Small Gestational Age (SGA)Phase 3SGA OPTIMISNCT00249821

Optimization of the Dosage Regimen of Growth Hormone Therapy in Children Born Small for Gestational Age

A Phase 3 study testing Saizen® in people with Small Gestational Age (SGA), run by Merck KGaA, Darmstadt, Germany.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

Multicentric, open-label, randomized, pilot comparative study in parallel groups comparing 1 group of subjects receiving 0.057 milligram/kilogram/day (mg/kg/day) or 0.40 mg/kg/week of Saizen® during 1 year to 1 group receiving 0.035 mg/kg/day (0.24 mg/kg/week) of Saizen® during 1 year after an initial 3-year treatment… Read more about this study

Who is it for?: Ages 6 to 11 years
Conditions the study is for: Small Gestational Age (SGA)
What is being tested?: Saizen®
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 22 participants
Where: No sites listed yet
Study dates: 28 Feb 2005to 30 Sep 2007
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 6 to 11 years
All sexes
No healthy volunteers
Child

Who may be able to join

To be eligible for inclusion / randomization into this study, the subjects must fulfill all of the following criteria (if there is no inclusion phase, the inclusion criteria will be considered as inclusion criteria for randomization):
Written consent form signed by the parents / legal guardian, and child if possible
Subject born SGA and receiving a r-hGH therapy for this pathology
Recombinant human growth hormone (r-hGH) started at the maximal chronological age of 7 years for girls and 8 years for boys
Treatment with r-hGH started for at least 30 months and less than 36 months at 0.057 mg/kg/day
Height gain during the first 2 years of GH treatment > 1 SD compared with the initial value

Who cannot join

To be eligible for inclusion in this study the subjects must not meet any of the following criteria:
Known hypersensitivity to Somatropin or any of the excipients
Active neoplasia (either newly diagnosed or recurrent)
Intracranial hypertension
Known diabetes mellitus
Proliferative or preproliferative diabetic retinopathy

Show all 13Show fewer

Evidence of any progression or recurrence of an underlying intra-cranial space occupying lesion
Obesity defined as degree 1 on the corpulence curves
Precocious puberty
Pubertal status: Tanner breast development stage > 2 for girls, and testicular volume > 4 milliliter (mL) or testicular length > 3 centimeter (cm) and/or testosterone value >1 nanomole/liter [nmol/L] (0.29 gram/mL [g/mL]) for boys For girls > 9 years and Tanner breast development stage 1: uterine size > 35 millimeter (mm)
Severe chronic concomitant illness such as chronic renal failure, cystic fibrosis
Concomitant corticoid treatment or levothyroxine treatment other than substitutive treatment, topical or inhaled treatment
Participation to any clinical study within the 30 days preceding study entry

About this study

Official title: Optimization of the Dosage Regimen With Growth Hormone Therapy in Children Born Small for Gestational Age. An Open Label, Randomized, Pilot Study, Comparing in Children Treated for 3 Years, the Efficacy of a Saizen® Treatment at the Same Dose Versus a Lower Maintenance Dose Prolonged During 1 Additional Year.

Conditions: Small Gestational Age (SGA)
Being tested: Saizen®

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Saizen®Drug
Also called somatropin
Saizen® [somatropin (recombinant deoxyribonucleic acid [rDNA] origin) for injection], a r-hGH, is indicated for the treatment of children with growth failure due to inadequate secretion of endogenous growth hormone.
Saizen®Drug
Also called somatropin
Saizen® [somatropin (rDNA origin) for injection], a r-hGH, is indicated for the treatment of children with growth failure due to inadequate secretion of endogenous growth hormone.

Study groups in NCT00249821
Study group	Role	What they receive
Saizen® 0.057 mg/kg/day	Experimental	Subjects who met all inclusion/exclusion criteria were randomly assigned to 0.057 mg/kg/day in this multi-center study. Subjects were stratified at randomization according to the Height-Standard Deviation Score (H-SDS) at this time (less than [<] -2 SDS or greater than [>] -2 SDS) Receives: Drug: Saizen®
Saizen® 0.035 mg/kg/day	Experimental	Subjects who met all inclusion/exclusion criteria were randomly assigned to 0.035 mg/kg/day in this multi-center study. Subjects were stratified at randomization according to the H-SDS at this time (< -2 SDS or > -2 SDS) Receives: Drug: Saizen®

What is being measured

The primary outcome is the main question the study is designed to answer.

Height Velocity
Height Velocity (HV) is the change in height since the previous year´s measurement and more precisely: HV = {(h-hp)/(d-dp)} * 365.25 [centimeter (cm)/year] where h is current height in cm, hp is previous height in cm, closest to 1 year previous, d is the current date and dp is the date of measurement of previous height, closest to 1 year previous. Additionally, d and dp have to be within 0.6 years and 1.5 years. HV is the mean height velocity over the interval between d and dp but is displayed as HV at d.
Measured: Month 12

Secondary outcomes (7)

Change From Baseline in Height-Standard Deviation Score (H-SDS) at Month 6 and Month 12
Height-Standard Deviation Score (H-SDS) was calculated as height minus mean (age-and sex-matched reference) divided by standard deviation (SD) [age and sex-matched reference]. Greater H-SDS indicates greater height.
Measured: Baseline (randomization), Month 6 and Month 12
Height Velocity-Standard Deviation Score (HV-SDS)
Height Velocity-Standard Deviation Score (HV-SDS) was calculated as height velocity minus reference mean height velocity divided by SD of the reference mean height velocity. Greater HV-SDS indicates greater height velocity.
Measured: Month 6 and Month 12
Change From Baseline in Height at Month 6
Measured: Baseline (randomization) and Month 6
Change From Baseline in Bone Age at Month 12
Bone age was assessed by a left wrist X-Ray and evaluated by the investigator according to the Greulich and Pyle method.
Measured: Baseline (randomization) and Month 12
Insulin Like Growth Factor-1 (IGF-1) Levels
Measured: Baseline (randomization), Month 6 and Month 12
Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) Levels
Measured: Baseline (randomization), Month 6 and Month 12
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Adverse Events (AEs): Any untoward medical occurrence in the form of signs, clinically significant abnormalities in laboratory findings, diseases, symptoms, or worsening of complications. TEAEs: AEs that occur during treatment with the Investigational Medicinal Product (IMP).
Measured: Baseline (randomization) until Month 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

28 Feb 200530 Sep 2007

First posted7 Nov 2005 (estimated)
Study start28 Feb 2005
Primary completion30 Sep 2007
Study completion30 Sep 2007

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.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Small Gestational Age (SGA)Studies 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: 22 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00249821
Sponsor's protocol ID: IMP 25735
Acronym: SGA OPTIMIS
Status last verified: Aug 2017
First submitted: 4 Nov 2005
Last updated: 26 Sep 2017
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Chatelain P, Colle M, Nako JP, Le Luyer B, Wagner K, Berlier P, Tauber M. Optimization of growth hormone dosing in children born small for gestational age: an open-label, randomized study of children during the fourth year of therapy. Horm Res Paediatr. 2012;77(3):156-63. doi: 10.1159/000337216. Epub 2012 Apr 12.
PubMed 22508151

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

Official record

Enrolling by invitationObservational study
Influence of Adherence to Growth Hormone Therapy (GHT) With Norditropin® on Near Final Height in Patients With Growth Hormone Deficiency (GHD) and Born Small for Gestational Age (SGA)
Growth Hormone Deficiency in Children, Born Small for Gestational Age
Active, not recruitingPhase 2
A Research Study in Children Born Small and Who Stayed Small. Treatment is Somapacitan Once a Week Compared to Norditropin® Once a Day
Short Stature Children Born Small for Gestational Age (SGA)
Not yet recruitingPhase 3
A Phase III Clinical Study of Jinpei Growth Hormone Injection in the Treatment of Short Stature Children With Small for Gestational Age (SGA)
Small for Gestational Age
Active, not recruitingPhase 3
A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9
SGA, Turner Syndrome
Active, not recruitingPhase 3
A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow
SGA, Turner Syndrome, Noonan Syndrome, ISS

All Small Gestational Age (SGA) trials

Study at a glance

From the registry record, last updated by the sponsor on 26 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Optimization of the Dosage Regimen With Growth Hormone Therapy in Children Born Small for Gestational Age. An Open Label, Randomized, Pilot Study, Comparing in Children Treated for 3 Years, the Efficacy of a Saizen® Treatment at the Same Dose Versus a Lower Maintenance Dose Prolonged During 1 Additional Year.
ClinicalTrials.gov ID: NCT00249821
Acronym / sponsor ID: SGA OPTIMIS / IMP 25735
Conditions: Small Gestational Age (SGA)
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 22 participants (actual)
Lead sponsor: Merck KGaA, Darmstadt, GermanyIndustry (pharma, biotech, devices)
Collaborators: Merck Serono S.A.S., an affiliate of Merck KGaA, Darmstadt, Germany
Study start: 28 Feb 2005 (actual)
Primary completion: 30 Sep 2007 (actual)
Study completion: 30 Sep 2007 (actual)
First posted: 7 Nov 2005 (estimated)
Results: Posted 10 Oct 2012

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Saizen® 0.057 mg/kg/day	Experimental	Drug: Saizen®Subjects who met all inclusion/exclusion criteria were randomly assigned to 0.057 mg/kg/day in this multi-center study. Subjects were stratified at randomization according to the Height-Standard Deviation Score (H-SDS) at this time (less than [<] -2 SDS or greater than [>] -2 SDS)
Saizen® 0.035 mg/kg/day	Experimental	Drug: Saizen®Subjects who met all inclusion/exclusion criteria were randomly assigned to 0.035 mg/kg/day in this multi-center study. Subjects were stratified at randomization according to the H-SDS at this time (< -2 SDS or > -2 SDS)

Who may qualify

Ages: 6 to 11 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (6 listed)

To be eligible for inclusion / randomization into this study, the subjects must fulfill all of the following criteria (if there is no inclusion phase, the inclusion criteria will be considered as inclusion criteria for randomization):
Written consent form signed by the parents / legal guardian, and child if possible
Subject born SGA and receiving a r-hGH therapy for this pathology
Recombinant human growth hormone (r-hGH) started at the maximal chronological age of 7 years for girls and 8 years for boys
Treatment with r-hGH started for at least 30 months and less than 36 months at 0.057 mg/kg/day

Key exclusion criteria (13 listed)

To be eligible for inclusion in this study the subjects must not meet any of the following criteria:
Known hypersensitivity to Somatropin or any of the excipients
Active neoplasia (either newly diagnosed or recurrent)
Intracranial hypertension
Known diabetes mellitus

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
Saizen®Also: somatropinSaizen® [somatropin (recombinant deoxyribonucleic acid [rDNA] origin) for injection], a r-hGH, is indicated for the treatment of children with growth failure due to inadequate secretion of endogenous growth hormone.	Drug	Saizen® 0.057 mg/kg/day
Saizen®Also: somatropinSaizen® [somatropin (rDNA origin) for injection], a r-hGH, is indicated for the treatment of children with growth failure due to inadequate secretion of endogenous growth hormone.	Drug	Saizen® 0.035 mg/kg/day

Primary outcome

Height Velocity
Time frame: Month 12
Height Velocity (HV) is the change in height since the previous year´s measurement and more precisely: HV = {(h-hp)/(d-dp)} * 365.25 [centimeter (cm)/year] where h is current height in cm, hp is previous height in cm, closest to 1 year previous, d is the current date and dp is the date of measurement of previous height, closest to 1 year previous. Additionally, d and dp have to be within 0.6 years and 1.5 years. HV is the mean height velocity over the interval between d and dp but is displayed as HV at d.

7 secondary outcomes

Change From Baseline in Height-Standard Deviation Score (H-SDS) at Month 6 and Month 12
Time frame: Baseline (randomization), Month 6 and Month 12
Height Velocity-Standard Deviation Score (HV-SDS)
Time frame: Month 6 and Month 12
Change From Baseline in Height at Month 6
Time frame: Baseline (randomization) and Month 6
Change From Baseline in Bone Age at Month 12
Time frame: Baseline (randomization) and Month 12
Insulin Like Growth Factor-1 (IGF-1) Levels
Time frame: Baseline (randomization), Month 6 and Month 12
Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) Levels
Time frame: Baseline (randomization), Month 6 and Month 12
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Time frame: Baseline (randomization) until Month 12

Results and safety

Results posted to the registry on 10 Oct 2012, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Saizen® 0.057 mg/kg/Day	10	10
Saizen® 0.035 mg/kg/Day	12	11

Primary outcome: Height Velocity

Time frame: Month 12. Mean (Standard Deviation). Unit: centimeter (cm)/year

Measure	Saizen® 0.057 mg/kg/Day n = 10	Saizen® 0.035 mg/kg/Day n = 12
Value	6.40 (1.35)	4.40 (1.15)

Saizen® 0.057 mg/kg/Day vs Saizen® 0.035 mg/kg/Day: p = 0.001, ANCOVA

Population analysed: Full Analysis (FA) set included all the participants who received at least 1 dose of study medication and had at least 1 height evaluation post randomization. Last observation carried forward (LOCF) was used to impute missing values.

7 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse Events (AEs) are collected on an ongoing basis from day of written informed consent. All new AEs must be recorded until 4 weeks post drug administration. AEs are classified as pre-treatment, treatment-emergent and post-treatment.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Saizen® 0.057 mg/kg/Day	0 of 10 (0%)	2 of 10 (20%)	Not reported
Saizen® 0.035 mg/kg/Day	0 of 12 (0%)	1 of 12 (8.3%)	Not reported

Limitations and caveats (from the sponsor): Participants' inclusion period was extended but due to remaining low recruitment rate, it was finally decided to stop enrollment even if only 22 participants were enrolled.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 26 Sep 2017.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 22508151Chatelain P, Colle M, Nako JP, Le Luyer B, Wagner K, Berlier P, Tauber M. Optimization of growth hormone dosing in children born small for gestational age: an open-label, randomized study of children during the fourth year of therapy. Horm Res Paediatr. 2012;77(3):156-63. doi: 10.1159/000337216. Epub 2012 Apr 12.
Related InfoLink provided by the sponsor.

Related trials for Small Gestational Age (SGA)

Enrolling by invitationObservational study
Influence of Adherence to Growth Hormone Therapy (GHT) With Norditropin® on Near Final Height in Patients With Growth Hormone Deficiency (GHD) and Born Small for Gestational Age (SGA)
Growth Hormone Deficiency in Children, Born Small for Gestational Age
Active, not recruitingPhase 2
A Research Study in Children Born Small and Who Stayed Small. Treatment is Somapacitan Once a Week Compared to Norditropin® Once a Day
Short Stature Children Born Small for Gestational Age (SGA)
Not yet recruitingPhase 3
A Phase III Clinical Study of Jinpei Growth Hormone Injection in the Treatment of Short Stature Children With Small for Gestational Age (SGA)
Small for Gestational Age
Active, not recruitingPhase 3
A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9
SGA, Turner Syndrome
Active, not recruitingPhase 3
A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow
SGA, Turner Syndrome, Noonan Syndrome, ISS

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.A.S, France

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Saizen® in people with Small Gestational Age (SGA), run by Merck KGaA, Darmstadt, Germany.

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 6 inclusion and 13 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: finished, as of the sponsor's last update on 26 Sep 2017. Status can change between updates, so confirm with the study contact before making plans.

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 10 Oct 2012). 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?

Height Velocity (time frame: Month 12). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00249821, which also lists 1 linked publication.

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 26 Sep 2017.