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Trial record 1 of 1 for:    NCT01367834
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Effect of Growth Hormone on Early Brain Development in Girls With Turner Syndrome

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ClinicalTrials.gov Identifier: NCT01367834
Recruitment Status : Completed
First Posted : June 7, 2011
Results First Posted : March 24, 2017
Last Update Posted : March 24, 2017
Sponsor:
Collaborator:
Pfizer
Information provided by (Responsible Party):
Rebecca Knickmeyer Santelli, PhD, University of North Carolina, Chapel Hill

Tracking Information
First Submitted Date  ICMJE June 2, 2011
First Posted Date  ICMJE June 7, 2011
Results First Submitted Date  ICMJE December 12, 2016
Results First Posted Date  ICMJE March 24, 2017
Last Update Posted Date March 24, 2017
Study Start Date  ICMJE May 2010
Actual Primary Completion Date May 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 6, 2012)
Total Brain Volume [ Time Frame: Change in volume from 12 months of age scan in 24 months of age scan ]
Percent change in total brain volume as determined by magnetic resonance imaging (MRI)
Original Primary Outcome Measures  ICMJE
 (submitted: June 6, 2011)
Total Brain Volume [ Time Frame: 12 and 24 months of age ]
Percent change in total brain volume as determined by magnetic resonance imaging (MRI)
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 3, 2017)
  • Volume of Brain Lobes (Occipital) [ Time Frame: Change in volume from 12 months of age scan in 24 months of age scan ]
    Percent change in volumes of occipital lobes as determined by MRI.
  • White Matter Tracts (SLF) [ Time Frame: Change in FA from 12 months of age scan in 24 months of age scan ]
    Change in the fractional anisotropy (FA) of white matter tracts using Diffusion Tensor Imaging (DTI); superior longitudinal fasciculus
  • Volume of Brain Lobes (Central) [ Time Frame: Change in volume from 12 months of age scan in 24 months of age scan ]
    Percent change in volumes of central brain region (precentral gyrus, postcentral gyrus, rolandic operculum) as determined by MRI.
  • Volume of Brain Lobes (Frontal) [ Time Frame: Change in volume from 12 months of age scan in 24 months of age scan ]
    Percent change in volumes of frontal lobes as determined by MRI.
  • Volume of Brain Lobes (Temporal) [ Time Frame: Change in volume from 12 months of age scan in 24 months of age scan ]
    Percent change in volumes of temporal lobes as determined by MRI.
  • Volume of Brain Lobes (Parietal) [ Time Frame: Change in volume from 12 months of age scan in 24 months of age scan ]
    Percent change in volumes of parietal lobes as determined by MRI.
  • Volume of Brain Lobes (Limbic) [ Time Frame: Change in volume from 12 months of age scan in 24 months of age scan ]
    Percent change in volumes of parietal lobes as determined by MRI.
  • Volume of Brain Lobes (Insular Cortex) [ Time Frame: Change in volume from 12 months of age scan in 24 months of age scan ]
    Percent change in volumes of parietal lobes as determined by MRI.
Original Secondary Outcome Measures  ICMJE
 (submitted: June 6, 2011)
  • Volume of brain lobes [ Time Frame: 12 and 24 months of age ]
    Percent change in volumes of prefrontal, frontal, temporal, parietal, and occipital lobes as determined by MRI.
  • White matter tracts [ Time Frame: 12 months and 24 months ]
    Change in the fractional anisotropy of white matter tracts using Diffusion Tensor Imaging (DTI)
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Effect of Growth Hormone on Early Brain Development in Girls With Turner Syndrome
Official Title  ICMJE Effect of Growth Hormone on Early Brain Development in Girls With Turner Syndrome
Brief Summary The purpose of this research study is to learn about brain development in very young girls with Turner syndrome (TS) and the effect that growth hormone (GH) therapy has on early brain development.
Detailed Description

Studies in older children and adults have found that IQ (intelligence quotient) in individuals with Turner syndrome (TS) and the general population are similar. However, many individuals with TS have a nonverbal learning disability which may cause problems with imagining and working with objects in space (for example: building blocks, working puzzles, copying designs, driving a car) as well as problems in planning, paying attention, and getting along with people their own age. Studies of brain structure have found changes in the size and function of specific areas of the brain that are involved in these nonverbal processes. However, there are no data published on brain structure or function in girls with Turner Syndrome in the first few years of life when brains are learning and growing most rapidly.

In a recent study, very young, short girls with Turner Syndrome (TS)(averaging 2 years in age) were treated with Growth Hormone (GH) and almost reached an average height after 2 years. The risks of GH for the young girls in that study appeared to be the same as those for older girls. Therefore, it is now recommended that GH therapy be considered as soon as a child with TS has growth failure. Growth failure often occurs during infancy; therefore, more children with TS are now receiving GH therapy as young as 12 months of age. In this study, the investigators wish to start understanding what effect GH has on learning and brain growth when given between the ages of 12 and 24 months. In this study, the girls with TS will have developmental studies, a physical examination, magnetic resonance imaging (MRI), and blood drawn at one and two years of age. Some of the girls in this study will receive GH from 12 until 24 months of life, while others will not. Brain growth and development will be compared between those who have been treated with GH and those who have not. This is a pilot study in which the investigators will gather data to design a larger study that can answer these questions.

Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Condition  ICMJE Turner Syndrome
Intervention  ICMJE Drug: somatotropin
Subjects will receive 5 mg somatotropin (growth hormone) pens with cartridges. Subcutaneous injections are to be given every evening around bedtime. Dosing regimen: 50 mcg/kg/day to be adjusted at 4 month intervals to the closest 0.1 mg. Subjects will be given 12 months of treatment (from 12 to 24 months of life). Subjects will visit their pediatrician or pediatric endocrinologist at 4 and 8 months of life.
Other Names:
  • Genotropin
  • Growth hormone
Study Arms  ICMJE
  • Experimental: Growth Hormone
    Subjects in the somatotropin (growth hormone, GH) arm will receive GH injections from 12-24 months of life.
    Intervention: Drug: somatotropin
  • No Intervention: Control
    Subjects will receive no GH or placebo.
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  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 3, 2017)
17
Original Estimated Enrollment  ICMJE
 (submitted: June 6, 2011)
25
Actual Study Completion Date  ICMJE May 2014
Actual Primary Completion Date May 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Diagnosis of Turner syndrome
  • Less than the 50th percentile for length for the general female population

Exclusion Criteria:

  • Prior Growth Hormone (GH) therapy
  • Diabetes
  • Allergy to metacresol (a preservative in the GH liquid that is injected)
  • Contraindications for Magnetic Resonance Imaging (MRI) (such as metal in the body)
  • Part of a Y chromosome in child's karyotype
  • Parent/guardian is not willing for child to be randomized to be in the treatment group (receives Growth Hormone injections for one year) or the control group (receives no Growth Hormone during the study)
  • Parent/guardian is not willing for child to have some of her developmental testing digitally recorded for scoring
Sex/Gender  ICMJE
Sexes Eligible for Study: Female
Ages  ICMJE 11 Months to 13 Months   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01367834
Other Study ID Numbers  ICMJE 09-2171
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Rebecca Knickmeyer Santelli, PhD, University of North Carolina, Chapel Hill
Study Sponsor  ICMJE Rebecca Knickmeyer Santelli, PhD
Collaborators  ICMJE Pfizer
Investigators  ICMJE
Principal Investigator: Rebecca Knickmeyer University of North Carolina, Chapel Hill
PRS Account University of North Carolina, Chapel Hill
Verification Date February 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP