Phase 3
N=91
Somatropin + Leuprorelin vs Somatropin Alone in Pubertal Children With Idiopathic Short Stature
Idiopathic Short Stature (ISS)
Bottom Line
View on ClinicalTrials.gov: NCT00355030 ↗Enrolled (actual)
91
Serious AEs
20.9%
Results posted
Oct 2016
Primary outcome: Primary: Number of Participants With One or More Drug-related Adverse Events — 42; 38; 11; 9 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- somatropin (Drug); leuprorelin (Drug)
- Age
- Pediatric · 8+ yrs
- Sex
- All
- Sponsor
- Eli Lilly and Company
- Primary completion
- Jul 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With One or More Drug-related Adverse Events |
42; 38; 11; 9 | — |
| PRIMARY Adult Height Standard Deviation Score (SDS) |
-1.8; -1.9 | — |
| SECONDARY Height Velocity |
7.8; 7.4; 9.9; 10.6; 8.1; 9.7 | — |
| SECONDARY Height SDS |
-2.5; -2.5; -2.4; -2.4; -2.3; -2.2 | — |
| SECONDARY Difference Between Adult Height SDS and Target Height SDS |
-0.6; -1.2 | — |
| SECONDARY Difference Between Adult Height SDS and Baseline Predicted Height SDS |
1.1; 1.2 | — |
| SECONDARY Difference Between Adult Height SDS and Baseline Height SDS |
0.6; 0.6 | — |
| SECONDARY Percentage of Children With Normal Adult Height SDS |
26.7; 25.6 | — |
| SECONDARY Bone Age |
10.8; 11.0; 11.7; 12.1; 12.4; 13.4 | — |
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.
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT00355030). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.