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

Phase 1

Completed

• Conditions: Fabry Disease
• Interventions: Drug: IV migalastat HCl; Drug: IV placebo; Drug: oral migalastat HCl

• Registration Number: NCT02082327
• Lead Sponsor: Amicus Therapeutics

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2014-03
• Study First Submitted: 2014-03-06
• Study First Submitted QC: 2014-03-06
• Study First Posted: 2014-03-10
• Status Verified: 2014-04
• Primary Completion: 2014-06
• Study Completion: 2014-06
• Last Update Submitted: 2014-06-17
• Last Update Posted: 2014-06-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 31

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
0.3 mg/kg	IV migalastat HCl	IV infusion of migalastat HCl or placebo
0.3 mg/kg	IV placebo	IV infusion of migalastat HCl or placebo
1 mg/kg	IV migalastat HCl	IV infusion of migalastat HCl or placebo
1 mg/kg	IV placebo	IV infusion of migalastat HCl or placebo
10 mg/kg	IV migalastat HCl	IV infusion of migalastat HCl or placebo
10 mg/kg	IV placebo	IV infusion of migalastat HCl or placebo
150 mg IV	IV migalastat HCl	150 mg single IV infusion
150 mg IV	oral migalastat HCl	150 mg single IV infusion
150 mg oral	IV migalastat HCl	150 mg single oral dose
150 mg oral	oral migalastat HCl	150 mg single oral dose

Primary Outcome Measures

Name	Time	Method
Plasma pharmacokinetics of migalastat	Pre-dose, 0.25, 0.5, 1, 1.5, 2.0, 2.5, 3, 4, 6, 8, 10, 12, 16, 24, 48	To investigate the effect on the body of migalastat following a single 2 hour IV infusion in healthy subjects.
Safety and tolerability of migalastat	48 hours	Adverse events, clinical laboratory test values, vital signs, ECG, physical examinations

Secondary Outcome Measures

Name	Time	Method
Urinary pharmacokinetics	Pre dose, between 0-6, 6-12 and 12-24 hours after start of infusion	To estimate the urinary excretion of unchanged migalastat following a single 2 hour IV infusion in healthy subjects
Plasma pharmacokinetics of migalastat	Pre-dose, 0.25, 0.5, 1, 1.5, 2.0, 2.5, 3, 4, 6, 8, 10, 12, 16, 24, 48	To assess the dose proportionality of migalastat following a single 2 hour IV infusion

Trial Locations

Locations (1)

PRA International; 🇳🇱 Groningen, Netherlands