RecruitingNCT03789721

Adrenoleukodystrophy National Registry Study

Sponsor

Masonic Cancer Center, University of Minnesota

Enrollment

1,000 participants

Start Date

May 1, 2019

Study Type

OBSERVATIONAL

Conditions

Adrenoleukodystrophy ALD (Adrenoleukodystrophy)Cerebral Adrenoleukodystrophy

Summary

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.

Eligibility

Inclusion Criteria5

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 Criteria5

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.)

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