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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Eli Lilly and Company
ClinicalTrials.gov Identifier:
NCT00266656
First received: December 15, 2005
Last updated: July 13, 2016
Last verified: July 2016
  Purpose
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.

Condition Intervention Phase
Turner Syndrome
Drug: Humatrope
Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Effect of Early Growth Hormone Treatment on Long-term Growth and Skeletal Maturation in Girls With Turner Syndrome

Resource links provided by NLM:


Further study details as provided by Eli Lilly and Company:

Primary Outcome Measures:
  • Most Mature Height Standard Deviation Score (SDS) [ Time Frame: Baseline through End of Study (10 years) ] [ Designated as safety issue: No ]
    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 Outcome Measures:
  • Height SDS at Various Ages [ Time Frame: Age 10, Age 13, Age 16 ] [ Designated as safety issue: No ]
    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) ] [ Designated as safety issue: No ]
    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) ] [ Designated as safety issue: No ]
    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) ] [ Designated as safety issue: No ]

    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) ] [ Designated as safety issue: Yes ]
    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) ] [ Designated as safety issue: Yes ]
    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) ] [ Designated as safety issue: Yes ]
    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) ] [ Designated as safety issue: Yes ]
    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.


Enrollment: 69
Study Start Date: December 2005
Study Completion Date: September 2015
Primary Completion Date: September 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

Drug: Humatrope
According to investigator's clinical practice and guided by the approved package insert
Other Names:
  • LY137998
  • Somatropin
  • Growth hormone
Experimental: Experimental 2 Humatrope
Humatrope according to investigator's clinical practice and guided by the approved package insert on whether treatment is given.
Drug: Humatrope
According to investigator's clinical practice and guided by the approved package insert
Other Names:
  • LY137998
  • Somatropin
  • Growth hormone

  Eligibility

Ages Eligible for Study:   4 Years to 20 Years   (Child, Adult)
Genders Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Criteria

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
  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: NCT00266656

Locations
United States, California
Childrens Hospital of Los Angeles
Los Angeles, California, United States, 90027
United States, Colorado
Children's Hospital
Aurora, Colorado, United States, 80045
United States, Connecticut
Connecticut Children's Medical Center
Hartford, Connecticut, United States, 06106
United States, Illinois
Children's Hospital of Chicago Research Center
Chicago, Illinois, United States, 60611
United States, Indiana
Riley Hosptial for Children
Indianapolis, Indiana, United States, 46202
United States, Missouri
Children's Mercy Hospital
Kansas City, Missouri, United States, 64108
United States, North Carolina
University of NC at Chapel Hill School of Medicine
Chapel Hill, North Carolina, United States, 27514
United States, Pennsylvania
Thomas Jefferson University
Philadelphia, Pennsylvania, United States, 19107
United States, Washington
Childrens Hospital and Medical Center
Seattle, Washington, United States, 98105
Sponsors and Collaborators
Eli Lilly and Company
Investigators
Study Director: 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) Eli Lilly and Company
  More Information

Additional Information:
Responsible Party: Eli Lilly and Company
ClinicalTrials.gov Identifier: NCT00266656     History of Changes
Other Study ID Numbers: 10088  B9R-US-GDGH 
Study First Received: December 15, 2005
Results First Received: July 13, 2016
Last Updated: July 13, 2016
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Turner Syndrome
Gonadal Dysgenesis
Primary Ovarian Insufficiency
Syndrome
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

ClinicalTrials.gov processed this record on August 24, 2016