A multicenter, open-label, single-arm, baseline-controlled trial to assess the efficacy and safety of lucerastat in treatment-naïve/pseudo-naïve adult male participants with Fabry disease
Fabry disease is a rare genetic condition where the body cannot properly break down a fatty substance known as Gb3. Over time, this fat builds up in blood vessels and organs, particularly the kidneys and heart, which can cause long-term damage. This Phase 3 clinical trial is testing a new oral medicine called lucerastat. It is designed for adult men with Fabry disease who have never received treatment before, or who have not had treatment for a long time. In this study, there is no dummy pill (placebo). Every participant will receive lucerastat. Doctors will monitor participants over 18 months using blood tests and kidney checks to see if the medicine reduces fat buildup in kidney cells and helps protect kidney health. The study also closely tracks safety and side effects.
At a glance
What is this study about?
Fabry disease is an inherited condition caused by a faulty gene. People with the condition do not produce enough of a vital enzyme that clears away a fatty substance called Gb3. Without this enzyme, the fat gradually accumulates in cells throughout the body. It especially affects the blood vessels in the kidneys, heart, and nervous system, leading to pain, kidney problems, and other serious health complications over time.
Current standard treatments usually involve regular hospital drips to replace the missing enzyme. This trial is investigating a different approach using a medicine called lucerastat. Instead of replacing the enzyme, lucerastat is designed to slow down the production of the fatty substance in the first place, helping to prevent it from piling up inside organs.
Because this is a Phase 3 trial, researchers are gathering detailed evidence to confirm how well lucerastat works and how safe it is over 18 months. The main goal is to measure whether the amount of fat trapped in tiny kidney blood vessels decreases. Finding effective new treatments gives people with Fabry disease more options to protect their organs and manage their condition in everyday life.
Key takeaways
- Phase 3 trial testing an oral medicine called lucerastat for Fabry disease.
- Open to adult men who have not recently received standard Fabry treatments.
- Aims to reduce fatty deposits (Gb3) in the kidneys over 18 months.
- All participants receive the study medicine; there is no placebo.
- Involves regular clinic visits, blood tests, and kidney health monitoring.
Who may be eligible?
This study is open to men aged 18 and older who have a confirmed diagnosis of Fabry disease.
To take part, you must be either 'treatment-naïve' or 'pseudo-naïve'. This means you have either never had specific Fabry disease treatments (such as enzyme replacement therapy) or you have been off these treatments for an agreed period before joining.
You will need to be able to attend regular hospital check-ups and be willing to undergo the required health checks, which include blood tests and specialised kidney assessments. Your study doctor will review your medical history and current health to confirm if this trial is safe and suitable for you.
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.
- I am a man aged 18 or older.
- I have been diagnosed with Fabry disease.
- I have never had enzyme replacement therapy, or have been off it for a required period.
- I am willing to take study medicine by mouth every day.
- I am able to attend regular hospital appointments over an 18-month period.
What does participation involve?
If you take part, you will be in the study for at least 18 months. You will take the study medicine, lucerastat, by mouth as instructed by the medical team. Because this is an 'open-label' study, you will know you are receiving the active drug, as there is no placebo.
You will need to visit the study clinic regularly. During these visits, doctors will check your overall health, monitor your heart and blood pressure, and take blood and urine samples. To see if the medicine is clearing fat from your kidneys, you will have specialised kidney tests, which may include a kidney biopsy at the start and after 18 months. The trial team will support you throughout the study and keep close track of any symptoms or side effects.
Potential risks and benefits
Locations (2)
- —UnverifiedGermany
- —UnverifiedFrance
Common questions
Will I receive a placebo (dummy pill)?
No. This is an open-label, single-arm study, which means every person who takes part receives the active medicine, lucerastat.
How is lucerastat taken?
Lucerastat is an oral medicine, meaning you take it by mouth rather than having it as a drip into a vein.
Why is the study only open to men?
Fabry disease is linked to the X chromosome, so men often experience more consistent and severe fatty buildup in kidney cells. This makes it easier for scientists to measure clear changes during this stage of research.
Does taking part cost anything?
No. The study medication and all trial-related tests are provided free of charge, and travel expenses are usually reimbursed.
What happens if I decide to leave the trial early?
You can leave the study at any point without giving a reason. Your standard medical care will continue as normal.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.