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

Cerebral Adrenoleukodystrophy (cALD) Clinical Trial

— CALYX  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05819866
• Other study ID #: MT-3-01
• Status: Recruiting
• Phase: Phase 3
• Start date: July 12, 2023
• Est. completion date: May 2027

Study information

• Verified date: October 2023
• Source: Minoryx Therapeutics, S.L.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: May 2027
• Est. primary completion date: May 2027
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Adrenoleukodystrophy
• Cerebral Adrenoleukodystrophy (cALD)

Intervention

Drug:
• 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

Locations

Country	Name	City	State
Argentina	Hospital Austral	Buenos Aires
Brazil	Federal University of Sao Paulo	São Paulo
United States	Kennedy Krieger Institute	Baltimore	Maryland
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	Neuro Medicine Hospital / UF Health	Gainesville	Florida
United States	University of Minnesota	Minnesota	Minnesota
United States	Stanford University Medical Center	Palo Alto	California
United States	Health University of Utah	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Minoryx Therapeutics, S.L.

Countries where clinical trial is conducted

United States,  Argentina,  Brazil, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)
Primary	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)
Primary	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	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)
Secondary	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)
Secondary	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)