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

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Brief Title

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

Official Title

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

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 Type

Observational


Primary Outcome

Loes score


Condition

X-Linked Adrenoleukodystrophy



Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

100

Start Date

February 16, 2018

Completion Date

June 1, 2021

Primary Completion Date

June 1, 2021

Eligibility Criteria

        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
      

Gender

Male

Ages

2 Years - 13 Years

Accepts Healthy Volunteers

No

Contacts

John Henderson, MD, 415-870-6967, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT03278899

Organization ID

NV1205-008


Responsible Party

Sponsor

Study Sponsor

NeuroVia, Inc.


Study Sponsor

John Henderson, MD, Study Director, NeuroVia, Inc.


Verification Date

May 2018