Timolol Maleate for Infantile Hemangioma

Evaluating timolol as a treatment for infantile hemangiomas, a common birthmark affecting infants.

Summary

Since first reported in 2008 that propranolol, a beta blocker, was effective in the treatment of Infantile Hemangiomas (IH)—a birthmark that most commonly appears as a rubbery, bright red nodule of extra blood vessels in the skin, commonly called a “strawberry”—this class of drug has been used as the first line of therapy for infants with IH. Timolol, also a beta blocker, is available in topical formulation, and increasingly used off-label for small, non-complicated IH. The popularity of timolol is likely due to its perceived safety as a topical drug. However, data on timolol efficacy, safety and pharmacokinetics are limited.

PTN’s timolol study will enroll 100 infants between the ages of ≥32 to <50 weeks postmenstrual age. The infants will be treated with timolol for 28 days then randomized into 2 groups. One group will continue timolol treatment while the other will be withdrawn from treatment. Both groups will remain on the study in their respective groups for up to 120 days. The study began enrolling in summer 2016 at 10 U.S. sites. The data received from the study will be presented to the FDA to support the use of timolol for the treatment of IH.

OVERVIEW

Status:
Enrolling

ClinicalTrials.gov Identifier:
NCT02913612

Principal Investigators:
Beth Ann Drolet, MD and Kristen Holland, MD
Medical College of Wisconsin
Milwaukee, WI

NEWS

  • Timolol for Infantile Hemangiomas under Review November 17, 2015 Since first reported in 2008 that propranolol, a beta blocker, was effective in the treatment of Infantile Hemangiomas (IH)—a birthmark that most commonly appears as a rubbery, bright red nodule of extra blood vessels in the skin, commonly called a “strawberry”—this class of drug has been used as the first line of therapy for infants ...