Disease Progression in Women With X-linked Adrenoleukodystrophy

X-linked Adrenoleukodystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06178120
• Other study ID #: MT-NH-01
• Status: Recruiting
• Start date: January 2, 2024
• Est. completion date: June 2027

Study information

• Verified date: December 2023
• Source: Minoryx Therapeutics, S.L.
• Contact: Arun Mistry
• Phone: +34935441466
• Email: amistry[@]minoryx.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: June 2027
• Est. primary completion date: December 2026
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Adrenoleukodystrophy
• Disease Progression
• X-linked Adrenoleukodystrophy

Locations

Country	Name	City	State
France	Paris Brain Institute (ICM) Centre Hospitalier Universitaire Pitié Salpêtrière	Paris

Sponsors (1)

Lead Sponsor	Collaborator
Minoryx Therapeutics, S.L.

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Spinal cord magnetization transfer ratio (MTR)	To assess the disease evolution on spinal cord MRI	Change from baseline up to 2 years
Secondary	Spinal cord morphometry. Cross sectional area (CSA) to cover the C1 to C7 vertebral levels.	To assess the disease evolution on spinal cord MRI	Change from baseline up to 2 years
Secondary	Spinal cord diffusion image analysis parameters: Fractional Anisotropy (FA), Radial Diffusivity (RD), Axial Diffusivity (AD), Mean Diffusivity (MD) at the cervical levels C1-C5.	To assess the disease evolution on spinal cord MRI	Change from baseline up to 2 years
Secondary	Brain diffusion image analysis parameters: Fixel-Based analysis (FBA) (Fiber Density (FD), Fiber cross-section (FC) and combination of FD and FC), Diffusion tensor imaging (DTI) (FA, RD, AD, MD, Markers of severity and markers of evolution).	To assess the disease evolution on brain MRI	Change from baseline up to 2 years
Secondary	Body sway amplitude (considering antero-posterior and medio-lateral sway with eyes closed, feet apart; eyes open, feet apart; eyes closed, feet together; eyes open, feet together)	To assess clinical evolution of myelopathy	Change from baseline up to 2 years
Secondary	Expanded Disability Status Scale (EDSS)	To assess the impact on Quality of Life	Change from baseline up to 2 years
Secondary	Activities of Daily Living (ADL)	To assess the impact on Quality of Life	Change from baseline up to 2 years
Secondary	Pain Visual Analogue Scale (Pain VAS)	To assess the impact on Quality of Life	Change from baseline up to 2 years
Secondary	Monthly falls	To assess the impact on Quality of Life	Change from baseline up to 2 years
Secondary	Short Form Health Survey (SF-36)	To assess the impact on Quality of Life	Change from baseline up to 2 years
Secondary	Plasma biomarkers (NFL, MMP-9, IL-18, MIP-1beta and IL-1Ra)	To assess the evolution of plasma biomarkers	Change from baseline up to 2 years