Ignite Creation Date:
2025-12-24 @ 11:00 PM
Ignite Modification Date:
2026-02-28 @ 10:56 PM
Study NCT ID:
NCT07008469
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2025-12-22
First Post:
2025-05-29
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Global Open-Label Extension Study of Del-desiran for the Treatment of DM1
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009223', 'term': 'Myotonic Dystrophy'}, {'id': 'D009222', 'term': 'Myotonia'}, {'id': 'D020967', 'term': 'Myotonic Disorders'}], 'ancestors': [{'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D020879', 'term': 'Neuromuscular Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 230}}, 'statusModule': {'overallStatus': 'ENROLLING_BY_INVITATION', 'startDateStruct': {'date': '2025-07-25', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2030-10', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-19', 'studyFirstSubmitDate': '2025-05-29', 'studyFirstSubmitQcDate': '2025-05-29', 'lastUpdatePostDateStruct': {'date': '2025-12-22', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-06-06', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2030-08', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'TEAEs', 'timeFrame': 'From enrollment to the end of treatment at 4 years', 'description': 'Incidence of treatment emergent adverse events'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['DM1', 'Myotonic Dystrophy', 'Myotonic Dystrophy 1', 'Myotonia', 'Myotonic Dystrophy Type 1 (DM1)', 'Dystrophy Myotonic', 'Myotonic Disorders', 'Steinert Disease', 'Steinert', 'Myotonic Muscular Dystrophy', 'HARBOR-OLE', 'Avidity Biosciences', 'Avidity', 'AOC 1001', 'Del-desiran', 'Delpacibart etedesiran', 'HARBOR'], 'conditions': ['Myotonic Dystrophy Type 1', 'DM1', 'Myotonic Dystrophy', 'Myotonia', 'Myotonic Dystrophy 1', 'Myotonic Disorders', 'Steinert Myotonic Dystrophy', 'Steinert Disease']}, 'descriptionModule': {'briefSummary': 'A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1', 'detailedDescription': 'The study consists of a Screening Period of up to either 4-weeks or 8-weeks depending on the prior parent trial, and up to a 4-year Treatment Period. The anticipated duration is 50 months and 2 weeks (4 years and 2.5 months).\n\nParticipants will receive an intravenous infusion of del-desiran at the clinical study site every 8 weeks for a total of 7 doses per year. The final dose will occur at Year 4, Visit 7, followed by a final assessment 8 weeks after the last dose.\n\nAn additional subgroup of de novo participants will also be included in a Fixed-Dose PK cohort.\n\nAn Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '16 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'FROM A PARENT STUDY\n\nKey Inclusion Criteria:\n\n* Completion of any prior AOC 1001 studies with satisfactory completion of dosing and follow-up assessments and satisfactory compliance with the protocol requirements of the parent study, as determined by the Investigator.\n\nKey Exclusion Criteria:\n\n* Breastfeeding, pregnancy, or intent to become pregnant during the study.\n* Unwilling to comply with contraceptive requirements.\n* Any new conditions or worsening of existing condition that in the opinion of the Investigator would make the participant unsuitable for the study.\n\nFIXED-DOSE PK COHORT\n\nKey Inclusion Criteria:\n\n* Clinical and genetic diagnosis of DM1 and clinically significant hand myotonia.\n\nKey Exclusion Criteria:\n\n* Breastfeeding, pregnancy, or intent to become pregnant during the study\n* Unwilling to comply with contraceptive requirements\n* Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study\n* Diabetes that is not adequately controlled\n* History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded\n* Body Mass Index \\> 35 kg/m2 at Screening\n* Recently treated with an investigational drug or biological agent\n\nNote: Additional protocol defined Inclusion and Exclusion criteria apply'}, 'identificationModule': {'nctId': 'NCT07008469', 'acronym': 'HARBOR-OLE', 'briefTitle': 'Global Open-Label Extension Study of Del-desiran for the Treatment of DM1', 'organization': {'class': 'INDUSTRY', 'fullName': 'Avidity Biosciences, Inc.'}, 'officialTitle': 'A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1', 'orgStudyIdInfo': {'id': 'AOC 1001-CS4'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Del-desiran', 'description': 'Del-desiran (AOC 1001) will be administered seven times per year for up to 4 years.', 'interventionNames': ['Drug: Del-desiran (AOC 1001)']}], 'interventions': [{'name': 'Del-desiran (AOC 1001)', 'type': 'DRUG', 'description': 'Del-desiran will be administered by intravenous (IV) infusion.', 'armGroupLabels': ['Del-desiran']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'University of California, Los Angeles (UCLA)', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94305', 'city': 'Stanford', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University', 'geoPoint': {'lat': 37.42411, 'lon': -122.16608}}, {'zip': '80045', 'city': 'Denver', 'state': 'Colorado', 'country': 'United States', 'facility': 'University of Colorado', 'geoPoint': {'lat': 39.73915, 'lon': -104.9847}}, {'zip': '32608', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Florida', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '46202', 'city': 'Indianapolis', 'state': 'Indiana', 'country': 'United States', 'facility': 'Indiana University (IU)', 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'zip': '66205', 'city': 'Kansas City', 'state': 'Kansas', 'country': 'United States', 'facility': 'Kansas University 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': '02215', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Beth Israel Deaconess Medical Center', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '14642', 'city': 'Rochester', 'state': 'New York', 'country': 'United States', 'facility': 'University of Rochester Medical Center', 'geoPoint': {'lat': 43.15478, 'lon': -77.61556}}, {'zip': '43221', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': 'Ohio State University', 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'University of Pennsylvania', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Houston Methodist Neurological Institute', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '23298', 'city': 'Richmond', 'state': 'Virginia', 'country': 'United States', 'facility': 'Virginia Commonwealth University', 'geoPoint': {'lat': 37.55376, 'lon': -77.46026}}, {'zip': '038-1331', 'city': 'Aomori', 'state': 'Aomori', 'country': 'Japan', 'facility': 'Aomori Hospital', 'geoPoint': {'lat': 40.81667, 'lon': 140.73333}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED', 'description': 'Not yet determined'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Avidity Biosciences, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}