A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy (CALYX)

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

Study Overview

• Status: Active, not recruiting
• Conditions: Cerebral Adrenoleukodystrophy (cALD)
• Intervention / Treatment: Drug: Leriglitazone; Drug: Placebo

• Study Type: Interventional
• Enrollment (Estimated): 40
• Phase: Phase 3

Contacts and Locations

Study Locations

• Argentina
  • Buenos Aires, Argentina
    • Hospital Austral
• Brazil
  • São Paulo, Brazil
    • Federal University of São Paulo
• France
  • Paris, France, 75013
    • ICM La Pitie Salpetriere
• Germany
  • Leipzig, Germany, 04103
    • Klinik und Poliklinik für Neurologie-Leipzig
• India
  • New Delhi, India, 110060
    • Sir Ganga Ram Hospital
• Spain
  • Madrid, Spain, 28041
    • Hospital 12 de Octubre
• United Kingdom
  • London, United Kingdom, WC1N 3AX
    • National Hospital For Neurology and Neurosurgery
• United States
  • California
    • Palo Alto, California, United States, 94304-5978
      • Stanford University Medical Center
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • Kennedy Krieger Institute
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
  • Minnesota
    • Minneota, Minnesota, United States, 55455
      • University of Minnesota

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Placebo will match the study drug visually and by taste
Active Comparator: Leriglitazone; Leriglitazone Treatment	Drug: Leriglitazone; Leriglitazone at a strength of 15 mg/ml. Once-daily dosing at an initial volume of 10 ml

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	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.	Final analysis (at 36 months of treatment)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from Baseline in Loes Score.	Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome	Interim analysis 1 (at 18 months of treatment)
Change from Baseline in Loes Score	Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome	Interim analysis 2 (at 27 months of treatment)
Change from Baseline in Loes Score	Change from Baseline in Loes Score. Loes Score Minimum = 0 Maximum =34 0 better outcome (healthy) 34 worst osutcome	Final analysis (at 36 months of treatment)

Collaborators and Investigators

• Sponsor: Minoryx Therapeutics, S.L.

Study record dates

Study Major Dates

• Study Start (Actual): July 12, 2023
• Primary Completion (Estimated): May 1, 2027
• Study Completion (Estimated): May 1, 2027

Study Registration Dates

• First Submitted: March 23, 2023
• First Submitted That Met QC Criteria: April 5, 2023
• First Posted (Actual): April 19, 2023

Study Record Updates

• Last Update Posted (Actual): July 29, 2026
• Last Update Submitted That Met QC Criteria: July 28, 2026
• Last Verified: July 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neurologic Manifestations; Endocrine System Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Neurobehavioral Manifestations; Demyelinating Diseases; Heredodegenerative Disorders, Nervous System; Adrenal Gland Diseases; Mental Retardation, X-Linked; Intellectual Disability; Genetic Diseases, X-Linked; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Hereditary Central Nervous System Demyelinating Diseases; Leukoencephalopathies; Adrenal Insufficiency; Peroxisomal Disorders; Adrenoleukodystrophy; leriglitazone
• Other Study ID Numbers: MT-3-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No