Idiopathic Short Stature
Conditions
Keywords
growth hormone
Brief summary
Growth hormone therapy will improve the height of idiopathic short statured children. DA-3002(Recombinant Human Growth hormone)treated group for 26 weeks, will be compared to non-treatment group in efficacy and safety.
Interventions
DRUGDA-3002
Sponsors
Dong-A ST Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
4 Years to 13 Years
Healthy volunteers
No
Inclusion criteria
* Chronological Age ≥ 4 * Bone Age \<11 (for girls) and \<13 (for boys) * Height \<3rd percentile for age * normal thyroid function
Exclusion criteria
* endocrine and/or metabolic disorders * growth failure caused by other disorders * previous use of drugs that could interfere with Growth Hormone treatment
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Annualized Height Velocity(cm/Year) After 26 Weeks | 26 weeks | Height Velocity calculated with height measured at Baseline and after 26 weeks was converted to annual growth rate. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Changes in Height Standard Deviation Score After 26 Weeks | 26 weeks | The Height Standard Deviation Score was calculated as height minus reference mean height divided by the standard deviation of the reference mean height, both given by a reference growth table for the corresponding chronological age at the height measurement. Greater Height Standard Deviation Score indicates greater height. |
Other
| Measure | Time frame |
|---|---|
| Changes in Anti-growth Hormone Antibody | baseline and 26 weeks |
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| DA-3002 Treatment Group 1.11 IU(0.37mg)/kg bodyweight of DA-3002 per week given by subcutaneous injections (six or seven times per week)
DA-3002 | 36 |
| Non-treatment Control Group Height be measured with no treatment | 34 |
| Total | 70 |
Baseline characteristics
| Characteristic | DA-3002 Treatment Group | Non-treatment Control Group | Total |
|---|---|---|---|
| Age, Continuous | 6.75 years STANDARD_DEVIATION 1.79 | 7.17 years STANDARD_DEVIATION 2.34 | 6.96 years STANDARD_DEVIATION 2.07 |
| Region of Enrollment Korea, Republic of | 36 participants | 34 participants | 70 participants |
| Sex: Female, Male Female | 14 Participants | 17 Participants | 31 Participants |
| Sex: Female, Male Male | 22 Participants | 17 Participants | 39 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 19 / 36 | 14 / 31 |
| serious Total, serious adverse events | 1 / 36 | 1 / 31 |
Outcome results
Primary
Annualized Height Velocity(cm/Year) After 26 Weeks
Height Velocity calculated with height measured at Baseline and after 26 weeks was converted to annual growth rate.
Time frame: 26 weeks
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DA-3002 Treatment Group | Annualized Height Velocity(cm/Year) After 26 Weeks | 10.68 cm/year | Standard Deviation 1.95 |
| Non-treatment Control Group | Annualized Height Velocity(cm/Year) After 26 Weeks | 5.72 cm/year | Standard Deviation 1.72 |
Secondary
Changes in Height Standard Deviation Score After 26 Weeks
The Height Standard Deviation Score was calculated as height minus reference mean height divided by the standard deviation of the reference mean height, both given by a reference growth table for the corresponding chronological age at the height measurement. Greater Height Standard Deviation Score indicates greater height.
Time frame: 26 weeks
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DA-3002 Treatment Group | Changes in Height Standard Deviation Score After 26 Weeks | 0.63 ratio | Standard Deviation 0.16 |
| Non-treatment Control Group | Changes in Height Standard Deviation Score After 26 Weeks | 0.06 ratio | Standard Deviation 0.15 |
Other Pre-specified
Changes in Anti-growth Hormone Antibody
Time frame: baseline and 26 weeks
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| DA-3002 Treatment Group | Changes in Anti-growth Hormone Antibody | 0.30 ng/mL | Standard Deviation 0.35 |
| Non-treatment Control Group | Changes in Anti-growth Hormone Antibody | 0.21 ng/mL | Standard Deviation 0.54 |