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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05819866
Other study ID # MT-3-01
Secondary ID
Status Recruiting
Phase Phase 3
First received
Last updated
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
Health authority
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.

Study Design


Related Conditions & MeSH terms


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)
See also
  Status Clinical Trial Phase
Recruiting NCT06224413 - A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
Completed NCT03852498 - A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD) Phase 3
Active, not recruiting NCT02698579 - Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
Completed NCT01896102 - A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD) Phase 2/Phase 3
Terminated NCT02204904 - Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)