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

NCT05911919 · Status: NOT_YET_RECRUITING · Type: OBSERVATIONAL · Enrollment: 200

Last updated 2023-07-03

No results posted yet for this study

Summary

CALD is an inflammatory demyelinating disease that causes severe motor and cognitive deficit leading to rapid death. Hematopoietic stem cell transplantation (HSCT) can halt neuroinflammation in CALD through the replacement of microglia (i.e., brain immune system) but only if performed during its early phase.

Using standard brain MRI, it is estimated that only 30% of adult CALD patients are identified. Complex and lengthy clinical evaluations together with MRI reading from experts improve CALD detection but are not available in routine clinical practice.

Diffusion tensor imaging is a quantitative microstructural technique that can identify neuroinflammation at a very early stage. Still, its implementation in clinical practice has been very limited due to high inter-center measurements variability and bias due to data quality issues. The approach we will use solves these problems by introducing an automatic calibration and standardization with systematic quality control enabling the use of all MRI scanners in clinical settings.

The innovative aspect of this project lies on the validation of an expert-independent prognosis biomarker able to specifically identify patients at high-risk to convert to CALD so that treatment can be initiated at the early stage of neuroinflammation.

We aim to demonstrate that this tool has at least a 2-fold sensitivity compared to the current standard of care.

Conditions

  • X-linked Adrenoleukodystrophy

Interventions

DEVICE

brainQuant module of brainTale-care platform

brainQuant is a software module of brainTale-care medical device (a medical device software as a service (web platform) ) for the automatic processing of brain diffusion Magnetic resonance images and the provision of the diffusion regional standardised parameters.

Sponsors & Collaborators

  • Braintale

    collaborator INDUSTRY

  • Assistance Publique - Hôpitaux de Paris

    lead OTHER

Principal Investigators

  • Fanny MOCHEL, MD, PhD · APHP - Pitié-Salpêtrière Hospital

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2023-09-30
Primary Completion
2028-09-30
Completion
2028-09-30

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05911919 on ClinicalTrials.gov