Topical Timolol Gel for the Treatment of Infantile Hemangiomas

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.

Study Overview

• Status: Completed
• Conditions: Hemangioma
• Intervention / Treatment: Drug: timolol maleate 0.5% gel

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.

• Study Type: Interventional
• Enrollment (Actual): 26
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • San Diego, California, United States, 92123
      • Pediatric and Adolescent Dermatology, Rady Children's Hospital/University of California, San Diego

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 week to 6 months (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Rady Children's Hospital, San Diego
• Collaborators: Valeant Pharmaceuticals
• Investigators: Principal Investigator: Sheila F Friedlander, MD, Rady Children's Hospital/ University of California, San Diego

Study record dates

Study Major Dates

• Study Start (Actual): May 1, 2014
• Primary Completion (Actual): October 1, 2016
• Study Completion (Actual): November 1, 2016

Study Registration Dates

• First Submitted: May 21, 2014
• First Submitted That Met QC Criteria: May 22, 2014
• First Posted (Estimate): May 23, 2014

Study Record Updates

• Last Update Posted (Actual): February 13, 2017
• Last Update Submitted That Met QC Criteria: February 9, 2017
• Last Verified: February 1, 2017

More Information

Terms related to this study

• Keywords: hemangioma; birthmark; strawberry hemangioma; timolol
• Additional Relevant MeSH Terms: Skin Diseases; Neoplasms by Histologic Type; Neoplasms; Congenital Abnormalities; Skin Abnormalities; Neoplasms, Vascular Tissue; Hemangioma, Capillary; Port-Wine Stain; Hemangioma; Physiological Effects of Drugs; Adrenergic beta-Antagonists; Adrenergic Antagonists; Adrenergic Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Anti-Arrhythmia Agents; Antihypertensive Agents; Timolol
• Other Study ID Numbers: PED3560; 20132793 (Other Identifier: ICN Pharmaceuticals)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No