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

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

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.

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.