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.
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.
France1 site, 1 recruiting
- 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
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.
- Conditions
- X-linked Adrenoleukodystrophy
- Medical subject headings
- Adrenoleukodystrophy
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
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
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
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
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
Expanded Disability Status Scale (EDSS)
To assess the impact on Quality of Life
Measured: Change from baseline up to 2 years
Activities of Daily Living (ADL)
To assess the impact on Quality of Life
Measured: Change from baseline up to 2 years
Pain Visual Analogue Scale (Pain VAS)
To assess the impact on Quality of Life
Measured: Change from baseline up to 2 years
Monthly falls
To assess the impact on Quality of Life
Measured: Change from baseline up to 2 years
Short Form Health Survey (SF-36)
To assess the impact on Quality of Life
Measured: Change from baseline up to 2 years
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.
- First posted20 Dec 2023
- Study start2 Jan 2024
- Primary completionDec 2026 (estimated)
- Study completionJun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for X-linked AdrenoleukodystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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)
Sponsor and investigators
- Lead sponsor
- Minoryx Therapeutics, S.L.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
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
Study at a glance
From the registry record, last updated by the sponsor on 31 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational Study to Assess Disease Progression in Women With X-linked Adrenoleukodystrophy
- ClinicalTrials.gov ID
- NCT06178120
- Acronym / sponsor ID
- MT-NH-01
- Conditions
- X-linked Adrenoleukodystrophy
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Minoryx Therapeutics, S.L.Industry (pharma, biotech, devices)
- Study start
- 2 Jan 2024 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Jun 2027 (estimated)
- First posted
- 20 Dec 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- 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.
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Spinal cord magnetization transfer ratio (MTR)
Time frame: Change from baseline up to 2 years
To assess the disease evolution on spinal cord MRI
10 secondary outcomes
Spinal cord morphometry. Cross sectional area (CSA) to cover the C1 to C7 vertebral levels.
Time frame: 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.
Time frame: 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).
Time frame: 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)
Time frame: Change from baseline up to 2 years
Expanded Disability Status Scale (EDSS)
Time frame: Change from baseline up to 2 years
Activities of Daily Living (ADL)
Time frame: Change from baseline up to 2 years
Pain Visual Analogue Scale (Pain VAS)
Time frame: Change from baseline up to 2 years
Monthly falls
Time frame: Change from baseline up to 2 years
Short Form Health Survey (SF-36)
Time frame: Change from baseline up to 2 years
Plasma biomarkers (NFL, MMP-9, IL-18, MIP-1beta and IL-1Ra)
Time frame: Change from baseline up to 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jan 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
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?
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 last refreshed its copy on 7 Nov 2026.