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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01056341
Recruitment Status : Completed
First Posted : January 26, 2010
Results First Posted : June 24, 2014
Last Update Posted : December 10, 2015
Information provided by (Responsible Party):
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.

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 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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Birthmarks
U.S. FDA Resources

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

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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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

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

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 identifier (NCT number): NCT01056341

  Hide Study Locations
United States, California
University of California
Irvine, California, United States, 92697-1385
Lucile Packard Children's Hospital
Redwood City, California, United States, 94063-5334
Rady Children's Hospital
San Diego, California, United States, 92123
United States, Florida
Miami Children's Hospital
Miami, Florida, United States, 33155
United States, Illinois
Children's Memorial Hospital
Chicago, Illinois, United States, 60614
United States, Missouri
Cardinal Glennon Children's Hospital
St.Louis, Missouri, United States, 63104
United States, New York
State University of NY
Brooklyn, New York, United States, 11203
United States, Oregon
Oregon Health Sciences University
Portland, Oregon, United States, 97239
United States, Texas
Dell Children's Medical center
Austin, Texas, United States, 78723
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Eastern Clinical Research Unit - Box Hill Hospital
Box Hill, Australia
Royal Children's Hospital
Melbourne, Australia
Sydney Children's Hospital
Randwick, Australia
CHU St.Justine
Montreal, Canada, H3T 1C5
The Hospital for Sick Children
Toronto, Canada, M5G 1H4
Czech Republic
Children Dermatology
Brno, Czech Republic
Clinic of Dermatovenerology, University
Prague, Czech Republic
Hôpital Pellegrin-Enfants
Bordeaux, France, 33076
Hôpital Femme Mère Enfant
Lyon, France, 69677
CHU Hôtel Dieu
Nantes, France, 44093
Hôpital Archet 2
Nice, France, 06202
Hôpital Armand Trousseau
Paris, France, 75012
Hôpital Necker Enfants malades
Paris, France, 75015
Hopital Robert Debre - Consultation de Dermatologie
Paris, France, 75019
Hopital Nord-CHU St Etienne
St-Etienne, France, 42055
Hôpital des enfants
Toulouse, France, 31100
Hôpital Clocheville
Tours, France, 37044
Universitätsklinikum Freiburg
Freiburg, Germany, D-79106
Kinderkrankenhaus Wilhelmstift
Hamburg, Germany, D-22149
Universitätsklinikum Schleswig-Holstein
Kiel, Germany, 24105
Kinderchirurgische Klinik Ludwig-Maximilians-Universität
München, Germany, D-30337
Heim Pál Gyermekkórház,
Budapest, Hungary
University of Bari
Bari, Italy, 70124
Clinica Dermatologica
Milano, Italy, 20122
Vilnius University Children's Hospital
Vilnius, Lithuania
Hospital Infantil de Mexico Federico Gomez
Mexico CIty, Mexico
New Zealand
Auckland Dermatology
Auckland, New Zealand
Waikato Clinical Research 2008 Ltd.
Hamilton, New Zealand
Clinica Internacional
Lima, Peru
Hospital Nacional Edgardo Rebagliati Martins
Lima, Peru
Instituto Nacional de Salud del Niño
Lima, Peru
Klinika Chirurgii i Urologii Dzieci i Mlodziezy Akademii Medycznej
Gdansk, Poland
University Children's Hospital
Krakow, Poland
Department of Pediatric Surgery and Oncology
Lodz, Poland
Klinika Onkologii, Centrum Zdrowia Dziecka
Warszawa, Poland
Spitalul Clinic Urgenta pentru Copii Grigore Alexandrescu
Bucharest, Romania, 011743
I.O.M.C Alfred Rusescu
Bucharest, Romania, 020395
Spitalul de Copii Dr. Victor Gomoiu
Bucharest, Romania, 022102
Spitalul Clinic de Urgenta pentu Copii Sf. Maria
Iasi, Romania, 700309
Spitalul de Urgenta Copii, Louis Turcanu
Timisoara, Romania, 300011
Russian Federation
Medical University - Filatov Pediatric Hospital
Moscow, Russian Federation
Medical Pediatric Academy
St-Peterburg, Russian Federation
Neonatal Intensive Care Department
St-Peterburg, Russian Federation
Servicio de Dermatologia del Hospital Infantil
A Coruna, Spain, 15006
Hospital Sant Pau de Barcelona
Barcelona, Spain, 08025
Hospital Universitario Infantil Niño Jesús
Madrid, Spain, 28009
Hospital La Paz
Madrid, Spain, 28056
Hospital Universitario Virgen del Rocio
Sevilla, Spain, 41013
Hospital Universitario de Valencia
Valencia, Spain, 15006
Sponsors and Collaborators
Pierre Fabre Dermatology
Study Chair: Christine Labreze, MD Hopital de Bordeaux

Publications of Results:
Other Publications:
Responsible Party: Pierre Fabre Dermatology 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

Additional relevant MeSH terms:
Hemangioma, Capillary
Port-Wine Stain
Neoplasms, Vascular Tissue
Neoplasms by Histologic Type
Skin Abnormalities
Congenital Abnormalities
Skin Diseases
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