Hemangioma, Infant
Conditions
Keywords
topical 0.5% Timolol, infantile hemangioma (IH)
Brief summary
The purpose of this study is to learn about a new potential use for topical timolol 0.5% aqueous solution that may help treat small uncomplicated infantile hemangiomas. This study would examine whether topical timolol could be a potential therapy.
Detailed description
At the first visit, subjects will fill out a questionnaire that asks about the child's history and the hemangioma. Photographs and measurements will be taken at this and each subsequent visit. At the first visit, subjects will have a physical exam, including vital signs, height and weight. An EKG will also be done. Study drug will be dispensed and instructions given. Half of the subjects will receive a placebo. This is a blinded randomized study. Follow-up visits will be weekly for the first two weeks (three visits total including the first visit), then monthly until the study is over. Photographs and measurements will again be taken at each visit. Subjects will be asked to evaluate the size and the color of the hemangioma against the first photographs and fill out forms regarding any changes in behavior and asked about any side effects.
Interventions
DRUGtopical 0.5% Timolol
topical 0.5% Timolol aqueous solution, 2-3 drops to cover the hemangioma, twice daily
DRUGControl (placebo) group
Control (placebo) group
Sponsors
Oregon Health and Science University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Months to 8 Months
Healthy volunteers
No
Inclusion criteria
Infants 1 month to 8 months of age with infantile hemangioma (IH) that are 3 cm or less on the scalp, trunk, or extremities.
Exclusion criteria
1. Subjects with facial, genital, perianal, hand, finger, feet, or toe IH 2. Subjects with PHACES (plaque-like hemangioma on the face awaiting imaging) syndrome (proven) or suspected PHACES . 3. Subjects with IH measuring more than 3cm in size or ulcerated. 4. Children with a history of hypersensitivity to beta blockers. 5. Children with a personal history of asthma. 6. Children with known renal impairment. 7. Children with known cardiac conditions that may predispose to heart block 8. Personal history of hypoglycemia 9. Children on medications that may interact with beta blockers
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. | at 6 months | 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%. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Compare Treatment Group to Control Group Improvement Assessments | 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. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 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 | 5 |
| Placebo Aqueous placebo, 2-3 drops to cover the hemangioma, twice daily
Control (placebo) group: Control (placebo) group | 1 |
| Total | 6 |
Baseline characteristics
| Characteristic | Topical 0.5% Timolol | Total | Placebo |
|---|---|---|---|
| Age, Continuous | 93.5 days | 123.2 days | 178 days |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 5 Participants | 6 Participants | 1 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 5 Participants | 6 Participants | 1 Participants |
| Region of Enrollment United States | 5 participants | 6 participants | 1 participants |
| Sex: Female, Male Female | 4 Participants | 4 Participants | 0 Participants |
| Sex: Female, Male Male | 1 Participants | 2 Participants | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 1 / 5 | 0 / 1 |
| serious Total, serious adverse events | 0 / 5 | 0 / 1 |
Outcome results
Primary
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.
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%.
Time frame: at 6 months
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Topical 0.5% Timolol | 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 |
| Placebo | 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. | 0 Participants |
Secondary
Compare Treatment Group to Control Group Improvement Assessments
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.
Time frame: at 6 months