Adrenoleukodystrophy National Registry Study

Launched by MASONIC CANCER CENTER, UNIVERSITY OF MINNESOTA · Dec 26, 2018

Keywords

ClinConnect Summary

The Adrenoleukodystrophy National Registry Study is designed to help researchers learn more about how Adrenoleukodystrophy (ALD) progresses over time. This study aims to collect information from patients and their families to understand the disease better and find potential markers that could help in diagnosing and treating ALD in the future. If you or a family member has been diagnosed with ALD, you may be eligible to participate in this study.

To qualify, participants should be between 0 and 100 years old, live in the United States, and either have a confirmed diagnosis of ALD or have a family history that suggests a risk for the disease. Those who cannot give consent, have had certain treatments like bone marrow transplants, or face language barriers may not be able to join. If you participate, you can expect to share your health information, which may help advance our understanding of ALD and improve care for future patients. Your involvement could make a significant difference in the lives of others affected by this condition.

Gender

ALL

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.)