RECRUITINGOBSERVATIONAL
Adrenoleukodystrophy National Registry Study
About This Trial
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.
Who May Be Eligible (Plain English)
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.)
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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.)
Treatments Being Tested
OTHER
Medical Record Abstraction
Collect clinical and epidemiological data through medical record abstraction and self-reported questionnaire survey semi-annually.
OTHER
Biospecimen Sample Collection
Collect research samples, when feasible for those diagnosed with ALD.
Locations (1)
Masonic Cancer Center at University of Minnesota
Minneapolis, Minnesota, United States