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

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2013 by University of North Carolina, Chapel Hill
Sponsor:
Collaborator:
Pfizer
Information provided by (Responsible Party):
Marsha Davenport, MD, University of North Carolina, Chapel Hill
ClinicalTrials.gov Identifier:
NCT01367834
First received: June 2, 2011
Last updated: May 23, 2013
Last verified: May 2013
  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: 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 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 ] [ Designated as safety issue: No ]
    Percent change in total brain volume as determined by magnetic resonance imaging (MRI)


Secondary Outcome Measures:
  • Volume of brain lobes [ Time Frame: Change in volume from 12 months of age scan in 24 months of age scan ] [ Designated as safety issue: No ]
    Percent change in volumes of occipital lobes as determined by MRI.

  • White matter tracts [ Time Frame: Change in volume from 12 months of age scan in 24 months of age scan ] [ Designated as safety issue: No ]
    Change in the fractional anisotropy of white matter tracts using Diffusion Tensor Imaging (DTI)

  • Volume of brain lobes [ Time Frame: Change in volume from 12 months of age scan in 24 months of age scan ] [ Designated as safety issue: No ]
    Percent change in volumes of prefrontal lobes as determined by MRI.

  • Volume of brain lobes [ Time Frame: Change in volume from 12 months of age scan in 24 months of age scan ] [ Designated as safety issue: No ]
    Percent change in volumes of frontal lobes as determined by MRI.

  • Volume of brain lobes [ Time Frame: Change in volume from 12 months of age scan in 24 months of age scan ] [ Designated as safety issue: No ]
    Percent change in volumes of temporal lobes as determined by MRI.

  • Volume of brain lobes [ Time Frame: Change in volume from 12 months of age scan in 24 months of age scan ] [ Designated as safety issue: No ]
    Percent change in volumes of parietal lobes as determined by MRI.


Estimated Enrollment: 25
Study Start Date: May 2010
Estimated Study Completion Date: September 2013
Estimated Primary Completion Date: September 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Growth Hormone
Subjects in the 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

Detailed Description:

Studies in older children and adults have found that IQ 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

Ages Eligible for Study:   11 Months to 13 Months
Genders 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01367834

Contacts
Contact: Marsha L Davenport, MD (919)966-4435 ext 232 mld@med.unc.edu

Locations
United States, North Carolina
University of North Carolina at Chapel Hill Recruiting
Chapel Hill, North Carolina, United States, 27599
Contact: Marsha L Davenport, MD    919-966-4435 ext 232    mld@med.unc.edu   
Sponsors and Collaborators
Marsha Davenport, MD
Pfizer
Investigators
Principal Investigator: Marsha L Davenport, MD University of North Carolina, Chapel Hill
  More Information

Additional Information:
No publications provided

Responsible Party: Marsha Davenport, MD, MD, University of North Carolina, Chapel Hill
ClinicalTrials.gov Identifier: NCT01367834     History of Changes
Other Study ID Numbers: 09-2171
Study First Received: June 2, 2011
Last Updated: May 23, 2013
Health Authority: United States: Institutional Review Board

Keywords provided by 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
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 18, 2014