Efficacy and Safety of Somatropin in Combination With Leuprorelin Compared to Somatropin Alone in Pubertal Children With Idiopathic Short Stature
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Sponsor
- Eli Lilly and Company
- Enrollment
- 91
- Locations
- 1
- Primary Endpoint
- Number of Participants With One or More Drug-related Adverse Events
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 8 Years to 171 Months (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Arms & Interventions
1
Intervention: somatropin (Drug)
1
Intervention: leuprorelin (Drug)
2
Intervention: somatropin (Drug)
Outcomes
Primary Outcomes
Number of Participants With One or More Drug-related Adverse Events
Time Frame: 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)
Time Frame: 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 Outcomes
- Difference Between Adult Height SDS and Target Height SDS(Baseline through End of Study (up to 9 years))
- Height SDS(Baseline through End of Study (up to 9 years))
- Difference Between Adult Height SDS and Baseline Height SDS(Baseline through End up Study (up to 9 years))
- Percentage of Children With Normal Adult Height SDS(Baseline through End of Study (up to 9 years))
- Height Velocity(Baseline through End of Study (up to 9 years))
- Bone Age(Baseline through End of Study (up to 9 years))
- Difference Between Adult Height SDS and Baseline Predicted Height SDS(Baseline through End up Study (up to 9 years))