Who is eligible for clinical trial NCT04020055?

Participants must meet protocol criteria for Fabry Disease. Confirm exact inclusion and exclusion requirements with the study coordinator.

Does clinical trial NCT04020055 provide travel or cost reimbursement?

Check with the site coordinator at the participating facility for travel and lodging stipend availability, and for which routine-care costs are covered.

🏛️ Verified NIH Record • Updated 2026-07-02

A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease (NCT04020055)

🔵 Active, Not Recruiting Phase3 💊 Investigational Drug / Biologic Sponsor: Amicus Therapeutics

🩺 Plain-English Summary (8th-Grade Level)

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)

Who can join: To join this study, participants generally need to meet the listed inclusion criteria (8 items) and avoid the listed exclusions (14 items).

Location: Emory University — Atlanta, Georgia

📋 "Do I Qualify?" — 1-Minute Self Screener

Instant Checklist

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

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

Usually NOT eligible if any of the following apply:

Exclusion Criteria:
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

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (12 Sites)

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

💰 Cost, Insurance & Patient Rights

100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

🏛️ View Official NIH Record ↗