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

• Conditions: X-Linked Adrenoleukodystrophy

• Registration Number: NCT03278899
• Lead Sponsor: NeuroVia, Inc.

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 patient treatment are necessary.

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.

Study Timeline

• Study First Submitted: 2017-08-30
• Study First Submitted QC: 2017-09-07
• Study First Posted: 2017-09-12
• Study Start: 2018-02-16
• Status Verified: 2018-05
• Last Update Submitted: 2018-08-07
• Last Update Posted: 2018-08-08
• Primary Completion: 2021-06-01
• Study Completion: 2021-06-01

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: Male
• Target Recruitment: 100

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

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Loes score	baseline and 24 weeks	Percent change from baseline in brain lesions assessed as Loes score will be calculated
Neurological symptoms	baseline and 24 weeks	Development of new neurological symptoms throughout the study
Plasma VLCFA levels	baseline and 24 weeks	Change from baseline plasma VLCFA levels

Trial Locations

Locations (3)

Stanford University; 🇺🇸 Palo Alto, California, United States

University of Pittsburgh; 🇺🇸 Pittsburgh, Pennsylvania, United States

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States