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Leriglitazone

Leriglitazone is a drug currently being studied in clinical trials for the treatment of cerebral adrenoleukodystrophy (cALD), a rare genetic disorder affecting adult males. This article explores the ongoing research to assess the efficacy and safety of Leriglitazone in patients with cALD, providing insights into the potential benefits and outcomes of this experimental treatment.

Table of Contents

What is Leriglitazone?

Leriglitazone is a new drug that is currently being studied for its potential to treat a rare genetic disorder called cerebral adrenoleukodystrophy (cALD). This medication is undergoing clinical trials to assess its effectiveness and safety in adult male patients with cALD.[1]

What Condition Does Leriglitazone Treat?

Leriglitazone is being developed to treat cerebral adrenoleukodystrophy (cALD). This is a rare genetic disorder that primarily affects males. cALD is characterized by the breakdown of myelin, the protective covering of nerve cells in the brain. This leads to progressive neurological symptoms and can be life-threatening if left untreated.[1]

The Clinical Study on Leriglitazone

A clinical study is currently underway to evaluate the efficacy and safety of Leriglitazone in adult male subjects with cALD. This study is designed to compare the effects of Leriglitazone against a placebo (a substance that looks like the medication but contains no active drug). The study includes two main groups:[1]

  • Active Comparator Group: This group receives Leriglitazone treatment.
  • Placebo Comparator Group: This group receives a placebo that matches the study drug visually and by taste.

How is Leriglitazone Administered?

In the clinical trial, Leriglitazone is administered as follows:[1]

  • The drug is provided at a strength of 15 mg/ml.
  • It is taken once daily.
  • The initial dosing volume is 10 ml.

Primary Outcome of the Study

The main goal of this study is to determine how effective Leriglitazone is in delaying the progression of cALD. The primary endpoint (main measure of success) of the study is:[1]

The time to either death or the subject becoming bedridden with a requirement for permanent ventilatory support (breathing assistance), whichever comes earlier. This will be compared between subjects treated with Leriglitazone and those given a placebo.

This primary outcome will be assessed at three time points:

  1. Interim analysis 1: After 18 months of treatment
  2. Interim analysis 2: After 27 months of treatment
  3. Final analysis: After 36 months of treatment

Secondary Outcome: The Loes Score

In addition to the primary outcome, the study will also look at changes in what’s called the Loes Score. This is a way to measure the severity of brain abnormalities in cALD using MRI (Magnetic Resonance Imaging) scans.[1]

The Loes Score ranges from 0 to 34:

  • A score of 0 represents a healthy brain (better outcome)
  • A score of 34 represents the most severe brain abnormalities (worst outcome)

The study will measure how the Loes Score changes from the beginning of the study (baseline) at the same three time points as the primary outcome: 18 months, 27 months, and 36 months of treatment.

Aspect Details
Drug Name Leriglitazone
Condition Studied Cerebral Adrenoleukodystrophy (cALD)
Study Type Randomized, Placebo-Controlled Clinical Trial
Study Duration 36 months
Primary Outcome Measure Time to death or becoming bedridden with permanent ventilatory support
Secondary Outcome Measure Change in Loes Score
Drug Administration Once-daily, 15 mg/ml, initial volume of 10 ml
Analysis Points 18 months, 27 months, and 36 months

FAQ

  • What is Leriglitazone? Leriglitazone is an experimental drug being tested for the treatment of cerebral adrenoleukodystrophy (cALD) in adult male patients. It is administered once daily at a strength of 15 mg/ml, with an initial volume of 10 ml.
  • What is the main goal of the clinical trial for Leriglitazone? The main goal of the clinical trial is to assess the efficacy and safety of Leriglitazone in adult male subjects with cerebral adrenoleukodystrophy (cALD). The study aims to determine if Leriglitazone can improve outcomes for patients with this condition.
  • How is the effectiveness of Leriglitazone being measured in the trial? The primary measure of effectiveness is the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, whichever comes earlier. This is compared between subjects treated with Leriglitazone and those receiving a placebo.
  • What is the Loes Score, and how is it used in the trial? The Loes Score is a measure used to assess the severity of cerebral adrenoleukodystrophy. It ranges from 0 (healthy) to 34 (worst outcome). The trial is measuring changes in the Loes Score from the beginning of treatment to assess the effect of Leriglitazone on the progression of the disease.
  • How long does the clinical trial for Leriglitazone last? The clinical trial for Leriglitazone lasts for 36 months (3 years) in total. There are three analysis points: an interim analysis at 18 months, another at 27 months, and the final analysis at 36 months of treatment.

Ongoing Clinical Trials on Leriglitazone

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

    Recruiting

    1 1
    Investigated drugs:
    France Germany Spain

  • Study of drug interactions between leriglitazone, gemfibrozil, itraconazole, and carbamazepine and food effects in healthy male volunteers for adrenoleukodystrophy treatment

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Poland

  • Study on the Safety and Tolerability of Leriglitazone for Children with Rett Syndrome

    Not recruiting

    1
    Investigated diseases:
    Investigated drugs:
    Spain

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

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    France Germany Spain

Glossary

  • Cerebral Adrenoleukodystrophy (cALD): A rare genetic disorder that primarily affects males, causing damage to the nervous system, particularly the brain and spinal cord. It is characterized by the buildup of very long-chain fatty acids in the body.
  • Leriglitazone: An experimental drug being tested for the treatment of cerebral adrenoleukodystrophy in adult male patients. It is administered orally once daily.
  • Placebo: A substance that looks and tastes like the actual medication but contains no active ingredients. It is used in clinical trials to compare the effects of the real drug against no treatment.
  • Loes Score: A scoring system used to measure the severity of cerebral adrenoleukodystrophy. It ranges from 0 (healthy) to 34 (worst outcome), helping to track the progression of the disease.
  • Efficacy: The ability of a treatment to produce the desired beneficial effect. In clinical trials, efficacy refers to how well the drug works under ideal conditions.
  • Safety: The extent to which a drug can be used without causing harmful effects. Safety assessments in clinical trials help identify potential risks and side effects of a new treatment.
  • Interim Analysis: A planned review of data that occurs before a clinical trial is completed. It allows researchers to evaluate the progress and early results of the study.
  • Ventilatory Support: The use of mechanical assistance to help a patient breathe. In severe cases of cALD, patients may require permanent ventilatory support.

References