Cerebral Adrenoleukodystrophy Clinical Trial
— NEXUSOfficial 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)
Verified date | January 2023 |
Source | Minoryx Therapeutics, S.L. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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)
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 |
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 |
Lead Sponsor | Collaborator |
---|---|
Minoryx Therapeutics, S.L. |
Argentina, France, Germany, Spain,
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 |
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT03789721 -
Adrenoleukodystrophy National Registry Study
|
||
Withdrawn |
NCT02410239 -
MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD)
|
Phase 1 | |
Terminated |
NCT03367546 -
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases
|
Phase 2 | |
Recruiting |
NCT06030648 -
MT2022-01: MSCs for ALD
|
Phase 1 |