Health Condition 1: G909- Disorder of the autonomic nervoussystem, unspecified
Conditions
Interventions
Intervention1: leriglitazone: leriglitazone is used to treat Cerebral Adrenoleukodystrophy
Composition is 15mg/ml
Administered once in a day.
Control Intervention1: Placebo: Administered once in a da
Sponsors
Minoryx Therapeutics S.L
Minoryx Therapeutics SL
Eligibility
Inclusion criteria
Inclusion criteria: 1. Subject is able to read and understand the ICF and has provided written informed consent to participate in the study. 2. Subject is male and aged 18 years. 3. Subject has genetic confirmation of X ALD. 4. Subject has progressive cALD, defined as GdE plus brain lesions. 5. Subjects for whom HSCT is not recommended by the investigator or subject is not willing to undergo HSCT. 6. Subject has a Loes score 0.5 and 12 at Screening. 7. Subject does not have major functional disability in the Major Functional Disabilities Neurological Function Score (MFD-NFS), other than wheelchair bound or total incontinence, which will be allowed as these are considered expected symptoms of AMN in the time course of the disease. 8. 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. 9. Subject has normal adrenal function or appropriate steroid replacement if adrenal insufficiency is present.
Exclusion criteria
Exclusion criteria: 1. Subject who had previous bone marrow transplantation (HSCT) or treatment with ex-vivo gene therapy (eli-Cel). 2. Subject has known type 1 or type 2 diabetes. 3. Subject has known hypersensitivity or intolerance to pioglitazone or any other thiazolidinedione. 4. Subject is taking or has taken honokiol, pioglitazone, or other thiazolidinediones within 3 months prior to Screening. 5. Subject has a requirement for treatment with a prohibited concomitant medication. 6. Subject has a previous or current history of congestive heart failure. 7. Subject has reduced left-ventricular ejection fraction or other clinically significant cardiac abnormalities on echocardiogram that in the opinion of the investigator could predispose the subject to volume overload or its associated consequences. 9. Subject has moderate or severe hepatic impairment (Child-Pugh classification groups B or C).2 10. Subject with chronic kidney disease (CKD) of stage 3 or higher (according to the Renal Association CKD staging).3 11. Subject has previous or current history of cancer, unless surgically resected and without evidence of recurrence for a minimum of 5 years. 12. Subjects who are not surgically sterilized. If not surgically sterilized, subjects should be willing to use adequate contraception and not donate sperm from the first dose of the study drug until 90 days after the EOT visit. 13. Subject has contraindications for MRI such as having paramagnetic material in the body (e.g., aneurysm clips, pacemakers, intraocular metal, or cochlear implants). 14. Subject with conditions that could modify absorption of the study drug. 15. Subject with current participation in another interventional clinical study or within 1 month prior to Screening. 16. 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To evaluate the efficacy of leriglitazone compared to placebo at increasing survival.Timepoint: To evaluate the efficacy of leriglitazone compared to placebo in 6 weeks. | — |
Secondary
| Measure | Time frame |
|---|---|
| To evaluate the efficacy of leriglitazone compared to placebo at slowing radiological progression.Timepoint: Change from Baseline in Loes score at 18 months | — |
Countries
Argentina, Brazil, Germany, India, Spain, United Kingdom, United States of America
Contacts
Public ContactBharat Doshi
BioDev Services Private Limited
Outcome results
None listed