Adrenoleukodystrophy National Registry Study
Status: Recruiting
Location: See location...
Intervention Type: Other
Study Type: Observational
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
Participation Requirements
Sex: All
Healthy Volunteers: f
View:
• 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
Locations
United States
Minnesota
Masonic Cancer Center at University of Minnesota
RECRUITING
Minneapolis
Contact Information
Primary
Ashish Gupta, MD
gupta461@umn.edu
612-626-2961
Backup
Paul Orchard, MD
orcha001@umn.edu
612-626-2961
Time Frame
Start Date: 2019-05-01
Estimated Completion Date: 2030-02
Participants
Target number of participants: 1000
Treatments
Adrenoleukodystrophy
All patients living in the United States diagnosed with adrenoleukodystrophy, either by newborn screen, based on family history or otherwise, are eligible to participate in this study.
Authors
Ashish Gupta
Related Therapeutic Areas
Sponsors
Leads: Masonic Cancer Center, University of Minnesota