🏛️ Verified NIH Record • Updated 2026-07-02
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease (NCT04020055)
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 or End-Stage Renal Disease Treated With Hemodialysis
🔵 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: Review the eligibility criteria below.
Location: Emory University — Atlanta, Georgia
Age range: 18 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 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.
More studies in Fabry Disease
- A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease — Birmingham, Alabama
- A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease — Birmingham, Alabama
- Long-Term Follow-up of Subjects Who Were Treated With ST-920 — Irvine, California
- An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease — La Jolla, California
- A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease — Birmingham, Alabama
Explore by condition: All Fabry Disease clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.