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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05939232
Other study ID # KY2023-011-02
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date July 20, 2023
Est. completion date December 1, 2028

Study information

Verified date July 2023
Source Beijing Tiantan Hospital
Contact Yilong Wang, MD, PhD
Phone 0086-010-67092222
Email yilong528@aliyun.com
Is FDA regulated No
Health authority
Study type Observational [Patient Registry]

Clinical Trial Summary

This study is a observational study conducted through recruiting X-linked adrenoleukodystrophy (X-ALD) patients, to build a comprehensive evaluation and long-term follow-up platform for X-ALD patients, and to provide a theoretical basis for the treatment and management of X-ALD patients.


Description:

In this study, X-ALD patients will be selected as study participants. Through a longitudinal collection of genetic, imaging and clinical symptoms data of the patients and carriers. The purpose of this study is to build a comprehensive evaluation and long-term follow-up platform for X-ALD patients. Through the long-term follow-up of imaging and clinical symptoms progress and outcome of X-ALD patients, combined with genetic data, we will improve the relationships between genes and phenotypes, and explore the deep insight in the pathophysiological mechanism of X-ALD, to provide a theoretical basis for the treatment and management of X-ALD patients.


Recruitment information / eligibility

Status Recruiting
Enrollment 200
Est. completion date December 1, 2028
Est. primary completion date December 1, 2028
Accepts healthy volunteers Accepts Healthy Volunteers
Gender All
Age group 6 Years to 70 Years
Eligibility 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.

Study Design


Related Conditions & MeSH terms


Locations

Country Name City State
China Beijing Tiantan Hospital Beijing

Sponsors (1)

Lead Sponsor Collaborator
Beijing Tiantan Hospital

Country where clinical trial is conducted

China, 

Outcome

Type Measure Description Time frame Safety issue
Primary Epidemiological characteristics in Chinese X-ALD patients Constructing a Chinese X-ALD epidemiological information database by collecting epidemiological information from X-ALD patients. 5 years
Primary Clinical characteristics in Chinese X-ALD patients Constructing a Chinese X-ALD clinical sympotoms information database by collecting clinical symptoms information from X-ALD patients. 5 years
Primary Imaging characteristics in Chinese X-ALD patients Constructing a Chinese X-ALD patient's head MRI information database by collecting head MRI information from X-ALD patients. 5 years
Primary The disease development in Chinese X-ALD patients Record the progress of patients' clinical symptoms from baseline through 5-year follow-up. 5 years
Primary New pathogenic loci of Chinese X-ALD patients in whole genome sequencing/whole exon sequencing To find and identify new pathogenic loci of X-ALD in whole genome sequencing/whole exon sequencing through comparing the differences of whole genome sequencing/whole exon sequencing between X-ALD patients and carrier-control individuals. 5 years
Secondary Etiology of Chinese X-ALD patients Etiological factors,including genetic and environmental etiological factors, will be explored by genomic analysis, lasso regression, multiple logistic regression, and cox proportional hazards regression. 5 years
Secondary The change of imagin features in Chinese X-ALD patients Record the change of the patient's head MRI from baseline through follow-up every year. day 1, year 1, year 2, year 3, year 4, year 5
Secondary The associations between genetic factors and clinical characteristics of Chinese X-ALD patients ß coefficient and 95%CI values in linear regression, OR and 95%CI values in logistic regression analysis, and HR and 95%CI values in cox proportional hazards regression analysis for the associations between genetic factors and clinical characteristics of X-ALD. 5 years
Secondary The associations between genetic factors and outcomes of Chinese X-ALD patients ß coefficient and 95%CI values in linear regression, OR and 95%CI values in logistic regression analysis, and HR and 95%CI values in cox proportional hazards regression analysis for the associations between genetic factors and outcomes of X-ALD. 5 years
Secondary The associations between genetic factors and imaging characteristics of Chinese X-ALD patients ß coefficient and 95%CI values in linear regression, OR and 95%CI values in logistic regression analysis, and HR and 95%CI values in cox proportional hazards regression analysis for the associations between genetic factors and imaging characteristics of X-ALD. 5 years
Secondary The associations between genetic factors and the long-term changes of imaging characteristics of Chinese X-ALD patients ß coefficient and 95%CI values in linear regression, OR and 95%CI values in logistic regression analysis, and HR and 95%CI values in cox proportional hazards regression analysis for the associations between genetic factors and the long-term changes of imaging characteristics of X-ALD. 5 years
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