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

X-Linked Adrenoleukodystrophy Clinical Trial

Official title:

A Non-interventional Study to Prospectively Assess Baseline Status and Disease Progression in Male Children With X-Linked Adrenoleukodystrophy

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03278899
• Other study ID #: NV1205-008
• Status: Recruiting
• Start date: February 16, 2018
• Est. completion date: June 1, 2021

Study information

• Verified date: May 2018
• Source: NeuroVia, Inc.
• Contact: Program Director
• Phone: 415-870-6967
• Email: patients[@]neurovia-inc.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 100
• Est. completion date: June 1, 2021
• Est. primary completion date: June 1, 2021
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 2 Years to 13 Years

Eligibility

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

Related Conditions & MeSH terms

• Adrenoleukodystrophy
• Disease Progression
• X-Linked Adrenoleukodystrophy

Locations

Country	Name	City	State
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	Stanford University	Palo Alto	California
United States	University of Pittsburgh	Pittsburgh	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
NeuroVia, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Loes score	Percent change from baseline in brain lesions assessed as Loes score will be calculated	baseline and 24 weeks
Primary	Plasma VLCFA levels	Change from baseline plasma VLCFA levels	baseline and 24 weeks
Primary	Neurological symptoms	Development of new neurological symptoms throughout the study	baseline and 24 weeks