Observational Study of Somatropin Treatment in Children (GeNeSIS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01088412
Recruitment Status : Completed
First Posted : March 17, 2010
Last Update Posted : October 26, 2015
Information provided by (Responsible Party):
Eli Lilly and Company

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

Condition or disease Intervention/treatment
Dwarfism, Growth Hormone Deficiency Turner Syndrome Infant, Small for Gestational Age SHOX Protein, Human Drug: Somatropin (rDNA origin)

Study Type : Observational
Actual Enrollment : 22929 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: The Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS)
Study Start Date : April 1999
Actual Primary Completion Date : September 2015
Actual Study Completion Date : September 2015

Group/Cohort Intervention/treatment
Patients treated with somatropin for improvement of growth
Drug: Somatropin (rDNA origin)
Dose, frequency and duration at discretion of attending physician.
Other Names:
  • Humatrope
  • LY137998

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

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

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

Biospecimen Retention:   Samples With DNA
If patient consented a DNA sample is kept until the end of the study

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Clinics and private practices

Inclusion Criteria:

All patients participating in GeNeSIS must be enrolled in the core study. Patients 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 patients with a history of neoplasia or in those with any SHOX-related disorder.

Exclusion Criteria:

  • Patients with closed epiphyses are not eligible for GeNeSIS entry. However, patients may remain in the study if epiphyseal closure occurs during study participation.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01088412

Sponsors and Collaborators
Eli Lilly and Company
Study Director: Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon-Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST) Eli Lilly and Company

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Eli Lilly and Company Identifier: NCT01088412     History of Changes
Other Study ID Numbers: 2712
B9R-EW-GDFC ( Other Identifier: ELI Lilly and Company )
First Posted: March 17, 2010    Key Record Dates
Last Update Posted: October 26, 2015
Last Verified: October 2015

Additional relevant MeSH terms:
Dwarfism, Pituitary
Turner Syndrome
Gonadal Dysgenesis
Primary Ovarian Insufficiency
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Endocrine
Pituitary Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Endocrine System Diseases
Disorders of Sex Development
Urogenital Abnormalities
Sex Chromosome Disorders of Sex Development
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Congenital Abnormalities
Sex Chromosome Disorders
Chromosome Disorders
Genetic Diseases, Inborn
Gonadal Disorders
Ovarian Diseases
Adnexal Diseases