Clinical Trial: Observational Study of Somatropin Treatment in Children

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: The Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS)

Brief 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
  • SHOX Deficiency Sub-study: Elucidating the clinical, endocrine and radiological features of patients with SHOX deficiency due to loss of, or mutation in the SHOX gene (including patients 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

Detailed Summary:
Sponsor: Eli Lilly and Company

Current Primary Outcome:

  • Standardized Incidence Ratio for Type 2 Diabetes Mellitus in Somatropin-Treated Children [ Time Frame: Year 15 ]
  • Standardized Incidence Ratio for De Novo Cancer in Somatropin-Treated Children [ Time Frame: Year 15 ]
  • Baseline Height, Final Height, Height Gain in Somatropin-Treated Children [ Time Frame: Baseline through Year 15 ]


Original Primary Outcome:

  • Incidence of type 2 diabetes mellitus in somatropin-treated children [ Time Frame: Will be analyzed at study completion ]
  • Incidence of de novo neoplasia in children without a prior history of neoplastic disease who receive treatment with somatropin. [ Time Frame: Will be analyzed at study completion ]
  • Identify factors associated with the magnitude of treatment-related height gains and/or attained final height in patients treated with somatropin [ Time Frame: Will be analyzed at study completion ]


Current Secondary Outcome:

  • Percentage of Participants with Defects in Genes Associated with Pituitary Development [ Time Frame: Baseline through Year 15 ]
  • Predicted First Year Height Gain Versus Actual First Year Height Gain [ Time Frame: Baseline through Year 15 ]
  • Change from Baseline to Final Height in Anthropometric Measures for Participants with SHOX Deficiency [ Time Frame: Baseline, Year 15 ]
  • Percentage of Participants with Recurrent Tumors and Second Neoplasms [ Time Frame: Baseline through Year 15 ]
  • Percentage of Participants with De Novo Neoplasms [ Time Frame: Baseline through Year 15 ]
  • Standardized Incidence Ratio for Diabetes Mellitus in Somatropin-Treated Children with Different Short Stature Diagnoses [ Time Frame: Baseline through Year 15 ]


Original Secondary Outcome:

  • Characterization of genetic defects and DNA sequence alterations associated with hypopituitarism, GH deficiency, growth disorders or short stature [ Time Frame: Data will be reviewed and analyzed periodically and at the end of the study ]
  • Development and validation of accurate growth prediction models using clinical data and biochemical /genetic data. [ Time Frame: Data will be reviewed and analyzed periodically and at the end of the study ]
  • Characterization of the clinical, endocrine and other features associated with SHOX deficiency and related disorders including Turner syndrome, Léri-Weill syndrome and Langer syndrome. [ Time Frame: Data will be reviewed and analyzed periodically and at the end of the study ]
  • Characterization of natural history of neoplastic disease,in relation to recurrence/progression of primary - or development of secondary neoplasia in children with a history of neoplasia evaluated/treated for an endocrine disorder or a growth disorder. [ Time Frame: Data will be reviewed and analyzed periodically and at the end of the study ]
  • Determine the occurrence of de novo neoplasms in both somatropin-treated and untreated patients. [ Time Frame: Data will be reviewed and analyzed periodically and at the end of the study ]
  • Determine the risk of diabetes mellitus or conditions associated with alterations in glucose metabolism in particular subgroups of somatropin-treated children [ Time Frame: Data will be reviewed and analyzed periodically and at the end of the study ]


Information By: Eli Lilly and Company

Dates:
Date Received: February 25, 2010
Date Started: April 1999
Date Completion:
Last Updated: October 23, 2015
Last Verified: October 2015