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



Status:Recruiting
Conditions:Endocrine
Therapuetic Areas:Endocrinology
Healthy:No
Age Range:2 - 13
Updated:8/10/2018
Start Date:February 16, 2018
End Date:June 1, 2021
Contact:Program Director
Email:patients@neurovia-inc.com
Phone:415-870-6967

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

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.

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.

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
We found this trial at
3
sites
Palo Alto, California 94304
Principal Investigator: Maura Ruzhnikov, MD
Phone: 650-498-9732
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185 Cambridge Street
Boston, Massachusetts 02114
617-724-5200
Principal Investigator: Florian S Eichler, MD
Phone: 617-724-6374
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4200 Fifth Ave
Pittsburgh, Pennsylvania 15260
(412) 624-4141
Principal Investigator: Maria L Escolar, MD, MS
Phone: 412-692-6351
University of Pittsburgh The University of Pittsburgh is a state-related research university, founded as the...
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