Trial Outcomes & Findings for Efficacy and Safety of DA-3002 in Children With Idiopathic Short Stature (NCT NCT01786902)

NCT ID: NCT01786902

Last Updated: 2017-10-19

Results Overview

Height Velocity calculated with height measured at Baseline and after 26 weeks was converted to annual growth rate.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 70 participants
• Primary outcome timeframe: 26 weeks
• Results posted on: 2017-10-19

Participant Flow

Participant milestones

Measure	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	Non-treatment Control Group Height be measured with no treatment
Overall Study STARTED	36	34
Overall Study COMPLETED	32	31
Overall Study NOT COMPLETED	4	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Efficacy and Safety of DA-3002 in Children With Idiopathic Short Stature

Baseline characteristics by cohort

Measure	DA-3002 Treatment Group n=36 Participants 1.11 IU(0.37mg)/kg bodyweight of DA-3002 per week given by subcutaneous injections (six or seven times per week) DA-3002	Non-treatment Control Group n=34 Participants Height be measured with no treatment	Total n=70 Participants Total of all reporting groups
Age, Continuous	6.75 years STANDARD_DEVIATION 1.79 • n=5 Participants	7.17 years STANDARD_DEVIATION 2.34 • n=7 Participants	6.96 years STANDARD_DEVIATION 2.07 • n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants	17 Participants n=7 Participants	31 Participants n=5 Participants
Sex: Female, Male Male	22 Participants n=5 Participants	17 Participants n=7 Participants	39 Participants n=5 Participants
Region of Enrollment Korea, Republic of	36 participants n=5 Participants	34 participants n=7 Participants	70 participants n=5 Participants

PRIMARY outcome

Timeframe: 26 weeks

Height Velocity calculated with height measured at Baseline and after 26 weeks was converted to annual growth rate.

Outcome measures

Measure	DA-3002 Treatment Group n=36 Participants 1.11 IU(0.37mg)/kg bodyweight of DA-3002 per week given by subcutaneous injections (six or seven times per week) DA-3002	Non-treatment Control Group n=32 Participants Height be measured with no treatment
Annualized Height Velocity(cm/Year) After 26 Weeks	10.68 cm/year Standard Deviation 1.95	5.72 cm/year Standard Deviation 1.72

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	DA-3002 Treatment Group n=36 Participants 1.11 IU(0.37mg)/kg bodyweight of DA-3002 per week given by subcutaneous injections (six or seven times per week) DA-3002	Non-treatment Control Group n=32 Participants Height be measured with no treatment
Changes in Height Standard Deviation Score After 26 Weeks	0.63 ratio Standard Deviation 0.16	0.06 ratio Standard Deviation 0.15

OTHER_PRE_SPECIFIED outcome

Timeframe: baseline and 26 weeks

Outcome measures

Measure	DA-3002 Treatment Group n=33 Participants 1.11 IU(0.37mg)/kg bodyweight of DA-3002 per week given by subcutaneous injections (six or seven times per week) DA-3002	Non-treatment Control Group n=31 Participants Height be measured with no treatment
Changes in Anti-growth Hormone Antibody	0.30 ng/mL Standard Deviation 0.35	0.21 ng/mL Standard Deviation 0.54

Adverse Events

DA-3002 Treatment Group

Serious events: 1 serious events

Other events: 19 other events

Deaths: 0 deaths

Non-treatment Control Group

Serious events: 1 serious events

Other events: 14 other events

Deaths: 0 deaths

Serious adverse events

Measure	DA-3002 Treatment Group n=36 participants at risk 1.11 IU(0.37mg)/kg bodyweight of DA-3002 per week given by subcutaneous injections (six or seven times per week) DA-3002	Non-treatment Control Group n=31 participants at risk Height be measured with no treatment
Infections and infestations Pharyngotonsillitis	2.8% 1/36	0.00% 0/31
Surgical and medical procedures Strabismus correction	0.00% 0/36	3.2% 1/31

Other adverse events

Measure	DA-3002 Treatment Group n=36 participants at risk 1.11 IU(0.37mg)/kg bodyweight of DA-3002 per week given by subcutaneous injections (six or seven times per week) DA-3002	Non-treatment Control Group n=31 participants at risk Height be measured with no treatment
Gastrointestinal disorders Enteritis	0.00% 0/36	3.2% 1/31
Gastrointestinal disorders Nausea	2.8% 1/36	0.00% 0/31
Gastrointestinal disorders Vomiting	0.00% 0/36	3.2% 1/31
Injury, poisoning and procedural complications Laceration	0.00% 0/36	3.2% 1/31
Nervous system disorders Dizziness	0.00% 0/36	3.2% 1/31
Nervous system disorders Headache	2.8% 1/36	0.00% 0/31
Respiratory, thoracic and mediastinal disorders Nasal congestion	2.8% 1/36	0.00% 0/31
Skin and subcutaneous tissue disorders Rash	2.8% 1/36	0.00% 0/31
Surgical and medical procedures Strabismus correction	0.00% 0/36	3.2% 1/31
General disorders Pyrexia	13.9% 5/36	0.00% 0/31
Immune system disorders Hypersensitivity	5.6% 2/36	0.00% 0/31
Infections and infestations Upper respiratory tract infection	22.2% 8/36	29.0% 9/31

Additional Information

Dr. Hanwook Yoo

Asan Medical Center

Phone: 82-2-3010-3374

Email: hwyoo@amc.seoul.kr

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place