A Clinical 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)

• Status: Recruiting
• Trial end date: 2021-02-28
• Target enrollment: 0
• Participant gender: Male

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)

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Minoryx Therapeutics, S.L.

Criteria

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