Clinical Trial: Efficacy, Safety and Pharmacokinetics of Topical Timolol in Infants With Infantile Hemangioma (IH)
Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional
Official Title: Efficacy, Safety, and Pharmacokinetics of Timolol in Infants With Infantile Hemangioma (IH)
Brief Summary: Multi-center, double-masked randomized, efficacy, safety and pharmacokinetic (PK) study.
Detailed Summary: Primary: Describe the efficacy of 0.25% and 0.5% topical timolol maleate GFS as assessed through IH changes in color. Secondary: Describe the safety and PK of topical timolol maleate GFS for treatment of IH.
Sponsor: Chiara Melloni
Current Primary Outcome: Comparison of partial response of hemangioma color within the treat arm compared to the untreated controls [ Time Frame: 180 days ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Efficacy Comparison of proportion of infants with partial response or greater change in volume [ Time Frame: 180 days ]Percentage of participants with partial response of change in volume from baseline to 180 days within each treatment arm compared with the untreated controls.
- Efficacy comparison of proportion of infants with partial response between the two treatment arms [ Time Frame: 180 days ]Percentage of participants with partial response of change in color from baseline to 180 days between the two treatment arms
- Efficacy comparison of partial response of infants with partial response of hemangioma volume between the treatment arms [ Time Frame: 180 days ]Percentage of participants with partial response of change in volume from baseline to 180 days between the two arms.
- Change in Efficacy Improvement of hemangioma complication within the treatment arms [ Time Frame: Baseline and Day 180 ]Change in the dynamic complication scale results within each treatment arm
- Efficacy assessment of time to partial response in both treatment arms [ Time Frame: 180 days ]Assess time to partial response by comparing baseline to day 30, day 60, day 120 and 120 in both treatment arms
- Change in Efficacy assessment of Quality of Life assessment for infants in both treatment arms [ Time Frame: Baseline and Day 180 ]Absolute change in the Quality of Life score within each treatment arm
- Rate of Serious Adverse Events and Adverse Events of special interest in treated infants in both arms [ Time Frame: 180 days ]Rate of serious adverse events and adverse events of special interest for infants in both treatment arms
- Pharmacokinetics (PK) Analysis measuring clearance of Timolol in Plasma specimen [ Time Frame: Up to 12 hours ]The PK blood samples will be 1.0 ml each and collected between the following timeframes after application of Timolol: within 2 hours, 2-4 hours, 5-7 hours, 8-10 hours or 11-12 hours.
- Pharmacokinetics (PK) Analysis measuring Volume of Distribution of Timolol in plasma specimen [ Time Frame: Up to 12 hours ]The PK blood samples will be 1.0 ml each and collected between the following timeframes after application of Timolol: within 2 hours, 2-4 hours, 5-7 hours, 8-10 hours or 11-12 hours.
- Pharmacokinetics (PK) Analysis measuring area under the curve of Timolol in plasma specimen [ Time Frame: Up to 12 hours ]The PK blood samples will be 1.0 ml each and collected between the following timeframes after application of Timolol: within 2 hours, 2-4 hours, 5-7 hours, 8-10 hours or 11-12 hours.
- Pharmacokinetics (PK) Analysis measuring maximum concentration of Timolol in plasma specimen [ Time Frame: Up to 12 hours ]The PK blood samples will be 1.0 ml each and collected between the following timeframes after application of Timolol: within 2 hours, 2-4 hours, 5-7 hours, 8-10 hours or 11-12 hours.
Original Secondary Outcome: Same as current
Information By: Duke University
Dates:
Date Received: August 12, 2016
Date Started: October 2016
Date Completion: September 2017
Last Updated: September 21, 2016
Last Verified: September 2016