Cerebral Adrenoleukodystrophy
Conditions
Brief summary
An Open-Label, multicenter study in male pediatric patients with cerebral x-linked adrenoleukodystrophy (cald) to assess the effects of MIN-102 treatment on disease progression prior to human stem cell transplant (HSCT)
Interventions
DRUGMIN-102
Once-daily dosing with a volume specified by the pharmacokineteic specialist to achive the desired plasma exposure. Min-102 Oral suspension, strength 15mg/ml.
Sponsors
Minoryx Therapeutics, S.L.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
2 Years to 12 Years
Healthy volunteers
No
Inclusion criteria
* Males aged ≥2 and ≤12 years with a diagnosis of X-linked ALD * White matter involvement as determined by cerebral MRI lesions without Gd enhancement at baseline (Population 1), or with Gd enhancement at baseline (Population 2). * Major Functional Disabilities (MFD) score of 0. * Baseline Loes score \>0 and ≤10 * Gadolinium Intensity Score \>2
Exclusion criteria
* Previous HSCT * Too far progressed inflammatory brain lesions
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| evaluate whether MIN-102 can halt disease progression of cALD if administered prior to hematopoietic stem-cell transplantation (HSCT), as determined by serial clinical and MRI investigations in pediatric subjects. | 6 months- 2 years | — |
| arrested disease will be assessed at 24 weeks and at 96 weeks | at 24 weeks and 96 weeks | arrested disease is defined using: Cahnge in NFS from Baseline, Free of MFD and lack of lesion progression on MRI |
Countries
Argentina, France, Germany, Spain
Outcome results
None listed