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) (NEXUS)

Minoryx

Status and phase: Active, not recruiting; Phase 2

Conditions: Cerebral Adrenoleukodystrophy

Treatments: Drug: MIN-102

Study type: Interventional

Funder types: Industry

Identifiers

NCT04528706

MT-2-02

Details and patient eligibility

About

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)

Enrollment: 13 estimated patients

Sex: Male

Ages: 2 to 12 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

13 participants in 1 patient group

MIN-102

Experimental group

Treatment:

Drug: MIN-102