A Study to Prospectively Assess Disease Progression in Male Children With X-ALD

Status unconfirmed

Conditions studied: X-Linked Adrenoleukodystrophy

In brief

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.

Key facts

Study ID

NCT03278899

Run by

NeuroVia, Inc.

People needed

100

Starts

2018-02-16

Expected to finish

2021-06-01

Last updated by the study team

2018-08-08

Who can join

Age: 2 and older, up to 13. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Stanford University — Palo Alto, California, United States (enrolling)
• Massachusetts General Hospital — Boston, Massachusetts, United States (enrolling)
• University of Pittsburgh — Pittsburgh, Pennsylvania, United States (enrolling)

Full record on ClinicalTrials.gov

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