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

Launched by ASSISTANCE PUBLIQUE - HÔPITAUX DE PARIS · Jun 12, 2023

Keywords

ClinConnect Summary

This clinical trial is focused on improving the detection of X-linked Adrenoleukodystrophy (ALD), a serious brain condition that can lead to severe physical and mental decline. The researchers aim to validate a new tool using advanced MRI technology to identify patients who are at high risk of developing a more severe form of ALD called Cerebral Adrenoleukodystrophy (CALD). This new method is designed to be more sensitive than the current standard tests, helping doctors start treatment earlier when it can be most effective.

To participate in this trial, individuals must be male, at least 18 years old, and genetically diagnosed with ALD. Healthy volunteers aged 18 to 65 are also welcome. Participants will undergo MRI scans to test this new technique, which will be done in a comfortable and supportive setting. It's important to note that certain individuals, such as those with other serious neurological conditions or those who cannot undergo an MRI for various reasons, may not be eligible. This trial is not yet recruiting but aims to offer a promising advancement in the early detection and treatment of ALD.

Gender

ALL

Eligibility criteria

• Patients:
• • Inclusion criteria
• • \> 18 years old
• • Male gender
• • Genetically diagnosed with ALD by carrying an ABCD1 pathogenic variant
• • Signed written informed consent
• • Affiliation to a social security regime (patient with AME cannot be included)
• • Exclusion criteria
• • Patient with non-arrested CALD
• • Other neurological conditions such as brain tumour, stroke or a traumatic head injury
• • Contraindication to MRI (claustrophobia, implanted metal components in the head, panic disorder, epilepsy)
• • People under legal protection measure (tutorship, curatorship or safeguard measures)
• • Patient included in another interventional clinical trial
• • Healthy volunteers
• • Inclusion criteria
• • Male or female subject from 18 to 65 years old
• • In general good health condition
• • Signed written informed consent
• • Affiliation to a social security regime (healthy volunteers with AME cannot be enrolled)
• • Exclusion criteria
• • People under legal protection measure (tutorship, curatorship or safeguard measures)
• • Contraindication to MRI (claustrophobia, implanted metal components in the head, panic disorder, epilepsy)
• • History of previous brain disease (including but not limited to cranial trauma in the last 12 months, glioma, stroke, neurodegenerative diseases)
• • Arterial hypertension as defined in WHO criteria
• • Volunteer included in another interventional clinical trial
• • Pregnancy and breastfeeding women