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| Name | Class |
|---|---|
| Merck Serono S.A.S., an affiliate of Merck KGaA, Darmstadt, Germany | UNKNOWN |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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) |
|
| 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) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Saizen® | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Month 12 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline (randomization), Month 6 and Month 12 |
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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):
Exclusion Criteria:
To be eligible for inclusion in this study the subjects must not meet any of the following criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Responsible | Merck Serono S.A.S, France | Study Director |
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| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 22508151 | Result | 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. |
| Label | URL |
|---|---|
| Related Info | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | Saizen® 0.057 mg/kg/Day | Saizen® (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. |
| FG001 | Saizen® 0.035 mg/kg/Day | Saizen® (r-hGH) subcutaneously administered at the daily dose of 0.035 mg/kg or 0.24 mg/kg/week for duration of 12 months. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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| ID | Title | Description |
|---|---|---|
| BG000 | Saizen® 0.057 mg/kg/Day | Saizen® (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. |
| BG001 | Saizen® 0.035 mg/kg/Day |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | 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. | Posted | Mean | Standard Deviation | centimeter (cm)/year | Month 12 |
|
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.
Pre-Treatment:Medical conditions present at initial study visit that did not worsen in severity or frequency during study;Treatment-Emergent: If onset date of AE was on or after the first dose date of the study medication; Post-Treatment: If the onset date of AE was post 4 weeks after drug administration for participants who completed the study.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Saizen® 0.057 mg/kg/Day | Saizen® (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. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Asthma | Immune system disorders | MedDRA (11.0) | Non-systematic Assessment |
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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Merck KGaA Communication Center | Merck Serono, a division of Merck KGaA | +49-6151-72-5200 | service@merckgroup.com |
| ID | Term |
|---|---|
| D019382 | Human Growth Hormone |
| ID | Term |
|---|---|
| D013006 | Growth Hormone |
| D010908 | Pituitary Hormones, Anterior |
| D010907 | Pituitary Hormones |
| D036361 | Peptide Hormones |
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|
| Saizen® | Drug | 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. |
|
|
| 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. | Month 6 and Month 12 |
| Change From Baseline in Height at Month 6 | 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. | Baseline (randomization) and Month 12 |
| Insulin Like Growth Factor-1 (IGF-1) Levels | Baseline (randomization), Month 6 and Month 12 |
| Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) Levels | 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). | Baseline (randomization) until Month 12 |
Saizen® (r-hGH) subcutaneously administered at the daily dose of 0.035 mg/kg or 0.24 mg/kg/week for duration of 12 months. |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| OG001 | Saizen® 0.035 mg/kg/Day | Saizen® (r-hGH) subcutaneously administered at the daily dose of 0.035 mg/kg or 0.24 mg/kg/week for duration of 12 months. |
|
|
|
| Secondary | 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. | FA set included all the participants who received at least 1 dose of study medication and had at least 1 height evaluation post randomization. LOCF was used to impute missing values. | Posted | Mean | Standard Deviation | standard deviation score | Baseline (randomization), Month 6 and Month 12 |
|
|
|
|
| Secondary | 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. | FA set included all the participants who received at least 1 dose of study medication and had at least 1 height evaluation post randomization. Here "n" signifies number of participants analyzed at that particular time point for each arm group respectively. LOCF was used to impute missing values. | Posted | Mean | Standard Deviation | standard deviation score | Month 6 and Month 12 |
|
|
|
|
| Secondary | Change From Baseline in Height at Month 6 | FA set included all the participants who received at least 1 dose of study medication and had at least 1 height evaluation post randomization. LOCF was used to impute missing values. | Posted | Mean | Standard Deviation | cm | Baseline (randomization) and Month 6 |
|
|
|
|
| Secondary | 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. | FA set included all participants who received at least 1 dose of study medication and had at least 1 height evaluation post randomization. 'N' (Number of participants analyzed) signified those participants who were evaluable for this measure and "n" = number of participants analyzed at that particular time point for each arm group respectively. | Posted | Mean | Standard Deviation | years | Baseline (randomization) and Month 12 |
|
|
|
|
| Secondary | Insulin Like Growth Factor-1 (IGF-1) Levels | FA set included all participants who received at least 1 dose of study medication and had at least 1 height evaluation post randomization. 'N' (Number of participants analyzed) signified those participants who were evaluable for this measure and "n" = number of participants analyzed at that particular time point for each arm group respectively. | Posted | Mean | Standard Deviation | microgram/liter (mcg/L) | Baseline (randomization), Month 6 and Month 12 |
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|
|
|
| Secondary | Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) Levels | FA set included all participants who received at least 1 dose of study medication and had at least 1 height evaluation post randomization. 'N' (Number of participants analyzed) signified those participants who were evaluable for this measure and "n" = number of participants analyzed at that particular time point for each arm group respectively. | Posted | Mean | Standard Deviation | milligram/L (mg/L) | Baseline (randomization), Month 6 and Month 12 |
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| Secondary | 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). | The safety population included all the participants who received at least 1 dose of study medication. | Posted | Number | participants | Baseline (randomization) until Month 12 |
|
|
|
| 0 |
| 10 |
| 2 |
| 10 |
| EG001 | Saizen® 0.035 mg/kg/Day | Saizen® (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 |
| Bronchitis | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
|
| Ear infection | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
|
| Insulin-like growth factor increased | Investigations | MedDRA (11.0) | Non-systematic Assessment |
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| D006728 |
| Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| Change at Month 12 |
|
| ANCOVA |
| 0.002 |
| 95 |
| Superiority or Other |
| ANCOVA |
| 0.002 |
| 95 |
| Superiority or Other |
| Month 12 (n=10,10) |
|
| ANCOVA |
| 0.026 |
| 95 |
| Superiority or Other |
| Month 12 (n= 9,10) |
|
| ANCOVA |
| 0.055 |
| 95 |
| Superiority or Other |