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

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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.