Adrenoleukodystrophy Clinical Trial
Official title:
Adrenoleukodystrophy National Registry Study
The aim of this registry to understand the natural history and disease progression in ALD and potentially develop bio-markers using the biospecimens collected using this registry.
Status | Recruiting |
Enrollment | 1000 |
Est. completion date | February 2030 |
Est. primary completion date | February 2030 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility | Inclusion Criteria - Age 0 - 100 - ALD patients or family member meeting any of the following criteria: - Any patient diagnosed with ALD (confirmed by positive VLCFA testing and/or genetic mutation). - Known or presumed mutation with ALD based on pedigree or confirmed mutation in ABCD1 gene - Participants living in the United States and territories Exclusion Criteria - Patients diagnosed with ALD who lack the capacity to consent/assent AND do not have a designated legally authorized representative or guardian. - Patients who have undergone BMT or other cellular therapy . - Patients not fluent in English who are unable to consent in-person at the BMT Journey Clinic. - Patients who are illiterate - Patient determined by the PI or designee to be unlikely to complete required study components (due to language barriers, compliance issues, etc.) |
Country | Name | City | State |
---|---|---|---|
United States | Masonic Cancer Center at University of Minnesota | Minneapolis | Minnesota |
Lead Sponsor | Collaborator |
---|---|
Masonic Cancer Center, University of Minnesota |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Collect Clinical and Epidemiological Data | Collect clinical and epidemiological data through medical record abstraction and self-reported questionnaire survey semi-annually. | 10 Years |
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