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

Fabry Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02082327
• Other study ID #: AT1001-018
• Secondary ID: 2013-005553-75
• Status: Completed
• Phase: Phase 1
• First received: March 6, 2014
• Last updated: June 17, 2014
• Start date: March 2014
• Est. completion date: June 2014

Study information

• Verified date: April 2014
• Source: Amicus Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Health authority: Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 31
• Est. completion date: June 2014
• Est. primary completion date: June 2014
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Both
• Age group: 18 Years to 45 Years

Eligibility

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

Allocation: Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Drug:
• IV migalastat HCl

• IV placebo

• oral migalastat HCl

Locations

Country	Name	City	State
Netherlands	PRA International	Groningen

Sponsors (1)

Lead Sponsor	Collaborator
Amicus Therapeutics

Country where clinical trial is conducted

Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Plasma pharmacokinetics of migalastat	To investigate the effect on the body of migalastat following a single 2 hour IV infusion in healthy subjects.	Pre-dose, 0.25, 0.5, 1, 1.5, 2.0, 2.5, 3, 4, 6, 8, 10, 12, 16, 24, 48	No
Primary	Safety and tolerability of migalastat	Adverse events, clinical laboratory test values, vital signs, ECG, physical examinations	48 hours	Yes
Secondary	Plasma pharmacokinetics of migalastat	To assess the dose proportionality of migalastat following a single 2 hour IV infusion	Pre-dose, 0.25, 0.5, 1, 1.5, 2.0, 2.5, 3, 4, 6, 8, 10, 12, 16, 24, 48	No
Secondary	Urinary pharmacokinetics	To estimate the urinary excretion of unchanged migalastat following a single 2 hour IV infusion in healthy subjects	Pre dose, between 0-6, 6-12 and 12-24 hours after start of infusion	No