Infant, Small for Gestational AgePhase 3SAIZEN in SGANCT01067352
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)
A Phase 3 study testing Recombinant human growth hormone (r-hGH) in people with Infant, Small for Gestational Age, run by Merck KGaA, Darmstadt, Germany at 1 site in Italy.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 4 to 6 years
- Conditions the study is for
- Infant, Small for Gestational Age
- What is being tested?
- Recombinant human growth hormone (r-hGH)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteItaly
- Study dates
- Feb 2004to Jul 2009
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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)
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- 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)
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
Read the detailed description
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
- Conditions
- Infant, Small for Gestational Age
- Being tested
- Recombinant human growth hormone (r-hGH)
- Keywords
- Infant, small for gestational age, Growth hormone, Saizen
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant human growth hormone (r-hGH)Drug
Also called Saizen
Recombinant human GH were administered subcutaneously (s.c) at the daily dose of 0.067 mg/kg of body weight to Group A1.
| Study group | Role | What they receive |
|---|---|---|
| Group A (A1) | Experimental | 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. Receives: Drug: Recombinant human growth hormone (r-hGH) |
| Group A (A2) | No intervention | 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. |
| Group B | No intervention | Participants were allocated to Group B being third percentile (thus showing a spontaneous catch-up growth). |
What is being measured
The primary outcome is the main question the study is designed to answer.
Correlation Between Gene Expression Profiling and Catch-up Growth in Small for Gestational Age (SGA) Children
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.
Measured: Baseline and Week 48
Secondary outcomes (2)
Percentage of Untreated Participants Who Showed a Spontaneous Catch-up Growth
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.
Measured: Baseline through Week 96
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Study Drug Discontinuation
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.
Measured: Baseline through Week 96
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2010 (estimated)
- Study startFeb 2004
- Primary completionJul 2009
- Study completionJul 2009
Why it stopped: This study was discontinued prematurely due to difficulty in participant recruitment.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Infant, Small for Gestational AgeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Merck KGaA, Darmstadt, Germany
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Merck Serono S.P.A., Italy
- Responsible party
- Sponsor
- Investigators
- Medical Responsible, Study director, Merck Serono S.P.A., Italy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01067352
- Sponsor's protocol ID
- IMP23681
- Acronym
- SAIZEN in SGA
- Status last verified
- Dec 2013
- First submitted
- 10 Feb 2010
- Last updated
- 27 Dec 2013
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 27 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT01067352
- Acronym / sponsor ID
- SAIZEN in SGA / IMP23681
- Conditions
- Infant, Small for Gestational Age
- Keywords
- Infant, small for gestational age, Growth hormone, Saizen
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: This study was discontinued prematurely due to difficulty in participant recruitment.
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Merck KGaA, Darmstadt, GermanyIndustry (pharma, biotech, devices)
- Collaborators
- Merck Serono S.P.A., Italy
- Study start
- Feb 2004
- Primary completion
- Jul 2009 (actual)
- Study completion
- Jul 2009 (actual)
- First posted
- 11 Feb 2010 (estimated)
- Results
- Posted 14 Dec 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Group A (A1) | Experimental | Drug: Recombinant human growth hormone (r-hGH)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. |
| Group A (A2) | No intervention | –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. |
| Group B | No intervention | –Participants were allocated to Group B being third percentile (thus showing a spontaneous catch-up growth). |
Who may qualify
- Ages
- 4 to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (10 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Recombinant human growth hormone (r-hGH)Also: SaizenRecombinant human GH were administered subcutaneously (s.c) at the daily dose of 0.067 mg/kg of body weight to Group A1. | Drug | Group A (A1) |
Primary outcome
Correlation Between Gene Expression Profiling and Catch-up Growth in Small for Gestational Age (SGA) Children
Time frame: Baseline and Week 48
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.
2 secondary outcomes
Percentage of Untreated Participants Who Showed a Spontaneous Catch-up Growth
Time frame: Baseline through Week 96
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Study Drug Discontinuation
Time frame: Baseline through Week 96
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Dec 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Responsible study director
Merck Serono S.P.A., Italy
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Recombinant human growth hormone (r-hGH) in people with Infant, Small for Gestational Age, run by Merck KGaA, Darmstadt, Germany at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 27 Dec 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Dec 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the 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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Dec 2013.