An observational study in X-linked Adrenoleukodystrophy, sponsored by Minoryx Therapeutics, S.L.. Recruiting at 1 site in France. Open to female participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-01-31.
Sponsored by Minoryx Therapeutics, S.L. · Observational
Observational, single-site prospective and minimally interventional study in women with X-linked adrenoleukodystrophy (ALD), conducted in France.
Patients accepting participation (after signing ICF) will be followed up to at least 2 years or until they started treatment for X-linked adrenoleukodystrophy (ALD) or withdraw consent, whichever occurs first. Follow-up will be extended beyond 2 years if deemed appropriate after an interim report. Tests and questionnaires will be assessed at baseline and yearly. If the study is extended, beyond 2 years, patients will be assessed at 1-year intervals.
At baseline visit and follow-up visits, patients will undergo an MRI of the brain and the spinal cord and assessments of body sway, EDSS, ADL, pain VAS and SF-36 questionnaire. Plasma biomarkers will be assessed from samples obtained through routine blood draw and a monthly falls diary will be provided each visit to be completed once a month.
This study will not assess any specific medicinal product or intervention, and the study will not interfere with that prescribed in clinical practice.
68 studies on the registry are indexed under Adrenoleukodystrophy; 19 are open to participants now.
This study's planned enrollment of 40 is below the median of 120 across 19 observational studies indexed under Adrenoleukodystrophy.
Browse Adrenoleukodystrophy studies →Minoryx Therapeutics, S.L. is the lead sponsor of 5 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Spinal cord magnetization transfer ratio (MTR)
To assess the disease evolution on spinal cord MRI
Time frame: Change from baseline up to 2 years
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 years
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 years
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 years
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 years
Expanded Disability Status Scale (EDSS)
To assess the impact on Quality of Life
Time frame: Change from baseline up to 2 years
Activities of Daily Living (ADL)
To assess the impact on Quality of Life
Time frame: Change from baseline up to 2 years
Pain Visual Analogue Scale (Pain VAS)
To assess the impact on Quality of Life
Time frame: Change from baseline up to 2 years
Monthly falls
To assess the impact on Quality of Life
Time frame: Change from baseline up to 2 years
Short Form Health Survey (SF-36)
To assess the impact on Quality of Life
Time frame: Change from baseline up to 2 years
Plasma biomarkers (NFL, MMP-9, IL-18, MIP-1beta and IL-1Ra)
To assess the evolution of plasma biomarkers
Time frame: Change from baseline up to 2 years
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
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Minoryx Therapeutics, S.L.