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

Amicus Therapeutics

Status and phase: Completed; Phase 1

Conditions: Fabry Disease

Treatments: Drug: IV migalastat HCl; Drug: IV placebo; Drug: oral migalastat HCl

Study type: Interventional

Funder types: Industry

Identifiers

NCT02082327

2013-005553-75 (EudraCT Number)

AT1001-018

Details and patient eligibility

About

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.

Enrollment: 31 patients

Sex: All

Ages: 18 to 45 years old

Volunteers: Accepts Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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31 participants in 5 patient groups

0.3 mg/kg

Experimental group

Description:

IV infusion of migalastat HCl or placebo

Treatment:

Drug: IV placebo

Drug: IV migalastat HCl

1 mg/kg

Experimental group

Description:

IV infusion of migalastat HCl or placebo

Treatment:

Drug: IV placebo

Drug: IV migalastat HCl

10 mg/kg

Experimental group

Description:

IV infusion of migalastat HCl or placebo

Treatment:

Drug: IV placebo

Drug: IV migalastat HCl

150 mg IV

Experimental group

Description:

150 mg single IV infusion

Treatment:

Drug: oral migalastat HCl

Drug: IV migalastat HCl

150 mg oral

Experimental group

Description:

150 mg single oral dose

Treatment:

Drug: oral migalastat HCl

Drug: IV migalastat HCl