An observational study in X-linked Adrenoleukodystrophy, sponsored by Beijing Tiantan Hospital. Recruiting at 1 site in China. Open to participants aged 6 Years to 70 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2023-07-11.
Sponsored by Beijing Tiantan Hospital · Observational
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.
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.
68 studies on the registry are indexed under Adrenoleukodystrophy; 19 are open to participants now.
This study's planned enrollment of 200 is above the median of 120 across 19 observational studies indexed under Adrenoleukodystrophy.
Browse Adrenoleukodystrophy studies →Beijing Tiantan Hospital is the lead sponsor of 489 studies on the registry; 293 are open to participants now.
Counted across the registry records on this site, refreshed daily.
X-linked adrenoleukodystrophy patients, and age,sex and education matched healthy carriers will be recruited from the hospital.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
X-ALD group：
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:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
X-linked adrenoleukodystrophy (X-ALD) patients.
Carriers of mutation in gene encoding ATP-binding cassette subfamily D member 1 (ABCD1), who have no X-linked adrenoleukodystrophy and are matched with the X-ALD group according to age, sex and education.
Epidemiological characteristics in Chinese X-ALD patients
Constructing a Chinese X-ALD epidemiological information database by collecting epidemiological information from X-ALD patients.
Time frame: 5 years
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.
Time frame: 5 years
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.
Time frame: 5 years
The disease development in Chinese X-ALD patients
Record the progress of patients' clinical symptoms from baseline through 5-year follow-up.
Time frame: 5 years
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.
Time frame: 5 years
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.
Time frame: 5 years
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.
Time frame: day 1, year 1, year 2, year 3, year 4, year 5
The associations between genetic factors and clinical characteristics of Chinese X-ALD patients
β 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.
Time frame: 5 years
The associations between genetic factors and outcomes of Chinese X-ALD patients
β 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.
Time frame: 5 years
The associations between genetic factors and imaging characteristics of Chinese X-ALD patients
β 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.
Time frame: 5 years
The associations between genetic factors and the long-term changes of imaging characteristics of Chinese X-ALD patients
β 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.
Time frame: 5 years
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