Actively Recruiting
Adrenoleukodystrophy National Registry Study
Led by Masonic Cancer Center, University of Minnesota · Updated on 2026-05-12
1000
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting a prospective, observational registry study on Adrenoleukodystrophy (ALD), including patients with confirmed or presumed ALD mutations. The study aims to understand the natural history, disease progression, and outcomes in affected individuals, including children, adolescents, adults, and women with ALD. The research also seeks to develop biomarkers from collected biospecimens to potentially predict disease progression and support future interventions. Participants provide medical histories and allow collection of various biospecimens such as buccal swabs, blood, stool, and urine samples, which are stored in a biorepository. The study collects clinical and epidemiological data through medical record reviews and self-reported questionnaires every six months. Eligible participants include those diagnosed by newborn screening, family history, or other means and those with confirmed or presumed mutations. During the study, participants may be asked to provide longitudinal samples and medical information over a period of up to 10 years. Researchers monitor clinical data to better understand disease progression and gather valuable biological data. The registry and biorepository serve as resources for ongoing and future ALD research, supporting efforts to identify prognostic markers and improve patient care.
CONDITIONS
Brief Title
Adrenoleukodystrophy National Registry Study
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 0 to 100 years
- Diagnosed with ALD confirmed by positive VLCFA testing or genetic mutation
- Known or presumed ALD mutation based on family history or confirmed mutation in ABCD1 gene
- Living in the United States or its territories
You will not qualify if you...
- Lack capacity to consent or assent without a legally authorized representative or guardian
- Have undergone bone marrow transplant or other cellular therapy
- Not fluent in English and unable to consent in person at the BMT Journey Clinic
- Illiterate
- Considered unlikely to complete study requirements due to language barriers or compliance issues
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 10 years
Participants are observed over time to collect clinical and epidemiological data related to Adrenoleukodystrophy (ALD).
Semi-annual visits for data collection and questionnaires
Duration - Ongoing during study participation
Participants provide biospecimen samples such as buccal swab, blood, stool, and urine when feasible for research purposes.
Sample collection occurs during visits as feasible
Trial Site Locations
Total: 1 location
1
Masonic Cancer Center at University of Minnesota
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
Research Team
A
Ashish Gupta, MD
P
Paul Orchard, MD
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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