Trial Outcomes & Findings for The Safety and Efficiency of Propranolol as an Initial Treatment for Pediatric Hemangioma (NCT NCT01908972)

NCT ID: NCT01908972

Last Updated: 2018-12-19

Results Overview

The primary efficacy variable was the clinical response at 16 weeks, classified as follows: when the volume did not increase or decreased by less than 25% after treatment began, we defined it as stop of progression; when the volume decreased by 25% or more compared with the original size, we defined it as regression. Both stop of progression and regressionwere defined as reaction. If the volume at the primary efficacy evaluation point was greater than the size measured when treatment started,we called it an increase. Increase was defined as a nonreaction.

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 34 participants
• Primary outcome timeframe: After 16weeks
• Results posted on: 2018-12-19

Participant Flow

Participant milestones

Measure	Prednisolone Patients who will receive prednisolone medication (2 mg/kg/day ) for 16 weeks Prednisolone: 2mg/kg/day for 16weeks	Propranolol Patients who will receive propranolol medication 2 mg/kg/day for 16 weeks Propranolol: 2mg/kg/day for 16weeks
Overall Study STARTED	17	17
Overall Study COMPLETED	15	17
Overall Study NOT COMPLETED	2	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Measure	Prednisolone n=17 Participants Patients who will receive prednisolone medication (2 mg/kg/day ) for 16 weeks Prednisolone: 2mg/kg/day for 16weeks	Propranolol n=17 Participants Patients who will receive propranolol medication 2 mg/kg/day for 16 weeks Propranolol: 2mg/kg/day for 16weeks	Total n=34 Participants Total of all reporting groups
Age, Categorical <=18 years	17 Participants n=17 Participants	17 Participants n=17 Participants	34 Participants n=34 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=17 Participants	0 Participants n=17 Participants	0 Participants n=34 Participants
Age, Categorical >=65 years	0 Participants n=17 Participants	0 Participants n=17 Participants	0 Participants n=34 Participants
Age, Continuous	3.0 months n=17 Participants	3.6 months n=17 Participants	3.3 months n=34 Participants
Sex: Female, Male Female	9 Participants n=17 Participants	10 Participants n=17 Participants	19 Participants n=34 Participants
Sex: Female, Male Male	8 Participants n=17 Participants	7 Participants n=17 Participants	15 Participants n=34 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.
Region of Enrollment South Korea	17 Participants n=17 Participants	17 Participants n=17 Participants	34 Participants n=34 Participants
Hemangioma volume by MRI	9,349 mm^3 STANDARD_DEVIATION 16,015 • n=17 Participants	14,125 mm^3 STANDARD_DEVIATION 18,246 • n=17 Participants	11,737 mm^3 STANDARD_DEVIATION 3348 • n=34 Participants

PRIMARY outcome

Timeframe: After 16weeks

The primary efficacy variable was the clinical response at 16 weeks, classified as follows: when the volume did not increase or decreased by less than 25% after treatment began, we defined it as stop of progression; when the volume decreased by 25% or more compared with the original size, we defined it as regression. Both stop of progression and regressionwere defined as reaction. If the volume at the primary efficacy evaluation point was greater than the size measured when treatment started,we called it an increase. Increase was defined as a nonreaction.

Outcome measures

Measure	Prednisolone n=17 Participants Patients who will receive prednisolone medication (2 mg/kg/day ) for 16 weeks Prednisolone: 2mg/kg/day for 16weeks	Propranolol n=17 Participants Patients who will receive propranolol medication 2 mg/kg/day for 16 weeks Propranolol: 2mg/kg/day for 16weeks
Number of Participants With Clinical Response From Baseline in Hemangioma Volume Measured by MRI or SONO	14 Participants	16 Participants

SECONDARY outcome

Timeframe: After 16 weeks

Percent Reduction in Hemangioma Volume from Baseline (measured by MRI or Sono (from basline to 16 weeks))

Outcome measures

Measure	Prednisolone n=14 Participants Patients who will receive prednisolone medication (2 mg/kg/day ) for 16 weeks Prednisolone: 2mg/kg/day for 16weeks	Propranolol n=16 Participants Patients who will receive propranolol medication 2 mg/kg/day for 16 weeks Propranolol: 2mg/kg/day for 16weeks
Percent Reduction in Hemangioma Volume From Baseline	46.52 % from baseline Standard Deviation 26.24	55.87 % from baseline Standard Deviation 18.92

SECONDARY outcome

Timeframe: up to 16weeks

Number of Participants in which, the Heart rate fell to <70% of acceptable age related minimum post-dose with child awake, anytime during the 16 weeks Count of patients whose Heart rate fall to <70% of acceptable age related minimum post-dose with child awake

Outcome measures

Measure	Prednisolone n=15 Participants Patients who will receive prednisolone medication (2 mg/kg/day ) for 16 weeks Prednisolone: 2mg/kg/day for 16weeks	Propranolol n=17 Participants Patients who will receive propranolol medication 2 mg/kg/day for 16 weeks Propranolol: 2mg/kg/day for 16weeks
Number of Participants in Which, the Heart Rate Fell to <70% of Acceptable Age Related Minimum Post-dose With Child Awake, Anytime During the 16 Weeks	1 Participants	5 Participants

SECONDARY outcome

Timeframe: After 16 weeks

Participants were observed for any change in color. The possible change in colors included change to Red/Purple/Blue/Gray/Apricot. Reported are the number of participants who experienced a change in color by the type of color

Outcome measures

Measure	Prednisolone n=15 Participants Patients who will receive prednisolone medication (2 mg/kg/day ) for 16 weeks Prednisolone: 2mg/kg/day for 16weeks	Propranolol n=17 Participants Patients who will receive propranolol medication 2 mg/kg/day for 16 weeks Propranolol: 2mg/kg/day for 16weeks
Number of Participants With Change in Color as Compared to Baseline Red	13 Participants	14 Participants
Number of Participants With Change in Color as Compared to Baseline Purple	0 Participants	0 Participants

SECONDARY outcome

Timeframe: After 16 weeks

size was measure the horizontal and vertical size (2-dimension) of ulceration (from baseline to 16 weeks after medication)

Outcome measures

Measure	Prednisolone n=17 Participants Patients who will receive prednisolone medication (2 mg/kg/day ) for 16 weeks Prednisolone: 2mg/kg/day for 16weeks	Propranolol n=17 Participants Patients who will receive propranolol medication 2 mg/kg/day for 16 weeks Propranolol: 2mg/kg/day for 16weeks
Number of Participants With Size Reduction of Ulceration	2 Participants	1 Participants

SECONDARY outcome

Timeframe: After 16 weeks

Number of participants with Reepithelialzation in 16weeks..

Outcome measures

Measure	Prednisolone n=17 Participants Patients who will receive prednisolone medication (2 mg/kg/day ) for 16 weeks Prednisolone: 2mg/kg/day for 16weeks	Propranolol n=17 Participants Patients who will receive propranolol medication 2 mg/kg/day for 16 weeks Propranolol: 2mg/kg/day for 16weeks
Number of Participants With Reepithelialzation in 16weeks	4 Participants	8 Participants

SECONDARY outcome

Timeframe: After 16 weeks

Number of participants whose hemangioma stop proliferating in 16weeks

Outcome measures

Measure	Prednisolone n=17 Participants Patients who will receive prednisolone medication (2 mg/kg/day ) for 16 weeks Prednisolone: 2mg/kg/day for 16weeks	Propranolol n=17 Participants Patients who will receive propranolol medication 2 mg/kg/day for 16 weeks Propranolol: 2mg/kg/day for 16weeks
Number of Participants With Stop of Proliferation	15 Participants	17 Participants

SECONDARY outcome

Timeframe: Within 16 weeks

Number of participants whose hemangioma showed regression in 16 weeks.

Outcome measures

Measure	Prednisolone n=17 Participants Patients who will receive prednisolone medication (2 mg/kg/day ) for 16 weeks Prednisolone: 2mg/kg/day for 16weeks	Propranolol n=17 Participants Patients who will receive propranolol medication 2 mg/kg/day for 16 weeks Propranolol: 2mg/kg/day for 16weeks
Number of Participants With Regression	9 Participants	13 Participants

SECONDARY outcome

Timeframe: After 16 weeks

We checked Number of participants with Drug compliance within 16 weeks

Outcome measures

Measure	Prednisolone n=17 Participants Patients who will receive prednisolone medication (2 mg/kg/day ) for 16 weeks Prednisolone: 2mg/kg/day for 16weeks	Propranolol n=17 Participants Patients who will receive propranolol medication 2 mg/kg/day for 16 weeks Propranolol: 2mg/kg/day for 16weeks
Number of Participants With Drug Compliance Within 16 Weeks	15 Participants	17 Participants

SECONDARY outcome

Timeframe: up to 16weeks

Number of Participants in Which, the Systolic blood pressure fall of >25% of baseline postdose with child awake, Anytime During the 16 Weeks..

Outcome measures

Measure	Prednisolone n=17 Participants Patients who will receive prednisolone medication (2 mg/kg/day ) for 16 weeks Prednisolone: 2mg/kg/day for 16weeks	Propranolol n=17 Participants Patients who will receive propranolol medication 2 mg/kg/day for 16 weeks Propranolol: 2mg/kg/day for 16weeks
Number of Participants in Which, the Systolic Blood Pressure Fall of >25% of Baseline Postdose With Child Awake, Anytime During the 16 Weeks	1 Participants	5 Participants

SECONDARY outcome

Timeframe: up to 16weeks

Number of Participants in Which, Glucose levels fall (to <50mg/dl), Anytime During the 16 Weeks..

Outcome measures

Measure	Prednisolone n=17 Participants Patients who will receive prednisolone medication (2 mg/kg/day ) for 16 weeks Prednisolone: 2mg/kg/day for 16weeks	Propranolol n=17 Participants Patients who will receive propranolol medication 2 mg/kg/day for 16 weeks Propranolol: 2mg/kg/day for 16weeks
Number of Participants in Which, Glucose Levels Fall (to <50mg/dl), Anytime During the 16 Weeks	0 Participants	0 Participants

SECONDARY outcome

Timeframe: up to 16weeks

Number of Participants in Which, facial edema occurs, Anytime During the 16 Weeks..

Outcome measures

Measure	Prednisolone n=17 Participants Patients who will receive prednisolone medication (2 mg/kg/day ) for 16 weeks Prednisolone: 2mg/kg/day for 16weeks	Propranolol n=17 Participants Patients who will receive propranolol medication 2 mg/kg/day for 16 weeks Propranolol: 2mg/kg/day for 16weeks
Number of Participants in Which, Facial Edema Occurs, Anytime During the 16 Weeks	5 Participants	0 Participants

SECONDARY outcome

Timeframe: up to 16weeks

Number of Participants with Growth Retardation within 16 weeks..

Outcome measures

Measure	Prednisolone n=17 Participants Patients who will receive prednisolone medication (2 mg/kg/day ) for 16 weeks Prednisolone: 2mg/kg/day for 16weeks	Propranolol n=17 Participants Patients who will receive propranolol medication 2 mg/kg/day for 16 weeks Propranolol: 2mg/kg/day for 16weeks
Number of Participants With Growth Retardation Within 16 Weeks	2 Participants	0 Participants

SECONDARY outcome

Timeframe: up to 16weeks

Number of Participants With Gastroesophageal reflux within 16 weeks..

Outcome measures

Measure	Prednisolone n=17 Participants Patients who will receive prednisolone medication (2 mg/kg/day ) for 16 weeks Prednisolone: 2mg/kg/day for 16weeks	Propranolol n=17 Participants Patients who will receive propranolol medication 2 mg/kg/day for 16 weeks Propranolol: 2mg/kg/day for 16weeks
Number of Participants With Gastroesophageal Reflux Within 16 Weeks	0 Participants	0 Participants

SECONDARY outcome

Timeframe: up to 16weeks

All symptoms associated adverse drug reaction will be checked

Outcome measures

Measure	Prednisolone n=17 Participants Patients who will receive prednisolone medication (2 mg/kg/day ) for 16 weeks Prednisolone: 2mg/kg/day for 16weeks	Propranolol n=17 Participants Patients who will receive propranolol medication 2 mg/kg/day for 16 weeks Propranolol: 2mg/kg/day for 16weeks
Number of Participants With Adverse Drug Reaction	15 Participants	16 Participants

Adverse Events

Prednisolone

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Propranolol

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Prednisolone n=17 participants at risk Patients who will receive prednisolone medication (2 mg/kg/day ) for 16 weeks Prednisolone: 2mg/kg/day for 16weeks	Propranolol n=17 participants at risk Patients who will receive propranolol medication 2 mg/kg/day for 16 weeks Propranolol: 2mg/kg/day for 16weeks
Blood and lymphatic system disorders Bradycardia	5.9% 1/17 • Number of events 1 • 20 weeks	29.4% 5/17 • Number of events 5 • 20 weeks

Additional Information

Prof. Tae Hyun Choi

Seoul National University College of Medicine

Phone: 82-10-5595-5829

Email: psthchoi@snu.ac.kr

Results disclosure agreements

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