'lat': 39.11417, 'lon': -94.62746}}, {'zip': '21205', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Kennedy Krieger Institute', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '01605', 'city': 'Worcester', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'University of Massachusetts Memorial Medical Center', 'geoPoint': {'lat': 42.26259, 'lon': -71.80229}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University School of Medicine', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '43205', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': "Nationwide Children's Hospital", 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '76104', 'city': 'Fort Worth', 'state': 'Texas', 'country': 'United States', 'facility': "Cook Children's Medical Center", 'geoPoint': {'lat': 32.72541, 'lon': -97.32085}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Edgewise Therapeutics, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}