Basic Studies

Open for recruitment
In progress

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

ClinicalTrials.gov ID NCT04925349

This research project studies the activation status of macrophages in patients with X-linked adrenoleukodystrophy, metachromatic leukodystrophy, and CSF1R-related leukodystrophy to better understand the mechanisms leading to neuroinflammation and demyelination in these diseases.

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Observational Studies

Open for recruitment
In progress

ARISE: Disease Progression in Women With X-linked Adrenoleukodystrophy

ClinicalTrials.gov ID NCT06178120

This single-center study focuses on women with adrenomyeloneuropathy and aims to identify biomarkers of disease progression to be used in future therapeutic trials.

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Open for recruitment
In progress

CALDIFF: Validation of a Prognostic Biomarker Using Brain Diffusion MRI in X-linked Adrenoleukodystrophy

ClinicalTrials.gov ID NCT05911919

This multicenter study focuses on men with a mutation in the ABCD1 gene, who have not yet developed the cerebral form of adrenoleukodystrophy. The study aims to demonstrate the diagnostic performance of a diffusion tensor imaging biomarker in detecting early-stage cerebral demyelination, independently of an expert panel.

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Therapeutic Trials

Open for recruitment
In progress

LEDIANT REGISTRY

As a condition of the marketing authorization under exceptional circumstances by the European Medicines Agency, the marketing authorization holder must maintain a patient registry for individuals with Cerebrotendinous xanthomatosis treated with chenodeoxycholic acid. The registry collects long-term real-world data on safety and effectiveness, including adverse events, liver function, and disease progression, to support ongoing evaluation of the medicine’s benefit–risk balance.

https://www.ema.europa.eu/en/medicines/human/EPAR/chenodeoxycholic-acid-leadiant

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Open for recruitment
In progress

CALYX : A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy.

Clinical Trial ID: NCT05819866

This ongoing phase 3 randomized, double-blind, placebo-controlled trial evaluates the efficacy and safety of leriglitazone in adult males with cerebral adrenoleukodystrophy who are not candidates for hematopoietic stem cell transplantation. Participants are assigned to receive either leriglitazone or placebo and are followed over 3 years. The primary endpoint is the time to major disease progression, defined as death or severe disability requiring permanent ventilatory support. Secondary outcomes include changes in MRI severity (Loes score).

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Recruitment closed
Finished

ADVANCE: A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients

ClinicalTrials.gov ID NCT03231878

This international multicenter therapeutic trial is testing the efficacy and safety of leriglitazone in men with adrenomyeloneuropathy. The study is now completed.

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Recruitment closed
Finished

IGNITE: A Study of VGL101 in Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia

ClinicalTrials.gov ID NCT05677659

This international multicenter therapeutic trial, conducted by VIGIL, aims to evaluate the therapeutic benefit of a monoclonal antibody targeting the TREM2 protein in CSF1R-related leukodystrophy.

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