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Topical Timolol Gel for the Treatment of Infantile Hemangiomas

Phase 2
Completed
Conditions
Hemangioma
Interventions
Registration Number
NCT02145884
Lead Sponsor
Rady Children's Hospital, San Diego
Brief Summary

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

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.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
26
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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
timolol maleate 0.5% geltimolol maleate 0.5% geltimolol gel 1 to 2 drops twice a day to lesions for 4 months
Primary Outcome Measures
NameTimeMethod
Change in color of hemangiomaBaseline, week 2, 4, 8, 12, 16

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.

Change in size of hemangiomaBaseline, week 2, 4, 8, 12, 16

At each visit, the size of the IH is assessed in three dimensions (length, width, and height) in cm.

Secondary Outcome Measures
NameTimeMethod
Measure the extent of systemic absorption and the factors which influence absorptionweek 2

A heel stick blood sample is collected to assess for systemic absorption.

Trial Locations

Locations (1)

Pediatric and Adolescent Dermatology, Rady Children's Hospital/University of California, San Diego

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San Diego, California, United States

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