Evaluating Migalastat for Fabry Disease Patients with Kidney Issues

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

This study is testing if migalastat can be safe and effective for people with Fabry disease who have serious kidney problems, whether they are on dialysis or not.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 14 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Amicus Therapeutics (industry)
• Locations: 13 sites (Atlanta, Georgia and 12 other locations)
• Trial ID: NCT04020055 on ClinicalTrials.gov

What this trial studies

This open-label, non-comparative study aims to evaluate the safety and pharmacokinetics of migalastat HCl in patients with Fabry disease who have severe renal impairment or are in end-stage renal disease. Participants will be divided into two cohorts: those not on dialysis and those receiving hemodialysis. The study will involve screening for eligibility, including GLA genotyping, followed by a baseline visit for those who qualify. The primary focus is on patients with an estimated glomerular filtration rate (eGFR) of less than 30 mL/min/1.73 m².

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older diagnosed with Fabry disease and having a GLA variant amenable to migalastat.

Not a fit: Patients with an eGFR greater than 30 mL/min/1.73 m² will not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new treatment option for patients with Fabry disease and significant renal impairment.

How similar studies have performed: Previous studies have shown promise with migalastat in treating Fabry disease, indicating potential for success in this specific population.

Eligibility criteria

Inclusion Criteria:

1. Male or female subjects aged 18 years or older, diagnosed with Fabry disease.
2. 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
3. Subject has a GLA variant that is amenable to migalastat recorded in their medical records
4. 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
5. 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
6. Subjects with ESRD must commit to completing at least 4 standard HD or HDF sessions during each 2-week dosing interval.
7. Subjects with ESRD must commit to completing the entire prescribed duration for each dialysis session.
8. If of reproductive potential, both male and female patients agree to use a medically accepted method of contraception

Exclusion Criteria:

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

Where this trial is running

Atlanta, Georgia and 12 other locations

• Emory University — Atlanta, Georgia, United States (RECRUITING)
• The Cleveland Clinic — Cleveland, Ohio, United States (RECRUITING)
• Renal Disease Research Institute — Dallas, Texas, United States (WITHDRAWN)
• Lysosomal and Rare Disorders Research and Treatment Center, Inc — Fairfax, Virginia, United States (RECRUITING)
• Royal Melbourne Hospital — Parkville, Victoria, Australia (RECRUITING)
• Royal Perth Hospital — Perth, Washington, Australia (RECRUITING)
• Internal Medicine Unit Croix Saint Simon Hospital — Paris, France (WITHDRAWN)
• Osaka University Hospital — Suita, Osaka, Japan (RECRUITING)
• Centro Hospitalar e Universitário de Coimbra (CHUC) — Coimbra, Portugal (RECRUITING)
• Hospital Universitari(o) de Bellvitge (HUB) Feixa Llarga — Barcelona, Spain (RECRUITING)
• Hospital Universitario Reina Sofia — Córdoba, Spain (WITHDRAWN)
• Hospital General Universitario de Elda — Elda, Spain (RECRUITING)
• Hospital General Universitario Gregorio Marañon — Madrid, Spain (RECRUITING)

Study contacts

• Study coordinator: Amicus Therapeutics Patient Advocacy

Conditions: Fabry Disease, Renal Disease, Severe Renal Impairment, End-Stage Renal Disease