Clinical trial NCT02731287: Topical Timolol for Infantile Hemangioma in Early Proliferative Phase
Official title: Efficacy and Safety of Topical Timolol Maleate 0.5% Solution for Superficial Infantile Hemangioma in Early Proliferative Phase. A Randomized Clinical Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hemangioma
Lead sponsor: Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
Sponsor class: OTHER
First posted: 2016-04-07
Enrollment: 70
Sex: ALL
Minimum age (years): 10
Maximum age (years): 60
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).

Eligibility criteria: 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

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.

Primary outcomes:
measure: Complete or almost complete resolution of the lesions
timeFrame: 24 weeks
Secondary outcomes:
measure: Adverse reactions
timeFrame: 24 weeks
Other outcomes:
Locations:
city: Barcelona
country: Spain
facility: Hospital de la Santa Creu i Sant Pau
city: Sevilla
country: Spain
facility: Hospital Virgen del Rocío
Study officials:
affiliation: Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
name: Eulalia Baselga, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: