M2 microglia and macrophages drive oligodendrocyte differentiation during CNS remyelination.
Veronique E Miron, Amanda Boyd, Jing-Wei Zhao...
https://pubmed.ncbi.nlm.nih.gov/23872599Actively Recruiting
Age: 15Months - 60Years
All Genders
Healthy Volunteers
ID04925349
MATRIX - Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
Led by Assistance Publique - Hôpitaux de Paris · Updated on 2025-09-24
100
Participants Needed
2
Research Sites
26 weeks
Total Duration
On this page
Sponsors
A
Assistance Publique - Hôpitaux de Paris
Lead Sponsor
U
URC-CIC Paris Descartes Necker Cochin
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying adults and children affected by several inherited brain diseases including adrenomyeloneuropathy (AMN), cerebral adrenoleukodystrophy (cALD), metachromatic leukodystrophy (MLD), and adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP). These conditions involve nerve damage and inflammation, and the study aims to understand how immune cells called macrophages contribute to disease progression. This observational study involves 49 participants and explores how genetic mutations affect immune responses linked to these diseases. Participants include affected children and adults as well as healthy controls. The main activity involves collecting one blood sample during a routine medical visit. This sample will be used to analyze macrophage function, including their ability to engulf myelin, their distribution, and certain immune markers. No treatments or interventions are given, as this is a minimal risk study focusing on immune system activity related to these conditions. During the study, researchers will monitor macrophage function and gene activity over a two-year period following blood collection. Participants will have one blood draw during their scheduled medical follow-up. The study also includes healthy children and adults for comparison. The research team will track immune cell profiles and metabolic patterns to better understand how immune responses may influence disease outcomes in these leukodystrophies.
CONDITIONS
Brief Title
Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
Who Can Participate
Age: 15Months - 60Years
All Genders
Healthy Volunteers
Eligibility Criteria
You may qualify if you...
- Boys aged between 3 and 18 years with childhood cerebral adrenoleukodystrophy (C-CALD)
- Boys or girls aged 15 months to 18 years diagnosed with metachromatic leukodystrophy (MLD)
- Presymptomatic boys aged 3 to 18 years carrying ABCD1 mutations (PRE-ALD)
- Adult males or females aged 18 to 60 years diagnosed with MLD
- Males aged 18 to 60 years diagnosed with adrenomyeloneuropathy (AMN)
- Males aged 18 to 60 years diagnosed with cerebral adrenoleukodystrophy (CALD)
- Adult males or females aged 18 to 60 years diagnosed with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP)
- Presymptomatic adult males or females carrying CSF1R mutations (PRE-ALSP)
- Children aged 15 months to 18 years without neurological disease
- Adults aged 18 to 60 years without neurological disease
- Informed consent obtained from parents or guardians for children, and from adults themselves
You will not qualify if you...
- Participation in any therapeutic clinical trial
- Treatment likely to modify the immune system
- Unable to have blood collected (e.g., low hemoglobin as judged by investigator)
- Any other reason at investigator's discretion
- Children or adults without health insurance or social security
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Diagnostic Evaluation
Duration - 1 day
Participants provide blood samples for analysis to assess macrophage activation and functionality related to their condition.
1 visit (in-person)
Long-term Monitoring
Duration - 2 years
Participants are observed over a 2-year period to evaluate macrophage functionality and related outcomes following blood collection.
Periodic assessments (visit frequency not specified)
Trial Site Locations
Total: 2 locations
1
AP-HP Hôpital Bicêtre
Le Kremlin-Bicêtre, France, 94275
Actively Recruiting
2
AP-HP Hôpital La Pitié Salpêtrière
Paris, France, 75013
Actively Recruiting
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Research Team
F
Fanny MOCHEL
C
Christelle AUGER
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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Published Research Related To This Trial
Impaired plasticity of macrophages in X-linked adrenoleukodystrophy.
Isabelle Weinhofer, Bettina Zierfuss, Simon Hametner...
https://pubmed.ncbi.nlm.nih.gov/29860501Pathophysiology of X-linked adrenoleukodystrophy.
J Berger, S Forss-Petter, F S Eichler
https://pubmed.ncbi.nlm.nih.gov/24316281Metachromatic leukodystrophy--an update.
V Gieselmann, I Krägeloh-Mann
https://pubmed.ncbi.nlm.nih.gov/20571983