A Study to Prospectively Assess Disease Progression in Male Children With X-ALD

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

100 participants

Study Classification

OBSERVATIONAL

Study Start Date

2018-02-16

Study Completion Date

2021-06-01

Brief Summary

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This is a non-interventional, multi-center study that follows general principles of periodic assessment of X-ALD patients in routine practice. No study drug treatment will be given and no changes to patient treatment are necessary.

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Detailed Description

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This is a non-interventional, multi-center study that follows general principles of periodic assessment of X-ALD patients in routine practice. No study drug treatment will be given and no changes to the patient treatment are necessary.

Patients with X-ALD will be recruited and invited to attend a baseline visit. After eligible patients are enrolled, retrospective and baseline data will be collected. After enrollment, patients will be seen approximately every 6 months until they meet withdrawal criteria or the Sponsor terminates the study.

Conditions

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X-Linked Adrenoleukodystrophy

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

* Signed informed consent by patient's parent(s)/legally acceptable representative(s). In addition, signed children's assent form according to local requirements.
* Males patients 2-13 years of age with a confirmed diagnosis of X-ALD that fall into one of the following:

* Asymptomatic patients without MRI evidence of cerebral involvement
* Patients with MRI evidence of cerebral involvement (Loes score ≥0.5) with or without clinical symptoms
* Patient who have HSCT within 3 months from enrollment

Exclusion Criteria

* Patients who are 14 years of age or older
* Patients who are in a vegetative state
* Patients (or their guardians) who are unwilling or unable to comply with the study procedures
* Patients who received HSCT more than 3 months before enrollment

Minimum Eligible Age

2 Years

Maximum Eligible Age

13 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No