Observational Study of Somatropin Treatment in Children (GeNeSIS)
This study is enrolling participants by invitation only.
Sponsor:
Eli Lilly and Company
Information provided by (Responsible Party):
Eli Lilly and Company
ClinicalTrials.gov Identifier:
NCT01088412
First received: February 25, 2010
Last updated: November 2, 2012
Last verified: November 2012
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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:
Genetics Home Reference related topics:
combined pituitary hormone deficiency
isolated growth hormone deficiency
metatropic dysplasia
persistent Müllerian duct syndrome
pseudoachondroplasia
tetrasomy 18p
Turner syndrome
Drug Information available for:
Somatropin
U.S. FDA Resources
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:
|
|
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 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: | November 2, 2012 |
| 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:
|
Dwarfism Dwarfism, Pituitary Turner Syndrome Gonadal Dysgenesis Primary Ovarian Insufficiency Endocrine System Diseases Bone Diseases, Developmental Bone Diseases Musculoskeletal Diseases Genetic Diseases, Inborn Bone Diseases, Endocrine Hypopituitarism Pituitary Diseases Hypothalamic Diseases Brain Diseases |
Central Nervous System Diseases Nervous 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 Gonadal Disorders Ovarian Diseases Adnexal Diseases |
ClinicalTrials.gov processed this record on May 16, 2013