Leriglitazone for Adrenoleukodystrophy (CALYX Trial)

Phase 3

Recruiting

Research Sponsored by Minoryx Therapeutics, S.L.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subject has progressive cALD, defined as GdE+ brain lesions.

Subjects for whom HSCT is not recommended by the investigator or subject is not willing to undergo HSCT.

Must not have

Subject who had previous bone marrow transplantation (HSCT) or treatment with ex-vivo gene therapy (eli-Cel).

Subject has known type 1 or type 2 diabetes.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up interim analysis 2 (at 27 months of treatment)

Awards & highlights

Pivotal Trial

Summary

This trial studies the effects of a drug on adults with a rare neurological disorder called Cerebral Adrenoleukodystrophy.

Who is the study for?

This trial is for adult males with cerebral adrenoleukodystrophy (cALD) who can't or choose not to undergo a bone marrow transplant. Participants should have progressing cALD, a specific brain lesion score, and no major functional disabilities except those common in AMN progression. They must not have diabetes, previous treatments like HSCT or certain drugs within 3 months, and shouldn't be part of another study.

What is being tested?

The trial is testing Leriglitazone's effectiveness and safety against a placebo in treating cALD. Men will randomly receive either the actual drug or an inactive substance to compare outcomes between the two groups.

What are the potential side effects?

Potential side effects are not specified here but based on similar medications; they could include weight gain, fluid retention leading to swelling, heart issues, and possible liver changes. Side effects vary from person to person.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My brain scans show active lesions due to my condition.

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I am not recommended or willing to undergo a stem cell transplant.

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I am a man and 18 years old or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had a bone marrow transplant or gene therapy treatment.

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I have been diagnosed with diabetes.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ interim analysis 2 (at 27 months of treatment)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~interim analysis 2 (at 27 months of treatment)

This trial's timeline: 3 weeks for screening, Varies for treatment, and interim analysis 2 (at 27 months of treatment) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.

Secondary study objectives

Change from Baseline in Loes Score

Change from Baseline in Loes Score.