Back to results

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

Primary Purpose

Fabry Disease

Status

Completed

Phase

Phase 1

Locations

Netherlands

Study Type

Interventional

Intervention

IV migalastat HCl

IV placebo

oral migalastat HCl

About this trial

This is an interventional treatment trial for Fabry Disease focused on measuring Fabry Disease, Fabry, Lysosomal storage disorders, LSD, Amicus, migalastat HCl, migalastat, AT1001

Eligibility Criteria

18 Years - 45 Years (Adult)All SexesAccepts Healthy Volunteers

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.

Sites / Locations

PRA International

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm Type

Experimental

Arm Label

0.3 mg/kg

1 mg/kg

10 mg/kg

150 mg IV

150 mg oral

Arm Description

IV infusion of migalastat HCl or placebo

150 mg single IV infusion

150 mg single oral dose

Outcomes

Primary Outcome Measures

Plasma pharmacokinetics of migalastat

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

Safety and tolerability of migalastat

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

Secondary Outcome Measures

Plasma pharmacokinetics of migalastat

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

Urinary pharmacokinetics

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

Full Information

NCT ID

NCT02082327

First Posted

March 6, 2014

Last Updated

June 17, 2014

Sponsor

Amicus Therapeutics

1. Study Identification

Unique Protocol Identification Number

NCT02082327

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2014

Overall Recruitment Status

Completed

Study Start Date

March 2014 (undefined)

Primary Completion Date

June 2014 (Actual)

Study Completion Date

June 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Amicus Therapeutics

4. Oversight

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Fabry Disease

Keywords

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

31 (Actual)

8. Arms, Groups, and Interventions

Arm Title

0.3 mg/kg

Arm Type

Experimental

Arm Description

IV infusion of migalastat HCl or placebo

Arm Title

1 mg/kg

Arm Type

Experimental

Arm Description

IV infusion of migalastat HCl or placebo

Arm Title

10 mg/kg

Arm Type

Experimental

Arm Description

IV infusion of migalastat HCl or placebo

Arm Title

150 mg IV

Arm Type

Experimental

Arm Description

150 mg single IV infusion

Arm Title

150 mg oral

Arm Type

Experimental

Arm Description

150 mg single oral dose

Intervention Type

Drug

Intervention Name(s)

IV migalastat HCl

Other Intervention Name(s)

AT1001

Intervention Type

Drug

Intervention Name(s)

IV placebo

Other Intervention Name(s)

Sodium Chloride 0.9%

Intervention Type

Drug

Intervention Name(s)

oral migalastat HCl

Other Intervention Name(s)

AT1001

Primary Outcome Measure Information:

Title

Plasma pharmacokinetics of migalastat

Description

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

Title

Safety and tolerability of migalastat

Description

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

Time Frame

48 hours

Secondary Outcome Measure Information:

Title

Plasma pharmacokinetics of migalastat

Description

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

Title

Urinary pharmacokinetics

Description

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

45 Years

Accepts Healthy Volunteers

Eligibility Criteria

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Medical Monitor Clinical Research

Organizational Affiliation

Amicus Therapeutics

Official's Role

Study Director

Facility Information:

Facility Name

PRA International

City

Groningen

ZIP/Postal Code

9713 GZ

Country

Netherlands

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs