Disease Progression in Women With X-linked Adrenoleukodystrophy

Minoryx

Status: Enrolling

Conditions: X-linked Adrenoleukodystrophy

Study type: Observational

Funder types: Industry

Identifiers

NCT06178120

MT-NH-01

Details and patient eligibility

About

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

Full 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.

Enrollment: 40 estimated patients

Sex: Female

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.