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)

Cerebral Adrenoleukodystrophy Clinical Trial

— NEXUS  

Official title:

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)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04528706
• Other study ID #: MT-2-02
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: September 13, 2019
• Est. completion date: December 31, 2023

Study information

• Verified date: January 2023
• Source: Minoryx Therapeutics, S.L.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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)

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 13
• Est. completion date: December 31, 2023
• Est. primary completion date: December 31, 2023
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 2 Years to 12 Years

Eligibility

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

Related Conditions & MeSH terms

• Adrenoleukodystrophy
• Cerebral Adrenoleukodystrophy
• Disease Progression

Intervention

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.

Locations

Country	Name	City	State
Argentina	Hernan Amartino	Bueno Aires	Buenos Aires
France	CHU Kremlin Bicêtre	Paris
Germany	Universitätsmedizin Göttingen Georg-August-Universität	Göttingen
Germany	Universitätsklinikum Hamburg-Eppendorf Klinik und Poliklinik für Kinder- und Jugendmedizin	Hamburg
Spain	Hospital Sant Joan de Déu	Barcelona

Sponsors (1)

Lead Sponsor	Collaborator
Minoryx Therapeutics, S.L.

Countries where clinical trial is conducted

Argentina,  France,  Germany,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	"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	at 24 weeks and 96 weeks