Assessing quality of life in women with X-linked adrenoleukodystrophy
Official title: Quality of Life in Female Carriers of X-linked Adrenoleukodystrophy
Run by Leipzig University Medical Center · NCT04675749 · Data updated 2026-11-12
This study is trying to see how symptoms of X-linked adrenoleukodystrophy affect the quality of life for women who are carriers of the condition.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 18 Years and up |
| Sex | Women only |
| Enrollment | 200 (estimated) |
| Sponsor | Leipzig University Medical Center Academic / other |
| Locations | 1 site (Leipzig, Saxony) |
| Status | Recruiting |
| Started | December 1, 2019 |
| Main results expected | January 1, 2028 |
| Study ends | March 1, 2028 |
| Registry record updated | August 27, 2026 |
| Trial ID | NCT04675749 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates for this study are women aged 18 and older who are confirmed carriers of X-ALD.
✕Probably not a fit
Patients who are not female or who are currently pregnant will not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This observational study focuses on women who are carriers of X-linked adrenoleukodystrophy (X-ALD), a genetic disorder that can lead to significant neurological symptoms. The study aims to evaluate the prevalence of symptoms associated with adrenomyeloneuropathy (AMN) in these women and to assess how these symptoms affect their quality of life. Participants must be female, at least 18 years old, and have confirmed X-ALD through genetic testing or elevated fatty acid levels. The study is conducted at Leipzig University Medical Center in Germany.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the impact of X-ALD on women's health and quality of life, potentially guiding future treatment and support strategies.
How similar studies have gone: While there have been studies on X-ALD, this specific focus on the quality of life in female carriers is relatively novel.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Informed consent obtained from the participant * Females ≥18 years at the time of consent, with proven X-ALD as defined by 1. Elevated VLCFA values, or 2. Mutation in ABCD1 gene Exclusion Criteria: * No informed consent and assent * Current pregnancy
Where it's running
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Leipzig University Medical Center, Leukodystrophy Outpatient Clinic, Department of Neurology, Leipzig, Germany
Leipzig, Saxony, Germany Recruiting
Study contacts
- Principal investigator: Wolfgang Köhler, MD — Leipzig University Medical Center, Leukodystrophy Outpatient Clinic, Department of Neurology, Leipzig, Germany
- Study coordinator: Lisa Schäfer, PhD
- Email: lisa.schaefer@medizin.uni-leipzig.de
- Phone: +49-341-9720086
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Observing disease progression in women with X-linked Adrenoleukodystrophy
This study follows women with X-linked Adrenoleukodystrophy for at least two years to see how the disease progresses by checking their health and doing regular brain scans.
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Registry for patients with X-linked Adrenoleukodystrophy
This study is gathering information from people with X-linked adrenoleukodystrophy to better understand the condition and improve treatment options.
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Collecting data and samples from patients with leukodystrophy
This study is collecting health information and samples from people with leukodystrophy to help find new causes and better treatments for the condition.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.