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Completed

NCT Number: NCT01056341

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

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.

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Key information

Conditions

Infantile Hemangioma Hemangioma Hemangioma, Capillary Neoplasms Neoplasms by Histologic Type Neoplasms, Vascular Tissue

Age range

35 day–150 day

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Eastern Clinical Research Unit - Box Hill Hospital, Box Hill, Australia

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Propranolol

Drug

Propranolol (1 or 3 mg/kg/day for 3 or 6 months)

Placebo

Drug

Treatment with placebo for 6 months

Primary outcomes

  1. 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.

    Time frame: 6 months

  2. 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.

    Time frame: 6 months

Secondary outcomes

  1. Success/Failure Based on the Investigator Qualitative Assessment of Complete Resolution at W48.

    Time frame: 6 months

    Time to first sustained improvement based on centralized qualitative assessments of paired patient-visits

Sponsors and collaborators

Lead sponsor

Pierre Fabre Dermatology

Industry

Registry information

Official study title

A Randomised, Controlled, Multidose, Multicentre, Adaptive Phase II/III Study in Infants With Proliferating Infantile Hemangiomas (IHs) Requiring Systemic Therapy to Compare 4 Regimens of Propranolol (1 or 3 mg/kg/Day for 3 or 6 Months) to Placebo (Double Blind).

Important dates

Study start
2010
Primary completion
2012
Study completion
2013
First posted
Jan 26, 2010
Registry last updated
Dec 10, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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