MIN-102 for Adrenoleukodystrophy

(Advance Trial)

No longer recruiting at 10 trial locations

Age: 18+

Sex: Male

Trial Phase: Phase 2 & 3

Sponsor: Minoryx Therapeutics, S.L.

Must not be taking: Honokiol, Thiazolidinediones

Disqualifiers: Chronic neurological disease, Diabetes, Cancer, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Phase II/III duration not specified

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the effectiveness and safety of a treatment called MIN-102 for men with X-linked adrenoleukodystrophy (X-ALD), a genetic condition affecting the spinal cord that can impair walking and balance. Participants will receive either the treatment or a placebo (a substance with no active drug) to compare results. Men diagnosed with X-ALD and experiencing spinal cord issues may be suitable for this study. As a Phase 2, Phase 3 trial, the research measures the treatment's effectiveness in an initial, smaller group and represents the final step before FDA approval. Participants have the opportunity to contribute to potentially groundbreaking advancements in X-ALD treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you've taken honokiol, pioglitazone, or similar drugs in the past 6 months.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that MIN-102, a treatment under study for a type of adrenoleukodystrophy (ALD), is generally safe and well-tolerated. Earlier studies found that MIN-102 can be administered at higher doses than required for efficacy without causing serious side effects. Testing revealed no major unexpected or harmful reactions. Additionally, MIN-102 effectively reaches the brain, which is crucial for treating ALD. Overall, the available evidence suggests that the safety of MIN-102 is promising. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for adrenoleukodystrophy, which often focus on managing symptoms and slowing disease progression, MIN-102 is unique because it targets the root cause at the cellular level. MIN-102 is a small molecule that crosses the blood-brain barrier and specifically targets the peroxisome proliferator-activated receptor gamma (PPARγ). This mechanism has the potential to directly address the neurological degeneration caused by the disease. Researchers are excited about MIN-102 because it offers a promising new approach that could significantly improve neurological outcomes for patients.

What evidence suggests that MIN-102 might be an effective treatment for adrenoleukodystrophy?

Research has shown that MIN-102, also known as leriglitazone, may help treat X-linked adrenoleukodystrophy (X-ALD), particularly the AMN type. Early studies suggest it works effectively on the brain and spinal cord, which is crucial for managing this condition. Previous research demonstrated that MIN-102 is safe even at high doses. Initial results indicated that patients noticed improvements while on this treatment. In this trial, participants will receive either MIN-102 or a placebo. Overall, these findings suggest that MIN-102 could be a helpful treatment option for those with AMN. ^{1 2 5 6 7}

Are You a Good Fit for This Trial?

This trial is for male patients aged 18-65 with X-linked adrenoleukodystrophy (X-ALD), confirmed by specific tests and showing spinal cord involvement. It's not open to those who've taken certain diabetes drugs recently, have a history of cancer (except treated skin cancer), inflammatory brain lesions, other neurological diseases causing similar symptoms, heart failure, or had bone marrow transplants.

Inclusion Criteria

I am a man aged between 18 and 65.

My cancer has spread to my spinal cord.

I have been diagnosed with X-ALD through specific blood and genetic tests.

Exclusion Criteria

I have been diagnosed with diabetes.

I have had cancer before, but not basal cell carcinoma.

You have abnormal MRI results that show inflammation or other issues not mentioned in the inclusion criteria.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive MIN-102 or placebo in a double-blind, placebo-controlled setting

Phase II/III duration not specified

Follow-up

Participants are monitored for safety and effectiveness after treatment

4-8 weeks

Open-label extension

Participants may opt into continuation of treatment with MIN-102 long-term

What Are the Treatments Tested in This Trial?

Interventions

• MIN-102
• Placebos

Trial Overview The study compares the effects of MIN-102 against a placebo in treating AMN phenotype of X-ALD. Participants are randomly assigned to one of two groups: one receiving MIN-102 and the other a placebo. The trial is double-blind meaning neither participants nor researchers know who gets the real treatment.

How Is the Trial Designed?

Group I: Active Active Control 1 Intervention

Group II: Placebo Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Minoryx Therapeutics, S.L.

Lead Sponsor

Trials

5

Recruited

240+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03231878

NCT03231878 | A Clinical Study to Evaluate the Efficacy ... To evaluate the efficacy of MIN-102 in terms of patient reported outcomes. in 96 weeks. SSPROM (Severity Score System for Progressive Myelopathy ), in 96 weeks.

2. minoryx.com minoryx.com/media/minoryx-therapeutics-successfully-completes-phase-1-clinical-trial-for-lead-candidate-min-102/

Minoryx Therapeutics successfully completes phase 1 ... MIN-102 was well-tolerated at much higher doses than those required for efficacy. MIN-102 exhibited linear pharmacokinetics and no food ...

3. neurology.org neurology.org/doi/10.1212/WNL.94.15_supplement.759

MIN-102 (leriglitazone), a Brain Penetrant PPAR gamma ... Conclusions: The preclinical data indicate that leriglitazone is a promising therapeutic approach to treat both CNS phenotypes of X-ALD, AMN ...

4. thelancet.com thelancet.com/journals/eclinm/article/PIIS2589-5370(25)00197-X/fulltext

Safety and efficacy of leriglitazone in childhood cerebral ... Cerebral adrenoleukodystrophy rapidly progresses in approximately 90% of untreated patients. Current treatment, haematopoietic stem-cell ...

5. aldconnect.org aldconnect.org/clinical-trials-and-research/clinical-trials/

Clinical Trials An Open-Label, multicenter study in male pediatric patients with cerebral x-linked adrenoleukodystrophy (CALD) to assess the effects of MIN-102 treatment on ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT03231878?cond=ADRENOLEUKODYSTROPHY&rank=5

A Clinical Study to Evaluate the Efficacy and Safety of MIN ... The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure.

7. minoryx.com minoryx.com/media/the-lancet-neurology-publishes-results-from-minoryx-therapeutics-phase2-3-advance-clinical-trial-of-leriglitazone-in-x-linked-adrenoleukodystrophy/

The Lancet Neurology publishes results from ... It has demonstrated sufficient brain penetration and a favorable safety profile.