Topical Timolol 0.5% Solution for Proliferating Infantile Hemangiomas

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01147601
Recruitment Status : Terminated
First Posted : June 22, 2010
Results First Posted : July 5, 2017
Last Update Posted : July 5, 2017
Information provided by (Responsible Party):
Dr. Alfons Krol, MD, FRCPC, Oregon Health and Science University

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.

Condition or disease Intervention/treatment Phase
Hemangioma Infant Drug: topical 0.5% Timolol Drug: Control (placebo) group Early Phase 1

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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 6 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Topical Timolol 0.5% Solution for Proliferating Infantile Hemangiomas: A Prospective Double Blinded Placebo Controlled Study
Study Start Date : March 2010
Actual Primary Completion Date : October 2012
Actual Study Completion Date : August 2014

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Birthmarks

Arm Intervention/treatment
Active Comparator: Topical 0.5% Timolol
Half of the enrolled subjects (intervention group) will receive topical 0.5% Timolol.
Drug: topical 0.5% Timolol
topical 0.5% Timolol aqueous solution, 2-3 drops to cover the hemangioma, twice daily

Placebo Comparator: Placebo
Aqueous placebo, 2-3 drops to cover the hemangioma, twice daily
Drug: Control (placebo) group
Control (placebo) group

Primary Outcome Measures :
  1. 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. [ Time Frame: 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 Outcome Measures :
  1. Compare Treatment Group to Control Group Improvement Assessments [ Time Frame: 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Month to 8 Months   (Child)
Sexes Eligible for Study:   All
Accepts 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01147601

United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239-4501
Sponsors and Collaborators
Oregon Health and Science University
Principal Investigator: Alfons L. Krol, M.D. Oregon Health and Science University

Responsible Party: Dr. Alfons Krol, MD, FRCPC, Professor, Oregon Health and Science University Identifier: NCT01147601     History of Changes
Other Study ID Numbers: 6114
First Posted: June 22, 2010    Key Record Dates
Results First Posted: July 5, 2017
Last Update Posted: July 5, 2017
Last Verified: April 2017

Keywords provided by Dr. Alfons Krol, MD, FRCPC, Oregon Health and Science University:
topical 0.5% Timolol
infantile hemangioma (IH)

Additional relevant MeSH terms:
Hemangioma, Capillary
Port-Wine Stain
Neoplasms, Vascular Tissue
Neoplasms by Histologic Type
Skin Abnormalities
Congenital Abnormalities
Skin Diseases
Adrenergic beta-Antagonists
Adrenergic Antagonists
Adrenergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Anti-Arrhythmia Agents
Antihypertensive Agents