A Phase 2 interventional study of MIN-102 in Cerebral Adrenoleukodystrophy, sponsored by Minoryx Therapeutics, S.L.. Status unknown at 5 sites in 4 countries. Open to male participants aged 2 Years to 12 Years. Per ClinicalTrials.gov, last updated 2023-07-18.
Sponsored by Minoryx Therapeutics, S.L. · Phase 2, Interventional, and Treatment
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)
Exclusion Criteria:
Drug: MIN-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.
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.
Time frame: 6 months- 2 years
"arrested disease" will be assessed at 24 weeks and at 96 weeks
arrested disease is defined using: Cahnge in NFS from Baseline, Free of MFD and lack of lesion progression on MRI
Time frame: at 24 weeks and 96 weeks
This study is status unknown, as verified in Jan 2023. You cannot join it, but the record below documents what was studied.
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Minoryx Therapeutics, S.L.