Ignite Creation Date:
2025-12-25 @ 2:11 AM
Ignite Modification Date:
2025-12-27 @ 11:54 PM
Study NCT ID:
NCT05683860
Status:
TERMINATED
Last Update Posted:
2023-10-23
First Post:
2023-01-04
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Open-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'submissionInfos': [{'resetDate': '2025-04-21', 'mcpReleaseN': 3, 'releaseDate': '2025-04-02'}], 'estimatedResultsFirstSubmitDate': '2025-04-02'}}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 8}}, 'statusModule': {'whyStopped': 'Despite robust, sustained reductions in poly(GP), no clinical benefit was seen at 24 weeks, and reductions in poly(GP) were not associated with stabilization in functional outcomes. Based on these data, Wave decided to stop development of WVE-004.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2022-12-14', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-01', 'completionDateStruct': {'date': '2023-06-30', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-10-18', 'studyFirstSubmitDate': '2023-01-04', 'studyFirstSubmitQcDate': '2023-01-04', 'lastUpdatePostDateStruct': {'date': '2023-10-23', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-01-13', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-06-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety: Number of patients with adverse events (AEs)', 'timeFrame': 'Day 1 to Week 120 (end of study)'}, {'measure': 'Safety: Number of patients with a severe AE', 'timeFrame': 'Day 1 to Week 120 (end of study)'}, {'measure': 'Safety: Number of patients with serious AEs (SAEs)', 'timeFrame': 'Day 1 to Week 120 (end of study)'}, {'measure': 'Safety: Number of patients who withdraw due to AEs', 'timeFrame': 'Day 1 to Week 120 (end of study)'}]}, 'oversightModule': {'isUsExport': True, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['ALS', 'FTD']}, 'descriptionModule': {'briefSummary': 'This is an OLE study conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of WVE-004 in adult patients with ALS, FTD, or mixed ALS/FTD phenotype with a documented mutation in the C9orf72 gene. To participate in the study, patients must have successfully completed Phase 1b/2a WVE-004-001 study.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patient successfully completed the Phase 1b/2a study with WVE-004, WVE-004-001.\n\nExclusion Criteria:\n\n* Patient has a clinically significant medical finding on the physical examination other than C9orf72-associated ALS or FTD that, in the judgment of the Investigator or Sponsor, will make the patient unsuitable for participation in and/or completion of the trial procedures.\n* Patient received any other investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Patient received an investigational oligonucleotide within the past 6 months or 5 half-lives of the drug, whichever is longer.'}, 'identificationModule': {'nctId': 'NCT05683860', 'briefTitle': 'Open-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Wave Life Sciences Ltd.'}, 'officialTitle': 'A Multicenter, Open-label Extension (OLE) Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)', 'orgStudyIdInfo': {'id': 'WVE-004-002'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Experimental: WVE-004 (Dose A)', 'interventionNames': ['Drug: WVE-004']}], 'interventions': [{'name': 'WVE-004', 'type': 'DRUG', 'description': 'WVE-004 is a stereopure antisense oligonucleotide. It is administered via intrathecal injection', 'armGroupLabels': ['Experimental: WVE-004 (Dose A)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '3015', 'city': 'Rotterdam', 'country': 'Netherlands', 'facility': 'Erasmus MC', 'geoPoint': {'lat': 51.9225, 'lon': 4.47917}}, {'city': 'Utrecht', 'country': 'Netherlands', 'facility': 'Universitair Medisch Centrum Utrecht', 'geoPoint': {'lat': 52.09083, 'lon': 5.12222}}, {'zip': 'OX3 7LF', 'city': 'Oxford', 'country': 'United Kingdom', 'facility': 'University of Oxford - Nuffield Department of Clinical Neurosciences', 'geoPoint': {'lat': 51.75222, 'lon': -1.25596}}], 'overallOfficials': [{'name': 'Medical Director, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Wave Life Sciences'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Wave Life Sciences Ltd.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}