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Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
Sponsor: Assistance Publique - Hôpitaux de Paris
Summary
This study is a national, non-randomized, open-label, multi-site with minimal risk study in adult with adrenomyeloneuropathy (AMN), childhood and adult subjects with cerebral ALD (cALD), juvenile/adult metachromatic leukodystrophy (MLD) and adults with leukoencephalopathy and axonal spheroids and pigmented glia (ALSP). 49 subjects will be enrolled with one blood sample collection during one of their medical follow-up visit. This trial will evaluate the role of innate immunity to influence disease progression in X-ALD, MLD and ALSP, and if the mutations related to these leukodystrophies result in a specific immune response leading to the pathogenesis.
Official title: MATRIX - "Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia"
Key Details
Gender
All
Age Range
15 Months - 60 Years
Study Type
OBSERVATIONAL
Enrollment
100
Start Date
2021-08-30
Completion Date
2027-02
Last Updated
2025-09-24
Healthy Volunteers
Yes
Conditions
Interventions
Blood sample collection
blood sample collection
Locations (2)
AP-HP Hôpital Bicêtre
Le Kremlin-Bicêtre, France
AP-HP Hôpital La Pitié Salpêtrière
Paris, France