Efficacy, Safety and Pharmacokinetics of Topical Timolol in Infants With Infantile Hemangioma (IH)

Kanecia Obie Zimmerman105 enrolled

Overview

The purpose of this study is to assess the safety and efficacy of Timolol 0.25% and 0.5% doses.

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

105

Conditions

Infantile Hemangioma

Interventions

0.25% Timolol Maleate Gel Forming SolutionDRUG

50:50 Randomized 0.25% Timolol Maleate Gel Forming Solution

0.5% Timolol Maleate Gel Forming SolutionDRUG

50:50 Randomized 0.5% Timolol Maleate Gel Forming Solution

Eligibility

Sex: ALLMax age: 84 Days

Medical Language ↔ Plain English

Inclusion Criteria 1. Documented informed consent from legal guardian 2. 0-84 days postnatal age at time of first study dose or when enrolled into the non-intervention cohort. 3. Clinical diagnosis of superficial cutaneous or mucosal infantile hemangioma (must include all of the following): 1. Superficial lesion in the dermis 2. Thin \<2 mm in thickness 3. Small \>=5 cm at its longest dimension and \<=10cm2 4. Involves skin or keratinized mucosa Exclusion Criteria 1. History of previous treatment with any pharmacologic or laser therapy for IH 2. Ongoing therapy with an oral beta blocker or oral corticosteroid (e.g., cardiac arrhythmia, adrenal insufficiency, upper airway obstruction, tetralogy of fallot (TOF), hypertension, reactive airways disease) 3. IH that requires systemic therapy (defined by dynamic complication scale \>3) 4. IH of the non-keratinized mucosa 5. Infants with more than one hemangioma that requires therapy 6. Hemodynamically significant cardiovascular disease, as determined by the investigator 7. Known allergy to beta blockers or vehicle 8. Heart rate \<100 beats per minute at screening visit 9. Known prenatal or postnatal diagnosis of 2nd/3rd degree atrioventricular block 10. History of Reactive Airways Disease (RAD) 11. Any condition which would make the participant, in the opinion of the investigator unsuitable for the study.

Locations (12)

Ann and Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Indiana University Health

Indianapolis, Indiana, United States

Johns Hopkins Medical Center

Baltimore, Maryland, United States

Outcomes

Primary Outcomes

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

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.

Time frame: 180 days

Secondary Outcomes

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

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.

Time frame: 180 days

Number of Participants With Partial Response of Hemangioma Volume as Measured by VAS (Visual Analog Scale) Within Each Treatment Arm

Time frame: 180 days

Comparison of Partial Response of Hemangioma Color From Baseline to 180 Days, Within Each Treatment Arm

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.

Time frame: 180 days

Change in Hemangioma Dynamic Complication Scale (HDCS)

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.

Data from ClinicalTrials.gov

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