Efficacy, Safety and Pharmacokinetics of Topical Timolol in Infants With Infantile Hemangioma (IH)
- Conditions
- Infantile Hemangioma
- Interventions
- Drug: 0.25% Timolol Maleate Gel Forming SolutionDrug: 0.5% Timolol Maleate Gel Forming Solution
- Registration Number
- NCT02913612
- Lead Sponsor
- Kanecia Obie Zimmerman
- Brief Summary
The purpose of this study is to assess the safety and efficacy of Timolol 0.25% and 0.5% doses.
- Detailed Description
Primary: Describe the efficacy of 0.25% and 0.5% topical timolol maleate Gel-forming solution (GFS) as assessed through Infantile Hemangioma (IH) changes in volume.
Secondary: Describe the safety of topical timolol maleate GFS for treatment of IH.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 105
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description 0.25% Timolol Treatment 0.25% Timolol Maleate Gel Forming Solution Subjects assigned to this arm will be randomized to 0.25% timolol for 180 days. If during the 180 days the subject is considered a treatment failure, the subject will be unblinded. If the subject is on 0.25% timolol they will be changed to 0.5% timolol. 0.5% Timolol Treatment 0.5% Timolol Maleate Gel Forming Solution Subjects assigned to this arm will be randomized to 0.5% timolol for 180 days. If during the 180 days the subject is considered a treatment failure, the subject will be unblinded. If the subject is on 0.5% timolol the treating physician will decide to either continue 0.5% timolol or withdraw the subject and begin an alternative treatment.
- Primary Outcome Measures
Name Time Method Number of Participants With Partial Response of Hemangioma Volume as Measured by VAS (Visual Analog Scale) Within Each Treatment Arm and Compared With Untreated Controls 180 days The VAS-volume is a 100 mm scale used to independently grade hemangioma volume. -100 indicates hemangioma has doubled in size, 0 indicates no change, and +100 indicates complete shrinkage. Partial response is defined as \>20% and up to 80% reduction in volumetric size of hemangioma.
- Secondary Outcome Measures
Name Time Method Number of Participants Who Reach Partial Response, Assessed by Hemangioma Color 180 days Assess time to partial response or greater by VAS-color, comparing baseline to day 30, day 60, day 120 and day 180. Partial response: \>30% and up to 80% reduction in color of hemangioma
Number of Participants With Partial Response in Hemangioma Color as Measured by VAS (Visual Analog Scale) Within Each Treatment Arm and Compared With Untreated Controls 180 days The VAS-color is a 100 mm scale used to independently grade hemangioma color. -100 indicates hemangioma is twice as intense, 0 indicates no change, and +100 indicates complete resolution. Partial response is defined as \>30% and up to 80% reduction in color of hemangioma.
Number of Participants Who Reach Partial Response, Assessed by Volume 30 days, 60 days, 120 days, 180 days Assess time to partial response or greater by VAS-volume, comparing baseline to day 30, day 60, day 120 and day 180. Partial response: \>20% and up to 80% reduction in volumetric size of hemangioma
Number of Participants With Partial Response of Hemangioma Volume as Measured by VAS (Visual Analog Scale) Within Each Treatment Arm 180 days The VAS-volume is a 100 mm scale used to independently grade hemangioma volume. -100 indicates hemangioma has doubled in size, 0 indicates no change, and +100 indicates complete shrinkage. Partial response is defined as \>20% and up to 80% reduction in volumetric size of hemangioma.
Comparison of Partial Response of Hemangioma Color From Baseline to 180 Days, Within Each Treatment Arm 180 days Comparison of partial response of hemangioma color (partial response or greater as assessed by VAS-color) between the two treatment arms. Partial response: \>30% and up to 80% reduction in color of hemangioma.
Change in Hemangioma Dynamic Complication Scale (HDCS) baseline, day 180 Absolute change in hemangioma dynamic complication scale from Day 0 to end of study within each treatment arm. The HDCS provides a 6-point severity grading system for 12 individual hemangioma-related complications (grade 0 represents absent to minimal; grade 5 = most severe). The total score ranges from 0-60.
Number of Serious Adverse Events and Adverse Events of Special Interest in Infants Treated With Topical Timolol Maleate up to 270 days Serious adverse events and adverse events of special interest from randomization to Day 180 in infants treated with topical timolol maleate (0.25% and 0.5%) GFS for the treatment of infantile hemangioma.
Change in Hemangioma Quality of Life (IH-QoL) Assessment for Infants baseline, day 180 Absolute change in IH-QoL score scale from Day 0 to end of study within each treatment arm. The IH-QoL score scale consists of 4 domains (physical symptom of patient, social functioning of patient, social and psychological functioning of caregiver, and emotional functioning of caregiver) and 29 items, with each item scored on a Likert scale : 0 = never a problem, 1 = almost never a problem, 2 = sometimes a problem, 3 = often a problem and 4 = almost always a problem). The total range is 0-116; the higher the total number indicates a worse outcome.
Trial Locations
- Locations (12)
Columbia University Medical Center
🇺🇸New York, New York, United States
DCOL Center for Clinical Research
🇺🇸Longview, Texas, United States
Dartmouth-Hitchcock Medical Center
🇺🇸Lebanon, New Hampshire, United States
The University of Texas Medical School at Houston
🇺🇸Houston, Texas, United States
Johns Hopkins Medical Center
🇺🇸Baltimore, Maryland, United States
Children's Hospital of Philadephia
🇺🇸Philadelphia, Pennsylvania, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Indiana University Health
🇺🇸Indianapolis, Indiana, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Medical College of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States