Child to Adult Neurodevelopment in Gene Expanded Huntington's Disease (ChANGE HD)
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| ClinicalTrials.gov Identifier: NCT01860339 |
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Recruitment Status :
Recruiting
First Posted : May 22, 2013
Last Update Posted : February 18, 2021
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| Tracking Information | |||||||||
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| First Submitted Date | May 16, 2013 | ||||||||
| First Posted Date | May 22, 2013 | ||||||||
| Last Update Posted Date | February 18, 2021 | ||||||||
| Actual Study Start Date | July 2005 | ||||||||
| Estimated Primary Completion Date | August 31, 2024 (Final data collection date for primary outcome measure) | ||||||||
| Current Primary Outcome Measures |
Volume of brain structures as measured by Magnetic Resonance Imaging (MRI) [ Time Frame: 1 hour out of 6-7 hour testing day, 3-4 annual visits ] Magnetic Resonance Imaging (MRI) and Diffusion Tensor Imaging (DTI) data will be analyzed to assess brain structure based upon variables including global volume, total cerebral spinal fluid, subregion volumes, cortical surface anatomy including cortical depth, surface area and gyral shape, and symmetry between brain hemispheres, all in consideration of age, gender, and height. Results will be evaluated for comparative differences between the GE group and the GNE group. In addition, these measures of brain structure will be paired with corresponding measures of brain function to evaluate brain development based upon growth and performance.
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| Original Primary Outcome Measures |
Volume of brain structures as measured by Magnetic Resonance Imaging (MRI) [ Time Frame: Participant age of 6-18 years ] Assessments of brain structure and function during developmental years will be evaluated for comparative differences between the GE group, the GNE group, and the healthy control group.
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| Change History | |||||||||
| Current Secondary Outcome Measures |
Quantitative assessment of cognitive skills and behavioral factors [ Time Frame: 4 hours out of 6-7 hour testing day, 3-4 annual visits ] Participants undergo a cognitive battery which will quantify skills such as attention, learning, memory. In addition, behavioral measures will be administered in both self and proxy report formats. Results will be analyzed for comparative differences between the GE group and the GNE group. In addition, these measures of brain function will be paired with appropriate measures of brain structure to evaluate brain development based upon growth and performance.
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| Original Secondary Outcome Measures |
Quantitative assessment of cognitive skills and motor skills [ Time Frame: Participant age 6-18 years ] Subject undergo a cognitive battery which will quantify skills such as attention, learning, memory. In addition, motor skill (both fine and gross) will be assessed and quantified.
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| Current Other Pre-specified Outcome Measures |
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| Original Other Pre-specified Outcome Measures | Not Provided | ||||||||
| Descriptive Information | |||||||||
| Brief Title | Child to Adult Neurodevelopment in Gene Expanded Huntington's Disease | ||||||||
| Official Title | Growth and Development of Striatal-Cerebellum Circuitry in Subjects at Risk for Huntington's Disease | ||||||||
| Brief Summary | Huntington's Disease (HD) is an autosomal dominant disease manifested in a triad of cognitive, psychiatric, and motor signs and symptoms. HD is caused by a triplet repeat (CAG)expansion in the gene Huntingtin (HTT). This disease has classically been conceptualized as a neurodegenerative disease. However, recent evidence suggests that abnormal brain development may play an important role in the etiology of HD. Huntingtin (HTT)is expressed during development and through life. In animal studies, the HTT gene has been shown to be vital for brain development. This suggests that a mutant form of HTT (gene-expanded or CAG repeats of 40 and above) would affect normal brain development. In addition, studies in adults who are gene-expanded for HD, but have not yet manifested the illness, (pre-HD subjects) have significant changes in the structure of their brain, even up to 20 years before onset of clinical diagnosis. How far back these changes are evident is unknown. One possibility is that these brain changes are present throughout life, due to changes in brain development,though initially associated with only subtle functional abnormalities. In an effort to better understand the developmental aspects of this brain disease, the current study proposes to evaluate brain structure and function in children, adolescents, and young adults (ages 6-30) who are at risk for developing HD - those who have a parent or grandparent with HD. Brain structure will be evaluating using Magnetic Resonance Imaging (MRI) with quantitative measures of the entire brain, cerebral cortex, as well as white matter integrity via Diffusion Tensor Imaging. Brain function will be assessed by cognitive tests, behavioral assessment, and physical and neurologic evaluation. Subjects that are gene-expanded (GE) will be compared to subjects who are gene non-expanded (GNE). Changes in brain structure and/or function in the GE group compared to the GNE group would lend support to the notion that this disease has an important developmental component. |
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| Detailed Description | Not Provided | ||||||||
| Study Type | Observational | ||||||||
| Study Design | Observational Model: Case-Control Time Perspective: Cross-Sectional |
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| Target Follow-Up Duration | Not Provided | ||||||||
| Biospecimen | Retention: Samples With DNA Description: Bio-specimens are collected and retained for all participants for genetic testing purposes and future research. Specimens are either a single sample of 3-4 teaspoons of blood drawn from the arm. Biospecimens will be tested for the number of CAG repeats in the Huntingtin Gene. Each sample obtained will be coded with a randomly assigned number and never linked with personal identifiers. The results from the genetic analysis will be sent directly to the data manager on the project, who is the ONLY research member with access to this data. This person has no direct contact with study participants. |
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| Sampling Method | Non-Probability Sample | ||||||||
| Study Population | Young people ages 6-30 years old who have a parent or grandparent that has been diagnosed with Huntington's Disease | ||||||||
| Condition | Huntington's Disease | ||||||||
| Intervention | Not Provided | ||||||||
| Study Groups/Cohorts |
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| Publications * | Not Provided | ||||||||
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||||||
| Recruitment Status | Recruiting | ||||||||
| Estimated Enrollment |
400 | ||||||||
| Original Estimated Enrollment | Same as current | ||||||||
| Estimated Study Completion Date | August 31, 2024 | ||||||||
| Estimated Primary Completion Date | August 31, 2024 (Final data collection date for primary outcome measure) | ||||||||
| Eligibility Criteria | Inclusion Criteria:
Exclusion Criteria:
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| Sex/Gender |
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| Ages | 6 Years to 30 Years (Child, Adult) | ||||||||
| Accepts Healthy Volunteers | Yes | ||||||||
| Contacts |
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| Listed Location Countries | United States | ||||||||
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| Administrative Information | |||||||||
| NCT Number | NCT01860339 | ||||||||
| Other Study ID Numbers | 200507759 2U01NS055903-10 ( U.S. NIH Grant/Contract ) |
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| Has Data Monitoring Committee | No | ||||||||
| U.S. FDA-regulated Product | Not Provided | ||||||||
| IPD Sharing Statement |
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| Responsible Party | Peggy C Nopoulos, University of Iowa | ||||||||
| Study Sponsor | Peggy C Nopoulos | ||||||||
| Collaborators | National Institute of Neurological Disorders and Stroke (NINDS) | ||||||||
| Investigators |
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| PRS Account | University of Iowa | ||||||||
| Verification Date | February 2021 | ||||||||