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Study on the Effects of Leriglitazone in Male Children with Cerebral X-Linked Adrenoleukodystrophy Before Stem Cell Transplant

Verified siteInvestigationalNo placebo
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What is this trial about?

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

This clinical trial is focused on studying a rare genetic disease called cerebral X-linked adrenoleukodystrophy (CALD), which affects the brain and is primarily found in young boys. The study is investigating the effects of a treatment using a medication known as Min-102, which contains the active substance leriglitazone. The purpose of the study is to see if Min-102 can slow down the progression of CALD before patients undergo a procedure called a human stem cell transplant.

Participants in the study will receive Min-102 as an oral suspension, which means it is taken by mouth in liquid form. The study will take place over a period of time, with regular check-ups and assessments to monitor the effects of the treatment. These assessments will include clinical evaluations and imaging tests like magnetic resonance imaging (MRI) to observe changes in the brain. The study aims to determine if the treatment can help stabilize the disease and improve the quality of life for those affected by CALD.

Throughout the study, researchers will collect data to evaluate the effectiveness of Min-102 in halting disease progression. This will involve tracking changes in specific symptoms and overall health. The study will also compare the outcomes of those receiving Min-102 with those who may receive a placebo, to better understand the treatment's impact. The ultimate goal is to provide valuable insights into managing CALD and potentially offer a new treatment option for patients with this challenging condition.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    The study involves male pediatric patients diagnosed with cerebral X-linked adrenoleukodystrophy (CALD).

    Participants must be between 2 and 12 years old and meet specific medical criteria, including genetic testing or family history confirmation.

  2. Step 2

    Treatment with leriglitazone

    Participants will receive leriglitazone, an oral suspension medication.

    The treatment aims to assess its effects on disease progression before a potential human stem cell transplant (HSCT).

  3. Step 3

    Monitoring and assessments

    The main objective is to evaluate whether leriglitazone can stop disease progression over a period of 96 weeks.

    This will be determined through regular clinical evaluations and magnetic resonance imaging (MRI) scans.

  4. Step 4

    Primary and secondary endpoints

    The primary endpoint is the number of patients meeting the 'arrested disease' criteria by Visit 12.

    Secondary endpoints include changes in neurological function scores, MRI severity scores, and overall survival without undergoing HSCT.

  5. Step 5

    Completion of the study

    The study is estimated to conclude by February 19, 2025.

    Participants will be monitored throughout the study to ensure safety and effectiveness of the treatment.

Who can join the trial?

8 criteria

  • Written informed consent must be provided by a parent, legal guardian, or authorized legal representative for the child to participate in the study.
  • The participant must be a male aged between 2 and 12 years old with a diagnosis of X-linked ALD. This can be confirmed through genetic testing or, if genetic testing is not available, by elevated levels of very long-chain fatty acids (VLCFA) and a family history of X-ALD with clinical symptoms.
  • The participant must have white matter involvement in the brain, as shown by an MRI scan. This can be without Gadolinium (Gd) enhancement (Population 1) or with Gd enhancement (Population 2).
  • The participant must have a Major Functional Disabilities (MFD) score of 0, which is determined by key measures in the Neurological Function Scale (NFS). This means there are no major functional disabilities.
  • The participant must have a baseline Loes score greater than 0 and less than or equal to 10. The Loes score is a way to measure the severity of brain changes in this condition.
  • The participant must have a baseline Gadolinium Intensity Score (GIS) of 3 or less. This score measures the intensity of certain changes seen on an MRI scan.
  • The participant must not show signs or symptoms of adrenal insufficiency, which is when the adrenal glands do not produce enough hormones. If adrenal insufficiency is present, it must be managed with appropriate steroid replacement. Morning cortisol and aldosterone levels must be within normal ranges for the participant's age.
  • The participant's glycated hemoglobin (HbA1c), which is a measure of blood sugar levels over time, must be within the normal range.

Who cannot join the trial?

4 criteria

  • Patients who are not male cannot participate in the study.
  • Patients who are not within the specified age range for the study cannot participate.
  • Patients who do not have the specific condition being studied, which is Cerebral X-linked Adrenoleukodystrophy (CALD), cannot participate.
  • Patients who are considered part of a vulnerable population may not be eligible to participate.
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Investigated drugs

MIN-102 is a medication being studied to see if it can slow down the progression of cerebral X-linked adrenoleukodystrophy (cALD) in young male patients. This condition affects the brain and can lead to severe neurological problems. The trial aims to determine if taking MIN-102 can help manage the disease before patients undergo a human stem cell transplant. The effects of the medication are monitored through clinical assessments and MRI scans over a period of time.

What is already known about the treatment

MIN-102 – This medication is administered orally and is currently being studied in clinical trials for its potential to treat cerebral X-linked adrenoleukodystrophy (CALD) in male pediatric patients. The main therapeutic indication is to slow or stop the progression of CALD, a rare genetic disorder affecting the brain. MIN-102 works by targeting specific molecular pathways involved in the disease, aiming to stabilize or improve neurological function. It is classified as a neuroprotective agent, and ongoing research is evaluating its effectiveness and safety in delaying disease progression before human stem cell transplant.

Investigated diseases

Cerebral X-linked Adrenoleukodystrophy (CALD) – This is a rare genetic disorder that primarily affects the nervous system and adrenal glands. It is caused by mutations in the ABCD1 gene, leading to the accumulation of very long-chain fatty acids in the brain and adrenal cortex. The disease typically manifests in childhood, with symptoms such as behavioral changes, learning difficulties, and progressive neurological decline. As the condition progresses, affected individuals may experience vision and hearing loss, difficulty with speech, and motor function impairment. Over time, the disease can lead to severe disability due to the progressive damage to the brain's white matter. CALD is an X-linked condition, meaning it predominantly affects males, while females may be carriers with milder symptoms.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IVTrial ID2024-513774-21-00Protocol codeMT-2-02Estimated enrolment16 patientsSponsorMinoryx Therapeutics S.L.

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