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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

Tracking Information
First Submitted Date December 14, 2018
First Posted Date December 31, 2018
Last Update Posted Date June 6, 2019
Actual Study Start Date May 1, 2019
Estimated Primary Completion Date February 2030   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: December 26, 2018)
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.
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT03789721 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Minnesota Adrenoleukodystrophy Registry Study (MARS) and Biobank
Official Title Minnesota Adrenoleukodystrophy Registry Study (MARS) and Biobank
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.
Detailed Description Not Provided
Study Type Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration 99 Years
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Patients diagnosed with ALD and their families.
Condition ALD (Adrenoleukodystrophy)
Intervention
  • 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.
Study Groups/Cohorts 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:
  • Other: Medical Record Abstraction
  • Other: Biospecimen Sample Collection
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: December 26, 2018)
1000
Original Estimated Enrollment Same as current
Estimated Study Completion Date February 2030
Estimated Primary Completion Date February 2030   (Final data collection date for primary outcome measure)
Eligibility 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.
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers Yes
Contacts
Contact: Paul Orchard, MD 612-626-2961 orcha001@umn.edu
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03789721
Other Study ID Numbers 2019NTLS027
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Responsible Party Masonic Cancer Center, University of Minnesota
Study Sponsor Masonic Cancer Center, University of Minnesota
Collaborators Not Provided
Investigators Not Provided
PRS Account Masonic Cancer Center, University of Minnesota
Verification Date June 2019