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

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

Status

UNKNOWN

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT03278899

Enrollment

100

Registered

2017-09-12

Start date

2018-02-16

Completion date

2021-06-01

Last updated

2018-08-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

X-Linked Adrenoleukodystrophy

Keywords

Adrenoleukodystropy, X-ALD, ALD

Brief summary

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

MALE

Age

2 Years to 13 Years

Healthy volunteers

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

Design outcomes

Primary

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

Countries

United States

Contacts

Primary ContactProgram Director

patients@neurovia-inc.com415-870-6967

Outcome results