Actively Recruiting
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
283 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
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.
CONDITIONS
Official Title
Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Boys aged 3 to 18 years diagnosed with childhood cerebral ALD (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 diagnosed with MLD
- Males aged 18 to 60 diagnosed with adrenomyeloneuropathy (AMN)
- Males aged 18 to 60 diagnosed with cerebral ALD (CALD)
- Adult males or females aged 18 to 60 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 without neurological disease
- Informed consent obtained from parents or guardians for children and from adult participants themselves
You will not qualify if you...
- Participation in a therapeutic clinical trial
- Treatment likely to modify the immune system
- Unable to have blood collection as judged by investigator (e.g., low hemoglobin levels)
- Any other reason at the discretion of the investigator
- Children or adults without health insurance or social security
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
Research Team
F
Fanny MOCHEL
CONTACT
C
Christelle AUGER
CONTACT
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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