Not yet recruitingRare disease

Efficacy and Safety of Lucerastat in Treatment‑Naïve Adult Male Patients with Fabry Disease

Verified siteInvestigationalNo placebo

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What is this trial about?

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

The study focuses on Fabry disease, a rare inherited condition that causes a fatty substance called Gb3 to build up in organs such as the kidneys, heart, and skin. The investigational medicine being tested is lucerastat, an oral capsule taken twice daily.

The purpose of the study is to learn how well the medicine works and how safe it is in adult men who have not received prior treatment for the condition. Participants will take the study drug every day for about a year and a half, with regular clinic visits where blood samples, urine samples, and simple kidney checks are performed to follow any changes.

The main goal is to see whether the amount of the fatty substance in the tiny blood vessels of the kidney decreases over time, and a secondary goal is to see if the level of the substance in the blood changes. Throughout the trial, doctors will monitor participants for any side effects and will collect information to assess both effectiveness and safety.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Baseline screening and assessments
You will attend an initial visit after joining the study. during this visit, the research team will confirm that you have Fabry disease and have not received prior treatment for it.
The team will perform measurements of kidney health, including a test that counts the number of Gb3 (a fatty substance that can accumulate in the kidneys) inclusions in kidney capillaries. a blood sample will also be taken to measure the amount of Gb3 in the plasma.
Step 2
Receive study medication
You will be given the investigational drug lucerastat in the form of hard capsules.
Each dose contains 2000 mg of the active substance and is intended for oral use.
Step 3
Start daily dosing
You will take the prescribed dose of lucerastat once each day, swallowing the capsule with water.
The medication should be taken consistently for the entire study period.
Step 4
Regular follow‑up visits
You will return to the study site at scheduled times to allow the team to check your safety and to repeat the kidney and blood measurements.
Typical visit times include approximately month 1, month 6, month 12, and month 18 after you start the medication.
Step 5
Continue treatment for 18 months
You will keep taking the daily dose of lucerastat for a total of 18 months.
During this period, you will continue the scheduled follow‑up visits and any additional safety checks that the study requires.
Step 6
Final assessment at month 18
At the 18‑month visit, the team will repeat the kidney Gb3 scoring and the blood Gb3 measurement to determine how the treatment has affected the disease.
The results from this final assessment will be compared with the baseline measurements taken at the first visit.

Who can join the trial?

6 criteria

Have a confirmed diagnosis of Fabry Disease, shown by either (a) plasma or white‑blood‑cell α‑galactosidase A enzyme activity less than 1% of normal, or (b) a known disease‑causing change in the GLA gene that also results in low enzyme activity (less than 30% of normal). α‑galactosidase A is a protein that helps break down certain fats in the body.
Have had at least one of these common signs of Fabry Disease: nerve pain (called neuropathic pain), a swirl‑like pattern on the surface of the eye (cornea verticillata), or small dark skin spots (angiokeratoma).
Be treatment‑naïve or pseudo‑naïve, meaning you have not taken any approved or experimental therapy for Fabry Disease for at least six months before the screening visit.
Have a blood level of plasma globotriaosylsphingosine that is 20 ng/ml or higher, as measured by a central laboratory. This substance builds up in Fabry Disease and is used to assess disease activity.
Have a kidney function test called estimated glomerular filtration rate (eGFR) of 45 mL/min/1.73 m² or higher, measured by a central laboratory. eGFR estimates how well the kidneys are filtering waste from the blood.
Be an adult male participant.

Who cannot join the trial?

12 criteria

Any current illness or medication that makes a kidney biopsy unsafe or could make the study results unclear.
Any condition that might stop you from following the study plan, such as drug or alcohol dependence, or mental‑health problems like moderate depression (a score > 20 and ≤ 28 on a standard questionnaire) if you do not agree to see a mental‑health professional, severe depression (score > 28), suicidal thoughts, or a suicide attempt in the past year.
Having received gene or cell therapy before.
Using certain medicines called cationic amphiphilic drugs (for example, amiodarone or hydroxychloroquine) within the last 6 months, because they can affect kidney biopsy results.
Having a urine test that shows an albumin‑to‑creatinine ratio higher than 300 mg/g at screening, unless you are already taking blood‑pressure medicines such as ACE inhibitors, ARBs, or SGLT2 inhibitors.
Having a bleeding or clotting problem, such as a low platelet count, an international normalized ratio (INR) greater than 1.5, or being unable to stop blood‑thinning medicines for the required time.
Having a hemoglobin level lower than 9.0 g/dL (a measure of the blood’s ability to carry oxygen).
Having had a sudden kidney injury in the past 12 months.
Having poorly controlled diabetes, defined as a Hemoglobin A1c greater than 8.0% (a test that shows average blood‑sugar levels over the last three months).
Having experienced a stroke, mini‑stroke (transient ischemic attack), heart attack, unstable chest pain, heart surgery, or a heart procedure (such as stent placement) within the past 6 months.
Having severe heart failure (New York Heart Association class IV) or being hospitalized for heart failure in the past 3 months.
Having a cardiac device implanted (such as a pacemaker, implantable cardioverter‑defibrillator, or cardiac resynchronization therapy device) or being hospitalized for an irregular heartbeat within the past 6 weeks.

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Verified sites

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

Charite Universitaetsmedizin Berlin KöR
Verified
Berlin, Germany
Oncopole Claudius Regaud
Verified
Toulouse, France
Raymond-Poincare Hospital
Verified
Garches, France

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Investigated drugs

Lucerastat is an oral capsule taken by mouth that is being studied as a new treatment for Fabry disease in adult men who have not received previous therapy. It is designed to help lower the amount of a fatty substance called globotriaosylceramide (Gb3) that builds up in the kidneys of people with Fabry disease. By reducing this buildup, the drug aims to protect kidney function and improve overall health. This medication is classified as an orphan drug, meaning it is intended for a rare condition and receives special support for development.

Lucerastat

What is already known about the treatment

Lucerastat - Lucerastat is taken as a hard capsule by mouth, typically 2000 mg each day. It is an orphan drug that is still under clinical investigation and has not yet received full market approval. The medicine is being studied for Fabry disease, a rare condition where a fatty substance builds up in the kidneys and other organs. It works by blocking a key enzyme in the pathway that creates the buildup, lowering the amount of the harmful substance, and it is classified as a substrate reduction therapy.

Investigated diseases

Fabry disease - Fabry disease is a rare inherited disorder caused by a deficiency of the enzyme alpha‑galactosidase A, which leads to buildup of a fatty substance called globotriaosylceramide in many body cells. The excess material gradually accumulates in blood vessels and organ tissues, causing pain in the hands and feet, small dark skin spots, and reduced sweating. Over time the storage material can affect the kidneys, heart, and brain, leading to progressive loss of kidney function, heart muscle thickening, and small strokes. The disease typically begins in childhood or adolescence and worsens slowly as more material is stored. Because the underlying enzyme defect is present from birth, the pathological changes continue unless intervened.