A Study to Prospectively Assess Disease Progression in Male Children With X-ALD
Purpose
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.
Condition
- X-Linked Adrenoleukodystrophy
Eligibility
- Eligible Ages
- Between 2 Years and 13 Years
- Eligible Genders
- Male
- Accepts Healthy Volunteers
- No
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
Study Design
- Phase
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Recruiting Locations
Boston, Massachusetts 02114
More Details
- Status
- Unknown status
- Sponsor
- NeuroVia, Inc.
Detailed Description
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.