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RECRUITING
NCT04925349

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

View on ClinicalTrials.gov

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

Interventions

DIAGNOSTIC_TEST

Blood sample collection

blood sample collection

Locations (2)

AP-HP Hôpital Bicêtre

Le Kremlin-Bicêtre, France

AP-HP Hôpital La Pitié Salpêtrière

Paris, France