Phase 3
N=316
Individualized, Target-Driven Treatment Of Children With Idiopathic Short Stature
Idiopathic Short Stature
Bottom Line
View on ClinicalTrials.gov: NCT00396097 ↗Enrolled (actual)
316
Serious AEs
6.3%
Results posted
Nov 2013
Primary outcome: Primary: Absolute On-target Difference (AOTD) at 24 Months — 0.603; 0.625 Standard Deviation Score (SDS) — p=0.5762
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Genotropin (Drug)
- Age
- Pediatric · 3+ yrs
- Sex
- All
- Sponsor
- Pfizer
- Primary completion
- Aug 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute On-target Difference (AOTD) at 24 Months |
0.603; 0.625 | 0.5762 |
| SECONDARY Variability of Height SDS at 24 Months |
1.12; 1.11; 1.03; 1.04 | 0.627 |
| SECONDARY Time Cost (Months Until >= -2 SDS) |
12.00; 12.00 | 0.8016 |
| SECONDARY Computed Cost of Height Gain at 48 Months |
72.77; 67.30; 63.07; 69.62 | 0.0101 sig |
| SECONDARY Estimated Cost of Height Gain Estimated Until Full Adult Height (FAH) at 48 Months |
127.99; 91.34; 80.06; 92.24 | <0.0001 sig |
| SECONDARY Change From Baseline in Height SDS at 48 Months. |
1.33; 1.24; 1.33; 1.34 | 0.2618 |
Summary
To demonstrate that an individualized, formula-based Genotropin regimen for children with Idiopathic Short Stature will lead to a targeted height gain (to reach the target of 10th percentile (%), or -1.3 SDS) during 24 months of treatment. The endpoint at 4 years is to explore treatment efficiency over four years of two formula-based dose regimens (sub-arms) compared to standard treatment
Eligibility Criteria
Inclusion Criteria
- Prepubertal children with bone ages between 3 and 10 years of age for males and 3 and 9 years of age for females
- Naive to Growth Hormone treatment
Exclusion Criteria
- Abnormal karyotype. Small Gestational Age and Skeletal dysplasia.
Data sourced from ClinicalTrials.gov (NCT00396097). 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.