An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease

CompletadoFase 3ClinicalTrials.gov

ID: NCT02194985Tipo: INTERVENTIONALInicio: 14 de mar de 2015Fin estimado: 23 de oct de 2019

Traducción no disponible, mostrando original

Resumen

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.

Elegibilidad

Edad mínima: 18 YearsSexo: ALL

Criterios de inclusión

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)

Criterios de exclusión

Participant required treatment with Glyset® (miglitol) or Zavesca® (miglustat)
Participants with severe or unsuitable concomitant medical condition
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)
Participants with clinically significant abnormal laboratory value(s) and/or clinically significant electrocardiogram (ECG) findings

Intervenciones

drug

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.

Ubicaciones

Clinical Study Site

Pilar, Argentina

Patrocinadores

PrincipalAmicus Therapeutics

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