Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function

• Status: Completed
• Trial end date: 2012-04-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Amicus Therapeutics

Criteria

Inclusion Criteria All subjects

• 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)

• body mass index 18.0 to 40.0 kilogram (kg)/square meter (m^2) inclusive

• 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

• males will be sterile or use approved methods of contraception

• understands and signs informed consent form Healthy subjects with normal renal function

• negative test for selected drugs of abuse (excludes alcohol) at Screening and Check-in

• good health with no clinically significant medical history, physical examination, vital signs, or 12-lead ECG

• clinical laboratory tests within the reference range or not clinically significant

• normal renal function (estimated CLcr >90 mL/min) at Screening Subjects with mild, moderate or severe renal impairment

• negative test for selected drugs of abuse (excludes alcohol) at Screening and Check-in or verification of a prescription for a positive test

• renal impairment (estimated CLcr <90 mL/min)

• evidence of stable renal impairment defined as two separate estimated CLcr values within 25%

• clinical laboratory results consistent with their renal condition or of no clinical significance for the study

• 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

• subjects with renal impairment must have stable underlying medical conditions < 90 days before study start

• stable medication regimen(s) (no new drug(s) or changed dosage(s) <30 days before study drug)

• in good general health, allowing for concurrent illnesses associated with chronic kidney disease

Exclusion Criteria:

All subjects:

• history of hypersensitivity or allergies to any drug, unless approved by the Investigator and reviewed by Sponsor/Medical Monitor

• participation in a study with receipt of an investigational drug < 5 half-lives or 30 days (whichever is longer) before Check-in

• 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

• poor peripheral venous access

• whole blood donation < 56 days before dosing or plasma donation < 14 days before dosing

• receipt of blood products < 2 months before Check-in

• history or presence of any clinically significant abnormal ECG

• history of alcoholism or drug addiction < 1 year before Check-in

• positive test for HIV antibody, HBsAg or anti-HCV

• pregnant or breastfeeding

Healthy subjects with normal renal function:

• use of any tobacco- or nicotine-containing products < 6 months before Check-in

• 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

• screening laboratory values outside normal range and deemed clinically significant by the Investigator

• 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

Subjects with mild, moderate, or severe renal impairment:

• unstable disease (concurrent medical conditions that have changed significantly < 90 days)

• changes in concomitant prescription medications < 30 days before dosing or expected changes during study

• use of new non-prescription medication < 30 days before dosing

• renal transplant

• acute or chronic non-renal condition limiting the subject's ability to complete and/or participate in the study