Study on the Effects of Leriglitazone for Adult Men with Cerebral Adrenoleukodystrophy

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What is this study about?

This clinical trial is focused on studying a rare genetic disease called cerebral adrenoleukodystrophy (cALD), which affects the brain and is primarily found in adult males. The study will evaluate the effectiveness and safety of a medication called leriglitazone, which is taken as an oral suspension. Participants in the study will either receive leriglitazone or a placebo, which looks like the medication but does not contain the active ingredient.

The main goal of the study is to see if leriglitazone can help increase survival in patients with cALD. Participants will be monitored over a period of time to assess their health and any changes in their condition. The study will track important health outcomes, such as the time it takes for a participant to become bedridden or require permanent ventilatory support. Additionally, changes in brain function and quality of life will be evaluated.

Throughout the study, participants will undergo various assessments to measure the effects of leriglitazone. These assessments will include tests for cognitive function, physical abilities, and overall health status. The study aims to provide valuable information on whether leriglitazone can be a beneficial treatment option for individuals with cerebral adrenoleukodystrophy.

1 joining the study

Upon joining the study, you will be asked to provide written informed consent. This means you agree to participate after understanding the study details.

You must meet certain criteria, such as being an adult male with a confirmed diagnosis of cerebral adrenoleukodystrophy (cALD).

2 initial assessment

An initial assessment will be conducted to evaluate your current health status. This includes checking your brain lesions and other health parameters.

Your eligibility will be confirmed based on specific criteria, such as your Loes score, which measures the severity of brain changes.

3 medication administration

You will be randomly assigned to receive either the study drug leriglitazone or a placebo. A placebo looks like the study drug but does not contain the active substance.

The medication is taken orally as a suspension. The dosage and frequency will be explained to you by the study team.

4 regular follow-up visits

You will attend regular follow-up visits to monitor your health and the effects of the medication. These visits will include various tests and assessments.

The study team will check for any changes in your condition and adjust the treatment plan if necessary.

5 final assessment

At the end of the study period, a final assessment will be conducted to evaluate the overall effects of the treatment.

This assessment will include a review of your health status and any changes in your condition since the start of the study.

Who Can Join the Study?

The participant must be able to read and understand the informed consent form and must have signed it to join the study.
The participant must be a male who is at least 18 years old.
The participant must have a genetic confirmation of X-ALD (a specific genetic condition).
The participant must have progressive cALD (a condition affecting the brain), shown by specific brain changes called GdE+ brain lesions.
The participant should not be recommended for HSCT (a type of stem cell transplant) by the doctor, or the participant must not be willing to undergo this procedure.
The participant must have a Loes score (a way to measure brain changes) between 0.5 and 12 at the start of the study.
The participant should not have major functional disabilities, except for being “wheelchair bound” or having “total incontinence,” which are allowed.
The participant should not have major cognitive impairment, meaning they should be able to understand and participate in the study.
The participant must have normal adrenal function or be on appropriate steroid treatment if they have adrenal insufficiency (a condition where the adrenal glands do not produce enough hormones).
If the participant is not surgically sterilized, they must agree to use adequate contraception during sexual intercourse with a partner who is pregnant or could become pregnant, and not donate sperm from the first dose of the study drug until at least 90 days after the last dose. Adequate contraception includes using a diaphragm, cervical cap, or male condom, combined with hormonal contraceptives or an intrauterine device for a non-pregnant partner. Total abstinence is also acceptable.

Who Cannot Join the Study?

Patients who do not have cerebral adrenoleukodystrophy (cALD) cannot participate. This is a specific condition affecting the brain.
Only male patients are eligible to participate. Female patients cannot join this study.
Patients who are not within the specified age range cannot participate. The study is for certain age groups only.
Patients who belong to a vulnerable population are not eligible. This means people who might need special protection or care.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country	Status
Universitaet Leipzig	Leipzig	Germany

Other Sites

Site Name	City	Country	Status
Hopitaux Universitaires Pitie Salpetriere	Paris	France
Hospital Universitario 12 De Octubre	Madrid	Spain

Trial status

Country	Status	Recruitment Start
France	Recruiting	01.06.2025
Germany	Recruiting	01.06.2025
Spain	Recruiting	01.06.2025

Trial locations

Investigated drugs:

Leriglitazone

Leriglitazone is a medication being studied for its potential to help people with a condition called cerebral adrenoleukodystrophy. This condition affects the brain and can lead to serious health problems. The main goal of using leriglitazone in this study is to see if it can help increase the survival of patients with this condition. Researchers are looking at how effective and safe this medication is for adult male patients. By participating in this trial, patients will help determine if leriglitazone can be a beneficial treatment option for this serious condition.

Cerebral adrenoleukodystrophy (cALD) – Cerebral adrenoleukodystrophy is a genetic disorder that affects the brain and adrenal glands. It is characterized by the buildup of very long-chain fatty acids in the brain, which leads to the destruction of the myelin sheath, the protective covering of nerve cells. This process results in progressive neurological deterioration, affecting motor skills, speech, and cognitive functions. As the disease progresses, individuals may experience behavioral changes, vision and hearing loss, and difficulty with coordination and balance. The condition primarily affects males and typically begins in childhood. Over time, the neurological decline can lead to severe disability.

Trial ID:

2024-515104-39-00

Protocol code:

MT-3-01

NCT ID:

NCT05819866

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Effects of Leriglitazone for Adult Men with Cerebral Adrenoleukodystrophy

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?