Disease Progression in Women With X-linked Adrenoleukodystrophy

NCT06178120 | Recruiting

• 1 other identifier: MT-NH-01
• Study Type: observational
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

X-linked Adrenoleukodystrophy

Outcome Measures

Primary Outcomes (1)

• Spinal cord magnetization transfer ratio (MTR)

  To assess the disease evolution on spinal cord MRI

  Change from baseline up to 2 years

Secondary Outcomes (10)

• Spinal cord morphometry. Cross sectional area (CSA) to cover the C1 to C7 vertebral levels.

  Change from baseline up to 2 years

• Spinal cord diffusion image analysis parameters: Fractional Anisotropy (FA), Radial Diffusivity (RD), Axial Diffusivity (AD), Mean Diffusivity (MD) at the cervical levels C1-C5.

  Change from baseline up to 2 years

• 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).

  Change from baseline up to 2 years

• 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)

  Change from baseline up to 2 years

• Expanded Disability Status Scale (EDSS)

  Change from baseline up to 2 years

Eligibility Criteria

• Age: 18 Years+
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study will include up to 40 adult women with X-linked ALD who attend to a regular visit, who meet all inclusion criteria and none of the exclusion criteria.

You may qualify if:

• Women aged 18 years old or older.
• Diagnosis of X-linked ALD based on genetic testing, altered VLCFA levels, or family history.
• Willing to undergo annual follow-up visits, including brain and spinal cord MRI scans.
• Provision of written informed consent.
• Affiliation or beneficiary of a French social security system or of such a regime.

You may not qualify if:

• 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.
• Under treatment or previous treatment with leriglitazone.
• Pregnant or lactating women.
• Subjects benefiting from laws aimed at protecting vulnerable adults: subjects being deprived of liberty by judicial or administrative decision, subjects under guardianship.
• Participation in an interventional clinical trial.

Sponsors & Collaborators

• Minoryx Therapeutics, S.L.: lead

Study Sites (1)

Paris Brain Institute (ICM) Centre Hospitalier Universitaire Pitié Salpêtrière

Paris, 75013, France

RECRUITING

MeSH Terms

Conditions

Adrenoleukodystrophy

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Hereditary Central Nervous System Demyelinating Diseases; Leukoencephalopathies; Demyelinating Diseases; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Metabolism, Inborn Errors; Peroxisomal Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Insufficiency; Adrenal Gland Diseases; Endocrine System Diseases

Central Study Contacts

Arun Mistry

CONTACT

+34935441466; amistry@minoryx.com

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 28, 2023
• First Posted: December 20, 2023
• Study Start: January 2, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): June 1, 2027
• Last Updated: January 31, 2024

Record last verified: 2023-12

Locations

FR (1)