Clinical Trial: Early Diagnosis Of Childhood Cerebral ALD

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Observational

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.

Detailed Summary:
Sponsor: Masonic Cancer Center, University of Minnesota

Current Primary Outcome: ALD Early Evaluation [ Time Frame: 5 years ]

To evaluate boys with adrenoleukodystrophy (ALD) diagnosed early in life, and to prospectively determine factors that have a high correlation with the emergence of cerebral ALD.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Emergence of cerebral disease through imaging [ Time Frame: 5 years ]
    The ability to discern the emergence of cerebral disease through imaging prior to the onset of classic T2 imaging. This will include diffusion tensor imaging (DTI), T1 and T2 rho, RAFF and spectroscopy, in addition to the standard MRI evaluations including gadolinium enhancement.
  • Biomarker Study: Inflammation Markers [ Time Frame: 5 years ]
    Markers of inflammation (chitotriosidase, lipidomics including arachidonic acid metabolites and inflammatory cytokines).
  • Biomarker Study: Oxidative Stress [ Time Frame: 5 years ]
    Determinations of oxidative stress (including but not limited to total and reduced/oxidized glutathione and 4-hydroxynonenal, or 4-HNE).
  • Biomarker Study: Immunologic Activation [ Time Frame: 5 years ]
  • Neuropsychology testing [ Time Frame: 5 years ]
    Extensive neuropsychology testing, including assessments of verbal comprehension, perceptual reasoning, working memory and processing speed.


Original Secondary Outcome: Same as current

Information By: Masonic Cancer Center, University of Minnesota

Dates:
Date Received: October 25, 2016
Date Started: July 2017
Date Completion: July 2022
Last Updated: November 8, 2016
Last Verified: November 2016