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CompletedNCT02082327Updated Jun 18, 2014

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

A Phase 1 interventional study of IV migalastat HCl and IV placebo in Fabry Disease, sponsored by Amicus Therapeutics. Completed at 1 site in Netherlands. Open to participants aged 18 Years to 45 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2014-06-18.

Sponsored by Amicus Therapeutics · Phase 1, Interventional, and Treatment

Phase
Phase 1
Study type
Interventional
Enrollment
31
Allocation
Randomized
Ages
18 Years to 45 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Fabry Disease

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Keywords

  • Fabry Disease
  • Fabry
  • Lysosomal storage disorders
  • LSD
  • Amicus
  • migalastat HCl
  • migalastat
  • AT1001
03

In context

Fabry Disease

242 studies on the registry are indexed under Fabry Disease; 54 are open to participants now.

This study's enrollment of 31 is above the median of 22 across 105 interventional studies indexed under Fabry Disease.

Browse Fabry Disease studies →

Lead sponsor

Amicus Therapeutics is the lead sponsor of 42 studies on the registry; 8 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 45 Years
Sexes eligible
All
Accepts 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

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.
05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Double (Participant, Investigator)
Enrollment
31 participants (actual)

Study arms

  • Experimental
    0.3 mg/kg

    IV infusion of migalastat HCl or placebo

    Drug: IV migalastat HCl · Drug: IV placebo

  • Experimental
    1 mg/kg

    IV infusion of migalastat HCl or placebo

    Drug: IV migalastat HCl · Drug: IV placebo

  • Experimental
    10 mg/kg

    IV infusion of migalastat HCl or placebo

    Drug: IV migalastat HCl · Drug: IV placebo

  • Experimental
    150 mg IV

    150 mg single IV infusion

    Drug: IV migalastat HCl · Drug: oral migalastat HCl

  • Experimental
    150 mg oral

    150 mg single oral dose

    Drug: IV migalastat HCl · Drug: oral migalastat HCl

Interventions

  • DrugIV migalastat HCl

    Also known as: AT1001

  • DrugIV placebo

    Also known as: Sodium Chloride 0.9%

  • Drugoral migalastat HCl

    Also known as: AT1001

06

What researchers measure

Primary outcomes

  1. Plasma pharmacokinetics of migalastat

    To investigate the effect on the body of migalastat following a single 2 hour IV infusion in healthy subjects.

    Time frame: Pre-dose, 0.25, 0.5, 1, 1.5, 2.0, 2.5, 3, 4, 6, 8, 10, 12, 16, 24, 48

  2. Safety and tolerability of migalastat

    Adverse events, clinical laboratory test values, vital signs, ECG, physical examinations

    Time frame: 48 hours

Secondary outcomes

  1. Plasma pharmacokinetics of migalastat

    To assess the dose proportionality of migalastat following a single 2 hour IV infusion

    Time frame: Pre-dose, 0.25, 0.5, 1, 1.5, 2.0, 2.5, 3, 4, 6, 8, 10, 12, 16, 24, 48

  2. Urinary pharmacokinetics

    To estimate the urinary excretion of unchanged migalastat following a single 2 hour IV infusion in healthy subjects

    Time frame: Pre dose, between 0-6, 6-12 and 12-24 hours after start of infusion

07

Study locations

1 site
  • PRA International
    Groningen, 9713 GZ, Netherlands
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jun 18, 2014, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT02082327
Lead sponsor
Amicus Therapeutics
Responsible party
Sponsor
First posted
Mar 10, 2014
Start date
Mar 2014
Primary completion
Jun 2014
Completion
Jun 2014
Last update
Jun 18, 2014

Study contacts

Medical Monitor Clinical Research
study director · Amicus Therapeutics

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Apr 2014. You cannot join it, but the record below documents what was studied.

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