Topical Timolol Gel for the Treatment of Infantile Hemangiomas

Study Overview
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Brief Summary
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We plan to conduct a study, to see how safe and effective timolol maleate 0.5% gel-forming solution is for infantile hemangiomas (IH) and the response of hemangiomas to timolol maleate 0.5% . Our hypothesis is that timolol will inhibit and possibly reverse growth of appropriate infantile hemangiomas.

Detailed Description
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Subjects will be assigned to open label timolol maleate 0.5%. Parents will be instructed to place 1 to 2 drops twice a day in the center of the hemangioma. Subjects are expected to use the study drug as directed every day for a 4 month period. Subjects will be assessed at baseline, 2 weeks, 4 weeks, and every 4 weeks thereafter for a total of 5 months. Physical exam, photographs to compare size and color intensity, and a scale of improvement will be completed at each visit. Parents will complete a quality of life questionnaire regarding their child's hemangioma and its impact on quality of life. Subjects who have evidence of worsening would be removed from the study and transferred to the clinic for evaluation and treatment.

Official Title
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Topical Timolol Gel for the Treatment of Infantile Hemangiomas

Conditions
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Hemangioma

Intervention / Treatment
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* Drug: timolol maleate 0.5% gel


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Written informed permission for study participation and the use of the patient's images are obtained from the patient's parent(s) or guardian(s), The patient is between 7 days and 6 months of age at the time of enrollment, and a proliferating HOI not requiring systemic therapy is present anywhere on the body, Multiple hemangiomas may be treated on same child, if the hemangioma meets this criteria. Exclusion Criteria: patients who are not otherwise generally healthy; at risk for imminent ulceration, life-threatening, function-threatening, or ulcerated; patients who have previously received systemic, intra-lesional or topical corticosteroids, imiquimod, vincristine, alpha-interferon, propranolol or other beta blockers; patients who have previously been treated for his/her HOI, including any surgical and/or medical procedures; patients whose mothers have been breastfeeding the patient while also being treated with beta-blockers, systemic corticosteroids, vincristine or alpha-interferon; patients who have previously experienced any anaphylactic reactions; patients with an unclear diagnosis of HOI; patients participating in another clinical study or living in the same household as an infant already participating in this study; patients born prematurely who have not yet reached his/her term equivalent age; and patients with parent(s) or guardian(s) who cannot be contacted by telephone in case of emergency.

Ages Eligible for Study
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Minimum Age: 7 Days
Maximum Age: 6 Months

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: timolol maleate 0.5% gel<br>timolol gel 1 to 2 drops twice a day to lesions for 4 months | Drug: timolol maleate 0.5% gel<br>* Apply timolol gel 1-2 drops twice a day to lesion<br>* Other names: timoptic;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in size of hemangioma | At each visit, the size of the IH is assessed in three dimensions (length, width, and height) in cm. | Baseline, week 2, 4, 8, 12, 16 | 
| Change in color of hemangioma | At each visit, the color intensity (e.g., barely perceptible; pale pink or mottled pink/red; red with central pallor; dull red; bright red) of the IH is assessed on a 6 point scale. | Baseline, week 2, 4, 8, 12, 16 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Measure the extent of systemic absorption and the factors which influence absorption | A heel stick blood sample is collected to assess for systemic absorption. | week 2 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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hemangioma, birthmark, strawberry hemangioma, timolol