Primary outcomeMean· Month 12
Height Velocity
| Group | Value (centimeter (cm)/year) | Spread |
|---|---|---|
| Saizen® 0.057 mg/kg/Day | 6.40 | 1.35 |
| Saizen® 0.035 mg/kg/Day | 4.40 | 1.15 |
Trial · Interventional
CompletedPhase 3Results postedRegistry dataOptimization of the Dosage Regimen of Growth Hormone Therapy in Children Born Small for Gestational Age
Short name SGA OPTIMIS
NCTNCT00249821 (opens ClinicalTrials.gov in a new tab)Other idIMP 25735
Overview
- Status
- Completed
- Phase
- Phase 3
- Enrollment
- 22 participants (actual)
- Sponsor
- Merck KGaA, Darmstadt, Germany · Industry
- Design
- Randomized · Open label (no masking)
- Countries
- Not stated
- Start
- 28 Feb 2005
- Primary completion
- 30 Sept 2007
- Completion
- 30 Sept 2007
- First posted
- 7 Nov 2005 (estimated)
- Last update posted
- 26 Sept 2017
- Conditions
- Small Gestational Age (SGA)
Timeline
1 trial · 1 completed · 1 with posted results
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
2005200620072008
- Ph 3
Registry summary
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
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 of recombinant human growth hormone (r-hGH) therapy with 0.057 mg/kg/day in both groups.
Interventions
- Saizen®Drug
- Saizen®Drug
Arms2
Experimental
Saizen® 0.057 mg/kg/day
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)
Experimental
Saizen® 0.035 mg/kg/day
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)
Primary outcomes1
Outcomes the sponsor listed as primary.
Height Velocity
Time frame · Month 12
Registry description
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.
Secondary outcomes(7)
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
Registry description
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.
Height Velocity-Standard Deviation Score (HV-SDS)
Time frame · Month 6 and Month 12
Registry description
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.
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
Registry description
Bone age was assessed by a left wrist X-Ray and evaluated by the investigator according to the Greulich and Pyle method.
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
Registry description
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).
Posted results
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
Primary outcome results
Secondary and other outcome results(7)
Secondary outcomeMean· Baseline (randomization), Month 6 and Month 12
Change From Baseline in Height-Standard Deviation Score (H-SDS) at Month 6 and Month 12
| Group | Value (standard deviation score) | Spread |
|---|---|---|
| Saizen® 0.057 mg/kg/Day | -1.30 | 0.42 |
| Saizen® 0.035 mg/kg/Day | -1.50 | 0.37 |
Secondary outcomeMean· Month 6 and Month 12
Height Velocity-Standard Deviation Score (HV-SDS)
| Group | Value (standard deviation score) | Spread |
|---|---|---|
| Saizen® 0.057 mg/kg/Day | 0.60 | 0.44 |
| Saizen® 0.035 mg/kg/Day | 0.10 | 0.22 |
Secondary outcomeMean· Baseline (randomization) and Month 6
Change From Baseline in Height at Month 6
| Group | Value (cm) | Spread |
|---|---|---|
| Saizen® 0.057 mg/kg/Day | 121.10 | 7.99 |
| Saizen® 0.035 mg/kg/Day | 119.20 | 5.29 |
Secondary outcomeMean· Baseline (randomization) and Month 12
Change From Baseline in Bone Age at Month 12
| Group | Value (years) | Spread |
|---|---|---|
| Saizen® 0.057 mg/kg/Day | 7.50 | 0.82 |
| Saizen® 0.035 mg/kg/Day | 7.00 | 1.85 |
Secondary outcomeMean· Baseline (randomization), Month 6 and Month 12
Insulin Like Growth Factor-1 (IGF-1) Levels
| Group | Value (microgram/liter (mcg/L)) | Spread |
|---|---|---|
| Saizen® 0.057 mg/kg/Day | 319.60 | 146.27 |
| Saizen® 0.035 mg/kg/Day | 275.40 | 98.82 |
Secondary outcomeMean· Baseline (randomization), Month 6 and Month 12
Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) Levels
| Group | Value (milligram/L (mg/L)) | Spread |
|---|---|---|
| Saizen® 0.057 mg/kg/Day | 3.40 | 0.70 |
| Saizen® 0.035 mg/kg/Day | 3.40 | 0.60 |
Secondary outcomeNumber· Baseline (randomization) until Month 12
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
| Group | Value (participants) |
|---|---|
| Saizen® 0.057 mg/kg/Day | 2 |
| Saizen® 0.035 mg/kg/Day | 1 |
Adverse events, as posted
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| Saizen® 0.057 mg/kg/DaySaizen® (recombinant human growth hormone, r-hGH) subcutaneously administered at the daily dose of 0.057 milligram/kilogram (mg/kg) or 0.40 mg/kg/week for duration of 12 months. | – | 0 / 10 | 2 / 10 |
| Saizen® 0.035 mg/kg/DaySaizen® (r-hGH) subcutaneously administered at the daily dose of 0.035 mg/kg or 0.24 mg/kg/week for duration of 12 months. | – | 0 / 12 | 1 / 12 |
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.
Other registry references
References listed at the registry that are not in PepBank. They link to PubMed.
- ResultChatelain 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. PMID 22508151
Eligibility
Who could join, as posted.
- Sex
- All
- Age
- from 6 Years up to 11 Years
- Healthy volunteers
- Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria:
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
Exclusion Criteria:
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
* 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
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