Observational Study of Somatropin Treatment in Children (GeNeSIS)

Lilly

Status: Completed

Conditions: SHOX Deficiency-related Disorder; Short Stature Homeobox Containing Gene (SHOX) Deficiency; Non-GH-deficient Growth Disorders; Growth Hormone (GH) Deficiency

Treatments: Drug: Somatropin (recombinant deoxyribonucleic acid [rDNA] origin)

Study type: Observational

Funder types: Industry

Identifiers

NCT01088412

2712

B9R-EW-GDFC (Other Identifier)

Details and patient eligibility

About

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

Enrollment: 22,845 patients

Sex: All

Ages: 1+ day old

Volunteers: No Healthy Volunteers

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.

Trial design

22,845 participants in 2 patient groups

Treated

Description:

Participants treated with somatropin for improvement of growth

Treatment:

Drug: Somatropin (recombinant deoxyribonucleic acid [rDNA] origin)

Untreated

Description:

Untreated participants with presence or history of neoplastic disease evaluated for endocrine or growth disorder or with any SHOX deficiency related disorder