Trial Outcomes & Findings for Topical Timolol 0.5% Solution for Proliferating Infantile Hemangiomas (NCT NCT01147601)

Last Updated: 2017-07-05

Results Overview

This will be generated by asking each of the assessors to score the improvement using a visual analog scale (VAS) assessing the decrease in size of hemangioma by comparing photographs at different times of treatment. The assessors will score this improvement into one of the following categories: 0-24%, 25-49%, 50-74%, \>75%.

Recruitment status

TERMINATED

Study phase

EARLY_PHASE1

Target enrollment

6 participants

Primary outcome timeframe

at 6 months

Results posted on

2017-07-05

Participant Flow

Participant milestones

Participant milestones
Measure	Topical 0.5% Timolol Half of the enrolled subjects (intervention group) will receive topical 0.5% Timolol. topical 0.5% Timolol: topical 0.5% Timolol aqueous solution, 2-3 drops to cover the hemangioma, twice daily	Placebo Aqueous placebo, 2-3 drops to cover the hemangioma, twice daily Control (placebo) group: Control (placebo) group
Overall Study STARTED	5	1
Overall Study COMPLETED	2	1
Overall Study NOT COMPLETED	3	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Topical Timolol 0.5% Solution for Proliferating Infantile Hemangiomas

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Topical 0.5% Timolol n=5 Participants Half of the enrolled subjects (intervention group) will receive topical 0.5% Timolol. topical 0.5% Timolol: topical 0.5% Timolol aqueous solution, 2-3 drops to cover the hemangioma, twice daily	Placebo n=1 Participants Aqueous placebo, 2-3 drops to cover the hemangioma, twice daily Control (placebo) group: Control (placebo) group	Total n=6 Participants Total of all reporting groups
Age, Continuous	93.5 days n=99 Participants	178 days n=107 Participants	123.2 days n=206 Participants
Sex: Female, Male Female	4 Participants n=99 Participants	0 Participants n=107 Participants	4 Participants n=206 Participants
Sex: Female, Male Male	1 Participants n=99 Participants	1 Participants n=107 Participants	2 Participants n=206 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=99 Participants	1 Participants n=107 Participants	6 Participants n=206 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Asian	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Black or African American	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) White	5 Participants n=99 Participants	1 Participants n=107 Participants	6 Participants n=206 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Region of Enrollment United States	5 participants n=99 Participants	1 participants n=107 Participants	6 participants n=206 Participants

PRIMARY outcome

Timeframe: at 6 months

Outcome measures

Outcome measures
Measure	Topical 0.5% Timolol n=2 Participants Half of the enrolled subjects (intervention group) will receive topical 0.5% Timolol. topical 0.5% Timolol: topical 0.5% Timolol aqueous solution, 2-3 drops to cover the hemangioma, twice daily	Placebo n=1 Participants Aqueous placebo, 2-3 drops to cover the hemangioma, twice daily Control (placebo) group: Control (placebo) group
Proportion of Subjects in Treatment Group Compared to Placebo Group With at Least 75% Improvement in the Extent of the Hemangioma as Compared to Baseline Photos.	1 Participants	0 Participants

SECONDARY outcome

Timeframe: at 6 months

1. The proportion of subjects in the treatment group as compared to the placebo control group with at least 50% improvement in the extent of the hemangioma. 2. The difference between the extent/size of the hemangioma as an outcome measure versus color changes. 3. Frequency of adverse events (e.g. hypotension, behavioral changes, etc.), collected by the investigator and reported by the parents.

Outcome measures

Outcome data not reported

Adverse Events

Topical 0.5% Timolol

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Topical 0.5% Timolol n=5 participants at risk Half of the enrolled subjects (intervention group) will receive topical 0.5% Timolol. topical 0.5% Timolol: topical 0.5% Timolol aqueous solution, 2-3 drops to cover the hemangioma, twice daily	Placebo n=1 participants at risk Aqueous placebo, 2-3 drops to cover the hemangioma, twice daily Control (placebo) group: Control (placebo) group
Respiratory, thoracic and mediastinal disorders Upper Respiratory Infection	20.0% 1/5 • Number of events 1	0.00% 0/1

Additional Information

Dr. Alfons Krol

Oregon Health & Science University

Phone: 503-494-9993

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place