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

November 12, 2015 updated by: Pierre Fabre Dermatology

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).

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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

Enrollment (Actual)

512

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 month to 4 months (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
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.
Time Frame: 6 months
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Pierre Fabre Dermatology

Investigators

  • Study Chair: Christine Labreze, MD, Hopital de Bordeaux

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2010

Primary Completion (Actual)

May 1, 2012

Study Completion (Actual)

November 1, 2013

Study Registration Dates

First Submitted

January 24, 2010

First Submitted That Met QC Criteria

January 24, 2010

First Posted (Estimate)

January 26, 2010

Study Record Updates

Last Update Posted (Estimate)

December 10, 2015

Last Update Submitted That Met QC Criteria

November 12, 2015

Last Verified

November 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • V00400 SB 201 Study

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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