An observational study in ALD (Adrenoleukodystrophy), Adrenoleukodystrophy and Cerebral Adrenoleukodystrophy, sponsored by Masonic Cancer Center, University of Minnesota. Recruiting at 1 site in United States. Per ClinicalTrials.gov, last updated 2026-05-12.
Sponsored by Masonic Cancer Center, University of Minnesota · Observational
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.
This is a prospective, non-therapeutic protocol designed to create and maintain a registry of participants with Adrenoleukodystrophy (ALD) and known/presumed mutation for ALD. This study also involves maintaining a prospective biorepository to collect and store buccal swab, blood, stool and urine samples as well. In this protocol, pediatric (including infants), adolescents and adult patients with confirmed or presumed ALD (based on positive VLCFA testing and/or confirmed mutation) will be offered potential study participation. Additionally, presumed mutation for ALD (based on pedigree or confirmed mutation) will be offered potential study participation. After appropriate consent (online or in-person), subjects will be requested to provide a medical history (with authorization of release of medical records), longitudinal biospecimens, and permission to perform laboratory analyses on these samples. The overall goal is to understand the natural disease course in affected and unaffected patients (identified patients and relatives with a diagnosis of ALD), as well as women with ALD to assemble a resource of clinical, medical, and biological data from the participants. This study also aims to understand the outcomes of this disease, as well as possibly develop biomarkers to identify prognostic markers for disease progression, which may help develop effective interventions. The biospecimen bank and registry will provide access to samples and data for the ongoing studies as well as will provide an important resource for the future research.
68 studies on the registry are indexed under Adrenoleukodystrophy; 19 are open to participants now.
This study's planned enrollment of 1,000 is above the median of 120 across 19 observational studies indexed under Adrenoleukodystrophy.
Browse Adrenoleukodystrophy studies →Masonic Cancer Center, University of Minnesota is the lead sponsor of 286 studies on the registry; 35 are open to participants now.
Of its 39 completed or terminated interventional studies of FDA-regulated products, 28 (72%) have results posted.
Counted across the registry records on this site, refreshed daily.
participants with Adrenoleukodystrophy (ALD) and known/presumed mutation for ALD.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
ALD patients or family member meeting any of the following criteria:
Exclusion Criteria
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.
Other: Medical Record Abstraction · Other: Biospecimen Sample Collection
Collect clinical and epidemiological data through medical record abstraction and self-reported questionnaire survey semi-annually.
Collect research samples, when feasible for those diagnosed with ALD.
Collect Clinical and Epidemiological Data
Collect clinical and epidemiological data through medical record abstraction and self-reported questionnaire survey semi-annually.
Time frame: 10 Years
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
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Masonic Cancer Center, University of Minnesota →