NCT06178120

Disease Progression in Women With X-linked Adrenoleukodystrophy

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

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 40 (estimated)
• Sponsor: Minoryx Therapeutics, S.L. · Industry
• Sex: Female
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed 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.

Conditions

• X-linked Adrenoleukodystrophy

Timeline

• Start date: 2024-01-02
• Primary completion: 2026-12-01
• Completion: 2027-06-01
• First posted: 2023-12-20
• Last updated: 2024-01-31

Locations

1 site across 1 country: France