Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruitingRare disease

Study on the Safety of Migalastat for Patients with Fabry Disease and Severe Kidney Problems on Dialysis

Verified siteRegistered drugNo placebo
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Fabry disease, a rare genetic disorder that affects the body's ability to break down a specific type of fat, leading to a buildup that can cause various health issues. The study is particularly interested in patients with severe kidney problems, including those with end-stage kidney disease who are undergoing hemodialysis, a treatment that helps filter waste from the blood when the kidneys are not functioning properly.

The treatment being tested in this study is a medication called migalastat, which is taken in the form of hard capsules. The purpose of the study is to evaluate the safety and how the body processes this medication in patients with Fabry disease who have severe kidney impairment. The study will help determine the appropriate dosing of migalastat for these patients.

Participants in the study will take migalastat and undergo regular monitoring to assess how their bodies handle the medication. This will involve checking the levels of migalastat in their blood and urine. The study will also monitor for any side effects and changes in health indicators such as blood pressure, heart rate, and body temperature. The study aims to provide valuable information on the use of migalastat in patients with Fabry disease and significant kidney issues.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial visit and consent

    Upon joining the study, you will attend an initial visit. During this visit, you will be asked to provide written informed consent. This means you agree to participate in the study and allow the use of your health information for research purposes.

    Your medical history will be reviewed to confirm your diagnosis of Fabry disease and the presence of a GLA variant that is suitable for treatment with migalastat.

  2. Step 2

    Genetic testing

    If you have a known GLA variant, confirmatory genetic testing will be conducted during the first visit. If your GLA variant is not known, genetic testing will be performed, and results must be received before the second visit.

  3. Step 3

    Baseline assessments

    Baseline assessments will be conducted to evaluate your health status. These assessments include laboratory tests, vital signs, and a physical examination.

    Your kidney function will be assessed through a test called eGFR (estimated glomerular filtration rate).

  4. Step 4

    Medication administration

    You will begin taking Galafold 123 mg hard capsules, which contain the active substance migalastat. The medication is taken orally.

    The dosing regimen will be based on your specific health condition and will be confirmed during the study.

  5. Step 5

    Ongoing assessments

    Throughout the study, regular assessments will be conducted to monitor your health and the effects of the medication. These include laboratory tests, vital signs, and physical examinations.

    Your kidney function will be monitored regularly, and you will be required to complete dialysis sessions if you have end-stage renal disease.

  6. Step 6

    Safety monitoring

    The study will monitor for any side effects or adverse events. This includes changes in laboratory parameters, vital signs, and any new symptoms you may experience.

    You will be asked to report any side effects or concerns to the study team promptly.

  7. Step 7

    Study completion

    Upon completion of the study, a final assessment will be conducted to evaluate your overall health and the effects of the medication.

    You will be provided with information about the study results and any necessary follow-up care.

Who can join the trial?

8 criteria

  • Male or female subjects aged 18 years or older, diagnosed with Fabry disease.
  • Subject is willing and able to provide written informed consent, which means they agree to participate in the study and allow the use of their health information for research. If the subject cannot provide consent, a legally-authorized representative can do so, and the subject must agree if possible.
  • Subject has a GLA variant in their medical records that can be treated with the study medication, migalastat. This will be confirmed with a genetic test at the first visit. If the subject's GLA variant is not known, the test results must be available before the second visit.
  • Subject has a documented eGFR (a measure of kidney function) value of less than 30 mL/min/1.73 m² in their medical history within the last 3 months and at the first visit, or the subject is on hemodialysis (HD), which is a treatment for kidney failure.
  • Subjects with end-stage renal disease (ESRD) must have been on a stable hemodialysis schedule, either 2 or 3 times a week, for at least 2 months before the first visit.
  • Subjects with ESRD must agree to complete at least 4 standard hemodialysis sessions during each 2-week dosing period.
  • Subjects with ESRD must agree to complete the entire prescribed duration for each dialysis session.
  • If the subject can have children, both male and female subjects must agree to use a medically accepted method of birth control during the study and for up to 30 days after the last dose of migalastat.

Who cannot join the trial?

8 criteria

  • Patients who do not have **Fabry Disease** with specific genetic changes (GLA variants) cannot participate.
  • Patients who do not have **severe kidney problems** or are not on **hemodialysis** (a treatment for kidney failure) cannot participate.
  • Patients who are not in the specified age range for the study cannot participate.
  • Patients who are not willing or able to follow the study procedures cannot participate.
  • Patients who have other medical conditions that might interfere with the study cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who are participating in another clinical trial cannot participate.
  • Patients who have a history of allergic reactions to the study medication cannot participate.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

Migalastat HCl is a medication used in this clinical trial to help people with Fabry disease. Fabry disease is a genetic condition that can cause a buildup of certain substances in the body, leading to problems with the heart, kidneys, and other organs. Migalastat works by helping the body break down these substances more effectively. In this study, the medication is being tested to see how it behaves in the body and to ensure that the dosing is correct for patients who have severe kidney problems or are undergoing hemodialysis, which is a treatment that helps clean the blood when the kidneys are not working well.

What is already known about the treatment

Migalastat HCl – Migalastat HCl is administered orally and is currently being studied for its safety and how it moves through the body in people with Fabry disease who have specific genetic variants and severe kidney problems, including those on dialysis. This medication is in the clinical trial phase, and researchers are working to confirm the best dosing regimens. It is primarily used to treat Fabry disease, a rare genetic disorder. At the molecular level, migalastat works by stabilizing certain enzymes that are deficient in people with Fabry disease, helping them function more effectively. It is classified as a pharmacological chaperone, which means it helps proteins fold correctly to perform their intended functions.

Investigated diseases

Fabry Disease – Fabry Disease is a genetic disorder caused by the buildup of a particular type of fat, called globotriaosylceramide, in the body's cells. This buildup occurs due to a deficiency of the enzyme alpha-galactosidase A. The disease progresses as these fats accumulate, leading to symptoms such as pain, skin rashes, and problems with the heart and kidneys. Over time, the accumulation can cause significant damage to organs, particularly the kidneys, heart, and nervous system. The progression of the disease can vary widely among individuals, with some experiencing severe symptoms early in life and others having milder symptoms that develop later. The disease is inherited in an X-linked manner, meaning it primarily affects males, but females can also be affected.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2022-500488-10-00Protocol codeAT1001-025Estimated enrolment15 patientsSponsorAmicus Therapeutics Inc.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.