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

NeuroVia, Inc.100 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

100

Conditions

X-Linked Adrenoleukodystrophy

Eligibility

Sex: MALEMin age: 2 YearsMax age: 13 Years

Medical Language ↔ Plain English

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

Locations (3)

Stanford University

Palo Alto, California, United States

RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, United States

RECRUITING

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

RECRUITING

Outcomes

Primary Outcomes

Loes score

Percent change from baseline in brain lesions assessed as Loes score will be calculated

Time frame: baseline and 24 weeks

Plasma VLCFA levels

Change from baseline plasma VLCFA levels

Time frame: baseline and 24 weeks

Neurological symptoms

Development of new neurological symptoms throughout the study

Time frame: baseline and 24 weeks

Central Contacts

Program Director

CONTACT

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

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.