Long-Term Growth and Skeletal Effects of Early Growth Hormone Treatment in Turner Syndrome

Overview

This is an extension study that will gather long-term data on the effect of early growth hormone (GH) treatment on adult height and other aspects of health and development in girls with Turner syndrome. The main purpose is to determine whether girls who received 2 years of GH treatment before 6 years of age achieve taller adult height than girls who were untreated during this time. The study will also look at middle ear and hearing function, and cognitive and behavioral development. Protocol completion is defined as attainment of height velocity less than or equal to 1.0 cm/year, or bone age greater than or equal to 15 years.

Full Title of Study: “Effect of Early Growth Hormone Treatment on Long-term Growth and Skeletal Maturation in Girls With Turner Syndrome”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Non-Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: September 2015

Interventions

  • Drug: Humatrope
    • According to investigator’s clinical practice and guided by the approved package insert

Arms, Groups and Cohorts

  • Experimental: Experimental 1 Control
    • No drug administration in B9R-US-GDFG (NCT00406926). Humatrope according to investigator’s clinical practice and guided by the approved package insert on whether treatment is given.
  • Experimental: Experimental 2 Humatrope
    • Humatrope according to investigator’s clinical practice and guided by the approved package insert on whether treatment is given.

Clinical Trial Outcome Measures

Primary Measures

  • Most Mature Height Standard Deviation Score (SDS)
    • Time Frame: Baseline through End of Study (10 years)
    • SDS reports the number of standard deviations from the mean for age and sex for an individual measurement (normal range is -2 to +2 SDS). Height SDS is derived by subtracting the population mean from individual’s height value and then dividing that difference by the population standard deviation. Greater height SDS values indicate greater height.

Secondary Measures

  • Height SDS at Various Ages
    • Time Frame: Age 10, Age 13, Age 16
    • SDS reports the number of standard deviations from the mean for age and sex for an individual measurement (normal range is -2 to +2 SDS). Height SDS is derived by subtracting the population mean from individual’s height value and then dividing that difference by the population standard deviation. Greater height SDS values indicate greater height.
  • Age at Attainment of Tanner 2 Breast Development
    • Time Frame: Baseline through End of Study (10 years)
    • The Tanner 2 breast development is the age at first evidence of breast development.
  • Chronological Age at First Visit Participant Attained Bone Age of 14.5 Years
    • Time Frame: Baseline through End of Study (10 years)
    • Bone age was measured by standard radiograph, x-ray at baseline and annually for 10 years or until attainment of height velocity less than or equal to 1.0 centimeter per year (cm/year) and bone age greater or equal to 15 years.
  • Reports of Serious Adverse Events
    • Time Frame: Baseline through End of Study (10 years)
    • Number of serious adverse events (SAEs) reported. Any adverse event from this study that results in one of the following outcomes, or is significant for any other reason were reported as an SAE: death, initial or prolonged inpatient hospitalization, a life-threatening experience (that is, immediate risk of dying), persistent or significant disability/incapacity, congenital anomaly/birth defect in the offspring of a study subject, significant for any other reason (includes cancer, other than superficial, and basal cell or squamous cell carcinomas of the skin, that did not meet other serious adverse event criteria). A summary of other nonserious AEs, and all SAE’s, regardless of causality, is located in the Reported Adverse Events section.
  • Percentage of Participants With Occurrence of Pre-specified Clinically Relevant Events
    • Time Frame: Baseline through End of Study (10 years)
    • Percentage of participants for whom certain non-serious, pre-specified adverse events (AEs; those that are commonly observed in Turner syndrome or are known to be related to GH treatment: impaired glucose tolerance, diabetes mellitus, hypothyroidism, benign intracranial hypertension, scoliosis, slipped capital femoral epiphysis, solid tumor/leukemia, pancreatitis, ear infections, and high blood pressure) are reported.
  • Percentage of Participants With Abnormal Tympanometry Results
    • Time Frame: Baseline, Age 10, Age 16, End of Study (10 years)
    • Percentage of participants with abnormal tympanometry [defined as middle ear dysfunction / middle ear effusion / patent pressure equalizer tube or possible tympanic membrane perforation] results at baseline, age 10 years, and age 16 years or endpoint.
  • Percentage of Participants With Prevalence of Abnormal Audiometry Results
    • Time Frame: Baseline, Age 10, Age 16, End of Study (10 years)
    • Percentage of participants with abnormal Audiometry results at baseline, age 10 years, and age 16 years or endpoint. Prevalence was calculated as number of participants with abnormal hearing divided by number of participants with measurable pure tone audiometry results at that visit.
  • Percentage of Participants With Abnormal Audiometry Results Based on Pure Tone Average (PTA)
    • Time Frame: Baseline, Age 10, Age 16, End of Study (10 years)
    • Percentage of participants with abnormal Audiometry results at baseline, age 10 years, and age 16 years or endpoint. PTA is defined as the average of pure tone hearing thresholds at 500, 1000 and 2000 Hz (Hertz), calculated separately for each ear and for each testing method (air or bone); normal PTA is defined as pure tone hearing threshold less than or equal to 20 dB HL (decibels Hearing Level), and abnormal PTA is defined as pure tone hearing threshold greater than 20 DB HL.

Participating in This Clinical Trial

Inclusion Criteria

  • Previously randomized in study B9R-US-GDFG (NCT00406926) – Karyotype-proven Turner syndrome Exclusion Criteria:

  • Immediate family members of study site personnel directly affiliated with the study

Gender Eligibility: Female

Minimum Age: 4 Years

Maximum Age: 20 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Eli Lilly and Company
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon-Fri 9 AM – 5 PM Eastern Time (UTC/GMT – 5 hrs), Study Director, Eli Lilly and Company

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