Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia

Recruiting Observational Study
Adrenoleukodystrophy Adrenomyeloneuropathy Metachromatic Leukodystrophy Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Healthy Volunteers Welcome You do not need to have the condition being studied to take part. No Study Drug Researchers observe your health over time — no experimental treatment is given.
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At a Glance
Age
15 Months – 60
Sex
Any
Study type
Observational
Participants needed
100 (estimated)
Sponsor
Assistance Publique - Hôpitaux de Paris · Other
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About This Trial
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 the…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: Boys aged between 3 and 18 years (inclusive) diagnosed with C-CALD (elevated levels of VLCFA and leukodystrophy at brain MRI) * Boys or girls aged between 15 months and 18 years (inclusive) diagnosed with MLD (low ARSA activity and accumulation of sulfatides in urine) * Presymp…
Contacts

Fanny MOCHEL

01 57 27 44 82

fanny.mochel@icm-institut.org

CONTACT

Christelle AUGER

christelle.auger@aphp.fr

CONTACT