Status:
UNKNOWN
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)
Lead Sponsor:
Minoryx Therapeutics, S.L.
Conditions:
Cerebral Adrenoleukodystrophy
Eligibility:
MALE
2-12 years
Phase:
PHASE2
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 cel...
Eligibility Criteria
Inclusion
- 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
- Previous HSCT
- Too far progressed inflammatory brain lesions
Key Trial Info
Start Date :
September 13 2019
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 31 2023
Estimated Enrollment :
13 Patients enrolled
Trial Details
Trial ID
NCT04528706
Start Date
September 13 2019
End Date
December 31 2023
Last Update
July 18 2023
Active Locations (5)
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1
Hernan Amartino
Bueno Aires, Buenos Aires, Argentina, B16641NZ
2
CHU Kremlin Bicêtre
Paris, France
3
Universitätsmedizin Göttingen Georg-August-Universität
Göttingen, Germany
4
Universitätsklinikum Hamburg-Eppendorf Klinik und Poliklinik für Kinder- und Jugendmedizin
Hamburg, Germany