Observing disease progression in women with X-linked Adrenoleukodystrophy

An Observational Study to Assess Disease Progression in Women With X-linked Adrenoleukodystrophy

This study follows women with X-linked Adrenoleukodystrophy for at least two years to see how the disease progresses by checking their health and doing regular brain scans.

Quick facts

• Study type: Observational
• Enrollment: 40 (estimated)
• Ages: 18 Years and up
• Sex: Female
• Sponsor: Minoryx Therapeutics, S.L. (Industry-sponsored)
• Locations: 1 site (Paris)
• Trial ID: NCT06178120 on ClinicalTrials.gov

What this trial studies

This observational study follows women diagnosed with X-linked Adrenoleukodystrophy (ALD) over a period of at least two years to monitor disease progression. Participants will undergo annual assessments, including brain and spinal cord MRIs, physical evaluations, and questionnaires to gather data on their health status. The study aims to collect valuable information on the natural history of ALD without administering any specific treatments. Follow-up may extend beyond two years based on interim findings.

Who should consider this trial

Good fit: Ideal candidates are women aged 18 and older with a confirmed diagnosis of X-linked ALD.

Not a fit: Patients currently undergoing treatment with leriglitazone or those with conditions that may interfere with study compliance will not benefit from this study.

Why it matters

Potential benefit: If successful, this study could enhance understanding of X-linked ALD progression, leading to improved patient management and care strategies.

How similar studies have performed: While observational studies on ALD exist, this specific approach focusing on women with X-linked ALD is relatively novel.

Eligibility criteria

Inclusion Criteria:

1. Women aged 18 years old or older.
2. Diagnosis of X-linked ALD based on genetic testing, altered VLCFA levels, or family history.
3. Willing to undergo annual follow-up visits, including brain and spinal cord MRI scans.
4. Provision of written informed consent.
5. Affiliation or beneficiary of a French social security system or of such a regime.

Exclusion Criteria:

1. 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.
2. Under treatment or previous treatment with leriglitazone.
3. Pregnant or lactating women.
4. Subjects benefiting from laws aimed at protecting vulnerable adults: subjects being deprived of liberty by judicial or administrative decision, subjects under guardianship.
5. Participation in an interventional clinical trial.

Where this trial is running

Paris

• Paris Brain Institute (ICM) Centre Hospitalier Universitaire Pitié Salpêtrière — Paris, France (Recruiting)

Study contacts

• Study coordinator: Arun Mistry

Conditions X-linked Adrenoleukodystrophy