Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
Condition(s) studied
Investigational drug(s) / intervention(s)
Blood sample collection: blood sample collection
Study 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.
Eligibility
Primary outcome measure(s)
- Macrophages functionality - distribution of monocytes — 2 years after blood collection
distribution of monocytes in the CD14-/+/++ and CD16-/+ classification using flow cytometry (% of CD14++/16-; CD14++/16+; CD14+/16+; CD14-/16-) - Macrophages functionality - myelin phagocytosis capacity — 2 years after blood collection
percentage of myelin high, myelin low and myelin negative cells using flow cytometry - Macrophages functionality - HLA levels — 2 years after blood collection
maximum fluorescence intensity for HLA markers using flow cytometry
Trial sites (2)
| Facility | City | Region | Status |
|---|---|---|---|
| AP-HP Hôpital Bicêtre | Le Kremlin-Bicêtre | France | Recruiting |
| AP-HP Hôpital La Pitié Salpêtrière | Paris | France | Recruiting |
More Assistance Publique - Hôpitaux de Paris trials in France
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT04925349 on ClinicalTrials.gov ↗ ← All trials in France