Disease Progression in Women With X-linked Adrenoleukodystrophy

Last updated: January 29, 2024
Sponsor: Minoryx Therapeutics, S.L.
Overall Status: Active - Recruiting

Phase

N/A

Condition

Bone Marrow Disorder

Treatment

N/A

Clinical Study ID

NCT06178120
MT-NH-01
  • Ages > 18
  • Female

Study Summary

Observational, single-site prospective and minimally interventional study in women with X-linked adrenoleukodystrophy (ALD), conducted in France.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Women aged 18 years old or older.
  2. Diagnosis of X-linked ALD based on genetic testing, altered VLCFA levels, or familyhistory.
  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

Exclusion Criteria:

  1. Any condition that in the opinion of the investigator are likely to adversely affectthe study participation, interfere with study compliance, or confound the studyresults.
  2. Under treatment or previous treatment with leriglitazone.
  3. Pregnant or lactating women.
  4. Subjects benefiting from laws aimed at protecting vulnerable adults: subjects beingdeprived of liberty by judicial or administrative decision, subjects underguardianship.
  5. Participation in an interventional clinical trial.

Study Design

Total Participants: 40
Study Start date:
January 02, 2024
Estimated Completion Date:
June 30, 2027

Study Description

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.

Connect with a study center

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

    Paris, 75013
    France

    Active - Recruiting

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.