Topical Timolol for Infantile Hemangioma in Early Proliferative Phase

Phase 2

Completed

• Conditions: Hemangioma
• Interventions: Drug: Placebo; Drug: Timolol

• Registration Number: NCT02731287
• Lead Sponsor: Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau

Brief Summary

Infantile hemangioma (IH) is the most common benign vascular tumor in pediatric population. Oral propranolol is the treatment of choice for complicated hemangiomas. Topical timolol, a non-selective beta-blocker, is an emerging treatment which has been reported to be effective and safe for the treatment of IH, especially for superficial hemangiomas. Investigators hypothesize that treatment with topical timolol in the first two months of life, before the proliferative phase or in early proliferative phase, may prevent from further growing and the need to treat with oral propranolol.

Detailed Description

A multicentric, randomized, double-blind, placebo-controlled, phase II clinical trial to evaluate the efficacy and safety of 0.5% timolol maleate solution for the early treatment of infantile hemangioma.

Patients with less of 60 days of life with focal or segmental hemangiomas, superficial, mixed or abortive will be treated with topical timolol 0.5% twice a day for 24 weeks.

Changes in lesion size, color and thickness will be evaluated from photographs taken at 2,4,8,12,24 and 36 weeks. Vital signs and side effects will be recorded at each visit. Response to treatment will be evaluated by a blinded investigator in a semiquantitative scale (complete resolution, improve, stabilization, worsening).

Study Timeline

• Study Start: 2014-03
• Study First Submitted: 2015-11-17
• Study First Submitted QC: 2016-04-06
• Study First Posted: 2016-04-07
• Primary Completion: 2017-01
• Study Completion: 2017-03
• Status Verified: 2018-11
• Last Update Submitted: 2018-11-13
• Last Update Posted: 2018-11-14

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 70

Inclusion Criteria

• Written informed consent for study participation and the use of the patient's photographs from the patient's parent(s) or guardian(s),

• The patient is between 10 and 60 days of age at the time of enrollment,

• The patient should have:

  • at least one focal or segmental hemangioma, mixed or superficial, present anywhere on the body of at least 0.3 cm of minimum diameter and not greater of 5 cm.
  • abortive or minimal growth hemangioma
  • Infantile hemangioma precursors

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Exclusion Criteria

• Patients > 60 days of age
• Complicated hemangiomas (life-threatening, function-threatening, or ulcerated)
• Children who have previously received systemic, intra-lesional or topical corticosteroids, imiquimod, vincristine, alpha-interferon, propranolol or other beta blockers, PDL laser.
• Patients with PHACES, LUMBAR, SACRAL or PELVIS syndrome.
• Internal involvement hemangiomas (liver, GI tract, larynx)
• Children with a personal history of asthma or cardiac conditions that may predispose to heart block.
• Children with congenital birth defects
• Children with Cancer (leukemia, sarcoma, neuroblastoma, retinoblastoma)
• Children with a history of hypersensitivity to beta blockers or excipients.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Saline topical solution; 50% of the patients treated (randomized)
Timolol	Timolol	Timolol maleate 0.5% topical solution; 50% of the patients treated (randomized)

Primary Outcome Measures

Name	Time	Method
Complete or almost complete resolution of the lesions	24 weeks	Efficacy to treatment at 24 weeks, categorized as complete or almost complete resolution of the lesions (almost complete resolution defined as faint macular erythema, telangiectasia and no palpable component).; This will be generated by independent blind evaluations by comparing photographs at baseline and 24 weeks. The investigators will score this improvement into one of the following categories (complete resolution, improve, stabilization, worsening)

Secondary Outcome Measures

Name	Time	Method
Adverse reactions	24 weeks	Evaluation of safety and adverse reactions. The investigators will monitor vital signs of the patients every follow-up visit and ask the caretakers about side effects and adverse reactions to treatment.

Trial Locations

Locations (2)

Hospital de la Santa Creu i Sant Pau; 🇪🇸 Barcelona, Spain

Hospital Virgen del Rocío; 🇪🇸 Sevilla, Spain