N/A
N=22,845
Observational Study of Somatropin Treatment in Children
Growth Hormone (GH) Deficiency · Short Stature Homeobox Containing Gene (SHOX) Deficiency · SHOX Deficiency-related Disorder · Non-GH-deficient Growth Disorders
Bottom Line
View on ClinicalTrials.gov: NCT01088412 ↗Enrolled (actual)
22,845
Serious AEs
2.6%
Results posted
May 2019
Primary outcome: Primary: Type 2 Diabetes Mellitus in GH-treated Participants — 1; 9; 8 Incident Cases
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- Somatropin (recombinant deoxyribonucleic acid [rDNA] origin) (Drug)
- Age
- Pediatric, Adult, Older Adult · 0+ yrs
- Sex
- All
- Sponsor
- Eli Lilly and Company
- Primary completion
- Sep 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Type 2 Diabetes Mellitus in GH-treated Participants |
1; 9; 8 | — |
| PRIMARY Primary Malignancies in Participant Without Previous Cancer History |
14 | — |
| PRIMARY Final Height (FH) Gain by Diagnostic Group |
-2.42; -1.18; 1.24; -2.35; -0.96; 1.39 | — |
| SECONDARY Percentage of Participants With Defects in Genes Associated With Pituitary Development |
6.7; 14.8; 0.0 | — |
| SECONDARY Predicted First Year Height Gain Versus Actual First Year Height Gain |
0.261; 0.408 | — |
| SECONDARY Change From Baseline to Final Height in Anthropometric Measures for Participants With SHOX Deficiency |
0.8; 1.4; -0.6; -0.7; 1.0; 10.2 | — |
| SECONDARY Percentage of Participants With Recurrent Neoplasms and Second Neoplasms in Childhood Cancer Survivors |
6.81; 4.98 | — |
| SECONDARY Percentage of Participants With De Novo Neoplasms |
0.07 | — |
| SECONDARY Diabetes Mellitus (DM) in Somatropin-Treated Children With Different Short Stature Diagnoses |
38; 22; 6; 4; 0; 2 | — |
Summary
GeNeSIS is an open-label, multinational, multicenter, observational study to evaluate the safety and effectiveness of Humatrope treatment.
GeNeSIS is a modular program that includes:
* Core study: Evaluating the safety and effectiveness of Humatrope in the observational setting
* Genetic Analysis Sub-study: Investigating the genetic defects underlying growth hormone (GH) deficiency and non-GH-deficient growth disorders
* Growth Prediction Sub-study: Working to validate and refine specific models to accurately predict growth response to GH
* Short Stature Homeobox containing gene (SHOX) Deficiency Sub-study: Elucidating the clinical, endocrine and radiological features of participants with SHOX deficiency due to loss of, or mutation in the SHOX gene (including participants with Turner syndrome)
* Neoplasia Sub-study: To characterize the natural history of neoplastic disease, especially in relation to recurrence/progression of primary neoplasia or development of secondary neoplasia in children with a history of neoplasia
Eligibility Criteria
Inclusion Criteria
All participants participating in GeNeSIS must be enrolled in the core study. Participants for whom written consent to release information is provided may enter the core study if they meet any of the following inclusion guidelines:
- Treatment with Humatrope for improvement of growth.
- No treatment with somatropin in participants with a history of neoplasia or in those with any SHOX deficiency-related disorder.
Exclusion Criteria
- Participants with closed epiphyses are not eligible for GeNeSIS entry. However, participants may remain in the study if epiphyseal closure occurs during study participation.
Data sourced from ClinicalTrials.gov (NCT01088412). 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.