A study to evaluate the effects of a new drug called MIN-102 on the progression of brain lesions in boys with cerebral X-linked adrenoleukodystrophy (cALD)

Phase 1

• Conditions: Cerebral X-linked Adrenoleukodystrophy (cALD); MedDRA version: 20.0 Level: PT Classification code 10051260 Term: Adrenoleukodystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2019-000654-59-ES
• Lead Sponsor: Minoryx Therapeutics S.L.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-04-30
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 10

Inclusion Criteria

1. Written informed consent by parent/legal guardian, or authorized legal representative to participate in the study
2. Males aged =2 and =12 years with a diagnosis of X-linked ALD based on genetic testing
3. White matter involvement as determined by cerebral MRI lesions without Gd enhancement at baseline (Population 1), or with Gd enhancement at baseline (Population 2).
4. Major Functional Disabilities (MFD) score of 0, as determined by key measures in the Neurological Function Scale (NFS)
5. Baseline Loes score >0 and =10
6. Baseline GIS =2
7. No signs or symptoms of adrenal insufficiency and morning cortisol and aldosterone levels within normal laboratory ranges for age, or appropriate steroid replacement if adrenal insufficiency is present. A history of adrenal insufficiency is not exclusionary if the foregoing is currently met.
8. Glycated hemoglobin (HbA1c) within normal range
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Other chronic neurological disease
2. Known intolerance to pioglitazone or other thiazolidinediones
3. Use of pioglitazone or other thiazolidinediones within the past 6 months prior to screening
4. Use of biotin at a daily dose of >50 mg per day within the past 3 months prior to screening
5. Current participation in another interventional clinical study or participation in such a study within 6 months prior to screening
6. Previous HSCT
7. Current use of immunosuppressant medication, excluding corticosteroids
8. Requirement for a prohibited concomitant medication
9. Previous or current history of bladder polyps, bladder cell hyperplasia, or cancer (other than successfully treated basal cell carcinoma)
10. Previous or current history of congestive heart failure
11. Clinically significant anemia with hemoglobin <10 g/dL
12. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) level >2 times the ULN or total bilirubin >1.5 times the ULN (unless due to Gilbert's syndrome)
13. Moderate or severe hepatic impairment (groups B and C according to Child-Pugh classification)
14. Chronic kidney disease stage 3 or higher (according to chronic kidney disease staging by The Renal Association)
15. Pulmonary disease or cardiac disease of sufficient severity to limit participation in the study and/or completion of study procedures
16. Reduced left-ventricular ejection fraction or other clinically significant cardiac abnormalities on echocardiogram that, in the investigator's opinion, could predispose the subject to volume overload or its attendant consequences
17. Contraindication to MRI procedure, such as presence of paramagnetic materials (aneurysm clips, pacemaker, intraocular metal, cochlear implant) in the body
18. Conditions that could modify the absorption of the study drug
19. Inability or unwillingness of parent/legal guardian or subject to comply with the study procedures
20. Other medical, neurologic, psychiatric, or social condition that, in the opinion of the investigator, is likely to unfavorably alter risk-benefit of study participation, confound interpretation of safety or efficacy results, or interfere with the satisfactory completion of study requirements

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified