Observational Study of Somatropin Treatment in Children - Full Text View

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 Meier-Gorlin syndrome metatropic dysplasia pseudoachondroplasia Turner syndrome

MedlinePlus related topics: Dwarfism Growth Disorders Turner Syndrome

Drug Information available for: Somatropin

Genetic and Rare Diseases Information Center resources: Children's Interstitial Lung Disease Gonadal Dysgenesis Growth Hormone Deficiency Hypopituitarism Turner Syndrome

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: 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):

Blum WF, Deal C, Zimmermann AG, Shavrikova EP, Child CJ, Quigley CA, Drop SL, Cutler GB Jr, Rosenfeld RG. Development of additional pituitary hormone deficiencies in pediatric patients originally diagnosed with idiopathic isolated GH deficiency. Eur J Endocrinol. 2013 Nov 22;170(1):13-21. doi: 10.1530/EJE-13-0643. Print 2014 Jan.

Deal C, Hasselmann C, Pfäffle RW, Zimmermann AG, Quigley CA, Child CJ, Shavrikova EP, Cutler GB Jr, Blum WF. Associations between pituitary imaging abnormalities and clinical and biochemical phenotypes in children with congenital growth hormone deficiency: data from an international observational study. Horm Res Paediatr. 2013;79(5):283-92. doi: 10.1159/000350829. Epub 2013 May 16.

Child CJ, Zimmermann AG, Scott RS, Cutler GB Jr, Battelino T, Blum WF; GeNeSIS International Advisory Board. Prevalence and incidence of diabetes mellitus in GH-treated children and adolescents: analysis from the GeNeSIS observational research program. J Clin Endocrinol Metab. 2011 Jun;96(6):E1025-34. doi: 10.1210/jc.2010-3023. Epub 2011 Apr 13. Erratum in: J Clin Endocrinol Metab. 2011 Aug;96(8):2624.


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 18, 2015
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, Pituitary Dwarfism Bone Diseases Bone Diseases, Developmental Bone Diseases, Endocrine Brain Diseases Central Nervous System Diseases	Endocrine System Diseases Hypopituitarism Hypothalamic Diseases Musculoskeletal Diseases Nervous System Diseases Pituitary Diseases

ClinicalTrials.gov processed this record on February 27, 2015

Observational Study of Somatropin Treatment in Children (GeNeSIS)