Study to Demonstrate the Efficacy and Safety of Propranolol Oral Solution in Infants With Proliferating Infantile Hemangiomas Requiring Systemic Therapy

Phase 2

Completed

• Conditions: Infantile Hemangioma
• Interventions: Drug: Placebo; Drug: Propranolol

• Registration Number: NCT01056341
• Lead Sponsor: Pierre Fabre Dermatology

Brief Summary

There is an unsatisfied medical need for a first-line treatment of proliferating IHs with a good benefit/risk profile. Based on the recent findings of encouraging results obtained with propranolol in a series of infants with severe Infantile Hemangioma (IH), propranolol is expected to be of significant benefit in the management of the condition. The present study has been designed to confirm efficacy of propranolol in severe IH by demonstrating superiority over placebo and to document the safety profile of propranolol in this indication.

Detailed Description

Primary objective The primary objective of this study is to identify the appropriate dose and duration of propranolol treatment and demonstrate its superiority over placebo based on the complete/nearly complete resolution of target IH at W24.

Study Timeline

• Study Start: 2010-01
• Study First Submitted: 2010-01-24
• Study First Submitted QC: 2010-01-24
• Study First Posted: 2010-01-26
• Primary Completion: 2012-05
• Disposition First Submitted: 2013-03-27
• Disposition First Submitted QC: 2013-03-28
• Disposition First Posted: 2013-04-04
• Study Completion: 2013-11
• Results First Submitted: 2014-04-09
• Results First Submitted QC: 2014-05-21
• Results First Posted: 2014-06-24
• Status Verified: 2015-11
• Last Update Submitted: 2015-11-12
• Last Update Posted: 2015-12-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 512

Inclusion Criteria

• Proliferating IH (target hemangioma) requiring systemic therapy anywhere on the body except on the diaper area with largest diameter of at least 1.5 cm

Exclusion Criteria

• The patient presents with one or more of the following medical conditions: Congenital hemangioma; Kasabach-Merritt syndrome; bronchial asthma; bronchospasm; hypoglycaemia (< 40 mg/dl or at risk); untreated phaeochromocytoma; hypotension (< 50/30 mmHg); second or third degree heart block; cardiogenic shock; metabolic acidosis; bradycardia (< 80 bpm); severe peripheral arterial circulatory disturbances; Raynaud's phenomenon; sick sinus syndrome; uncontrolled heart failure or Prinzmetal's angina; documented PHACES syndrome with central nervous system involvement

• The patient has previously been treated for IH, including any surgical and/or medical procedures (e.g. laser therapy)

• The patient is known to have a hypersensitivity to propranolol and/or any other beta-blockers

• One or more of the following types of IH are present:

  • Life-threatening IH
  • Function-threatening IH (e.g. those causing impairment of vision, respiratory compromise caused by airway lesions, etc.)
  • Ulcerated IH (whatever the localisation) with pain and lack of response to simple wound care measures

• The patient was born prematurely and has not yet reached his/her term equivalent age (e.g. an infant born 2 months prematurely cannot be included before the age of 2 months)

• LVEF (left ventricular systolic function) ≤40% and/or cardiomyopathy and/or hereditary arrhythmia disorder

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	-
Propranolol oral solution	Propranolol	-

Primary Outcome Measures

Name	Time	Method
Interim Analysis : Complete/Nearly Complete Resolution of the Target Infantile Hemangioma at Week 24 Compared to Baseline Based on the Intra-patient Blinded Centralized Independent Qualitative Assessments of Week 24 Photographs.	6 months
Primary Analysis : Complete/Nearly Complete Resolution of the Target Infantile Hemangioma at W24 Compared to Baseline Based on the Intra-patient Blinded Centralized Independent Qualitative Assessments of W24 Photographs.	6 months

Secondary Outcome Measures

Name	Time	Method
Success/Failure Based on the Investigator Qualitative Assessment of Complete Resolution at W48.	6 months	Time to first sustained improvement based on centralized qualitative assessments of paired patient-visits

Trial Locations

Locations (59)

University of California; 🇺🇸 Irvine, California, United States

Lucile Packard Children's Hospital; 🇺🇸 Redwood City, California, United States

Rady Children's Hospital; 🇺🇸 San Diego, California, United States

Miami Children's Hospital; 🇺🇸 Miami, Florida, United States

Children's Memorial Hospital; 🇺🇸 Chicago, Illinois, United States

Cardinal Glennon Children's Hospital; 🇺🇸 St.Louis, Missouri, United States

State University of NY; 🇺🇸 Brooklyn, New York, United States

Oregon Health Sciences University; 🇺🇸 Portland, Oregon, United States

Dell Children's Medical center; 🇺🇸 Austin, Texas, United States

Seattle Children's Hospital; 🇺🇸 Seattle, Washington, United States

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