Long Term Follow up Study of Predictive Markers in GHD and TS Children (PREDICT LT FUP)
Primary Purpose
Growth Hormon Deficiency, Turner Syndrome in Pre-pubertal Children
Status
Completed
Phase
Locations
Study Type
Observational
Intervention
Sponsored by
About this trial
This is an observational trial for Growth Hormon Deficiency focused on measuring Growth Hormon Deficiency and, Turner Syndrome in pre-pubertal children, Long term follow-up of predictive markers
Eligibility Criteria
Inclusion Criteria:
- Have completed the PREDICT study (NCT 00256126)
- Followed up at least 1 year when still under treatment after completion of PREDICT Trial
- Parent's or guardian's written consent given before any data collection
Exclusion Criteria:
- Use of an investigational drug or participation in another interventional clinical trial since discontinuation of PREDICT trial
Sites / Locations
Outcomes
Primary Outcome Measures
Data such as auxological parameters (height, weight, Tanner stage, bone age) will be collected as well as GH treatment use (including dose and adherence to the treatment).
Secondary Outcome Measures
When available laboratory parameters such as IGF-1, IGFPB-3, fasting glucose, fasting insulin, TSH and T4 will also be collected.
Full Information
NCT ID
NCT00699855
First Posted
June 13, 2008
Last Updated
February 17, 2014
Sponsor
Merck KGaA, Darmstadt, Germany
1. Study Identification
Unique Protocol Identification Number
NCT00699855
Brief Title
Long Term Follow up Study of Predictive Markers in GHD and TS Children
Acronym
PREDICT LT FUP
Official Title
Observational Long-term Follow-up of the Phase IV Open-label Trial of Predictive Markers in GHD and TS Pre-pubertal Children Treated With Saizen
Study Type
Observational
2. Study Status
Record Verification Date
February 2014
Overall Recruitment Status
Completed
Study Start Date
September 2008 (undefined)
Primary Completion Date
August 2012 (Actual)
Study Completion Date
August 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Merck KGaA, Darmstadt, Germany
4. Oversight
5. Study Description
Brief Summary
Primary objective is to assess the relationship between changes from serum biomarkers observed after 1 month of Saizen® therapy and change in height, weight after up to 5 years of treatment with Growth Hormone in children with Growth Hormone Deficiency (GHD) and Turner Syndrome (TS).
Detailed Description
This study is an observational study that will collect data from patients enrolled in a previous study (PREDICT, NCT 00256126). Data such as auxological parameters (height, weight, Tanner stage, bone age will be collected as well as GH treatment use (including dose and adherence to the treatment).
Because for some countries the start of this long term follow up study will take place more than one year after subjects have completed the initial study (PREDICT) retrospective data may be collected (if subjects agree) as well as prospective data.
When available laboratory parameters such as IGF-1, IGFPB-3, fasting glucose, fasting insulin, TSH and T4 will also be collected.
This data will be collected yearly during the normal follow up visits during 5 years.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Growth Hormon Deficiency, Turner Syndrome in Pre-pubertal Children
Keywords
Growth Hormon Deficiency and, Turner Syndrome in pre-pubertal children, Long term follow-up of predictive markers
7. Study Design
Enrollment
182 (Actual)
Primary Outcome Measure Information:
Title
Data such as auxological parameters (height, weight, Tanner stage, bone age) will be collected as well as GH treatment use (including dose and adherence to the treatment).
Time Frame
Yearly
Secondary Outcome Measure Information:
Title
When available laboratory parameters such as IGF-1, IGFPB-3, fasting glucose, fasting insulin, TSH and T4 will also be collected.
Time Frame
Yearly
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Have completed the PREDICT study (NCT 00256126)
Followed up at least 1 year when still under treatment after completion of PREDICT Trial
Parent's or guardian's written consent given before any data collection
Exclusion Criteria:
Use of an investigational drug or participation in another interventional clinical trial since discontinuation of PREDICT trial
Study Population Description
Subjects initially enrolled in PREDICT (NCT 00256126) clinical study and continuing a growth hormone treatment.
Sampling Method
Non-Probability Sample
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Gilles Della Corte
Organizational Affiliation
Merck Serono S.A., Geneva
Official's Role
Study Director
12. IPD Sharing Statement
Citations:
PubMed Identifier
23761422
Citation
Clayton P, Chatelain P, Tato L, Yoo HW, Ambler GR, Belgorosky A, Quinteiro S, Deal C, Stevens A, Raelson J, Croteau P, Destenaves B, Olivier C. A pharmacogenomic approach to the treatment of children with GH deficiency or Turner syndrome. Eur J Endocrinol. 2013 Jul 29;169(3):277-89. doi: 10.1530/EJE-13-0069. Print 2013 Sep.
Results Reference
result
PubMed Identifier
34045667
Citation
Stevens A, Murray P, De Leonibus C, Garner T, Koledova E, Ambler G, Kapelari K, Binder G, Maghnie M, Zucchini S, Bashnina E, Skorodok J, Yeste D, Belgorosky A, Siguero JL, Coutant R, Vangsoy-Hansen E, Hagenas L, Dahlgren J, Deal C, Chatelain P, Clayton P. Gene expression signatures predict response to therapy with growth hormone. Pharmacogenomics J. 2021 Oct;21(5):594-607. doi: 10.1038/s41397-021-00237-5. Epub 2021 May 27.
Results Reference
derived
PubMed Identifier
29618660
Citation
Murray PG, Stevens A, De Leonibus C, Koledova E, Chatelain P, Clayton PE. Transcriptomics and machine learning predict diagnosis and severity of growth hormone deficiency. JCI Insight. 2018 Apr 5;3(7):e93247. doi: 10.1172/jci.insight.93247. eCollection 2018 Apr 5.
Results Reference
derived
PubMed Identifier
26503811
Citation
De Leonibus C, Chatelain P, Knight C, Clayton P, Stevens A. Effect of summer daylight exposure and genetic background on growth in growth hormone-deficient children. Pharmacogenomics J. 2016 Nov;16(6):540-550. doi: 10.1038/tpj.2015.67. Epub 2015 Oct 27.
Results Reference
derived
PubMed Identifier
26340968
Citation
Valsesia A, Chatelain P, Stevens A, Peterkova VA, Belgorosky A, Maghnie M, Antoniazzi F, Koledova E, Wojcik J, Farmer P, Destenaves B, Clayton P; PREDICT Investigator group. GH deficiency status combined with GH receptor polymorphism affects response to GH in children. Eur J Endocrinol. 2015 Dec;173(6):777-89. doi: 10.1530/EJE-15-0474. Epub 2015 Sep 4.
Results Reference
derived
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Long Term Follow up Study of Predictive Markers in GHD and TS Children
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