A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers

A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Dose Escalation Trial to Evaluate the Safety, Tolerability and Pharmacokinetics of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers With an Open-Label, Randomized, Two-Way Crossover Arm

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of IV migalastat HCl alone and to determine absolute bioavailability for oral migalastat HCl as compared to IV administered migalastat HCl in healthy volunteers. The data from this study will help us understand how migalastat works in the body and will help us determine what would be an effective dose in future studies with migalastat hydrochloride.

Inclusion Criteria:

• Males and females between 18 and 45 years of age.
• Body weight range ≥ 50 kg ≤ 100 kg and BMI within the range 18.5 - 29.9 kg/m2.
• Healthy as determined by a responsible and experienced physician, based on a medial evaluation.
• Male and female subjects of childbearing potential agree to adhere to the contraception requirements.
• Capable of giving written informed consent.

Exclusion Criteria:

• History of sensitivity to migalastat HCl or related iminosugars (eg, miglitol, miglustat), or other significant drug allergy.
• Past medical history, or physical examination findings, of clinically significant abnormalities that may put the subject at risk or interfere with outcome variables.
• Positive pre-study drug/alcohol screen.
• Pregnant or lactating females.
• The subject has participated in a clinical trial and has received an investigational product within 60 days prior to the first dosing day in the current study.