Clinical trial NCT00699855: Long Term Follow up Study of Predictive Markers in GHD and TS Children
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
Overall status: COMPLETED
Conditions: 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
Lead sponsor: Merck KGaA, Darmstadt, Germany
Sponsor class: INDUSTRY
First posted: 2008-06-18
Enrollment: 182
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Subjects initially enrolled in PREDICT (NCT 00256126) clinical study and continuing a growth hormone treatment.

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.

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

Primary outcomes:
measure: 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).
timeFrame: Yearly
Secondary outcomes:
measure: When available laboratory parameters such as IGF-1, IGFPB-3, fasting glucose, fasting insulin, TSH and T4 will also be collected.
timeFrame: Yearly
Other outcomes:
Locations:
Study officials:
affiliation: Merck Serono S.A., Geneva
name: Gilles Della Corte
role: STUDY_DIRECTOR
References:
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.
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.
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.
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.
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.