Ignite Creation Date:
2025-12-24 @ 11:05 PM
Ignite Modification Date:
2026-01-02 @ 9:44 AM
Study NCT ID:
NCT01730469
Status:
COMPLETED
Last Update Posted:
2017-08-03
First Post:
2012-11-08
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000795', 'term': 'Fabry Disease'}], 'ancestors': [{'id': 'D013106', 'term': 'Sphingolipidoses'}, {'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D059345', 'term': 'Cerebral Small Vessel Diseases'}, {'id': 'D002561', 'term': 'Cerebrovascular Disorders'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C525167', 'term': 'larazotide acetate'}, {'id': 'C090092', 'term': 'migalastat'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 32}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2011-08'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-08', 'completionDateStruct': {'date': '2012-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-08-02', 'studyFirstSubmitDate': '2012-11-08', 'studyFirstSubmitQcDate': '2012-11-15', 'lastUpdatePostDateStruct': {'date': '2017-08-03', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2012-11-21', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2012-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of subjects with adverse events to assess safety and tolerability', 'timeFrame': 'Day 1 to Day 10 (+1)', 'description': 'Adverse events will be evaluated from Day 1 to the end of study (Day 10 +1).'}, {'measure': 'Clinical laboratory test values to assess safety and tolerability', 'timeFrame': 'Day -28 to Day 10 (+1)', 'description': 'Clinical laboratory evaluations (hematology, clinical chemistry, urinalysis, Hepatitis A and HIV screen) will be evaluated from screening to the end of the study.'}, {'measure': 'Vital signs to assess safety and tolerability', 'timeFrame': 'Day -28 to Day 10 (+1)', 'description': 'Vital signs (oral temperature, respiratory rate, and seated blood pressure) will be performed from screening to the end of the study.'}, {'measure': 'Physician examination to assess safety and tolerability', 'timeFrame': 'Day -28 to Day 10 (+1)', 'description': 'Physical examination (general appearance, skin, thorax/lungs, cardiovascular and abdomen) will be performed from screening to the end of the study.'}, {'measure': 'Measure of ECG to assess safety and tolerability', 'timeFrame': 'Day -28 to Day 10 (+1)', 'description': "Electrocardiogram (ECG) measures the electrical activity of the heart and the hearts' rhythm. All subjects will undergo ECG testing."}], 'secondaryOutcomes': [{'measure': 'Maximum observed concentration (Cmax) of AT1001', 'timeFrame': 'Day 1 to Day 6', 'description': 'Blood samples will be collected at predose, and 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 15, 24, 48, 72, 96, and 120 hours post-dose and the resultant maximum plasma concentration (Cmax) will be measured in subjects with impaired renal function and normal renal function.'}, {'measure': 'Time to achieve maximum concentration (Tmax) of AT1001', 'timeFrame': 'Day 1 to Day 6', 'description': 'Blood samples will be collected at predose, and 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 15, 24, 48, 72, 96, and 120 hours post-dose and time to maximum concentration (tmax) will be measured in subjects with impaired renal function and normal renal function.'}, {'measure': 'Apparent terminal elimination half life (t1/2 ) of AT1001', 'timeFrame': 'Day 1 to Day 6', 'description': 'Blood samples will be collected at predose, and 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 15, 24, 48, 72, 96, and 120 hours post-dose and and apparent terminal elimination half-life (t1/2) will be measured in subjects with impaired renal function and normal renal function.'}, {'measure': 'Area under the concentration-time curve from time zero to the last measurable concentration (AUC 0-t ) of AT1001', 'timeFrame': 'Day 1 to Day 6', 'description': 'Blood samples will be collected at predose, and 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 15, 24, 48, 72, 96, and 120 hours post-dose and AUC 0-t will be measured in subjects with impaired renal function and normal renal function'}, {'measure': 'Area under the concentration-time curve extrapolated to infinity (AUC 0-inf) of AT1001', 'timeFrame': 'Day 1 to Day 6', 'description': 'Blood samples will be collected at predose, and 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 15, 24, 48, 72, 96, and 120 hours post-dose and AUC 0-inf will be measured in subjects with impaired renal function and normal renal function'}, {'measure': 'Apparent terminal elimination rate constant for AT1001', 'timeFrame': 'Day 1 to Day 6', 'description': 'Blood samples will be collected at predose, and 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 15, 24, 48, 72, 96, and 120 hours post-dose and the apparent terminal elimination rate constant will be measured in subjects with impaired renal function and normal renal function'}, {'measure': 'Oral clearance of AT1001', 'timeFrame': 'Day 1 to Day 6', 'description': 'Blood samples will be collected at predose, and 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 15, 24, 48, 72, 96, and 120 hours post-dose and the oral clearance will be measured in subjects with impaired renal function and normal renal function'}, {'measure': 'Oral volume of distribution of AT1001', 'timeFrame': 'Day 1 to Day 6', 'description': 'Blood samples will be collected at predose, and 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, 12, 15, 24, 48, 72, 96, and 120 hours post-dose and the oral volume of distribution will be measured in subjects with impaired renal function and normal renal function'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Pharmacokinetics', 'GR181413', 'Safety', 'AT1001', 'Migalastat hydrochloride'], 'conditions': ['Fabry Disease']}, 'referencesModule': {'references': [{'pmid': '27136905', 'type': 'DERIVED', 'citation': 'Johnson FK, Mudd PN Jr, DiMino T, Vosk J, Sitaraman S, Boudes P, France N, Barlow C. An open-label study to determine the pharmacokinetics and safety of migalastat HCl in subjects with impaired renal function and healthy subjects with normal renal function. Clin Pharmacol Drug Dev. 2015 Jul;4(4):256-61. doi: 10.1002/cpdd.149. Epub 2014 Dec 22.'}]}, 'descriptionModule': {'briefSummary': 'This study will assess the safety, tolerability, and pharmacokinetics (PK) study of a single dose of 150 mg AT1001 (migalastat HCl, GR181413A) administered orally to healthy subjects with normal renal function and to subjects with mild, moderate, and severe renal impairment.', 'detailedDescription': 'This will be an open-label, non-randomized, multiple-center, sequential group, safety, tolerability, and PK study of a single dose of AT1001 (migalastat HCl, GR181413A) administered orally as a 150 mg dose in fasted healthy control male and female subjects with normal renal function compared to mild, moderate, and severe renally-impaired subjects (classified by level of creatinine clearance \\[CLcr\\] as determined by the Cockcroft-Gault formula).\n\nScreening will occur from Day -28 to Day -2. Subjects will check-in to the clinic on Day -1 and receive a single oral dose of 150 mg AT1001 on Day 1. Subjects will be discharged from the clinic on Day 2 (if stable as determined by the Investigator) and return for daily visits on Day 3 through Day 6 for a safety assessment and PK sampling. Subjects will undergo a follow-up visit on Day 7 (+1) and an end of study visit on Day 10 (+1).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '18 Years', 'healthyVolunteers': True, 'eligibilityCriteria': "Inclusion Criteria All subjects\n\n* males or females aged 18 to 70 years inclusive (subjects with normal renal function, mild or moderate renal impairment), and 18 to 75 years inclusive (subjects with severe renal impairment)\n* body mass index 18.0 to 40.0 kilogram (kg)/square meter (m\\^2) inclusive\n* females who are non-pregnant, non-lactating, or postmenopausal for \\>=1 year, surgically sterile for \\>= 90 days, or agree to use approved methods of contraception\n* males will be sterile or use approved methods of contraception\n* understands and signs informed consent form Healthy subjects with normal renal function\n* negative test for selected drugs of abuse (excludes alcohol) at Screening and Check-in\n* good health with no clinically significant medical history, physical examination, vital signs, or 12-lead ECG\n* clinical laboratory tests within the reference range or not clinically significant\n* normal renal function (estimated CLcr \\>90 mL/min) at Screening Subjects with mild, moderate or severe renal impairment\n* negative test for selected drugs of abuse (excludes alcohol) at Screening and Check-in or verification of a prescription for a positive test\n* renal impairment (estimated CLcr \\<90 mL/min)\n* evidence of stable renal impairment defined as two separate estimated CLcr values within 25%\n* clinical laboratory results consistent with their renal condition or of no clinical significance for the study\n* abnormal laboratory values must not be clinically significant. Anemia secondary to renal disease is acceptable if hemoglobin is ≥9 g/dL and no clinically significant symptoms. Liver enzymes and bilirubin must be below twice the upper normal level\n* subjects with renal impairment must have stable underlying medical conditions \\< 90 days before study start\n* stable medication regimen(s) (no new drug(s) or changed dosage(s) \\<30 days before study drug)\n* in good general health, allowing for concurrent illnesses associated with chronic kidney disease\n\nExclusion Criteria:\n\nAll subjects:\n\n* history of hypersensitivity or allergies to any drug, unless approved by the Investigator and reviewed by Sponsor/Medical Monitor\n* participation in a study with receipt of an investigational drug \\< 5 half-lives or 30 days (whichever is longer) before Check-in\n* use of alcohol, grapefruit, or caffeine-containing foods or beverages \\< 72 hours before Check-in, unless approved by the Investigator and reviewed by the Sponsor/Medical Monitor\n* poor peripheral venous access\n* whole blood donation \\< 56 days before dosing or plasma donation \\< 14 days before dosing\n* receipt of blood products \\< 2 months before Check-in\n* history or presence of any clinically significant abnormal ECG\n* history of alcoholism or drug addiction \\< 1 year before Check-in\n* positive test for HIV antibody, HBsAg or anti-HCV\n* pregnant or breastfeeding\n\nHealthy subjects with normal renal function:\n\n* use of any tobacco- or nicotine-containing products \\< 6 months before Check-in\n* clinically significant (history of or active) cardiac, hepatic, pulmonary, endocrine, neurological, infectious, gastrointestinal, hematologic, oncologic, or psychiatric disease putting the subject at increased risk or could interfere with study objectives\n* screening laboratory values outside normal range and deemed clinically significant by the Investigator\n* use of a prescription drug \\< 14 days of dosing or a non-prescription drug \\< 7 days before dosing or need of concomitant medication during the study\n\nSubjects with mild, moderate, or severe renal impairment:\n\n* unstable disease (concurrent medical conditions that have changed significantly \\< 90 days)\n* changes in concomitant prescription medications \\< 30 days before dosing or expected changes during study\n* use of new non-prescription medication \\< 30 days before dosing\n* renal transplant\n* acute or chronic non-renal condition limiting the subject's ability to complete and/or participate in the study"}, 'identificationModule': {'nctId': 'NCT01730469', 'briefTitle': 'Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function', 'organization': {'class': 'INDUSTRY', 'fullName': 'Amicus Therapeutics'}, 'officialTitle': 'An Open-Label Study to Determine the Safety and Pharmacokinetics of AT1001 in Subjects With Impaired Renal Function and Healthy Subjects With Normal Renal Function (AT1001-015)', 'orgStudyIdInfo': {'id': '116431'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'AT1001 150 mg', 'description': 'Each subject will receive a single oral dose of AT1001 150 mg administered orally with 240 mL room temperature water after at least a 4-hour fast', 'interventionNames': ['Drug: AT1001 150 mg']}], 'interventions': [{'name': 'AT1001 150 mg', 'type': 'DRUG', 'otherNames': ['migalastat HCl', 'GR181413A'], 'description': 'AT1001 150mg is available as a capsule', 'armGroupLabels': ['AT1001 150 mg']}]}, 'contactsLocationsModule': {'locations': [{'zip': '92626', 'city': 'Costa Mesa', 'state': 'California', 'country': 'United States', 'facility': 'GSK Investigational Site', 'geoPoint': {'lat': 33.64113, 'lon': -117.91867}}, {'zip': '33014', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'GSK Investigational Site', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '33169', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'GSK Investigational Site', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '32809', 'city': 'Orlando', 'state': 'Florida', 'country': 'United States', 'facility': 'GSK Investigational Site', 'geoPoint': {'lat': 28.53834, 'lon': -81.37924}}], 'overallOfficials': [{'name': 'Medical Monitor, Clinical Research', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Amicus Therapeutics'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Amicus Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}