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Adrenoleukodystrophy National Registry Study
RecruitingObservational study
Who can join
All ages · All sexes
Full eligibility criteria
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.)
About the 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.
What is being tested
- Medical Record Abstraction (other)
- Biospecimen Sample Collection (other)
Sponsor: Masonic Cancer Center, University of Minnesota · Participants: 1,000 · Started: May 1, 2019
Contact the study team
- Ashish Gupta, MD · Phone: 612-626-2961
- Paul Orchard, MD · Phone: 612-626-2961
Official record on ClinicalTrials.gov — NCT03789721
Locations in the U.S.
| Minnesota | Masonic Cancer Center at University of Minnesota, Minneapolis |
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.