Ignite Creation Date:
2025-12-24 @ 11:16 PM
Ignite Modification Date:
2026-01-25 @ 4:52 AM
Study NCT ID:
NCT03062956
Status:
COMPLETED
Last Update Posted:
2018-03-23
First Post:
2017-02-16
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Single Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of MYK-491
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D002311', 'term': 'Cardiomyopathy, Dilated'}], 'ancestors': [{'id': 'D006332', 'term': 'Cardiomegaly'}, {'id': 'D006331', 'term': 'Heart Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D009202', 'term': 'Cardiomyopathies'}, {'id': 'D000083083', 'term': 'Laminopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR'], 'maskingDescription': 'Subjects will be randomized 6:2 to MYK-491:placebo within each cohort.'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 64}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2017-01-16', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2018-03', 'completionDateStruct': {'date': '2017-11-28', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2018-03-21', 'studyFirstSubmitDate': '2017-02-16', 'studyFirstSubmitQcDate': '2017-02-20', 'lastUpdatePostDateStruct': {'date': '2018-03-23', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2017-02-24', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2017-11-28', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'The change from baseline in LVFS, LVEF, LVSV and SET by TTE', 'timeFrame': '7 days'}, {'measure': 'SET while using photoplethysmography', 'timeFrame': '7 days'}, {'measure': 'Relationship between MYK-491 plasma concentrations/PK parameters and PD parameters', 'timeFrame': '7 days'}, {'measure': 'Plasma concentrations of metabolites of MYK-491 in plasma and urine', 'timeFrame': '7 days'}, {'measure': 'Relationship between MYK-491 plasma concentration and QTc interval', 'timeFrame': '7 days'}], 'primaryOutcomes': [{'measure': 'Safety and tolerability assessments will include treatment emergent AEs and SAEs, ECG recordings, vital signs, hs-troponin 1 concentrations, laboratory abnormalities and physical exam abnormalities', 'timeFrame': '7 days'}], 'secondaryOutcomes': [{'measure': 'Maximum observed plasma drug concentration (Cmax)', 'timeFrame': '7 days'}, {'measure': 'Maximum observed plasma concentration (Tmax)', 'timeFrame': '7 days'}, {'measure': 'Area under the plasma concentration-time curve (AUC)', 'timeFrame': '7 days'}, {'measure': 'First-order terminal elimination half-life (t1/2)', 'timeFrame': '7 days'}, {'measure': 'Mean retention time (MRT)', 'timeFrame': '7 days'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Dilated Cardiomyopathy']}, 'descriptionModule': {'briefSummary': 'Up to 72 healthy volunteers will be given a single dose of MYK-491 or placebo and be monitored for safety and tolerability over a 7 day period.', 'detailedDescription': 'Up to 72 healthy volunteers will be given a single dose of MYK-491 or placebo and be monitored for safety and tolerability over a 7 day period. After the 28 day screening period, the eligible subject will be admitted to the clinical site and will receive a single dose of study drug or placebo. Subjects will be confined to the clinical site for five days (Day -1 to Day 4) and will return to the clinic on Day 7 for a safety follow-up.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT'], 'maximumAge': '55 Years', 'minimumAge': '18 Years', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Weight between 60 and 90 kg inclusive\n* Resting heart rate of \\< 80 beats per minute\n* Documented LVEF greater than or equal to 55% during Screening\n* Normal electrocardiogram (ECG) at Screening\n* Normal acoustic windows on transthoracic echocardiograms at Screening\n* All safety laboratory parameters within normal limits at Screening\n* History or evidence of another clinically significant disorder, in the opinion of the investigator.\n\nExclusion Criteria:\n\n* Active infection\n* History of coronary artery disease\n* History of malignancy with the exception of in situ cervical cancer more than 5 years prior to Screening or surgically-excised non-melanomatous skin cancers more than 2 years prior to Screening\n* Positive serology tests at screening\n* Current use of tobacco or nicotine-containing products exceeding 10 per day.'}, 'identificationModule': {'nctId': 'NCT03062956', 'briefTitle': 'A Single Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of MYK-491', 'organization': {'class': 'INDUSTRY', 'fullName': 'MyoKardia, Inc.'}, 'officialTitle': 'Randomized, Placebo-Controlled Study of Safety, Tolerability, Preliminary Pharmacokinetics and Pharmacodynamics of Single Ascending Oral Doses of MYK-491 in Healthy Adult Volunteers', 'orgStudyIdInfo': {'id': 'MYK-491-001'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Single oral dose of MYK-491', 'description': 'single-dose, oral suspension', 'interventionNames': ['Drug: MYK-491 or placebo']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Single oral dose of placebo', 'description': 'single-dose, oral suspension', 'interventionNames': ['Drug: MYK-491 or placebo']}], 'interventions': [{'name': 'MYK-491 or placebo', 'type': 'DRUG', 'description': 'Oral suspension', 'armGroupLabels': ['Single oral dose of MYK-491', 'Single oral dose of placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '3004', 'city': 'Melbourne', 'state': 'Victoria', 'country': 'Australia', 'facility': 'Nucleus Network', 'geoPoint': {'lat': -37.814, 'lon': 144.96332}}], 'overallOfficials': [{'name': 'Jason Lickliter, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Nucleus Network'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'MyoKardia, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}