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

A Non-interventional Study to Prospectively Assess Baseline Status and Disease Progression in Male Children With X-Linked Adrenoleukodystrophy

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.

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.

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