Recruiting

Observational Study

Sponsor:

Masonic Cancer Center, University of Minnesota

Code:

NCT03789721

Conditions

ALD (Adrenoleukodystrophy)

Adrenoleukodystrophy

Cerebral Adrenoleukodystrophy

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Interventions

Medical Record Abstraction

Biospecimen Sample Collection

Study Details

Brief 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.

Conditions

ALD (Adrenoleukodystrophy)

Adrenoleukodystrophy

Cerebral Adrenoleukodystrophy

Study ID

NCT03789721

Start date

May 1, 2019

Status verified date

May, 2026

Completion date

Feb, 2030

Anticipated

Primary completion date

Feb, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

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.)

Study Design

Enrollment

1000 participants

Anticipated

Interventions and Outcome Measures

Arms

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.

Interventions

Medical Record Abstraction

Collect clinical and epidemiological data through medical record abstraction and self-reported questionnaire survey semi-annually.

Biospecimen Sample Collection

Collect research samples, when feasible for those diagnosed with ALD.

Primary outcome measure

  • Collect Clinical and Epidemiological Data [ Time Frame: 10 Years ]

Central Contacts and Locations

Central contacts

Ashish Gupta, MD

612-626-2961gupta461@umn.edu

Paul Orchard, MD

612-626-2961orcha001@umn.edu

Locations

Masonic Cancer Center at University of Minnesota

Recruiting

Minneapolis, Minnesota, United States, 55455

Contacts

Phil Lacher

612-626-2977placher@umn.edu

More Information

Sponsor

Masonic Cancer Center, University of Minnesota

Last update posted

May 12, 2026

Last verified

May, 2026

Keywords

  • Registry, VLCFA, ABCD1, X-chromosome

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Masonic Cancer Center, University of Minnesota on 2026-05-12.