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

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ClinicalTrials.gov Identifier: NCT01056341
Recruitment Status : Completed
First Posted : January 26, 2010
Results First Posted : June 24, 2014
Last Update Posted : December 10, 2015
Sponsor:
Information provided by (Responsible Party):
Pierre Fabre Dermatology

Study Description
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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.

Condition or disease Intervention/treatment Phase
Infantile Hemangioma Drug: Propranolol Drug: Placebo Phase 2 Phase 3

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 512 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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).
Study Start Date : January 2010
Actual Primary Completion Date : May 2012
Actual Study Completion Date : November 2013

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Propranolol oral solution Drug: Propranolol
Propranolol (1 or 3 mg/kg/day for 3 or 6 months)
Placebo Comparator: Placebo Drug: Placebo
Treatment with placebo for 6 months


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  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


Eligibility Criteria
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Ages Eligible for Study:   35 Days to 150 Days   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01056341


  Show 59 Study Locations
Sponsors and Collaborators
Pierre Fabre Dermatology
Investigators
Study Chair: Christine Labreze, MD Hopital de Bordeaux
More Information
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Publications of Results:

Other Publications:

Responsible Party: Pierre Fabre Dermatology
ClinicalTrials.gov Identifier: NCT01056341     History of Changes
Other Study ID Numbers: V00400 SB 201 Study
First Posted: January 26, 2010    Key Record Dates
Results First Posted: June 24, 2014
Last Update Posted: December 10, 2015
Last Verified: November 2015

Keywords provided by Pierre Fabre Dermatology:
Infantile Hemangioma
Propranolol

Additional relevant MeSH terms:
Hemangioma
Hemangioma, Capillary
Port-Wine Stain
Neoplasms, Vascular Tissue
Neoplasms by Histologic Type
Neoplasms
Skin Abnormalities
Congenital Abnormalities
Skin Diseases
Propranolol
 Adrenergic beta-Antagonists
Adrenergic Antagonists
Adrenergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Anti-Arrhythmia Agents
Antihypertensive Agents
Vasodilator Agents