Status:
UNKNOWN
A Study to Prospectively Assess Disease Progression in Male Children With X-ALD
Lead Sponsor:
NeuroVia, Inc.
Conditions:
X-Linked Adrenoleukodystrophy
Eligibility:
MALE
2-13 years
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 p...
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 t...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
February 16 2018
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
June 1 2021
Estimated Enrollment :
100 Patients enrolled
Trial Details
Trial ID
NCT03278899
Start Date
February 16 2018
End Date
June 1 2021
Last Update
August 8 2018
Active Locations (3)
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1
Stanford University
Palo Alto, California, United States, 94304
2
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
3
University of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224