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)

Phase 2

Active, not recruiting

• Conditions: Cerebral Adrenoleukodystrophy
• Interventions: Drug: MIN-102

• Registration Number: NCT04528706
• Lead Sponsor: Minoryx Therapeutics, S.L.

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)

Detailed Description

Not available

Study Timeline

• Study Start: 2019-09-13
• Study First Submitted: 2020-07-31
• Study First Submitted QC: 2020-08-24
• Study First Posted: 2020-08-27
• Status Verified: 2023-01
• Last Update Submitted: 2023-07-17
• Last Update Posted: 2023-07-18
• Primary Completion: 2023-12-31
• Study Completion: 2023-12-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 13

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
MIN-102	MIN-102	-

Primary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (5)

CHU Kremlin Bicêtre; 🇫🇷 Paris, France

Universitätsklinikum Hamburg-Eppendorf Klinik und Poliklinik für Kinder- und Jugendmedizin; 🇩🇪 Hamburg, Germany

Hospital Sant Joan de Déu; 🇪🇸 Barcelona, Spain

Universitätsmedizin Göttingen Georg-August-Universität; 🇩🇪 Göttingen, Germany

Hernan Amartino; 🇦🇷 Bueno Aires, Buenos Aires, Argentina