A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
Status: Recruiting
Location: See all (13) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy.
Eligibility
Participation Requirements
Sex: Male
Minimum Age: 18
Healthy Volunteers: f
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• 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.