Disease Progression in Women With X-linked Adrenoleukodystrophy

Status Recruiting

Clinical Trial Summary

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

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT06178120
Study type	Observational
Source	Minoryx Therapeutics, S.L.
Contact	Arun Mistry
Phone	+34935441466
Email	amistry@minoryx.com
Status	Recruiting
Phase
Start date	January 2, 2024
Completion date	June 2027

View Details

See also
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No longer available	`NCT02233257` - Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy	N/A