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

Massachusetts General Hospital
Boston, Massachusetts 02114
Contact:
Camille Corre
617-724-6374
ccorre@partners.org

More Details

Status
Unknown status
Sponsor
NeuroVia, Inc.

Study Contact

Program Director
415-870-6967
patients@neurovia-inc.com

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.