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This is an open-label, multi-center, randomized, two-arm parallel, no-treatment group controlled (only for the first 6 months), Phase 3 study in children with ISS. The subjects will be treated with 0.067 milligram/kilogram/day (mg/kg/day) of Saizen®, weight base dose, for 12 months (12 months of treatment in the test group, and 6 months of no treatment and then 6 months of treatment in the control group).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Saizen Test Group | Experimental |
| |
| Saizen Control Group | Active Comparator |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Saizen® | Drug | Subjects in the Saizen test group will receive Saizen (recombinant-human growth hormone [r-hGH]) subcutaneously 6 days per week at a weight based dose of 0.067 milligram per kilogram of body weight per day (mg/kg/day) for 12 months. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Height Velocity at Month 6 Using Last Observation Carried Forward (LOCF) Method | Baseline height is defined as the last available height measurement before randomization. Baseline height velocity = ([Baseline height minus height measurement obtained at least 6 months prior] / 6) * 12. Height velocity at Month 6 = ([Month 6 height minus height measurement obtained at least 6 months prior] / 6) * 12. | Baseline, Month 6 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Height Velocity at Month 12 | Baseline height is defined as the last available height measurement before randomization. Baseline height velocity = ([Baseline height minus height measurement obtained at least 12 months prior] / 12) * 12. Height velocity at Month 12 = ([Month 12 height minus height measurement obtained at least 12 months prior] / 12) * 12. | Baseline, Month 12 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Responsible | Merck Ltd. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Please contact Merck KGaA Communication Center for Recruiting Sites | Located in | South Korea |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 30110706 | Derived | Chung WY, Yoo HW, Hwang JS, Ko CW, Kim HS, Jin DK, Lee KH, Han HS, Paranchothy P, Suh BK. Effect of Growth Hormone Therapy on Height Velocity in Korean Children with Idiopathic Short Stature: A Phase III Randomised Controlled Trial. Horm Res Paediatr. 2018;90(1):44-53. doi: 10.1159/000491016. Epub 2018 Aug 15. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Saizen Test Group | Subjects in the Saizen test group received Saizen (recombinant-human growth hormone [r-hGH]) subcutaneously 6 days per week at a weight based dose of 0.067 milligram per kilogram of body weight per day (mg/kg/day) for 12 months. |
| FG001 | Saizen Control Group | Subjects in the Saizen control group received no treatment for the first 6 months and thereafter received Saizen (r-hGH) subcutaneously 6 days per week at a weight based dose of 0.067 mg/kg/day for the next 6 months. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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|
Safety analysis set (SAS) included all randomized subjects who received at least 1 dose of planned trial treatment and had follow-up safety data. SAS also included the safety data for all subjects during non-treatment (the first 6 months after enrollment) period who belonged to control group.
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| ID | Title | Description |
|---|---|---|
| BG000 | Saizen Test Group | Subjects in the Saizen test group received Saizen (recombinant-human growth hormone [r-hGH]) subcutaneously 6 days per week at a weight based dose of 0.067 milligram per kilogram of body weight per day (mg/kg/day) for 12 months. |
| BG001 | Saizen Control Group |
| 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 | Change From Baseline in Height Velocity at Month 6 Using Last Observation Carried Forward (LOCF) Method | Baseline height is defined as the last available height measurement before randomization. Baseline height velocity = ([Baseline height minus height measurement obtained at least 6 months prior] / 6) * 12. Height velocity at Month 6 = ([Month 6 height minus height measurement obtained at least 6 months prior] / 6) * 12. | Intent-to-treat (ITT) analysis set included all randomized subjects, regardless of whether or not they received the treatment to which they were randomized, completed the study or had any protocol deviations. Here "n" signifies those subjects who were evaluable for this outcome measure at the specified time points. | Posted | Mean | Standard Deviation | centimeter/year (cm/yr) | Baseline, Month 6 |
|
Baseline up to Month 13
Safety analysis set (SAS) included all randomized subjects who received at least 1 dose of planned trial treatment and had follow-up safety data. SAS also included the safety data for all subjects during non-treatment (the first 6 months after enrollment) period who belonged to control group.
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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 Test Group | Subjects in the Saizen test group received Saizen (recombinant-human growth hormone [r-hGH]) subcutaneously 6 days per week at a weight based dose of 0.067 milligram per kilogram of body weight per day (mg/kg/day) for 12 months. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyrexia | General disorders | MedDRA 18.0 | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Motion sickness | Ear and labyrinth disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Merck KGaA Communication Center | Merck Healthcare, a business of Merck KGaA, Darmstadt, Germany | +49-6151-72-5200 | service@merckgroup.com |
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| 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 | Subjects in the Saizen control group will receive no treatment for the first 6 months and thereafter will receive Saizen (r-hGH) subcutaneously 6 days per week at a weight based dose of 0.067 mg/kg/day for the next 6 months. |
|
|
| Change From Baseline in Height at Month 6 and 12 | Baseline, Month 6, Month 12 |
| Change From Baseline in Height Standard Deviation Score (SDS) at Month 6 and 12 | Height SDS was calculated as: Height SDS = (measured height - population mean) / population standard deviation, where mean and standard deviation were based on the Korean standard growth charts. SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) a subject's value was relative to the mean of the reference population. The scores were centred around zero. Negative score indicated a subject was smaller for their age/gender. | Baseline, Month 6, Month 12 |
| Change From Baseline in Serum Concentration of Insulin-like Growth Factor-I (IGF-I) at Month 6 and 12 | Baseline, Month 6, Month 12 |
| Change From Baseline in Serum Concentration of Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) at Month 6 and 12 | Baseline, Month 6, Month 12 |
| Percentage of Adherence to Study Treatment | Percentage of adherence to study treatment (adherence rate) was defined as the actual number of received treatments divided by the scheduled number of treatments multiplied by 100. The adherence rate for 6 months was calculated from Baseline to 6 months for the Saizen Test Group and from Month 6 to Month 12 for the Saizen Control Group. | 6 months post-dose (Saizen Test Group and Saizen Control Group); 12 months post-dose (Saizen Test Group) |
| Number of Subjects With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs | An adverse event (AE) was defined as any untoward medical occurrence which does not necessarily have a causal relationship with this the study drug. An AE was defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of study drug, whether or not considered related to the study drug. A serious adverse event (SAE) was an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. For the Saizen Test Group, TEAEs were defined as events that occurred or worsened at or after the first administration of treatment and for the Saizen Control Group, TEAEs were defined as events that occurred or worsened at or after the randomization. | Baseline up to Month 13 |
| Consent withdrawal by parent's guardian |
|
| Entered puberty during the study |
|
Subjects in the Saizen control group received no treatment for the first 6 months and thereafter received Saizen (r-hGH) subcutaneously 6 days per week at a weight based dose of 0.067 mg/kg/day for the next 6 months. |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| OG001 | Saizen Control Group | Subjects in the Saizen control group received no treatment for the first 6 months and thereafter received Saizen (r-hGH) subcutaneously 6 days per week at a weight based dose of 0.067 mg/kg/day for the next 6 months. |
|
|
|
| Secondary | Change From Baseline in Height Velocity at Month 12 | Baseline height is defined as the last available height measurement before randomization. Baseline height velocity = ([Baseline height minus height measurement obtained at least 12 months prior] / 12) * 12. Height velocity at Month 12 = ([Month 12 height minus height measurement obtained at least 12 months prior] / 12) * 12. | ITT analysis set included all randomized subjects, regardless of whether or not they received the treatment to which they were randomized, completed the study or had any protocol deviations. Here "Number of participants analyzed" signifies those subjects who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | cm/year | Baseline, Month 12 |
|
|
|
| Secondary | Change From Baseline in Height at Month 6 and 12 | ITT analysis set included all randomized subjects, regardless of whether or not they received the treatment to which they were randomized, completed the study or had any protocol deviations. Here "n" signifies those subjects who were evaluable for this outcome measure at the specified time points. | Posted | Mean | Standard Deviation | centimeter (cm) | Baseline, Month 6, Month 12 |
|
|
|
| Secondary | Change From Baseline in Height Standard Deviation Score (SDS) at Month 6 and 12 | Height SDS was calculated as: Height SDS = (measured height - population mean) / population standard deviation, where mean and standard deviation were based on the Korean standard growth charts. SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) a subject's value was relative to the mean of the reference population. The scores were centred around zero. Negative score indicated a subject was smaller for their age/gender. | ITT analysis set included all randomized subjects, regardless of whether or not they received the treatment to which they were randomized, completed the study or had any protocol deviations. Here "n" signifies those subjects who were evaluable for this outcome measure at the specified time points. | Posted | Mean | Standard Deviation | standard deviation score | Baseline, Month 6, Month 12 |
|
|
|
| Secondary | Change From Baseline in Serum Concentration of Insulin-like Growth Factor-I (IGF-I) at Month 6 and 12 | ITT analysis set included all randomized subjects, regardless of whether or not they received the treatment to which they were randomized, completed the study or had any protocol deviations. Here "n" signifies those subjects who were evaluable for this outcome measure at the specified time points. | Posted | Mean | Standard Deviation | microgram/liter (mcg/L) | Baseline, Month 6, Month 12 |
|
|
|
| Secondary | Change From Baseline in Serum Concentration of Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) at Month 6 and 12 | ITT analysis set included all randomized subjects, regardless of whether or not they received the treatment to which they were randomized, completed the study or had any protocol deviations. Here "n" signifies those subjects who were evaluable for this outcome measure at the specified time points. | Posted | Mean | Standard Deviation | mcg/L | Baseline, Month 6, Month 12 |
|
|
|
| Secondary | Percentage of Adherence to Study Treatment | Percentage of adherence to study treatment (adherence rate) was defined as the actual number of received treatments divided by the scheduled number of treatments multiplied by 100. The adherence rate for 6 months was calculated from Baseline to 6 months for the Saizen Test Group and from Month 6 to Month 12 for the Saizen Control Group. | ITT analysis set included all randomized subjects, regardless of whether or not they received the treatment to which they were randomized, completed the study or had any protocol deviations. Here "n" signifies those subjects who were evaluable for this outcome measure at the specified time points. | Posted | Mean | Standard Deviation | percentage of adherance | 6 months post-dose (Saizen Test Group and Saizen Control Group); 12 months post-dose (Saizen Test Group) |
|
|
|
| Secondary | Number of Subjects With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs | An adverse event (AE) was defined as any untoward medical occurrence which does not necessarily have a causal relationship with this the study drug. An AE was defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of study drug, whether or not considered related to the study drug. A serious adverse event (SAE) was an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. For the Saizen Test Group, TEAEs were defined as events that occurred or worsened at or after the first administration of treatment and for the Saizen Control Group, TEAEs were defined as events that occurred or worsened at or after the randomization. | Safety analysis set (SAS) included all randomized subjects who received at least 1 dose of planned trial treatment and had follow-up safety data. SAS also included the safety data for all subjects during non-treatment (the first 6 months after enrollment) period who belonged to control group. | Posted | Number | subjects | Baseline up to Month 13 |
|
|
|
| 3 |
| 59 |
| 42 |
| 59 |
| EG001 | Saizen Control Group | Subjects in the Saizen control group received no treatment for the first 6 months and thereafter received Saizen (r-hGH) subcutaneously 6 days per week at a weight based dose of 0.067 mg/kg/day for the next 6 months. | 1 | 30 | 18 | 30 |
| Chronic sinusitis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Chronic tonsillitis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Croup infectious | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Adenoidal hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Tonsillar hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| T-cell lymphoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 18.0 | Non-systematic Assessment |
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| Hypothyroidism | Endocrine disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Pectus carinatum | Congenital, familial and genetic disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Chalazion | Eye disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Conjunctivitis allergic | Eye disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Eye inflammation | Eye disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Pyrexia | General disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Enteritis | Gastrointestinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Colitis | Gastrointestinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Oral mucosal eruption | Gastrointestinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Rhinitis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Bronchitis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Conjunctivitis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Gastroenteritis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Otitis media | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Influenza | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Pharyngitis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Acute tonsillitis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Hand-foot-and-mouth disease | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Hordeolum | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Impetigo | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Laryngitis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Lower respiratory tract infection | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Molluscum contagiosum | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Otitis media acute | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Perineal infection | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Pulpitis dental | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Subcutaneous abscess | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Tinea infection | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Tonsillitis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Tooth abscess | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Tracheitis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Varicella | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Contusion | Injury, poisoning and procedural complications | MedDRA 18.0 | Non-systematic Assessment |
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| Joint injury | Injury, poisoning and procedural complications | MedDRA 18.0 | Non-systematic Assessment |
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| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 18.0 | Non-systematic Assessment |
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| Radius fracture | Injury, poisoning and procedural complications | MedDRA 18.0 | Non-systematic Assessment |
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| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 18.0 | Non-systematic Assessment |
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| Blood thyroid stimulating hormone increased | Investigations | MedDRA 18.0 | Non-systematic Assessment |
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| Haemoglobin decreased | Investigations | MedDRA 18.0 | Non-systematic Assessment |
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| Dehydration | Metabolism and nutrition disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Scoliosis | Musculoskeletal and connective tissue disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Breast mass | Reproductive system and breast disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Skin papilloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 18.0 | Non-systematic Assessment |
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| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Rash generalised | Skin and subcutaneous tissue disorders | MedDRA 18.0 | 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 (n=52, 27) |
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| Change at Month 12 (n=52, 27) |
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| Change at Month 12 (n=52, 27) |
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| Change at Month 12 (n=52, 27) |
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