Active not recruitingPHASE3INTERVENTIONAL

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

This research is testing a medicine called migalastat for people living with Fabry disease. It's specifically for those whose Fabry disease is linked to certain genetic changes that migalastat can help, and who also have significant kidney problems. The study will look at two groups: one with severe kidney damage who are not on dialysis, and another group who are already receiving dialysis treatment. The main goals are to understand how safe migalastat is and how the body processes it in these situations. This is an 'open-label' study, meaning both patients and doctors will know they are receiving migalastat. It's an important step to gather more information about using this treatment in people with advanced kidney disease tied to Fabry disease.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Amicus Therapeutics

Enrolment target

Start

31 Oct 2022

Estimated completion

31 Dec 2026

Fabry Disease

What is this study about?

This study is about a medicine called migalastat, used for a rare genetic condition called Fabry disease. Fabry disease can cause lots of health problems, including serious damage to the kidneys. This study is specifically for people with Fabry disease who have certain genetic changes that migalastat is known to work for, and who also have severe kidney problems.

We want to find out more about how safe migalastat is and how the body handles it in people whose kidneys are severely affected. This includes people with very advanced kidney disease who are not yet on dialysis, and also those who are already receiving dialysis treatment. We hope this study will give us valuable information to help doctors best support people with Fabry disease and kidney issues.

It's important to understand that this is an 'open-label' study. This means everyone involved – the patients and the doctors – will know that migalastat is being given. There isn't a comparison group receiving a dummy pill or a different medicine in this particular study.

Key takeaways

This study is for adults with a specific type of Fabry disease and severe kidney problems.
It tests a medicine called migalastat to check its safety and how it works in people with kidney damage or on dialysis.
Participation involves taking migalastat and having regular check-ups.
You cannot join if you've had a kidney transplant or are on peritoneal dialysis.
This is an 'open-label' study, so everyone knows you're receiving migalastat.

Who may be eligible?

To join this study, you need to be an adult (18 years or older) with a diagnosis of Fabry disease. Your doctors must have noted that your specific type of Fabry disease, based on your genetic test results, is one that migalastat can help. You also need to have severe kidney problems, meaning your kidney function is quite low, as measured by a specific kidney test within the last three months and again at the start of the study.

If you have very severe kidney disease and are on dialysis, you might still be able to join. However, you would need to be on a regular dialysis schedule (2 or 3 times a week) for at least two months before starting the study. It's also important that you can receive your dialysis sessions at the study clinic or a nearby centre that the study doctor works with, and that you commit to completing all your prescribed dialysis sessions.

Unfortunately, you can't join if you've already had a kidney transplant, or if you're on a different type of dialysis called peritoneal dialysis. You also can't be taking other experimental drugs, or certain specific Fabry disease medications. If you've had certain heart problems recently, or have serious unstable heart or other medical conditions, or a history of allergic reactions to migalastat or similar drugs, you wouldn't be able to take part.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years or older with Fabry disease?
Has your doctor confirmed your Fabry disease type can be treated with migalastat?
Do you have severe kidney problems (eGFR below 30)?
If on dialysis, are you on hemodialysis 2-3 times a week for at least 2 months?
Have you NOT had a kidney transplant or been on peritoneal dialysis?
Are you NOT taking other study drugs or certain Fabry medications currently?

Answer every question to see your result.

What does participation involve?

If you decide to take part, your first step will be a screening visit to check if you're suitable for the study. This will likely involve confirming your Fabry disease type and your kidney function. If you meet all the requirements, you'll have a main starting visit within a month. During this study, you'll be taking migalastat as prescribed.

For those on dialysis, special arrangements will be made to ensure your dialysis sessions can happen at the study clinic or an affiliated centre, and you'll need to commit to attending all your scheduled dialysis sessions. The study will involve regular check-ups and assessments to monitor your health, how the medicine is working, and to ensure your safety. The total duration of your participation will be explained to you during the consent process.

Potential risks and benefits

Participating in this study might offer a potential benefit by giving you access to migalastat, which could help manage your Fabry disease, especially if you have significant kidney involvement. However, like all medicines, migalastat can have side effects, and some tests or procedures during the study might have their own small risks. The study team will carefully monitor your health throughout. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (12)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Emory University
Verified postcode
Atlanta, United States
The Cleveland Clinic
Verified postcode
Cleveland, United States
Lysosomal and Rare Disorders Research and Treatment Center, Inc
Verified postcode
Fairfax, United States
Royal Melbourne Hospital
Verified postcode
Parkville, Australia
Royal Perth Hospital
Verified postcode
Perth, Australia
Osaka University Hospital
Verified postcode
Suita, Japan
Shizuoka General Hospital
Verified postcode
Shizuoka, Japan
Centro Hospitalar e Universitário de Coimbra (CHUC)
Verified postcode
Coimbra, Portugal
Hospital Universitari(o) de Bellvitge (HUB) Feixa Llarga
Verified postcode
Barcelona, Spain
Hospital General Universitario de Elda
Verified postcode
Elda, Spain
Hospital General Universitario Gregorio Marañon
Verified postcode
Madrid, Spain
Salford Royal Hospital
Verified postcode
Salford, United Kingdom

Common questions

What is Fabry disease?

Fabry disease is a rare inherited condition that can affect many parts of the body, including the kidneys, heart, and skin, because the body can't break down a certain type of fat properly.

What is migalastat?

Migalastat is a medication used to treat certain types of Fabry disease where the specific genetic change allows the drug to help the body's own enzymes work better.

Why is this study only for people with kidney problems?

This study is specifically looking at how migalastat works and its safety in people with Fabry disease who also have severe kidney damage, including those on dialysis, as there's less information about how the drug behaves in these situations.

What does 'open-label' mean?

Open-label means that everyone involved in the study – both the patient and the doctors – will know that the patient is receiving the study medication, migalastat.

Will I have to pay to be in the study?

No, you will not have to pay for the study medication or any study-related tests and appointments. Travel costs may also be reimbursed.