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A Trial to Evaluate the Correlation Between Spontaneous Catch-up Growth, Clinical Response to Saizen (Recombinant Human Growth Hormone, r-hGH) and Gene Expression Profiling in Children Small for Gestational Age (SGA) (SAIZEN in SGA)

This study has been terminated.
(This study was discontinued prematurely due to difficulty in participant recruitment.)
Sponsor:
Collaborator:
Merck Serono S.P.A., Italy
Information provided by (Responsible Party):
Merck KGaA
ClinicalTrials.gov Identifier:
NCT01067352
First received: February 10, 2010
Last updated: December 2, 2013
Last verified: December 2013

February 10, 2010
December 2, 2013
February 2004
July 2009   (final data collection date for primary outcome measure)
Correlation Between Gene Expression Profiling and Catch-up Growth in Small for Gestational Age (SGA) Children [ Time Frame: Baseline and Week 48 ] [ Designated as safety issue: No ]
Gene expression profiling:analysis of ribonucleic acid (RNA) extracted from body tissue or fluids using Clontech Atlas Human Array to study level of activation of genes in tissue analyzed. Analysis was performed to identify possible correlation between catch-up growth (either spontaneous or drug-induced after Week 48) and therapeutic response to rhGH. Spontaneous catch up growth:shown by SGA participants having length more than third percentile at Week 96 without any treatment;drug induced growth was by SGA participants having length more than third percentile at Week 96 with drug treatment.
Not Provided
Complete list of historical versions of study NCT01067352 on ClinicalTrials.gov Archive Site
  • Percentage of Untreated Participants Who Showed a Spontaneous Catch-up Growth [ Time Frame: Baseline through Week 96 ] [ Designated as safety issue: No ]
    Spontaneous catch up growth was the growth shown by SGA participants having length more than third percentile at Week 96 without any study drug treatment.
  • Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Study Drug Discontinuation [ Time Frame: Baseline through Week 96 ] [ Designated as safety issue: Yes ]
    AEs: any new untoward medical occurrences/worsening of pre-existing medical condition, whether or not related to study drug , SAE: any AE that resulted in death; was life threatening; resulted in persistent/significant disability/incapacity; resulted in/prolonged an existing in-patient hospitalization; was a congenital anomaly/birth defect; or was an overdose. Participants who discontinued from the study due to AE were also recorded.
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Not Provided
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A Trial to Evaluate the Correlation Between Spontaneous Catch-up Growth, Clinical Response to Saizen (Recombinant Human Growth Hormone, r-hGH) and Gene Expression Profiling in Children Small for Gestational Age (SGA)
An Open, Multicenter, Randomized, Controlled Trial to Evaluate the Correlation Between Spontaneous Catch-up Growth, Clinical Response to Saizen (Recombinant Human Growth Hormone, r-hGH) and Gene Expression Profiling in Children Small for Gestational Age (SGA)

This open, multicentric, randomized, controlled study is planned to evaluate the correlation between gene expression, spontaneous catch-up growth and therapeutic response to Saizen in SGA children.

This open, multicentric, randomized, controlled study was planned to identify genes activated by hGH in SGA children responders to treatment (making it possible in the near future to better identify SGA children likely to benefit from hGH treatment). Furthermore, the study would hopefully allow to verify which genes were responsible of spontaneous catch-up growth in children with diagnosis of SGA at birth but above the third percentile for height at the age of 24 months, and if these genes were the same activated by hGH during the treatment in participants responders. Sixty children born at term (i.e. after the 37th completed week of gestation) and with a diagnosis of SGA (defined as a length less than tenth percentile according to the Italian reference table published by Bertini and Fabris) were planned to be enrolled in the study. Forty participants (group A) were still less than third percentile for height (according to the Tanner reference table) at the age of 24 months, the remaining 20 (group B) being more than or equal to third percentile (thus showing a spontaneous catch-up growth). Group A was randomized to receive Saizen at the daily dose of 0.067 mg/kg (Group A1) or no treatment (Group A2) for two years. All participants were to undergo full clinical examination and blood chemistry at baseline visit and visit after 1,6,12,18 and 24 months for a period of two years. Gene expression analysis using the Clontech Atlas Human Array was performed in all participants at baseline and after one year in order to identify the possible correlation between catch-up growth (either spontaneous or drug-induced) and expression of some genes.

OBJECTIVES

Primary objective:

  • To evaluate the correlation between gene expression profiling and catch-up growth (either spontaneous or drug induced after one year of treatment) in SGA children.

Secondary Objectives:

  • To evaluate the percentage of participants not treated who show a spontaneous catch-up growth during the two years of observation.
  • To assess the safety and tolerability of early treatment with Saizen
Interventional
Phase 3
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Infant, Small for Gestational Age
Drug: Recombinant human growth hormone (r-hGH)
Recombinant human GH were administered subcutaneously (s.c) at the daily dose of 0.067 mg/kg of body weight to Group A1.
Other Name: Saizen
  • Experimental: Group A (A1)
    Participants were allocated to Group A if were still third percentile for height (according to the Tanner reference table) at the age of 4-6 years. Group A would be then randomized to receive Saizen at the daily dose of 0.035 milligram (mg)/kilogram (kg) (Group A1) or no treatment (Group A2) for two years.
    Intervention: Drug: Recombinant human growth hormone (r-hGH)
  • No Intervention: Group A (A2)
    Participants were allocated to Group A if were still third percentile for height (according to the Tanner reference table) at the age of 4-6 years. Group A would be then randomized to receive no treatment (Group A2) for two years.
  • No Intervention: Group B
    Participants were allocated to Group B being third percentile (thus showing a spontaneous catch-up growth).
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Terminated
25
July 2009
July 2009   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • SGA at birth (defined as a length at birth equal or below the tenth percentile according to the Italian reference table of Bertini and Fabris)
  • Age of 24 Months
  • Caucasic
  • Born at term (i.e. after the 37th completed week of gestation)
  • Height equal or below (Group A) or up (Group B) the third percentile at the age of 24 months according to the Tanner reference table
  • Sufficient GH secretion (more than 10 nanogram (ng)/milliliter (ml)) at least to one of the tests commonly used at that age (glucagon, Levo-dopa, arginine, clonidine, Growth Hormone Releasing Hormone (GHRH), GH integrated secretion)
  • Normal level of Thyroid-stimulating hormone (THS), Free Triiodothyronine (FT3), Free Thyroxine (FT4), Insulin-like growth factor 1(IGF-1), insulin and haemoglobin A1c (HbA1c)
  • Normal level of Immunoglobulin A (IgA)
  • Children parents willing to comply with the protocol for the whole duration of the study
  • A written Informed Consent before the baseline visit must be obtained from the parent(s) / legal guardian(s)

Exclusion Criteria:

  • Congenital malformations (including Silver-Russel syndrome)
  • Known abnormal karyotype, especially in girls
  • Twins
  • Severe psychomotor retardation
  • Previous or ongoing treatment with anabolic steroids or r-hGH
  • Treatments interfering with the immune system (including bacterial lysate)
  • Severe chronic illnesses
  • Autoimmune diseases
Both
4 Years to 6 Years
No
Contact information is only displayed when the study is recruiting subjects
Italy
 
NCT01067352
IMP23681
Not Provided
Merck KGaA
Merck KGaA
Merck Serono S.P.A., Italy
Study Director: Medical Responsible Merck Serono S.P.A., Italy
Merck KGaA
December 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP