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

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02082327

Enrollment

Registered

2014-03-10

Start date

2014-03-31

Completion date

2014-06-30

Last updated

2014-06-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Fabry Disease

Keywords

Fabry Disease, Fabry, Lysosomal storage disorders, LSD, Amicus, migalastat HCl, migalastat, AT1001

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.

Interventions

DRUGIV migalastat HCl

DRUGIV placebo

DRUGoral migalastat HCl

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

DOUBLE (Subject, Investigator)

Eligibility

Sex/Gender

ALL

Age

18 Years to 45 Years

Healthy volunteers

Yes

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.

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
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
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

Countries

Netherlands

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026