Actively Recruiting
An Observational Study to Assess Disease Progression in Women With X-linked Adrenoleukodystrophy
Led by Minoryx Therapeutics, S.L. · Updated on 2024-01-31
40
Participants Needed
1
Research Sites
26 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This research investigates disease progression in women diagnosed with X-linked adrenoleukodystrophy (ALD) through an observational, minimally interventional study conducted in France. Participants will be followed for at least two years or until they begin treatment for ALD or withdraw, with possible extension beyond two years based on interim findings. The study aims to gather detailed information on disease changes over time without altering prescribed clinical care. Participants will undergo yearly assessments, including MRI scans of the brain and spinal cord, and evaluations of body sway, disability status (EDSS), daily living activities (ADL), pain levels (VAS), and quality of life (SF-36 questionnaire). Blood samples will be collected to measure plasma biomarkers, and participants will maintain a monthly falls diary. These assessments will occur at baseline and annually, continuing with yearly visits if the study extends beyond two years. During the study, participants will complete questionnaires and undergo imaging and physical assessments at scheduled visits. Researchers will monitor changes in spinal cord magnetization transfer ratio, spinal cord and brain imaging parameters, body sway, disability, pain, falls frequency, quality of life, and specific plasma biomarkers over time. The study does not involve any specific drug or intervention and will not interfere with treatments prescribed by the participants' healthcare providers.
CONDITIONS
Brief Title
Disease Progression in Women With X-linked Adrenoleukodystrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Women aged 18 years or older
- Diagnosis of X-linked ALD confirmed by genetic testing, altered VLCFA levels, or family history
- Willingness to attend annual follow-up visits including brain and spinal cord MRI scans
- Ability to provide written informed consent
- Affiliation or beneficiary of a French social security system or equivalent regime
You will not qualify if you...
- Any condition likely to negatively affect study participation, compliance, or results as judged by the investigator
- Current or past treatment with leriglitazone
- Pregnant or lactating women
- Individuals under legal protection such as guardianship or deprived of liberty by judicial or administrative decision
- Participation in any interventional clinical trial
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 1 day
Participants undergo baseline assessments including brain and spinal cord MRI scans and various clinical questionnaires and tests.
1 baseline visit (in-person)
Duration - At least 2 years
Participants are followed for at least 2 years or until they start treatment or withdraw consent, with yearly assessments including MRI scans, clinical tests, questionnaires, and plasma biomarker measurements. If the study extends beyond 2 years, assessments continue annually.
Annual visits for at least 2 years
Trial Site Locations
Total: 1 location
1
Paris Brain Institute (ICM) Centre Hospitalier Universitaire Pitié Salpêtrière
Paris, France, 75013
Actively Recruiting
Research Team
A
Arun Mistry
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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