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Effect of Growth Hormone on Early Brain Development in Girls With Turner Syndrome

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01367834
First Posted: June 7, 2011
Last Update Posted: March 24, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Pfizer
Information provided by (Responsible Party):
Rebecca Knickmeyer Santelli, PhD, University of North Carolina, Chapel Hill
  Purpose
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.

Condition Intervention
Turner Syndrome Drug: somatotropin

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Official Title: Effect of Growth Hormone on Early Brain Development in Girls With Turner Syndrome

Resource links provided by NLM:


Further study details as provided by Rebecca Knickmeyer Santelli, PhD, University of North Carolina, Chapel Hill:

Primary Outcome Measures:
  • 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)


Secondary Outcome Measures:
  • 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.


Enrollment: 17
Study Start Date: May 2010
Study Completion Date: May 2014
Primary Completion Date: May 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Growth Hormone
Subjects in the somatotropin (growth hormone, GH) arm will receive GH injections from 12-24 months of life.
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
No Intervention: Control
Subjects will receive no GH or placebo.

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.

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   11 Months to 13 Months   (Child)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01367834


Locations
United States, North Carolina
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27599
Sponsors and Collaborators
Rebecca Knickmeyer Santelli, PhD
Pfizer
Investigators
Principal Investigator: Rebecca Knickmeyer University of North Carolina, Chapel Hill
  More Information

Responsible Party: Rebecca Knickmeyer Santelli, PhD, Associate Professor, University of North Carolina, Chapel Hill
ClinicalTrials.gov Identifier: NCT01367834     History of Changes
Other Study ID Numbers: 09-2171
First Submitted: June 2, 2011
First Posted: June 7, 2011
Results First Submitted: December 12, 2016
Results First Posted: March 24, 2017
Last Update Posted: March 24, 2017
Last Verified: February 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Rebecca Knickmeyer Santelli, PhD, University of North Carolina, Chapel Hill:
Turner Syndrome
growth hormone
brain development

Additional relevant MeSH terms:
Syndrome
Turner Syndrome
Gonadal Dysgenesis
Primary Ovarian Insufficiency
Disease
Pathologic Processes
Disorders of Sex Development
Urogenital Abnormalities
Sex Chromosome Disorders of Sex Development
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Congenital Abnormalities
Sex Chromosome Disorders
Chromosome Disorders
Genetic Diseases, Inborn
Gonadal Disorders
Endocrine System Diseases
Ovarian Diseases
Adnexal Diseases
Genital Diseases, Female
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs