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Minnesota Adrenoleukodystrophy Registry Study (MARS) and Biobank

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ClinicalTrials.gov Identifier: NCT03789721
Recruitment Status : Recruiting
First Posted : December 31, 2018
Last Update Posted : June 6, 2019
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

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.

Condition or disease Intervention/treatment
ALD (Adrenoleukodystrophy) Other: Medical Record Abstraction Other: Biospecimen Sample Collection

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 1000 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 99 Years
Official Title: Minnesota Adrenoleukodystrophy Registry Study (MARS) and Biobank
Actual Study Start Date : May 1, 2019
Estimated Primary Completion Date : February 2030
Estimated Study Completion Date : February 2030


Group/Cohort Intervention/treatment
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.
Other: Medical Record Abstraction
Collect clinical and epidemiological data through medical record abstraction and self-reported questionnaire survey semi-annually.

Other: Biospecimen Sample Collection
Collect research samples, when feasible for those diagnosed with ALD.




Primary Outcome Measures :
  1. Collect Clinical and Epidemiological Data [ Time Frame: 10 Years ]
    Collect clinical and epidemiological data through medical record abstraction and self-reported questionnaire survey semi-annually.



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
Patients diagnosed with ALD and their families.
Criteria

Inclusion Criteria:

  • Any patient diagnosed with ALD (confirmed by genetic mutation) with no cognitive impairment will be eligible to participate in this study. Patients of all ages will be included in this registry study.
  • Proband Inclusion criteria: A diagnosis of ALD on new born screen, confirmed by pathogenic mutation in ABCD1-gene.
  • Family member inclusion criteria: All male family members of proband identified based on family pedigree, with elevated VLCFAs and confirmed by pathogenic mutation in ABCD1 gene.
  • Female carriers with pathogenic mutation in ABCD1 gene will also be included in this registry study.

Exclusion Criteria:

  • Patients diagnosed with ALD with cognitive impairment will be excluded from this registry.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03789721


Contacts
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Contact: Paul Orchard, MD 612-626-2961 orcha001@umn.edu

Locations
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United States, Minnesota
Masonic Cancer Center at University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Lauren Bigalke    612-626-2977    bigal016@umn.edu   
Contact: Ashish Gupta, MBBS, MPH    612-626-2961    gupta461@umn.edu   
Sub-Investigator: Ashish Gupta, MBBS, MPH         
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota

Additional Information:

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Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT03789721     History of Changes
Other Study ID Numbers: 2019NTLS027
First Posted: December 31, 2018    Key Record Dates
Last Update Posted: June 6, 2019
Last Verified: June 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Adrenoleukodystrophy
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hereditary Central Nervous System Demyelinating Diseases
Leukoencephalopathies
Demyelinating Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Peroxisomal Disorders
Metabolic Diseases
Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases