A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

Running, not enrolling · Phase 3

Conditions studied: Fabry Disease

In brief

An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects with Fabry Disease and Amenable GLA Variants and Severe Renal Impairment (SRI) or End Stage Renal Disease (ESRD)

Key facts

Study ID: NCT04020055
Run by: Amicus Therapeutics
People needed: 14
Starts: 2022-10-31
Expected to finish: 2026-12-31
Last updated by the study team: 2026-07-06

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Male or female subjects aged 18 years or older, diagnosed with Fabry disease.
Subject (or legally authorized representative as applicable) is willing and able to provide written informed consent and authorization for use and disclosure of Personal Health Information
Subject has a GLA variant that is amenable to migalastat recorded in their medical records
Subject has at least 1 documented eGFR value of < 30 mL/min/1.73 m2 within the last 3 months and has an eGFRMDRD value of < 30 mL/min/1.73 m2 at Visit 1
Subjects with ESRD have been on a stable 2- or 3-times a week HD (standard or HDF) regimen for at least 2 months prior to the screening visit
Subjects with ESRD must commit to completing at least 4 standard HD or HDF sessions during each 2-week dosing interval.
Subjects with ESRD must commit to completing the entire prescribed duration for each dialysis session.
If of reproductive potential, both male and female patients agree to use a medically accepted method of contraception

You may not qualify if…

Subject has undergone kidney transplantation
Subject is on peritoneal dialysis
Subject is treated or has been treated with another investigational drug (except migalastat) within the 30 days
Subject has undergone any gene therapy at any time prior to the study or anticipates undergoing gene therapy during the study.
Subject has had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction
Subject has clinically significant unstable cardiac disease
Subject has any intercurrent illness or condition that may preclude the subject from fulfilling the protocol requirements
Subject has a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol)
Subject requires concurrent treatment with Glyset® (miglitol), Replagal® (agalsidase alfa), or Fabrazyme® (agalsidase beta)
Subject requires concurrent treatment with Zavesca® (miglustat) or has been treated with Zavesca
Female subject is pregnant or breast-feeding
Subject is unable to comply with study requirements
In France only, protected persons as defined by the Public Health Code

Where it is running

Emory University — Atlanta, Georgia, United States
The Cleveland Clinic — Cleveland, Ohio, United States
Lysosomal and Rare Disorders Research and Treatment Center, Inc — Fairfax, Virginia, United States
Royal Melbourne Hospital — Parkville, Victoria, Australia
Royal Perth Hospital — Perth, Washington, Australia
Osaka University Hospital — Suita, Osaka, Japan
Shizuoka General Hospital — Shizuoka, Shizuoka, Japan
Centro Hospitalar e Universitário de Coimbra (CHUC) — Coimbra, Portugal
Hospital Universitari(o) de Bellvitge (HUB) Feixa Llarga — Barcelona, Spain
Hospital General Universitario de Elda — Elda, Spain
Hospital General Universitario Gregorio Marañon — Madrid, Spain
Salford Royal Hospital — Salford, England, United Kingdom

Full record on ClinicalTrials.gov

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