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

NCT01367834 | Completed Results

• 1 other identifier: 09-2171
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 1

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.

Conditions

Turner Syndrome

Keywords

Turner Syndrome; growth hormone; brain development

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (8)

• Volume of Brain Lobes (Occipital)

  Change in volume from 12 months of age scan in 24 months of age scan

• White Matter Tracts (SLF)

  Change in FA from 12 months of age scan in 24 months of age scan

• Volume of Brain Lobes (Central)

  Change in volume from 12 months of age scan in 24 months of age scan

• Volume of Brain Lobes (Frontal)

  Change in volume from 12 months of age scan in 24 months of age scan

• Volume of Brain Lobes (Temporal)

  Change in volume from 12 months of age scan in 24 months of age scan

Study Arms (2)

Growth Hormone

EXPERIMENTAL

Subjects in the somatotropin (growth hormone, GH) arm will receive GH injections from 12-24 months of life.

Drug: somatotropin

Control

NO INTERVENTION

Subjects will receive no GH or placebo.

Interventions

somatotropin DRUG

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.

Also known as: Genotropin, Growth hormone

Growth Hormone

Eligibility Criteria

• Age: 11 Months - 13 Months
• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Rebecca Knickmeyer Santelli, PhD: lead
• Pfizer: collaborator

Study Sites (1)

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

MeSH Terms

Conditions

Turner Syndrome

Interventions

Growth Hormone; Human Growth Hormone

Condition Hierarchy (Ancestors)

Gonadal Dysgenesis; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Sex Chromosome Disorders of Sex Development; Male Urogenital Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Sex Chromosome Disorders; Chromosome Disorders; Genetic Diseases, Inborn; Gonadal Disorders; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Limitations and Caveats

Low scan success rate and subject drop out resulted in small sample size (5 GH treated, 1 control).

Results Point of Contact

• Title: Rebecca Knickmeyer Santelli
• Organization: UNC Chapel Hill

rebecca_knickmeyer@med.unc.edu

9199662216

Study Officials

• Rebecca Knickmeyer

  University of North Carolina, Chapel Hill

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: June 2, 2011
• First Posted: June 7, 2011
• Study Start: May 1, 2010
• Primary Completion: May 1, 2014
• Study Completion: May 1, 2014
• Last Updated: March 24, 2017
• Results First Posted: March 24, 2017

Record last verified: 2017-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)