Observing disease progression in women with X-linked Adrenoleukodystrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

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

Study contacts

• Study coordinator: Arun Mistry
• Email: amistry@minoryx.com
• Phone: +34935441466

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Registry for patients with X-linked Adrenoleukodystrophy

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  Recruiting Beijing Tiantan Hospital · Beijing · NCT05939232

• Assessing quality of life in women with X-linked adrenoleukodystrophy

  This study is trying to see how symptoms of X-linked adrenoleukodystrophy affect the quality of life for women who are carriers of the condition.

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• Collecting data and samples from patients with leukodystrophy

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  Recruiting at 23 sites Children's Hospital of Philadelphia · Los Angeles, California and 22 other locations · NCT03047369

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.