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

Amicus Therapeutics

Status and phase

Completed

Phase 3

Conditions

Fabry Disease

Treatments

Drug: migalastat HCl 150 mg

Study type

Interventional

Funder types

Industry

Identifiers

NCT02194985

AT1001-042

2014-002701-38 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment

84 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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