Observational Study of Somatropin Treatment in Children (GeNeSIS)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Eli Lilly and Company
ClinicalTrials.gov Identifier:
NCT01088412
First received: February 25, 2010
Last updated: February 17, 2014
Last verified: February 2014
  Purpose

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

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: The Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS)

Resource links provided by NLM:


Further study details as provided by Eli Lilly and Company:

Primary Outcome Measures:
  • Incidence of type 2 diabetes mellitus in somatropin-treated children [ Time Frame: Will be analyzed at study completion ] [ Designated as safety issue: Yes ]
  • 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 ] [ Designated as safety issue: Yes ]
  • 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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: Yes ]
  • 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 ] [ Designated as safety issue: Yes ]
  • 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 ] [ Designated as safety issue: Yes ]

Biospecimen Retention:   Samples With DNA

If patient consented a DNA sample is kept until the end of the study


Estimated Enrollment: 23500
Study Start Date: April 1999
Estimated Study Completion Date: September 2015
Estimated Primary Completion Date: September 2015 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Treated
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
Untreated patients with presence or history of neoplastic disease evaluated for endocrine or growth disorder or with any SHOX deficiency related disorder

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Clinics and private practices

Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01088412

Sponsors and Collaborators
Eli Lilly and Company
Investigators
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
  More Information

No publications provided by Eli Lilly and Company

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Eli Lilly and Company
ClinicalTrials.gov Identifier: NCT01088412     History of Changes
Other Study ID Numbers: 2712, B9R-EW-GDFC
Study First Received: February 25, 2010
Last Updated: February 17, 2014
Health Authority: Australia: Human Research Ethics Committee
Austria: Ethikkommission
Belgium: Institutional Review Board
Canada: Ethics Review Committee
Czech Republic: Ethics Committee
Finland: Ethics Committee
Finland: Finnish Medicines Agency
France: French Data Protection Authority
Germany: Federal Institute for Drugs and Medical Devices
Greece: Ethics Committee
Greece: National Organization of Medicines
Hungary: Institutional Ethics Committee
India: Institutional Review Board
India: Drugs Controller General of India
Italy: Ethics Committee
Japan: Institutional Review Board
Lithuania: Bioethics Committee
Lithuania: State Medicine Control Agency - Ministry of Health
Netherlands: Independent Ethics Committee
Slovak Republic: Ethics Committee
Spain: Ethics Committee
Taiwan: Institutional Review Board
Taiwan: Department of Health
United States: Institutional Review Board

Additional relevant MeSH terms:
Turner Syndrome
Gonadal Dysgenesis
Primary Ovarian Insufficiency
Dwarfism, Pituitary
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
Endocrine System Diseases
Ovarian Diseases
Adnexal Diseases
Genital Diseases, Female
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Endocrine
Hypopituitarism
Pituitary Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases

ClinicalTrials.gov processed this record on September 16, 2014