Ignite Creation Date:
2025-12-24 @ 10:01 PM
Ignite Modification Date:
2025-12-25 @ 7:38 PM
Study NCT ID:
NCT07209332
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-10-07
First Post:
2025-08-14
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
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': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 175}}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2025-11', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-09', 'completionDateStruct': {'date': '2029-03', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-10-03', 'studyFirstSubmitDate': '2025-08-14', 'studyFirstSubmitQcDate': '2025-10-03', 'lastUpdatePostDateStruct': {'date': '2025-10-07', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-10-07', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2029-03', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of participants with treatment related adverse events as assessed by the following parameters:', 'timeFrame': 'Time Frame: Week 0 through Week 96', 'description': '* Complete Physical examination, including height and weight\n* Vital Signs (via blood pressure, temperature and pulse)\n* Safety Laboratory Tests (including complete blood cell count, urinalysis and clinical chemistry)\n* ECG (12- Lead single tracing)\n* ECHO (including left ventricular ejection fraction)\n* Pulmonary Function Tests (including Peak Flow Rate, Cough Peak Flow, Forced Vital Capacity and Maximum Inspiratory Pressure)'}], 'secondaryOutcomes': [{'measure': 'North Star Ambulatory Assessment (NSAA) (Version 2.0) composite score, each item assessed using a 3 point scale, including:', 'timeFrame': 'At baseline Week 0 through Week 96', 'description': '* Time to stand (measured in seconds )\n* Timed 10-meter walk/run (measured in seconds)\n* Four-stair climb time (measured in time to climb 4 stairs in seconds)'}, {'measure': 'Performance of the Upper Limb (PUL) (Version 2.0) (measured by total score and score of high level shoulder dimension, Mid-level Elbow Dimension, Distal Wrist and Hand Dimension)', 'timeFrame': 'At baseline Week 0 through Week 96'}, {'measure': 'Stride Velocity 95th Centile (SV95C) (collected in 3 consecutive weeks)', 'timeFrame': 'At baseline Week 0 through Week 96'}, {'measure': 'Upper limb proximal strength (assessed by handheld myometer measured in Kilograms)', 'timeFrame': 'At baseline Week 0 through Week 96'}]}, 'oversightModule': {'isUsExport': True, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Duchenne Muscular Dystrophy']}, 'descriptionModule': {'briefSummary': 'This is a Phase 2 open-label extension (OLE) study to evaluate the long-term safety, tolerability, efficacy, pharmacokinetics, and the pharmacodynamics (PD) through potential exploratory biomarker(s) of intravenous (IV) WVE-N531 in patients with DMD who participated in another study of WVE-N531. All patients will have rolled over from a previous study of WVE-N531.', 'detailedDescription': 'The study will include up to 175 patients from planned and ongoing WVE-N531 studies globally. All patients will continue to receive 10 mg/kg WVE-N531 IV every 4 weeks (Q4W), through Week 96. Safety monitoring will occur for approximately 11 months after the final dose. The primary objective of the study is to evaluate the long-term safety and tolerability of WVE-N531.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Participated in a prior study of WVE-N531 and has not experienced any significant toxicities due to WVE-N531 or significant clinical deterioration of general health since the last dose or early discontinuation visit.\n2. Of note: if there will be a greater than 31-day gap between the last dose on the prior study and the FD on this study, the case should be discussed between the Investigator and Medical Monitor.\n\nExclusion Criteria:\n\n1. Any clinically significant medical finding or change during or following participation in the prior WVE-N531 study, other than DMD that, in the judgment of the Investigator, would affect the potential safety of the patient to receive WVE-N531 or interfere with participation in the study.\n2. Any recreational substance use (including prescribed cannabinoids), with the exception of alcohol and nicotine, irrespective of legality, within 2 months prior to FD and/or unwilling to refrain from such use for the duration of the study.'}, 'identificationModule': {'nctId': 'NCT07209332', 'briefTitle': 'Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy', 'organization': {'class': 'INDUSTRY', 'fullName': 'Wave Life Sciences Ltd.'}, 'officialTitle': 'An Open-Label Extension Study to Investigate the Long-term Safety, Tolerability, Pharmacokinetics, and Efficacy of WVE-N531 in Patients With Duchenne Muscular Dystrophy Who Participated in Another Study of WVE-N531', 'orgStudyIdInfo': {'id': 'WVE-N531-002'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'WVE-N531', 'interventionNames': ['Drug: WVE-N531']}], 'interventions': [{'name': 'WVE-N531', 'type': 'DRUG', 'description': 'WVE-N531 is an antisense oligonucleotide (ASO)', 'armGroupLabels': ['WVE-N531']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Amman', 'country': 'Jordan', 'contacts': [{'name': 'Muath Alqurashi, Dr', 'role': 'CONTACT', 'email': 'drmuathalqurashi@yahoo.com'}, {'name': 'Muath Alqurashi, Dr', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Istiklal Hospital/ Clinical Research Unit', 'geoPoint': {'lat': 31.95522, 'lon': 35.94503}}, {'city': 'Amman', 'country': 'Jordan', 'contacts': [{'name': 'Mai Bader, Dr', 'role': 'CONTACT', 'email': 'dr.mai.bader@hotmail.com'}, {'name': 'Mai Bader, Dr', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'The Specialty Hospital (TSH)/ Advanced Clinical Center', 'geoPoint': {'lat': 31.95522, 'lon': 35.94503}}, {'zip': 'OX3 9DU', 'city': 'Headington', 'state': 'Oxford', 'country': 'United Kingdom', 'contacts': [{'name': 'Laurent Servais, MD, PhD', 'role': 'CONTACT', 'email': 'Laurent.Servais@ouh.nhs.uk', 'phone': '01865 227503'}, {'name': 'Laurent Servais, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust", 'geoPoint': {'lat': 51.75737, 'lon': -1.21974}}], 'centralContacts': [{'name': 'Clinical Operations', 'role': 'CONTACT', 'email': 'clinicaltrials@wavelifesci.com', 'phone': '855-215-4687'}], 'overallOfficials': [{'name': 'Medical Director, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Wave Life Sciences'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Wave Life Sciences Ltd.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}