Efficacy, Safety and Pharmacokinetics of Topical Timolol in Infants With Infantile Hemangioma (IH) (TIM01)

Kanecia Obie Zimmerman

Status and phase: Completed; Phase 2

Conditions: Infantile Hemangioma

Treatments: Drug: 0.5% Timolol Maleate Gel Forming Solution; Drug: 0.25% Timolol Maleate Gel Forming Solution

Study type: Interventional

Funder types: Other; Industry; NIH

Identifiers

NCT02913612

Pro00068212

Details and patient eligibility

About

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

Full 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.

Enrollment: 105 patients

Sex: All

Ages: Under 84 days old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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105 participants in 3 patient groups

0.25% Timolol Treatment

Experimental group

Description:

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.

Treatment:

Drug: 0.25% Timolol Maleate Gel Forming Solution

0.5% Timolol Treatment

Experimental group

Description:

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.

Treatment:

Drug: 0.5% Timolol Maleate Gel Forming Solution

Non-Intervention Group

No Intervention group

Description:

Subjects assigned to this group will not receive treatment. The subject will only be photographed on the same schedule as the intervention group.