Registry for understanding Adrenoleukodystrophy
Official title: Adrenoleukodystrophy National Registry Study
Run by Masonic Cancer Center, University of Minnesota · NCT03789721 · Data updated 2026-11-12
This study is collecting health information and samples from people with Adrenoleukodystrophy to better understand the disease and find ways to improve treatment.
Quick facts
| Study type | Observational |
|---|---|
| Sex | All |
| Enrollment | 1000 (estimated) |
| Sponsor | Masonic Cancer Center, University of Minnesota Academic / other |
| Locations | 1 site (Minneapolis, Minnesota) |
| Status | Recruiting |
| Started | May 1, 2019 |
| Main results expected | February 2030 |
| Study ends | February 2030 |
| Registry record updated | May 12, 2026 |
| Trial ID | NCT03789721 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include individuals diagnosed with ALD or family members with known or presumed mutations in the ABCD1 gene.
✕Probably not a fit
Patients who have undergone bone marrow transplantation or other cellular therapies may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This registry aims to understand the natural history and disease progression of Adrenoleukodystrophy (ALD) by collecting and maintaining biospecimens from participants with confirmed or presumed ALD. Participants, including pediatric and adult patients, will provide medical histories and longitudinal biospecimens such as blood and urine samples. The study seeks to assemble a comprehensive resource of clinical, medical, and biological data to identify prognostic markers for disease progression and potentially develop effective interventions. The registry will also facilitate ongoing studies by providing access to samples and data.
Why it matters
Potential benefit: If successful, this study could lead to improved understanding and management of Adrenoleukodystrophy, potentially resulting in better treatment options for patients.
How similar studies have gone: Other studies focusing on registries for genetic conditions have shown success in understanding disease progression and developing biomarkers.
Who can join: the full rules
Show full inclusion / exclusion 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.)
Where it's running
-
Masonic Cancer Center at University of Minnesota
Minneapolis, Minnesota, United States Recruiting
Study contacts
- Study coordinator: Ashish Gupta, MD
- Email: gupta461@umn.edu
- Phone: 612-626-2961
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.