Early Diagnosis Of Childhood Cerebral ALD

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Brief Title

Early Diagnosis Of Childhood Cerebral ALD

Official Title

Early Diagnosis Of Childhood Cerebral Adrenoleukodystrophy

Brief Summary

      The goal of this single institution study is to evaluate boys with adrenoleukodystrophy (ALD)
      diagnosed early in life, and to prospectively monitor them to determine parameters that will
      facilitate earlier detection of the childhood cerebral form of the disease. These at-risk
      subjects will be assessed yearly through travel to the University of Minnesota, where plasma
      and cerebral spinal fluid (CSF) biomarker studies, MRI based imaging and neuropsychological
      assessments will be performed at the University of Minnesota Masonic Children's Hospital and
      Clinics. The MRI and lumbar puncture to obtain CSF will be obtained under sedation. In
      addition, at intervening 6 months intervals information will be obtained remotely, including
      surveys and MRI's in their home location. Also at that time blood samples will be obtained
      locally and shipped to the University of Minnesota for study. There is no therapeutic intent
      in this study.

Study Type


Primary Outcome

ALD Early Evaluation

Secondary Outcome

 Emergence of cerebral disease through imaging




* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Estimated Enrollment


Start Date

November 2018

Completion Date

July 2023

Primary Completion Date

July 2023

Eligibility Criteria

        Inclusion Criteria:

          -  Boys with confirmed adrenoleukodystrophy, as determined by very long chain fatty acid
             (VLCFA) analysis and/or genotyping. Genotyping is not necessary for diagnosis.

          -  Between 1 and 5 years of age, inclusive at the time of consent.

          -  Able to undergo a sedation

          -  English as primary language of the household, to maximize consistency of the
             neuropsychological/developmental testing.

          -  Voluntary written parental/guardian consent

        Exclusion Criteria:

          -  Evidence of cerebral disease at time of enrollment - patients over 3 years of age must
             have an MRI within 4 months of signing consent to confirm that there is no evidence of
             cerebral disease

          -  Inability or unwillingness to travel to the University of Minnesota once a year for
             the duration of the study

          -  Evidence of cerebral disease by standard T2/FLAIR MRI. If a subject develops cerebral
             ALD during the study, they will come off study, as it is anticipated that they would
             be considered for transplantation.




1 Year - 5 Years

Accepts Healthy Volunteers



Paul Orchard, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Secondary IDs


Responsible Party


Study Sponsor

Masonic Cancer Center, University of Minnesota

Study Sponsor

Paul Orchard, MD, Principal Investigator, University of Minnesota

Verification Date

July 2018