Early Diagnosis Of Childhood Cerebral ALD
| Status: | Withdrawn |
|---|---|
| Conditions: | Endocrine |
| Therapuetic Areas: | Endocrinology |
| Healthy: | No |
| Age Range: | 1 - 5 |
| Updated: | 7/8/2018 |
| Start Date: | November 2018 |
| End Date: | July 2023 |
Early Diagnosis Of Childhood Cerebral Adrenoleukodystrophy
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.
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.
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.
We found this trial at
1
site
425 E River Pkwy # 754
Minneapolis, Minnesota 55455
Minneapolis, Minnesota 55455
612-624-2620
Principal Investigator: Paul Orchard, MD
Phone: 612-626-2313
Masonic Cancer Center at University of Minnesota The Masonic Cancer Center was founded in 1991....
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