Disease Progression in Women With X-linked Adrenoleukodystrophy
ConditionX-linked Adrenoleukodystrophy
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexFemale
Age18+
SponsorMinoryx Therapeutics, S.L.
About this trial
Observational, single-site prospective and minimally interventional study in women with X-linked adrenoleukodystrophy (ALD), conducted in France.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Women aged 18 years old or older.
Diagnosis of X-linked ALD based on genetic testing, altered VLCFA levels, or family history.
Willing to undergo annual follow-up visits, including brain and spinal cord MRI scans.
Provision of written informed consent.
Disqualifiers
Any condition that in the opinion of the investigator are likely to adversely affect the study participation, interfere with study compliance, or confound the study results.
Under treatment or previous treatment with leriglitazone.
Pregnant or lactating women.
Subjects benefiting from laws aimed at protecting vulnerable adults: subjects being deprived of liberty by judicial or administrative decision, subjects under guardianship.
Trial population
The study will include up to 40 adult women with X-linked ALD who attend to a regular visit, who meet all inclusion criteria and none of the exclusion criteria.
Trial design
Design model
Case-only
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
Spinal cord magnetization transfer ratio (MTR)
To assess the disease evolution on spinal cord MRI
Time frame
Change from baseline up to 2 yearsSecondary outcomes
1
Spinal cord morphometry. Cross sectional area (CSA) to cover the C1 to C7 vertebral levels.
To assess the disease evolution on spinal cord MRI
Time frame
Change from baseline up to 2 years2
Spinal cord diffusion image analysis parameters: Fractional Anisotropy (FA), Radial Diffusivity (RD), Axial Diffusivity (AD), Mean Diffusivity (MD) at the cervical levels C1-C5.
To assess the disease evolution on spinal cord MRI
Time frame
Change from baseline up to 2 years3
Brain diffusion image analysis parameters: Fixel-Based analysis (FBA) (Fiber Density (FD), Fiber cross-section (FC) and combination of FD and FC), Diffusion tensor imaging (DTI) (FA, RD, AD, MD, Markers of severity and markers of evolution).
To assess the disease evolution on brain MRI
Time frame
Change from baseline up to 2 years4
Body sway amplitude (considering antero-posterior and medio-lateral sway with eyes closed, feet apart; eyes open, feet apart; eyes closed, feet together; eyes open, feet together)
To assess clinical evolution of myelopathy
Time frame
Change from baseline up to 2 yearsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov