About this trial

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.

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

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.

Disqualifiers

Other hereditary diseases;

Other severe central nervous diseases;

History of surgery of brain or eye;

Psychiatric and psychological diseases, such as anxiety and depression;

Trial population

X-linked adrenoleukodystrophy patients, and age,sex and education matched healthy carriers will be recruited from the hospital.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

200 Participants

are grouped into 2 trial groups

Group A: X-ALD

Group B: Carrier-control (CC)

Trial outcomes

Primary outcomes

1

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

2

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

3

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

4

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

Secondary outcomes

1

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

2

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

3

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

4

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

Registry of X-linked Adrenoleukodystrophy

About this trial

Eligibility criteria

Qualifiers

Disqualifiers

Trial population

Trial design

Treatments tested in this trial

Trial groups

Trial outcomes

Primary outcomes

Secondary outcomes

Other outcomes

Sponsors and contacts