Observational Study of Somatropin Treatment in Children

NCT01088412 | Completed Results

• 2 other identifiers: 2712B9R-EW-GDFC
• Study Type: observational
• Target: 22,845
• Locations: 0 countries
• Sites: N/A

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
• Short Stature Homeobox containing gene (SHOX) Deficiency Sub-study: Elucidating the clinical, endocrine and radiological features of participants with SHOX deficiency due to loss of, or mutation in the SHOX gene (including participants 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

Conditions

Growth Hormone (GH) Deficiency; Short Stature Homeobox Containing Gene (SHOX) Deficiency; SHOX Deficiency-related Disorder; Non-GH-deficient Growth Disorders

Outcome Measures

Primary Outcomes (3)

• Type 2 Diabetes Mellitus in GH-treated Participants

  Year 15

• Primary Malignancies in Participant Without Previous Cancer History

  Due to the small number of participants involved, untreated and unknown treatment groups, data was not provided and could not be calculated.

  Year 15

• Final Height (FH) Gain by Diagnostic Group

  The standard deviation score (SDS) reports the number of standard deviations from the mean for age and sex for an individual measurement (normal range is -2 to +2 SDS). Height SDS is derived by subtracting the population mean from individual's height value and then dividing that difference by the population standard deviation. Greater height SDS values indicate greater height. Due to the small number of participants involved, untreated and unknown treatment groups, data was not provided and could not be calculated.

  Baseline through Year 15

Secondary Outcomes (6)

• Percentage of Participants With Defects in Genes Associated With Pituitary Development

  Baseline through Year 15

• Predicted First Year Height Gain Versus Actual First Year Height Gain

  Baseline through Year 15

• Change From Baseline to Final Height in Anthropometric Measures for Participants With SHOX Deficiency

  Baseline, Year 15

• Percentage of Participants With Recurrent Neoplasms and Second Neoplasms in Childhood Cancer Survivors

  Baseline through Year 15

• Percentage of Participants With De Novo Neoplasms

  Baseline through Year 15

Study Arms (2)

Treated

Participants treated with somatropin for improvement of growth

Drug: Somatropin (recombinant deoxyribonucleic acid [rDNA] origin)

Untreated

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

Interventions

Somatropin (recombinant deoxyribonucleic acid [rDNA] origin) DRUG

Dose, frequency and duration at discretion of attending physician.

Also known as: Humatrope, LY137998

Treated

Eligibility Criteria

• Age: 1 Day+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Clinics and private practices

You may qualify if:

• Treatment with Humatrope for improvement of growth.
• No treatment with somatropin in participants with a history of neoplasia or in those with any SHOX deficiency-related disorder.

You may not qualify if:

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

Sponsors & Collaborators

• Eli Lilly and Company: lead

Related Publications (6)

• Child CJ, Zimmermann AG, Chrousos GP, Cummings E, Deal CL, Hasegawa T, Jia N, Lawrence S, Linglart A, Loche S, Maghnie M, Perez Sanchez J, Polak M, Predieri B, Richter-Unruh A, Rosenfeld RG, Yeste D, Yorifuji T, Blum WF. Safety Outcomes During Pediatric GH Therapy: Final Results From the Prospective GeNeSIS Observational Program. J Clin Endocrinol Metab. 2019 Feb 1;104(2):379-389. doi: 10.1210/jc.2018-01189.

  PMID: 30219920 DERIVED

• Deal C, Kirsch S, Chanoine JP, Lawrence S, Cummings E, Rosolowsky ET, Marks SD, Jia N, Child CJ; GeNeSIS National Board on behalf of the GeNeSIS Canada Investigators. Growth hormone treatment of Canadian children: results from the GeNeSIS phase IV prospective observational study. CMAJ Open. 2018 Sep 10;6(3):E372-E383. doi: 10.9778/cmajo.20180020. Print 2018 Jul-Sep.

  PMID: 30201821 DERIVED

• Quigley CA, Child CJ, Zimmermann AG, Rosenfeld RG, Robison LL, Blum WF. Mortality in Children Receiving Growth Hormone Treatment of Growth Disorders: Data From the Genetics and Neuroendocrinology of Short Stature International Study. J Clin Endocrinol Metab. 2017 Sep 1;102(9):3195-3205. doi: 10.1210/jc.2017-00214.

  PMID: 28575299 DERIVED

• 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.

  PMID: 24088548 DERIVED

• Deal C, Hasselmann C, Pfaffle 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.

  PMID: 23689058 DERIVED

• 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.

  PMID: 21490076 DERIVED

Biospecimen

Retention: SAMPLES WITH DNA

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

MeSH Terms

Interventions

Human Growth Hormone; DNA, Recombinant; DNA, Ribosomal; Growth Hormone

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins; DNA; Nucleic Acids; Nucleic Acids, Nucleotides, and Nucleosides

Results Point of Contact

• Title: Chief Medical Officer
• Organization: Eli Lilly and Company

ClinicalTrials.gov@lilly.com

800-545-5979

Study Officials

• 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

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 25, 2010
• First Posted: March 17, 2010
• Study Start: April 1, 1999
• Primary Completion: September 1, 2015
• Study Completion: September 1, 2015
• Last Updated: May 13, 2019
• Results First Posted: May 13, 2019

Record last verified: 2018-07