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

Turner Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01367834
• Other study ID #: 09-2171
• Status: Recruiting
• Phase: N/A
• First received: June 2, 2011
• Last updated: May 23, 2013
• Start date: May 2010
• Est. completion date: September 2013

Study information

• Verified date: May 2013
• Source: University of North Carolina, Chapel Hill
• Contact: Marsha L Davenport, MD
• Phone: (919)966-4435
• Email: mld[@]med.unc.edu
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 25
• Est. completion date: September 2013
• Est. primary completion date: September 2013
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 11 Months to 13 Months

Eligibility

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

Study Design

Allocation: Randomized, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Basic Science

Related Conditions & MeSH terms

• Gonadal Dysgenesis
• Primary Ovarian Insufficiency
• Syndrome
• Turner Syndrome

Intervention

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.

Locations

Country	Name	City	State
United States	University of North Carolina at Chapel Hill	Chapel Hill	North Carolina

Sponsors (2)

Lead Sponsor	Collaborator
Marsha Davenport, MD	Pfizer

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Total brain volume	Percent change in total brain volume as determined by magnetic resonance imaging (MRI)	Change in volume from 12 months of age scan in 24 months of age scan	No
Secondary	Volume of brain lobes	Percent change in volumes of occipital lobes as determined by MRI.	Change in volume from 12 months of age scan in 24 months of age scan	No
Secondary	White matter tracts	Change in the fractional anisotropy of white matter tracts using Diffusion Tensor Imaging (DTI)	Change in volume from 12 months of age scan in 24 months of age scan	No
Secondary	Volume of brain lobes	Percent change in volumes of prefrontal lobes as determined by MRI.	Change in volume from 12 months of age scan in 24 months of age scan	No
Secondary	Volume of brain lobes	Percent change in volumes of frontal lobes as determined by MRI.	Change in volume from 12 months of age scan in 24 months of age scan	No
Secondary	Volume of brain lobes	Percent change in volumes of temporal lobes as determined by MRI.	Change in volume from 12 months of age scan in 24 months of age scan	No
Secondary	Volume of brain lobes	Percent change in volumes of parietal lobes as determined by MRI.	Change in volume from 12 months of age scan in 24 months of age scan	No

External Links

•   Website for the Turner Syndrome Study Group