Registry for patients with X-linked Adrenoleukodystrophy

Is this trial a fit for you?

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 aims to recruit patients diagnosed with X-linked adrenoleukodystrophy (X-ALD) to create a comprehensive evaluation and long-term follow-up platform. Participants will undergo longitudinal collection of genetic, imaging, and clinical symptom data to enhance understanding of the relationship between genes and phenotypes. The study seeks to explore the pathophysiological mechanisms of X-ALD, ultimately providing a theoretical basis for improved treatment and management strategies for affected individuals.

Why it matters

Who can join: the full rules

Inclusion Criteria:

X-ALD group：

* Meet the diagnostic criteria of X-ALD and supported by the results of genetic and very long chain fatty acid (VLCFA) test;
* Age: 6 - 70 years old;
* Able to communicate normally, and complete the test of scale as instructed (confirmed by the field test of scale);
* Sign the informed consent.

Carrier-control group:

Healthy people who have no significant difference in age, sex and education comparing with the X-ALD group, volunteer to participate in this study, could complete the test of scale as instructed, and meet the following criteria:

* Eligible for asymptomatic carriers in genetic tests (preference of patient's mother and close relatives);
* Age: 6 - 70 years old, able to complete the test of scale as instructed (confirmed by the field test of scale);
* No history of psychiatric diseases.

Exclusion Criteria:

* Other hereditary diseases;
* Other severe central nervous diseases;
* History of surgery of brain or eye;
* Psychiatric and psychological diseases, such as anxiety and depression;
* Metal foreign body or prosthesis in the human body (such as pacemaker and insulin pump), claustrophobia, and other MRI contraindications;
* History of surgery associated with gastrointestinal tract;
* No informed consent;
* Unable to tolerate MRI or eye related tests.

Where it's running

• Beijing Tiantan Hospital
  Beijing, China Recruiting

Study contacts

• Principal investigator: Yilong Wang, MD,PhD — Beijing Tiantan Hospital, Capital Medical University, Beijing, China
• Study coordinator: Yilong Wang, MD, PhD
• Email: yilong528@aliyun.com
• Phone: 0086-010-67092222

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• 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.

  Recruiting Minoryx Therapeutics, S.L. · Paris · NCT06178120

• Assessing quality of life in women with X-linked adrenoleukodystrophy

  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.

  Recruiting Leipzig University Medical Center · Leipzig, Saxony · NCT04675749

• 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.

  Recruiting at 23 sites Children's Hospital of Philadelphia · Los Angeles, California and 22 other locations · NCT03047369

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.