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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.
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| Measure | Description | Time Frame |
|---|---|---|
| Loes score | Percent change from baseline in brain lesions assessed as Loes score will be calculated | baseline and 24 weeks |
| Plasma VLCFA levels | Change from baseline plasma VLCFA levels | baseline and 24 weeks |
| Neurological symptoms | Development of new neurological symptoms throughout the study | baseline and 24 weeks |
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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:
Exclusion Criteria:
Males with a confirmed diagnosis of X-ALD with or without MRI evidence of cerebral involvement
Patients will be selected from participating sites, referring physicians, and outreach communities
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Program Director | Contact | 415-870-6967 | patients@neurovia-inc.com |
| Name | Affiliation | Role |
|---|---|---|
| John Henderson, MD | NeuroVia, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford University | Recruiting | Palo Alto | California | 94304 | United States |
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| ID | Term |
|---|---|
| D000326 | Adrenoleukodystrophy |
| ID | Term |
|---|---|
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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Biospecimen retained: Plasma
| Massachusetts General Hospital | Recruiting | Boston | Massachusetts | 02114 | United States |
|
| University of Pittsburgh | Recruiting | Pittsburgh | Pennsylvania | 15224 | United States |
|
| D009422 | Nervous System Diseases |
| D020279 | Hereditary Central Nervous System Demyelinating Diseases |
| D056784 | Leukoencephalopathies |
| D003711 | Demyelinating Diseases |
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D008661 | Metabolism, Inborn Errors |
| D018901 | Peroxisomal Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D000309 | Adrenal Insufficiency |
| D000307 | Adrenal Gland Diseases |
| D004700 | Endocrine System Diseases |