ClinConnect ClinConnect Logo
Search / Trial NCT04675749

Quality of Life in Women with X-linked Adrenoleukodystrophy

Launched by LEIPZIG UNIVERSITY MEDICAL CENTER · Dec 17, 2020

Trial Information

Current as of July 01, 2025

Recruiting

Keywords

Adrenoleukodystrophy Adrenomyeloneuropathy Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Demyelinating Diseases White Matter Disorder Genetic Diseases, X Linked Peroxisomal Disorders Metabolic Diseases Female Carrier Adrenoleukodystrophy, Women Heterozygous Carrier Quality Of Life

ClinConnect Summary

This clinical trial is studying the impact of X-linked adrenoleukodystrophy (X-ALD) on the quality of life for women who carry the gene for this condition. X-ALD is a rare genetic disorder that can lead to serious problems in the brain and spinal cord, and many women who are carriers experience symptoms related to a milder form of the disease known as adrenomyeloneuropathy (AMN). The researchers aim to find out how common these symptoms are in female carriers and how they affect daily activities, work, social life, sleep, mood, and intimacy.

To participate, women must be at least 18 years old and have a confirmed diagnosis of X-ALD, either through specific fatty acids in their blood or a genetic test showing a mutation in the ABCD1 gene. Participants will complete online questionnaires available in multiple languages to share their experiences. The trial is currently recruiting participants, and it's important to note that pregnant women cannot join. This study could provide valuable insights into the challenges faced by women living with X-ALD.

Gender

FEMALE

Eligibility 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

About Leipzig University Medical Center

Leipzig University Medical Center is a leading academic institution dedicated to advancing medical research and improving patient care through innovative clinical trials. With a strong emphasis on interdisciplinary collaboration, the center combines cutting-edge scientific research with practical clinical applications, fostering an environment that supports the development of new therapies and treatment protocols. Committed to ethical standards and patient safety, Leipzig University Medical Center plays a vital role in the healthcare community by conducting rigorous clinical studies that contribute to the understanding and management of various health conditions.

Locations

Leipzig, Saxony, Germany

Patients applied

0 patients applied

Trial Officials

Wolfgang Köhler, MD

Principal Investigator

Leipzig University Medical Center, Leukodystrophy Outpatient Clinic, Department of Neurology, Leipzig, Germany

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

Similar Trials