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Completed

NCT Number: NCT01088412

Observational Study of Somatropin Treatment in Children

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

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Key information

Conditions

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

Age range

1 day and older

Sex eligibility

All sexes

Study type

Observational

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Somatropin (recombinant deoxyribonucleic acid [rDNA] origin)

Drug

Dose, frequency and duration at discretion of attending physician.

Other names: Humatrope, LY137998

Primary outcomes

  1. Type 2 Diabetes Mellitus in GH-treated Participants

    Time frame: Year 15

  2. 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.

  3. 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.

Secondary outcomes

  1. 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).

  2. 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

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

    Time frame: Baseline, Year 15

  4. 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.

  5. 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.

  6. Diabetes Mellitus (DM) in Somatropin-Treated Children With Different Short Stature Diagnoses

    Time frame: Baseline through Year 15

Sponsors and collaborators

Lead sponsor

Eli Lilly and Company

Industry

Registry information

Official study title

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

Acronym: GeNeSIS

Important dates

Study start
1999
Primary completion
2015
Study completion
2015
First posted
Mar 17, 2010
Registry last updated
May 13, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.