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Trial · Interventional

TerminatedPhase 3Results postedRegistry data

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)

Short name SAIZEN in SGA

NCTNCT01067352 (opens ClinicalTrials.gov in a new tab)Other idIMP23681
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Terminated
Phase
Phase 3
Enrollment
25 participants (actual)
Sponsor
Merck KGaA, Darmstadt, Germany · Industry
Design
Randomized · Open label (no masking)
Countries
1 country

Italy

Start
Feb 2004
Primary completion
Jul 2009
Completion
Jul 2009
First posted
11 Feb 2010 (estimated)
Last update posted
27 Dec 2013 (estimated)
Conditions
Infant, Small for Gestational Age

Timeline

1 trial · 1 stopped early · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
2004200520062007200820092010
  1. Ph 3

    SAIZEN in SGA

    NCT01067352

    Feb 2004 to Jul 2009

    Terminated

Registry summary

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

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.

Interventions

  • Recombinant human growth hormone (r-hGH)Drug

Arms3

  • 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.

  • 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).

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Correlation Between Gene Expression Profiling and Catch-up Growth in Small for Gestational Age (SGA) Children

    Time frame · Baseline and Week 48

    Registry description

    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.

Secondary outcomes(2)

  1. Percentage of Untreated Participants Who Showed a Spontaneous Catch-up Growth

    Time frame · Baseline through Week 96

    Registry description

    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.

  2. Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Study Drug Discontinuation

    Time frame · Baseline through Week 96

    Registry description

    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.

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 outcome· Baseline and Week 48

Correlation Between Gene Expression Profiling and Catch-up Growth in Small for Gestational Age (SGA) Children

GroupValue
Group A, Less Than Third Percentile (Saizen)not posted
Group A2, Less Than Third Percentile (No Treatment)not posted
Group B, More Than Third Percentile (No Treatment)not posted
Secondary and other outcome results(2)
Secondary outcome· Baseline through Week 96

Percentage of Untreated Participants Who Showed a Spontaneous Catch-up Growth

GroupValue
Group A, Less Than Third Percentile (Saizen)not posted
Group A2, Less Than Third Percentile (No Treatment)not posted
Group B, More Than Third Percentile (No Treatment)not posted
Secondary outcomeNumber· Baseline through Week 96

Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Study Drug Discontinuation

GroupValue (participants)
Group A, Less Than Third Percentile (Saizen)6
Group A2, Less Than Third Percentile (No Treatment)4
Group B, More Than Third Percentile (No Treatment)9

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
Group A, Less Than Third Percentile (Saizen)Participants with less than third percentile for height (according to the Tanner reference table) at the age of 4-6 years received Saizen (recombinant human growth hormone, r-hGH) subcutaneously (s.c) at the daily dose of 0.035 milligram(mg)/kilogram(kg) for 2 years.–0 / 86 / 8
Group A2, Less Than Third Percentile (No Treatment)Participants with less than third percentile for height (according to the Tanner reference table) at the age of 4-6 years received no drug treatment for 2 years.–0 / 44 / 4
Group B, More Than Third Percentile (No Treatment)Participants with more than third percentile for height (according to the Tanner reference table) at the age of 4-6 years received no drug treatment for 2 years.–0 / 109 / 10

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

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
from 4 Years up to 6 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
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

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