Ignite Creation Date:
2025-12-24 @ 10:58 PM
Ignite Modification Date:
2026-01-01 @ 9:55 PM
Study NCT ID:
NCT05027269
Status:
COMPLETED
Last Update Posted:
2024-02-05
First Post:
2021-08-24
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009223', 'term': 'Myotonic Dystrophy'}, {'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'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 38}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2021-10-28', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-02', 'dispFirstSubmitDate': '2024-02-01', 'completionDateStruct': {'date': '2023-02-14', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-02-01', 'studyFirstSubmitDate': '2021-08-24', 'studyFirstSubmitQcDate': '2021-08-24', 'dispFirstPostDateStruct': {'date': '2024-02-05', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2024-02-05', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-08-30', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-02-14', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Frequency of treatment emergent adverse events (TEAEs)', 'timeFrame': 'Through study completion, up to Day 183'}], 'secondaryOutcomes': [{'measure': 'Plasma pharmacokinetic (PK) parameters', 'timeFrame': 'Through study completion, up to Day 183', 'description': 'Maximum plasma concentration (Cmax)'}, {'measure': 'Plasma pharmacokinetic (PK) parameters', 'timeFrame': 'Through study completion, up to Day 183', 'description': 'Time to maximum plasma concentration (Tmax)'}, {'measure': 'Plasma pharmacokinetic (PK) parameters', 'timeFrame': 'Through study completion, up to Day 183', 'description': 'Terminal Half-life (t1/2)'}, {'measure': 'Plasma pharmacokinetic (PK) parameters', 'timeFrame': 'Through study completion, up to Day 183', 'description': 'Area Under the Concentration-time Curve (AUC)'}, {'measure': 'Urine pharmacokinetic (PK) parameters', 'timeFrame': 'Through study completion, up to Day 183', 'description': 'fraction excreted (fe) in urine'}, {'measure': 'AOC 1001 levels in muscle tissue', 'timeFrame': 'Through study completion, up to Day 183'}, {'measure': 'Change and percentage change from baseline in DMPK mRNA knockdown', 'timeFrame': 'Through study completion, up to Day 183'}, {'measure': 'Change and percentage change from baseline in Spliceopathy', 'timeFrame': 'Through study completion, up to Day 183'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['DM1', 'Myotonic Dystrophy 1', 'Myotonic Dystrophy Type 1 (DM1)', 'Myotonic Dystrophy', 'DM', 'Dystrophy Myotonic', 'Myotonic Disorders', 'Steinert Disease', 'MARINA', 'Avidity Biosciences', 'Avidity', 'AOC 1001', 'Myotonic Muscular Dystrophy'], 'conditions': ['DM1', 'Myotonic Dystrophy 1', 'Myotonic Dystrophy', 'Myotonic Dystrophy Type 1 (DM1)', 'Dystrophy Myotonic', 'Myotonic Disorders', 'Steinert Disease', 'Myotonic Muscular Dystrophy']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://www.aviditybiosciences.com/', 'label': 'Related Info'}]}, 'descriptionModule': {'briefSummary': 'AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients (MARINA).\n\nPart A is a single dose design with 1 cohort (dose level). In Part A, the patient duration is 6 months as the treatment period is 1 day followed by a 6 month follow-up period.\n\nPart B is a multiple-ascending dose design with 2 cohorts (dose levels). In Part B, the patient duration is 6 months as the treatment period is 3 months followed by a 3 month follow-up period.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n* Genetic diagnosis of DM1 (CTG repeat length ≥ 100)\n* Clinician assessed signs of DM1\n* Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening\n\nKey Exclusion Criteria:\n\n* Diabetes that is not adequately controlled\n* BMI \\> 35 kg/m2\n* Uncontrolled hypertension\n* Congenital DM1\n* History of tibialis anterior (TA) biopsy within 3 months of Day 1 or planning to undergo TA biopsies during study period\n* Recently treated with an investigational drug\n* Treatment with anti-myotonic medication within 14 days of Day 1\n\nNote: Other protocol defined Inclusion/Exclusion criteria may apply.'}, 'identificationModule': {'nctId': 'NCT05027269', 'acronym': 'MARINA', 'briefTitle': 'Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients', 'organization': {'class': 'INDUSTRY', 'fullName': 'Avidity Biosciences, Inc.'}, 'officialTitle': 'A Randomized, Double-Blind, Placebo-Controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple-Doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients', 'orgStudyIdInfo': {'id': 'AOC 1001-CS1'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Part A Single Dose: AOC 1001 Dose Level 1', 'description': 'AOC 1001 will be administered once.', 'interventionNames': ['Drug: AOC 1001']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Part A Single Dose: Placebo', 'description': 'Saline will be administered once.', 'interventionNames': ['Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3', 'description': 'AOC 1001 will be administered three times.', 'interventionNames': ['Drug: AOC 1001']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Part B Multiple Ascending Dose: Placebo', 'description': 'Saline will be administered three times.', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'AOC 1001', 'type': 'DRUG', 'description': 'AOC 1001 will be administered by intravenous (IV) infusion.', 'armGroupLabels': ['Part A Single Dose: AOC 1001 Dose Level 1', 'Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo will be administered by intravenous (IV) infusion.', 'armGroupLabels': ['Part A Single Dose: Placebo', 'Part B Multiple Ascending Dose: Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'University of California Los Angeles', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94304', 'city': 'Palo Alto', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'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': '66205', 'city': 'Kansas City', 'state': 'Kansas', 'country': 'United States', 'facility': 'Kansas University Medical Center', 'geoPoint': {'lat': 39.11417, 'lon': -94.62746}}, {'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': '23298', 'city': 'Richmond', 'state': 'Virginia', 'country': 'United States', 'facility': 'Virginia Commonwealth University', 'geoPoint': {'lat': 37.55376, 'lon': -77.46026}}], 'overallOfficials': [{'name': 'Li Tai, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Avidity Biosciences, Inc.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Avidity Biosciences, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}