Trial · Observational

CompletedPhase not statedResults postedRegistry data

Observational Study of Somatropin Treatment in Children

Short name GeNeSIS

NCTNCT01088412Other id2712Other idB9R-EW-GDFC

Open at ClinicalTrials.govAsk about this trial

Overview

Status: Completed
Phase: Phase not stated
Enrollment: 22,845 participants (actual)
Sponsor: Eli Lilly and Company · Industry
Design: Not stated
Countries: Not stated
Start: Apr 1999
Primary completion: Sept 2015
Completion: Sept 2015
First posted: 17 Mar 2010 (estimated)
Last update posted: 13 May 2019
Conditions: Growth Hormone (GH) Deficiency · Short Stature Homeobox Containing Gene (SHOX) Deficiency · SHOX Deficiency-related Disorder · Non-GH-deficient Growth Disorders

Timeline

1 trial · 1 completed · 1 with posted results

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

2000200520102015

Ph –
GeNeSIS
NCT01088412
Apr 1999 to Sept 2015

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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

Interventions

Somatropin (recombinant deoxyribonucleic acid [rDNA] origin)Drug

Arms2

Other
Treated
Participants treated with somatropin for improvement of growth
Other
Untreated
Untreated participants with presence or history of neoplastic disease evaluated for endocrine or growth disorder or with any SHOX deficiency related disorder

Primary outcomes3

Outcomes the sponsor listed as primary.

Type 2 Diabetes Mellitus in GH-treated Participants
Time frame · Year 15
Primary Malignancies in Participant Without Previous Cancer History
Time frame · Year 15
Registry description
Due to the small number of participants involved, untreated and unknown treatment groups, data was not provided and could not be calculated.
Final Height (FH) Gain by Diagnostic Group
Time frame · Baseline through Year 15
Registry description
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(6)

Percentage of Participants With Defects in Genes Associated With Pituitary Development
Time frame · Baseline through Year 15
Registry description
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).
Predicted First Year Height Gain Versus Actual First Year Height Gain
Time frame · Baseline through Year 15
Registry description
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
Change From Baseline to Final Height in Anthropometric Measures for Participants With SHOX Deficiency
Time frame · Baseline, Year 15
Percentage of Participants With Recurrent Neoplasms and Second Neoplasms in Childhood Cancer Survivors
Time frame · Baseline through Year 15
Registry description
Percentage of participants with recurrence/progression of primary neoplastic disease and/or development of secondary neoplasms in childhood cancer survivors.
Percentage of Participants With De Novo Neoplasms
Time frame · Baseline through Year 15
Registry description
Percentage of participants with the development of de novo neoplastic disease with no history of prior neoplasia.
Diabetes Mellitus (DM) in Somatropin-Treated Children With Different Short Stature Diagnoses
Time frame · Baseline through Year 15

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeNumber· Year 15

Type 2 Diabetes Mellitus in GH-treated Participants

Group	Value (Incident Cases)
Treated	1

Primary outcomeNumber· Year 15

Primary Malignancies in Participant Without Previous Cancer History

Group	Value (Incident Cases)
Treated	14

Primary outcomeMean· Baseline through Year 15

Final Height (FH) Gain by Diagnostic Group

Group	Value (standard deviation score)	Spread
Treated	-2.42	1.01

Secondary and other outcome results(5)

Secondary outcomeNumber· Baseline through Year 15

Percentage of Participants With Defects in Genes Associated With Pituitary Development

Group	Value (percentage of participants)
Treated	6.7
Untreated	14.8
Unknown	0.0

Secondary outcomeNumber· Baseline through Year 15

Predicted First Year Height Gain Versus Actual First Year Height Gain

Group	Value (R Squared)
Treated	0.261

Secondary outcomeMean· Baseline, Year 15

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

Group	Value (standard deviation score)	Spread
Treated	0.8	0.9

Secondary outcomeNumber· Baseline through Year 15

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

Group	Value (percentage of participants)
Treated	6.81

Secondary outcomeNumber· Baseline through Year 15

Percentage of Participants With De Novo Neoplasms

Group	Value (percentage of participants)
Treated	0.07

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
Group	Deaths	Serious events	Other events
TreatedTreatment with Humatrope for improvement of growth.	–	547 / 21,177	6180 / 21,177
UntreatedNo treatment with somatropin in participants with a history of neoplasia or in those with any SHOX deficiency-related disorder.	–	14 / 306	126 / 306
GroupTreatment with Humatrope unknown.	–	0 / 40	0 / 40

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies2

Studies that cite this trial.

More studies

2 studies on this page

PubMed tag: none · 2018

Growth hormone treatment of Canadian children: results from the GeNeSIS phase IV prospective observational study.

Deal et al. 2018CMAJ opencited by 14

PMID 30201821PMC6182101DOI 10.9778/cmajo.20180020PUB-30201821

Rights unknown

Source link

Indexing hint: Observational study · 2017

Mortality in Children Receiving Growth Hormone Treatment of Growth Disorders: Data From the Genetics and Neuroendocrinology of Short Stature International Study.

Quigley et al. 2017The Journal of clinical endocrinology and metabolismcited by 28

PMID 28575299DOI 10.1210/jc.2017-00214PUB-28575299

Rights unknown

Source link

Other registry references

References listed at the registry that are not in PepBank. They link to PubMed.

DerivedChild 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
DerivedBlum 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
DerivedDeal 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
DerivedChild 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

Eligibility

Who could join, as posted.

Sex: All
Age: from 1 Day
Healthy volunteers: Not accepted

Inclusion and exclusion criteria, as posted

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.

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