Phase 2
N=141
Efficacy, Safety and Pharmacokinetics of Topical Timolol in Infants With Infantile Hemangioma (IH)
Infantile Hemangioma
Bottom Line
View on ClinicalTrials.gov: NCT02913612 ↗Enrolled (actual)
141
Serious AEs
4.8%
Results posted
Nov 2022
Primary outcome: Primary: 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 — 24; 28; 15 Participants — p=0.0205
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- 0.25% Timolol Maleate Gel Forming Solution (Drug); 0.5% Timolol Maleate Gel Forming Solution (Drug)
- Age
- Pediatric
- Sex
- All
- Sponsor
- Kanecia Obie Zimmerman
- Primary completion
- Oct 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY 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 |
24; 28; 15 | 0.0205 sig |
| SECONDARY 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 |
32; 34; 19 | — |
| SECONDARY Number of Participants With Partial Response of Hemangioma Volume as Measured by VAS (Visual Analog Scale) Within Each Treatment Arm |
24; 28 | — |
| SECONDARY Comparison of Partial Response of Hemangioma Color From Baseline to 180 Days, Within Each Treatment Arm |
32; 34 | — |
| SECONDARY Change in Hemangioma Dynamic Complication Scale (HDCS) |
0; -0.1; 0 | — |
| SECONDARY Number of Participants Who Reach Partial Response, Assessed by Volume |
12; 14; 0; 17; 19; 1 | — |
| SECONDARY Number of Participants Who Reach Partial Response, Assessed by Hemangioma Color |
17; 20; 0; 19; 21; 2 | — |
| SECONDARY Change in Hemangioma Quality of Life (IH-QoL) Assessment for Infants |
35.3; 37.4; 32.6 | — |
| SECONDARY Number of Serious Adverse Events and Adverse Events of Special Interest in Infants Treated With Topical Timolol Maleate |
14; 14; 3; 2 | — |
Summary
The purpose of this study is to assess the safety and efficacy of Timolol 0.25% and 0.5% doses.
Eligibility Criteria
Inclusion Criteria
- Documented informed consent from legal guardian
- 0-84 days postnatal age at time of first study dose or when enrolled into the non-intervention cohort.
- Clinical diagnosis of superficial cutaneous or mucosal infantile hemangioma (must include all of the following):
- Superficial lesion in the dermis
- Thin =5 cm at its longest dimension and 3)
- IH of the non-keratinized mucosa
- Infants with more than one hemangioma that requires therapy
- Hemodynamically significant cardiovascular disease, as determined by the investigator
- Known allergy to beta blockers or vehicle
- Heart rate <100 beats per minute at screening visit
- Known prenatal or postnatal diagnosis of 2nd/3rd degree atrioventricular block
- History of Reactive Airways Disease (RAD)
- Any condition which would make the participant, in the opinion of the investigator unsuitable for the study.
Data sourced from ClinicalTrials.gov (NCT02913612). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.