NCT02082327

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
31

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Mar 2014

Shorter than P25 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2014

Completed
5 days until next milestone

First Submitted

Initial submission to the registry

March 6, 2014

Completed
4 days until next milestone

First Posted

Study publicly available on registry

March 10, 2014

Completed
3 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2014

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2014

Completed
Last Updated

June 18, 2014

Status Verified

April 1, 2014

Enrollment Period

3 months

First QC Date

March 6, 2014

Last Update Submit

June 17, 2014

Conditions

Fabry Disease

Keywords

Fabry DiseaseFabryLysosomal storage disordersLSDAmicusmigalastat HClmigalastatAT1001

Outcome Measures

Primary Outcomes (2)

  • 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

  • Safety and tolerability of migalastat

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

    48 hours

Secondary Outcomes (2)

  • 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

  • Urinary pharmacokinetics

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

Study Arms (5)

0.3 mg/kg

EXPERIMENTAL

IV infusion of migalastat HCl or placebo

Drug: IV migalastat HClDrug: IV placebo

1 mg/kg

EXPERIMENTAL

IV infusion of migalastat HCl or placebo

Drug: IV migalastat HClDrug: IV placebo

10 mg/kg

EXPERIMENTAL

IV infusion of migalastat HCl or placebo

Drug: IV migalastat HClDrug: IV placebo

150 mg IV

EXPERIMENTAL

150 mg single IV infusion

Drug: IV migalastat HClDrug: oral migalastat HCl

150 mg oral

EXPERIMENTAL

150 mg single oral dose

Drug: IV migalastat HClDrug: oral migalastat HCl

Interventions

IV migalastat HClDRUG
Also known as: AT1001
0.3 mg/kg1 mg/kg10 mg/kg150 mg IV150 mg oral
IV placeboDRUG
Also known as: Sodium Chloride 0.9%
0.3 mg/kg1 mg/kg10 mg/kg
oral migalastat HClDRUG
Also known as: AT1001
150 mg IV150 mg oral

Eligibility Criteria

Age18 Years - 45 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

PRA International

Groningen, 9713 GZ, Netherlands

Location

MeSH Terms

Conditions

Fabry DiseaseLysosomal Storage Diseases

Interventions

migalastatlarazotide acetateSodium Chloride

Condition Hierarchy (Ancestors)

SphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesCerebral Small Vessel DiseasesCerebrovascular DisordersVascular DiseasesCardiovascular DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolism, Inborn ErrorsLipidosesLipid Metabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

ChloridesHydrochloric AcidChlorine CompoundsInorganic ChemicalsSodium Compounds

Study Officials

  • Medical Monitor Clinical Research

    Amicus Therapeutics

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 6, 2014

First Posted

March 10, 2014

Study Start

March 1, 2014

Primary Completion

June 1, 2014

Study Completion

June 1, 2014

Last Updated

June 18, 2014

Record last verified: 2014-04

Locations

NL(1)