Observational Study of Somatropin Treatment in Children (GeNeSIS)

February 11, 2019 updated by: Eli Lilly and Company

The Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS)

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

22845

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 day and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Clinics and private practices

Description

Inclusion Criteria:

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

Exclusion Criteria:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Treated
Participants treated with somatropin for improvement of growth
Drug: Somatropin (recombinant deoxyribonucleic acid [rDNA] origin)
Dose, frequency and duration at discretion of attending physician.
Other Names:
  • Humatrope
  • LY137998
Untreated
Untreated participants with presence or history of neoplastic disease evaluated for endocrine or growth disorder or with any SHOX deficiency related disorder

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Type 2 Diabetes Mellitus in GH-treated Participants
Time Frame: Year 15
Year 15
Primary Malignancies in Participant Without Previous Cancer History
Time Frame: Year 15
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
Time Frame: Baseline through Year 15
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of Participants With Defects in Genes Associated With Pituitary Development
Time Frame: Baseline through Year 15
Percentage of participants with genetic defects associated with pituitary development. Genes included but were not limited to GH1, Growth hormone releasing hormone receptor (GHRHR), Homeobox gene expressed in embryonic stem cells (HESX1), LIM homeobox 3 (LHX3), POU domain, class 1, transcription factor 1 (POU1F1), and Prophet of Pit1 (PROP1).
Baseline through Year 15
Predicted First Year Height Gain Versus Actual First Year Height Gain
Time Frame: Baseline through Year 15
The value for predicted and observed is of limited bearing, it is how each participant's predicted versus observed height gain compare and this is best estimated by the R-squared. An estimation parameter would not be a correct format for the R2 data. R2 can take value between 0 and 1 with values closer to 0 representing a poor fit while values closer to 1 representing a perfect fit
Baseline through Year 15
Change From Baseline to Final Height in Anthropometric Measures for Participants With SHOX Deficiency
Time Frame: Baseline, Year 15
Baseline, Year 15
Percentage of Participants With Recurrent Neoplasms and Second Neoplasms in Childhood Cancer Survivors
Time Frame: Baseline through Year 15
Percentage of participants with recurrence/progression of primary neoplastic disease and/or development of secondary neoplasms in childhood cancer survivors.
Baseline through Year 15
Percentage of Participants With De Novo Neoplasms
Time Frame: Baseline through Year 15
Percentage of participants with the development of de novo neoplastic disease with no history of prior neoplasia.
Baseline through Year 15
Diabetes Mellitus (DM) in Somatropin-Treated Children With Different Short Stature Diagnoses
Time Frame: Baseline through Year 15
Baseline through Year 15

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Eli Lilly and Company

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 1999

Primary Completion (Actual)

September 1, 2015

Study Completion (Actual)

September 1, 2015

Study Registration Dates

First Submitted

February 25, 2010

First Submitted That Met QC Criteria

March 16, 2010

First Posted (Estimate)

March 17, 2010

Study Record Updates

Last Update Posted (Actual)

May 13, 2019

Last Update Submitted That Met QC Criteria

February 11, 2019

Last Verified

July 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2712
  • B9R-EW-GDFC (Other Identifier: ELI Lilly and Company)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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