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Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease

NCT ID: NCT02194985

Last Updated: 2020-12-21

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

84 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-03-14

Study Completion Date

2019-10-23

Brief Summary

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This is an open-label extension study intended to provide continued treatment with migalastat hydrochloride (HCl) for participants with Fabry disease who completed treatment of a previous migalastat HCl study. The study assessed the long-term safety and effectiveness of migalastat HCl.

Detailed Description

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Conditions

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Fabry Disease

Keywords

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Amicus Therapeutics Galafold Migalastat AT1001

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Migalastat HCl 150 mg

Migalastat HCl 150 milligram (mg).

Group Type EXPERIMENTAL

migalastat HCl 150 mg

Intervention Type DRUG

Migalastat HCl 150 mg (equivalent to 123 mg migalastat) was provided as capsules in blister packs. One capsule was taken orally every other day.

Interventions

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migalastat HCl 150 mg

Migalastat HCl 150 mg (equivalent to 123 mg migalastat) was provided as capsules in blister packs. One capsule was taken orally every other day.

Intervention Type DRUG

Other Intervention Names

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AT1001 Galafold

Eligibility Criteria

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Inclusion Criteria

* Participant had completed treatment in a previous study of migalastat HCl given as a monotherapy
* Male and female participant agreed to use protocol-identified acceptable contraception
* Participant was willing to provide written informed consent and authorization for use and disclosure of Personal Health Information (PHI)

Exclusion Criteria

* Participant's last available estimated glomerular filtration rate (eGFR) in the previous study was \<30 milliliter (mL)/minute (min)/1.73 meters squared (m\^2); unless there was measured GFR available within 3 months of Baseline Visit, which was \>30 mL/min/1.73 m\^2
* Participant had undergone, or was scheduled to undergo kidney transplantation or was currently on dialysis
* Participant had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 3 months before Baseline Visit
* Participant had clinically significant unstable cardiac disease in the opinion of the investigator (for example, cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or New York Heart Association class III or IV congestive heart failure)
* Participant had a history of allergy or sensitivity to AT1001 (including excipients) or other iminosugars (for example, miglustat, miglitol)
* Participant required treatment with Glyset® (miglitol) or Zavesca® (miglustat)
* Participants with severe or unsuitable concomitant medical condition
* Participants with clinically significant abnormal laboratory value(s) and/or clinically significant electrocardiogram (ECG) findings
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Amicus Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Monitor Clinical Research

Role: STUDY_DIRECTOR

Amicus Therapeutics

Locations

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Clinical Study Site

Atlanta, Georgia, United States

Site Status

Clinical Study Site

Kansas City, Kansas, United States

Site Status

Clinical Study Site

Grand Rapids, Michigan, United States

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Clinical Study Site

New York, New York, United States

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Clinical Study Site

Portland, Oregon, United States

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Clinical Study Site

Dallas, Texas, United States

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Clinical Study Site

Fairfax, Virginia, United States

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Clinical Study Site

Pilar, , Argentina

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Clinical Study Site

Adelaide, , Australia

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Clinical Study Site

Parkville, , Australia

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Clinical Study Site

Vienna, , Austria

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Clinical Study Site

Edegem, , Belgium

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Clinical Study Site

Porto Alegre, , Brazil

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Clinical Study Site

Montreal, Quebec, Canada

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Clinical Study Site

Copenhagen, , Denmark

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Clinical Study Site

Cairo, , Egypt

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Clinical Study Site

Garches, , France

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Clinical Study Site

Lille, , France

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Clinical Study Site

Florence, , Italy

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Clinical Study Site

Roma, , Italy

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Clinical Study Site

Suita, Osaka, Japan

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Clinical Study Site

Niigata, , Japan

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Clinical Study Site

Osaka, , Japan

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Clinical Study Site

Tokyo, , Japan

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Clinical Study Site

Barcelona, , Spain

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Clinical Study Site

Ankara, , Turkey (Türkiye)

Site Status

Clinical Study Site

London, , United Kingdom

Site Status

Clinical Study Site

London, , United Kingdom

Site Status

Countries

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United States Argentina Australia Austria Belgium Brazil Canada Denmark Egypt France Italy Japan Spain Turkey (Türkiye) United Kingdom

References

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Narita I, Ohashi T, Sakai N, Hamazaki T, Skuban N, Castelli JP, Lagast H, Barth JA. Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study. Clin Exp Nephrol. 2020 Feb;24(2):157-166. doi: 10.1007/s10157-019-01810-w. Epub 2019 Dec 30.

Reference Type DERIVED
PMID: 31889231 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2014-002701-38

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

AT1001-042

Identifier Type: -

Identifier Source: org_study_id