Primary outcomeMean· Baseline, Month 6
Change From Baseline in Height Velocity at Month 6 Using Last Observation Carried Forward (LOCF) Method
| Group | Value (centimeter/year (cm/yr)) | Spread |
|---|---|---|
| Saizen Test Group | 5.63 | 1.62 |
| Saizen Control Group | 4.94 | 1.91 |
Trial · Interventional
CompletedPhase 3Results postedRegistry dataA Phase 3 Study to Evaluate the Safety and Efficacy of Saizen® in Children With Idiopathic Short Stature (ISS)
NCTNCT01746862 (opens ClinicalTrials.gov in a new tab)Other idEMR200104-533
Overview
- Status
- Completed
- Phase
- Phase 3
- Enrollment
- 90 participants (actual)
- Sponsor
- Merck KGaA, Darmstadt, Germany · Industry
- Design
- Randomized · Open label (no masking)
- Countries
1 country
South Korea
- Start
- Dec 2012
- Primary completion
- Aug 2014
- Completion
- Mar 2015
- First posted
- 11 Dec 2012 (estimated)
- Last update posted
- 18 Oct 2016 (estimated)
- Conditions
- Idiopathic Short Stature
Timeline
1 trial · 1 completed · 1 with posted results
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
20122013201420152016
- Ph 3
A Phase 3 Study to Evaluate the Safety and Efficacy of Saizen® in Children With Idiopathic Short Stature (ISS)
NCT01746862Dec 2012 to Mar 2015
Registry summary
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
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).
Interventions
- Saizen®Drug
- Saizen®Drug
Arms2
Experimental
Saizen Test Group
Active comparator
Saizen Control Group
Primary outcomes1
Outcomes the sponsor listed as primary.
Change From Baseline in Height Velocity at Month 6 Using Last Observation Carried Forward (LOCF) Method
Time frame · Baseline, Month 6
Registry description
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.
Secondary outcomes(7)
Change From Baseline in Height Velocity at Month 12
Time frame · Baseline, Month 12
Registry description
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.
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
Time frame · Baseline, Month 6, Month 12
Registry description
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.
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
Time frame · 6 months post-dose (Saizen Test Group and Saizen Control Group); 12 months post-dose (Saizen Test Group)
Registry description
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.
Number of Subjects With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs
Time frame · Baseline up to Month 13
Registry description
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.
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, Month 12
Change From Baseline in Height Velocity at Month 12
| Group | Value (cm/year) | Spread |
|---|---|---|
| Saizen Test Group | 3.77 | 2.21 |
| Saizen Control Group | 2.86 | 1.95 |
Secondary outcomeMean· Baseline, Month 6, Month 12
Change From Baseline in Height at Month 6 and 12
| Group | Value (centimeter (cm)) | Spread |
|---|---|---|
| Saizen Test Group | 108.27 | 7.67 |
| Saizen Control Group | 108.07 | 8.36 |
Secondary outcomeMean· Baseline, Month 6, Month 12
Change From Baseline in Height Standard Deviation Score (SDS) at Month 6 and 12
| Group | Value (standard deviation score) | Spread |
|---|---|---|
| Saizen Test Group | -2.26 | 0.36 |
| Saizen Control Group | -2.34 | 0.34 |
Secondary outcomeMean· Baseline, Month 6, Month 12
Change From Baseline in Serum Concentration of Insulin-like Growth Factor-I (IGF-I) at Month 6 and 12
| Group | Value (microgram/liter (mcg/L)) | Spread |
|---|---|---|
| Saizen Test Group | 112.62 | 43.06 |
| Saizen Control Group | 125.01 | 65.68 |
Secondary outcomeMean· 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
| Group | Value (mcg/L) | Spread |
|---|---|---|
| Saizen Test Group | 3,783.33 | 915.20 |
| Saizen Control Group | 3,935.67 | 866.42 |
Secondary outcomeMean· 6 months post-dose (Saizen Test Group and Saizen Control Group); 12 months post-dose (Saizen Test Group)
Percentage of Adherence to Study Treatment
| Group | Value (percentage of adherance) | Spread |
|---|---|---|
| Saizen Test Group | 94.38 | 14.12 |
| Saizen Control Group | 95.69 | 5.04 |
Secondary outcomeNumber· Baseline up to Month 13
Number of Subjects With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs
| Group | Value (subjects) |
|---|---|
| Saizen Test Group | 42 |
| Saizen Control Group | 18 |
Adverse events, as posted
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| Saizen Test GroupSubjects 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. | – | 3 / 59 | 42 / 59 |
| Saizen Control GroupSubjects 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 |
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
Studies1
Studies that cite this trial.
More studies
1 study on this page
Indexing hint: Randomized controlled trial · 2018
Effect of Growth Hormone Therapy on Height Velocity in Korean Children with Idiopathic Short Stature: A Phase III Randomised Controlled Trial.
Chung et al. 2018Hormone research in paediatricscited by 11
PMID 30110706PMC6214610DOI 10.1159/000491016PUB-30110706
Free to read · publisher termsCC BY-NC-ND
Eligibility
Who could join, as posted.
- Sex
- All
- Age
- from 5 Years
- Healthy volunteers
- Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria:
* Age greater than or equal to 5 years
* Pre-pubertal; testicular volume less than 4 milliliter (in males) and breast Stage 1 (in females)
* The official records of height (for example records measured in hospitals or schools) during previous 6 months or more preceding inclusion in the study (self-measurement of the height at home will not be considered as a valid record)
* Height less than or equal to 3rd percentile compared to same sex, same age
* Peak serum growth hormone (GH) greater than 10 microgram per liter (mcg/L) in GH stimulation test (results of peak serum GH greater than 10 mcg/L in GH stimulation test within 1 year can be used instead)
* Naive to GH therapy
* Normal birth weight (that is greater than or equal to 3rd percentile when compared to same sex)
* Normal thyroid function
* Normal karyotype in girls
* Written informed consent from parent/guardian
* Written informed consent from the subject who speaks, understand, read, and write Korean
* Bone age less than 10 years in boys and less than 9 years in girls, whose difference between the bone and chronological age is no more than 3 years
Exclusion Criteria:
* Puberty development (Tanner stage greater than or equal to 2)
* Skeletal dysplasia or abnormal body proportions
* Chronic systemic illness
* Dysmorphic syndrome
* Growth Hormone Deficiency
* Small for Gestational Age (SGA)
* Current medication for Attention deficit hyperactivity disorder (ADHD) or hyperactivity disorder
* Current medication with drugs that may influence secretion or action of growth hormone (such as estrogen, androgen, anabolic steroid, corticosteroid, thyroxine, aromatase inhibitors)
* Diabetes mellitus
* Kidney transplantation
* Acute critical illness, including complications following open heart surgery, abdominal surgery or multiple accidental trauma
* Acute respiratory failure
* Malignancy or previous therapy for malignancy
* Known hypersensitivity to somatotropin or any of its excipients including cresol or glycerol
* Closed epiphyses, progression or recurrence of an underlying intracranial tumor, chronic renal disease
* Endocrinologic or metabolic disorders such as Prader-Willi syndrome; Russel-Silver syndrome; Seckel syndrome; Down syndrome; Cushing syndrome; Noonan syndrome; short stature caused by other chromosomal abnormalities
* The disorders that explain short stature such as psychiatric disorders, nutritional disorders, and chronic debilitating diseases
* Participation in another clinical trial within the past 3 months
* Status of legal incapacity or limited legal capacity of the parents or legal guardian
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