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Not recruiting
Phase 3

Leriglitazone

Sponsor:

Minoryx Therapeutics, S.L.

Code:

NCT05819866

Conditions

Cerebral Adrenoleukodystrophy (cALD)

Eligibility Criteria

Sex: Male

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Leriglitazone

Placebo

Study Details

Brief summary:

A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy.

Conditions

Cerebral Adrenoleukodystrophy (cALD)

Study ID

NCT05819866

Start date

Jul 12, 2023

Status verified date

Jul, 2026

Completion date

May, 2027

Anticipated

Primary completion date

May, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Male

Age: 18+

Healthy Volunteers: Not accepted

Key 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 thiazolidinedione.
  • Subject is taking or has taken honokiol, pioglitazone, or other thiazolidinediones within 3 months prior to Screening.
  • Subject with current participation in another interventional clinical study or within 1 month prior to Screening.
  • Subject with other medical, neuropsychiatric or social conditions that, in the opinion of the investigator, are likely to adversely affect the risk-benefit of study participation, interfere with study compliance, or confound the study results.

Study Design

Enrollment

40 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

active comparator: Leriglitazone

Leriglitazone Treatment

placebo comparator: Placebo

Placebo

Interventions

Leriglitazone

Leriglitazone at a strength of 15 mg/ml. Once-daily dosing at an initial volume of 10 ml

Placebo

Placebo will match the study drug visually and by taste

Primary outcome measure

  • 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. [ 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. [ Time Frame: Final analysis (at 36 months of treatment) ]

Central Contacts and Locations

More Information

Sponsor

Minoryx Therapeutics, S.L.

Last update posted

Jul 29, 2026

Last verified

Jul, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-19. This information was provided to ClinicalTrials.gov by Minoryx Therapeutics, S.L. on 2026-07-29. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.