RecruitingNot Applicable

Adrenoleukodystrophy National Registry Study

United States1,000 participantsStarted 2019-05-01

Plain-language 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.

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.)

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Collect Clinical and Epidemiological Data

Timeframe: 10 Years

Trial details

NCT IDNCT03789721

SponsorMasonic Cancer Center, University of Minnesota

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2030-02

Contact for this trial

Ashish Gupta, MD

612-626-2961 gupta461@umn.edu

View on ClinicalTrials.gov More ALD (Adrenoleukodystrophy) trials

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.