Education and Training
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy.
Stanford is currently accepting patients for this trial.
Stanford Investigator(s):
Intervention(s):
- drug: Leriglitazone
- drug: Placebo
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.
Ages Eligible for Study
18 Years - N/A
Genders Eligible for Study
Male
Now accepting new patients
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Jacinda Sampson
650-723-0993
I'm interested