Somatropin + Leuprorelin vs Somatropin Alone in Pubertal Children With Idiopathic Short Stature

Phase 3

Completed

Conditions: Idiopathic Short Stature (ISS)

Interventions: Drug: somatropin
Drug: leuprorelin

Registration Number: NCT00355030

Lead Sponsor: Eli Lilly and Company

Brief Summary: The present randomized trial was initially intended to study the benefits of a combined treatment with growth hormone (GH) and a gonadotropin-releasing hormone (GnRH) agonist for pubertal children with idiopathic short stature. However, treatments were stopped in January 2012 at the request of the French drug agency. Therefore, a protocol amendment divided the study in two study periods.

Study Period 1 involved combined treatment with somatropin and leuprorelin or treatment with somatropin alone. Participants from France who participated in this Period 1 of the study were asked to participate in a long term safety follow up defined as a Period 2 of the study. Participants from the Netherlands were offered participation in Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS, clinicaltrials.gov Identifier: NCT01088412) for long term safety follow up independent of this study.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 91

Inclusion Criteria

male or female children with ISS
age greater than or equal to 8 years and less than or equal to 12 years and 3 months for girls and greater than or equal to 9 years and less than or equal to 14 years and 3 months for boys
bone age less than or equal to 12.0 years for girls and less than or equal to 14.0 years for boys based on a central reading of an X-ray of the left hand and wrist
Pubertal stage B2 and B3 for girls based on the Tanner method
Pubertal stage G2 and G3 for boys based on the Tanner method

Exclusion Criteria

Growth hormone deficiency (GHD)
Insulin-like growth factor-I levels greater than 3 SDS
Chromosomal abnormality diagnosed locally on a karyotype. For girls, the karyotype to eliminate a Turner syndrome, is mandatory
Small for gestational age (SGA)
Has reached menarche (had her first menstrual period)
Have any significant concomitant disease that is likely to interfere with growth or with the study, or is a known contraindication to Growth Hormone treatment

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
1	somatropin	-
1	leuprorelin	-
2	somatropin	-

Primary Outcome Measures

Name	Time	Method
Number of Participants With One or More Drug-related Adverse Events	Baseline through End of Study (up to 9 years)	A drug-related AE was an AE that occurred postdose or was present predose and became more severe postdose and was considered to be related to study treatment. A summary of other nonserious AEs, and all SAE's, regardless of causality, is located in the Reported Adverse Events section.
Adult Height Standard Deviation Score (SDS)	Baseline through End of Study (up to 9 years)	The height of the participants were measured barefoot using a standard wall-mounted Harpenden stadiometer. SDS report the number of standard deviations from the mean for age and sex for an individual measurement (normal range: -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

Name	Time	Method
Difference Between Adult Height SDS and Target Height SDS	Baseline through End of Study (up to 9 years)	This is the difference between the gender, age and country matched standard deviation score of adult height and standard deviation score of target height \[calculated as (mother's height (SDS) + father's height (SDS))/2\] for particular participant. The height of the participants were measured barefoot using a standard wall-mounted Harpenden stadiometer. SDS report the number of standard deviations from the mean for age and sex for an individual measurement (normal range: -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.
Height SDS	Baseline through End of Study (up to 9 years)	SDS report the number of standard deviations from the mean for age and sex for an individual measurement (normal range: -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.
Difference Between Adult Height SDS and Baseline Height SDS	Baseline through End up Study (up to 9 years)	This is the difference between the gender, age and country matched standard deviation score of adult height and standard deviation score of baseline height for particular participant. The height of the participants were measured barefoot using a standard wall-mounted Harpenden stadiometer. SDS report the number of standard deviations from the mean for age and sex for an individual measurement (normal range: -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.
Percentage of Children With Normal Adult Height SDS	Baseline through End of Study (up to 9 years)	Percentage of children with normal adult height SDS (greater than -2 SDS and less than +2 SDS)
Height Velocity	Baseline through End of Study (up to 9 years)	Height velocity is the difference between 2 height measurements, divided by years elapsed between measurements.
Bone Age	Baseline through End of Study (up to 9 years)	Bone age measured using the X-Ray of left hand and wrist.
Difference Between Adult Height SDS and Baseline Predicted Height SDS	Baseline through End up Study (up to 9 years)	This is the difference between the gender, age and country matched standard deviation score of adult height and standard deviation score of baseline predicted height \[calculated using the Bayley-Pinneau method based on height and bone age\] for particular participant. The height of the participants were measured barefoot using a standard wall-mounted Harpenden stadiometer. SDS report the number of standard deviations from the mean for age and sex for an individual measurement (normal range: -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.

Trial Locations

Locations (1): For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.
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Rotterdam, Netherlands