X-linked Adrenoleukodystrophy Clinical Trial
Official title:
Registry of X-linked Adrenoleukodystrophy
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.
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. |
Country | Name | City | State |
---|---|---|---|
China | Beijing Tiantan Hospital | Beijing |
Lead Sponsor | Collaborator |
---|---|
Beijing Tiantan Hospital |
China,
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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