AzTrials
AzTrials

X-linked AdrenoleukodystrophyNCT06178120

Disease Progression in Women With X-linked Adrenoleukodystrophy

An observational study of X-linked Adrenoleukodystrophy, run by Minoryx Therapeutics, S.L. at 1 site in France.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

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

Who is it for?
Ages 18 years and older, women only
Conditions the study is for
X-linked Adrenoleukodystrophy
How many people
40 participants
Where
1 siteFrance
Study dates
2 Jan 2024to Jun 2027
Phase
Observational
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • Women only
  • No healthy volunteers
  • Adult, Older adult

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.

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Arun Mistry contact

+34935441466

amistry@minoryx.com

France1 site, 1 recruiting

  • Paris Brain Institute (ICM) Centre Hospitalier Universitaire Pitié Salpêtrière

    Paris, 75013

    Fanny Mochel, Professor, +33 1 57 27 40 00, fanny.mochel@icm-institute.org

    Fanny Mochel, Professor

    Recruiting

Other trials in France

About this study

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

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

Read the 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.

Medical subject headings
Adrenoleukodystrophy

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Spinal cord magnetization transfer ratio (MTR)

    To assess the disease evolution on spinal cord MRI

    Measured: Change from baseline up to 2 years

Secondary outcomes (10)

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

    To assess the disease evolution on spinal cord MRI

    Measured: Change from baseline up to 2 years

  2. 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

    Measured: Change from baseline up to 2 years

  3. 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

    Measured: Change from baseline up to 2 years

  4. 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

    Measured: Change from baseline up to 2 years

  5. Expanded Disability Status Scale (EDSS)

    To assess the impact on Quality of Life

    Measured: Change from baseline up to 2 years

  6. Activities of Daily Living (ADL)

    To assess the impact on Quality of Life

    Measured: Change from baseline up to 2 years

  7. Pain Visual Analogue Scale (Pain VAS)

    To assess the impact on Quality of Life

    Measured: Change from baseline up to 2 years

  8. Monthly falls

    To assess the impact on Quality of Life

    Measured: Change from baseline up to 2 years

  9. Short Form Health Survey (SF-36)

    To assess the impact on Quality of Life

    Measured: Change from baseline up to 2 years

  10. Plasma biomarkers (NFL, MMP-9, IL-18, MIP-1beta and IL-1Ra)

    To assess the evolution of plasma biomarkers

    Measured: Change from baseline up to 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Dec 2023
  2. Study start2 Jan 2024
  3. Primary completionDec 2026 (estimated)
  4. Study completionJun 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Case only
Time perspective
Prospective
Enrolment
40 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06178120
Sponsor's protocol ID
MT-NH-01
Status last verified
Dec 2023
First submitted
28 Nov 2023
Last updated
31 Jan 2024
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

An observational study of X-linked Adrenoleukodystrophy, run by Minoryx Therapeutics, S.L. at 1 site in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 31 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Arun Mistry (+34935441466, amistry@minoryx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Spinal cord magnetization transfer ratio (MTR) (time frame: Change from baseline up to 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06178120.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Jan 2024.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in