NCT05819866 · Minoryx Therapeutics, S.L.
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
(CALYX)
What this study is about
A Clinical Study to Assess the effectiveness and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy.
View original scientific description
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy.
Interventions
DRUG
Leriglitazone
Leriglitazone at a strength of 15 mg/ml. Once-daily dosing at an initial volume of 10 ml
DRUG
Placebo
Placebo will match the study drug visually and by taste
Primary outcome measures
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.
Time frame: Interim analysis 1 (at 18 months of treatment)
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.
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.
Time frame: Interim analysis 2 (at 27 months of treatment)
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.
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.
Time frame: Final analysis (at 36 months of treatment)
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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Subject is male and aged ≥18 years.
- 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.
- Subject has a Loes score ≥0.5 and ≤12 at Screening.
- Subject does not have major functional disability in the Major Functional Disabilities-Neurological Function Score (MFD-NFS), except for "wheelchair bound" or "total incontinence", which will be allowed as these are considered expected symptoms of AMN in the time course of the disease
- Subject does not have major cognitive impairment which would impair his ability to take part in the study as determined by the investigator at screening. Key
Exclusion criteria
- 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.
- Subject has known hypersensitivity or intolerance to pioglitazone or any other thi
Where
- Palo Alto, California
- Gainesville, Florida
- Baltimore, Maryland
- Boston, Massachusetts
- Minneota, Minnesota
- Salt Lake City, Utah
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Nov 17, 2025 · Source of record for eligibility and locations