A Phase 3 interventional study of Saizen® and Saizen® in Idiopathic Short Stature, sponsored by Merck KGaA, Darmstadt, Germany. Completed at 1 site in Korea, Republic of. Open to participants aged 5 Years and older. Per ClinicalTrials.gov, last updated 2016-10-18.
Sponsored by Merck KGaA, Darmstadt, Germany · Phase 3, Interventional, and Treatment
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).
125 studies on the registry are indexed under Dwarfism; 19 are open to participants now.
This study's enrollment of 90 is above the median of 75 across 74 interventional studies indexed under Dwarfism.
Browse Dwarfism studies →Merck KGaA, Darmstadt, Germany is the lead sponsor of 269 studies on the registry; none are open to participants now.
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Exclusion Criteria:
Drug: Saizen®
Drug: Saizen®
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.
Also known as: Somatropin, r-hGH
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.
Also known as: Somatropin, r-hGH
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.
Time frame: Baseline, Month 6
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.
Time frame: Baseline, Month 12
Change From Baseline in Height at Month 6 and 12
Time frame: 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.
Time frame: Baseline, Month 6, Month 12
Change From Baseline in Serum Concentration of Insulin-like Growth Factor-I (IGF-I) at Month 6 and 12
Time frame: 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
Time frame: 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.
Time frame: 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.
Time frame: Baseline up to Month 13
| Milestone | Saizen Test Group | Saizen Control Group |
|---|---|---|
| Started | 60 | 30 |
| Treated | 59 | 30 |
| Completed | 52 | 27 |
| Not completed | 8 | 3 |
| Withdrew: Withdrawal by subject | 1 | 1 |
| Withdrew: Protocol violation | 1 | 0 |
| Withdrew: Consent withdrawal by parent's guardian | 5 | 2 |
| Withdrew: Entered puberty during the study | 1 | 0 |
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.
| centimeter/year (cm/yr) | Saizen Test Group | Saizen Control Group |
|---|---|---|
| Baseline (n=59,30) | 5.63 ± 1.62 | 4.94 ± 1.91 |
| Change at Month 6 (n=59,29) | 4.45 ± 2.70 | 1.01 ± 3.15 |
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.
| cm/year | Saizen Test Group | Saizen Control Group |
|---|---|---|
| Change From Baseline in Height Velocity at Month 12 | 3.77 ± 2.21 | 2.86 ± 1.95 |
| centimeter (cm) | Saizen Test Group | Saizen Control Group |
|---|---|---|
| Baseline (n=60, 30) | 108.27 ± 7.67 | 108.07 ± 8.36 |
| Change at Month 6 (n=55, 27) | 5.41 ± 1.04 | 3.10 ± 0.84 |
| Change at Month 12 (n=52, 27) | 9.78 ± 1.35 | 8.24 ± 1.47 |
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.
| standard deviation score | Saizen Test Group | Saizen Control Group |
|---|---|---|
| Baseline (n=60,30) | -2.26 ± 0.36 | -2.34 ± 0.34 |
| Change at Month 6 (n=55, 27) | 0.59 ± 0.21 | 0.08 ± 0.19 |
| Change at Month 12 (n=52, 27) | 0.96 ± 0.27 | 0.62 ± 0.27 |
| microgram/liter (mcg/L) | Saizen Test Group | Saizen Control Group |
|---|---|---|
| Baseline (n=60,30) | 112.62 ± 43.06 | 125.01 ± 65.68 |
| Change at Month 6 (n=55, 27) | 122.57 ± 96.99 | 8.30 ± 41.57 |
| Change at Month 12 (n=52, 27) | 164.48 ± 95.69 | 142.17 ± 112.94 |
| mcg/L | Saizen Test Group | Saizen Control Group |
|---|---|---|
| Baseline (n=60, 30) | 3,783.33 ± 915.20 | 3,935.67 ± 866.42 |
| Change at Month 6 (n=55, 27) | 857.64 ± 761.28 | -38.89 ± 616.88 |
| Change at Month 12 (n=52, 27) | 932.31 ± 666.13 | 545.56 ± 599.52 |
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.
| percentage of adherance | Saizen Test Group | Saizen Control Group |
|---|---|---|
| Adherence for 6 months (n=60, 27) | 94.38 ± 14.12 | 95.69 ± 5.04 |
| Adherence for 12 months (n=60, 0) | 93.27 ± 14.67 | NA ± NA |
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.
| subjects | Saizen Test Group | Saizen Control Group |
|---|---|---|
| TEAEs | 42 | 18 |
| Serious TEAEs | 3 | 1 |
Collected over Baseline up to Month 13. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Saizen Test Group | — | 3/59 (5.1%) | 42/59 (71.2%) |
| Saizen Control Group | — | 1/30 (3.3%) | 18/30 (60%) |
| Event | Saizen Test Group | Saizen Control Group |
|---|---|---|
| Adenoidal hypertrophyRespiratory, thoracic and mediastinal disorders | 2/59 | 0/30 |
| PyrexiaGeneral disorders | 0/59 | 1/30 |
| Chronic sinusitisInfections and infestations | 0/59 | 1/30 |
| T-cell lymphomaNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 0/59 | 1/30 |
| Chronic tonsillitisInfections and infestations | 1/59 | 0/30 |
| Croup infectiousInfections and infestations | 1/59 | 0/30 |
| Tonsillar hypertrophyRespiratory, thoracic and mediastinal disorders | 1/59 | 0/30 |
| Event | Saizen Test Group | Saizen Control Group |
|---|---|---|
| NasopharyngitisInfections and infestations | 24/59 | 9/30 |
| Upper respiratory tract infectionInfections and infestations | 14/59 | 9/30 |
| PyrexiaGeneral disorders | 4/59 | 3/30 |
| BronchitisInfections and infestations | 2/59 | 3/30 |
| RhinitisInfections and infestations | 4/59 | 2/30 |
| ConjunctivitisInfections and infestations | 4/59 | 0/30 |
| GastroenteritisInfections and infestations | 4/59 | 0/30 |
| Otitis mediaInfections and infestations | 2/59 | 2/30 |
| Rhinitis allergicRespiratory, thoracic and mediastinal disorders | 3/59 | 2/30 |
| Abdominal painGastrointestinal disorders | 2/59 | 0/30 |
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.
| Age, Continuous(years) | Saizen Test Group | Saizen Control Group | Total |
|---|---|---|---|
| Mean | 6.79 ± 1.54 | 6.83 ± 1.61 | 6.80 ± 1.55 |
| Sex: Female, Male(Participants) | Saizen Test Group | Saizen Control Group | Total |
|---|---|---|---|
| Female | 29 | 13 | 42 |
| Male | 30 | 17 | 47 |
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Merck KGaA, Darmstadt, Germany