RecruitingNCT06178120

Disease Progression in Women With X-linked Adrenoleukodystrophy

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


Sponsor

Minoryx Therapeutics, S.L.

Enrollment

40 participants

Start Date

Jan 2, 2024

Study Type

OBSERVATIONAL

Conditions

Summary

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


Eligibility

Sex: FEMALEMin Age: 18 Years

Inclusion Criteria5

  • 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.

Exclusion Criteria5

  • 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.

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Locations(1)

Paris Brain Institute (ICM) Centre Hospitalier Universitaire Pitié Salpêtrière

Paris, France

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NCT06178120


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