A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014)

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01730482
• Date of registration: 08/11/2012
• Prospective Registration: No
• Primary sponsor: Amicus Therapeutics
• Public title: A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014)
• Scientific title: A Phase 1 Study to Investigate the Absorption, Metabolism and Excretion of [14C] AT1001 (Migalastat Hydrochloride) Following a Single Oral Administration in Healthy Volunteers (AT1001-014)
• Date of first enrolment: August 2011
• Target sample size: 6
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT01730482
• Study type: Interventional
• Study design: Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Contacts

• Name: Medical Monitor, Clinical Research
• Affiliation: Amicus Therapeutics

Key inclusion & exclusion criteria

Inclusion Criteria:

- Healthy male subjects between the ages of 30 and 55 years, inclusive

- Body mass index (BMI) of >=18.0 to <=30.0 and weighing at least 60 kg

- Anticipated, regular, average bowel movements of 1-2 per day

- No clinically significant abnormal findings on the physical exam, vital signs, serum
chemistry, hematology, and urinalysis values, as deemed by the principal investigator

- Willing to avoid taking of all over-the-counter medications 7 days and all
prescription drugs 14 days prior to Day -1

- Willing to abstain from sexual intercourse or employ a barrier method of
contraception during the inpatient clinic confinement and until the follow-up visit

- Willing to avoid ingestion of broccoli, brussels sprouts, grapefruit, grapefruit
juice, or charbroiled meat during the period of confinement in the clinical unit

- Willingness to consume a fiber-rich diet during the period of confinement in the
clinic

- Willingness to avoid vigorous physical activity during inpatient clinic confinement
and through the follow-up visit

- Willingness and ability to comply with scheduled visits, treatment plan, laboratory
tests, other study procedures, and study restrictions

- Provide written informed consent to participate in the study

Exclusion Criteria:

- Any previous or ongoing clinically significant illness, medical condition, medical
history, physical findings, ECG finding, or laboratory abnormality that, in the
investigator's opinion, could affect the safety of the subject, or alter the
absorption, distribution, metabolism, or excretion of the study drug, or could impair
the assessment of study results

- History or presence of significant ophthalmic, cardiovascular, pulmonary, hepatic,
renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic,
neurologic or psychiatric disease

- History of a major surgical procedure within 30 days before screening

- History of blood or plasma donation or blood loss (>400 mL) within 60 days before
screening

- History of drug or alcohol abuse or addiction within 2 years before screening

- Intake of more than 2 alcoholic drinks per day within the past 7 days or use of
alcohol-, grapefruit-, or caffeine-containing foods or beverages within 72 hours
prior to Day -1

- Self-reported smoker (occasional or frequent) or positive urine cotinine test
(measured at screening and baseline) exceeding the local laboratory's lower limit of
detection.

- Presence or history of severe adverse reaction to any drug; history of
hypersensitivity or allergic reaction to AT1001 or related iminosugars

- Receipt of any investigational agent or participation in any other interventional
clinical trial within the past 30 days

- Participation in any clinical study involving administration of [14C] labeled
compound(s) within the last 12 months. A subject's previous effective dose will be
reviewed by the medical investigator to ensure there is no risk of
contamination/carryover into the current study

- At Day -1, a drug toxicology screen positive for any illicit substances, or alcohol

- Anticipated need for alcohol, tobacco, or any drug during the period of confinement
in the clinical unit



Age minimum: 30 Years
Age maximum: 55 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Drug: [14C] AT1001

Primary Outcome(s)

Recovery of total radioactivity in feces [Time Frame: Days 1 to 11]

Plasma total radioactivity pharmacokinetic parameters [Time Frame: Days 1 to 10]

Plasma AT1001 pharmacokinetic parameters [Time Frame: Days 1 to 10]

Presence of radioactivity in expired air [Time Frame: Day 1]

Recovery of total radioactivity in urine [Time Frame: Days 1 to 11]

Secondary Outcome(s)

Measure of ECG to access safety and tolerability [Time Frame: Upto 8 weeks]

Number of subjects with adverse events as a measure of safety and tolerability [Time Frame: Day 1 to Day 29]

Characterization of metabolites in plasma, urine, duodenal bile and fecal homogenates [Time Frame: Day -1 to Day 11]

Measure of clinical laboratory test values to access safety and tolerability [Time Frame: Upto 8 weeks]

Physical examination to access safety and tolerability [Time Frame: Upto 8 weeks]

The blood to plasma ratio of total radioactivity [Time Frame: Day 1]

Measure of vital signs to access safety and tolerability [Time Frame: Upto 8 weeks]

Secondary ID(s)

116435

Source(s) of Monetary Support

Please refer to primary and secondary sponsors