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New hope for fabry patients with kidney failure: drug dosing study underway

NCT ID NCT04020055

Disease control Sponsor: Amicus Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 07, 2026 · Updated 2 times

Summary

This study tests the safety and how the body processes migalastat in 14 adults with Fabry disease who have severe kidney impairment or are on dialysis. Participants take migalastat capsules, and researchers measure drug levels in blood and dialysate. The goal is to ensure proper dosing for this vulnerable group.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
migalastat (a drug that helps break down fatty substances in cells)
What this could lead to
If successful, this could confirm that migalastat is safe and properly dosed for Fabry patients with severe kidney problems, expanding treatment options for this group.
What could go wrong
This is a small, early-stage study (14 people) focused on safety and drug levels, not on whether the drug improves symptoms. It may not lead to a new treatment if safety issues arise.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 14 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2022

Expected to finish

Dec 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

chronic kidney disease Fabry disease kidney disorder kidney failure

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Fabry disease

Contacts and locations

Locations

  • Centro Hospitalar e Universitário de Coimbra (CHUC)

    Coimbra, 3041-801, Portugal

  • Emory University

    Atlanta, Georgia, 30322, United States

  • Hospital General Universitario Gregorio Marañon

    Madrid, 28007, Spain

  • Hospital General Universitario de Elda

    Elda, 03600, Spain

  • Hospital Universitari(o) de Bellvitge (HUB) Feixa Llarga

    Barcelona, 08907, Spain

  • Lysosomal and Rare Disorders Research and Treatment Center, Inc

    Fairfax, Virginia, 22030, United States

  • Osaka University Hospital

    Suita, Osaka, 565-0871, Japan

  • Royal Melbourne Hospital

    Parkville, Victoria, 3050, Australia

  • Royal Perth Hospital

    Perth, Washington, 6000, Australia

  • Salford Royal Hospital

    Salford, England, M6 8HD, United Kingdom

  • Shizuoka General Hospital

    Shizuoka, Shizuoka, 420-8527, Japan

  • The Cleveland Clinic

    Cleveland, Ohio, 44195, United States

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