A Non-interventional Study to Prospectively Assess Baseline Status and Disease Progression in Male Children With X-Linked Adrenoleukodystrophy
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- X-Linked Adrenoleukodystrophy
- Sponsor
- NeuroVia, Inc.
- Enrollment
- 100
- Locations
- 3
- Primary Endpoint
- Loes score
- Last Updated
- 7 years ago
Overview
Brief Summary
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.
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.
Investigators
Eligibility Criteria
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
Outcomes
Primary Outcomes
Loes score
Time Frame: baseline and 24 weeks
Percent change from baseline in brain lesions assessed as Loes score will be calculated
Neurological symptoms
Time Frame: baseline and 24 weeks
Development of new neurological symptoms throughout the study
Plasma VLCFA levels
Time Frame: baseline and 24 weeks
Change from baseline plasma VLCFA levels