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)

Minoryx Therapeutics, S.L.13 enrolled

Overview

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)

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Cerebral Adrenoleukodystrophy

Interventions

MIN-102DRUG

Once-daily dosing with a volume specified by the pharmacokineteic specialist to achive the desired plasma exposure. Min-102 Oral suspension, strength 15mg/ml.

Eligibility

Sex: MALEMin age: 2 YearsMax age: 12 Years

Medical Language ↔ Plain English

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

Locations (5)

Hernan Amartino

Bueno Aires, Buenos Aires, Argentina

CHU Kremlin Bicêtre

Paris, France

Universitätsmedizin Göttingen Georg-August-Universität

Göttingen, Germany

Universitätsklinikum Hamburg-Eppendorf Klinik und Poliklinik für Kinder- und Jugendmedizin

Hamburg, Germany

Outcomes

Primary Outcomes

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

Data from ClinicalTrials.gov

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