Study to Demonstrate the Efficacy and Safety of Propranolol Oral Solution in Infants With Proliferating Infantile Hemangiomas Requiring Systemic Therapy
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 512
- 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
- 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)
Cardinal Glennon Children's Hospital
🇺🇸St.Louis, Missouri, United States
The Hospital for Sick Children
🇨🇦Toronto, Canada
Hopital Nord-CHU St Etienne
🇫🇷St-Etienne, France
Kinderchirurgische Klinik Ludwig-Maximilians-Universität
🇩🇪München, Germany
I.O.M.C Alfred Rusescu
🇷🇴Bucharest, Romania
Spitalul de Copii Dr. Victor Gomoiu
🇷🇴Bucharest, Romania
Children Dermatology
🇨🇿Brno, Czech Republic
Hospital Nacional Edgardo Rebagliati Martins
🇵🇪Lima, Peru
Clinic of Dermatovenerology, University
🇨🇿Prague, Czech Republic
Spitalul Clinic Urgenta pentru Copii Grigore Alexandrescu
🇷🇴Bucharest, Romania
Heim Pál Gyermekkórház,
🇭🇺Budapest, Hungary
Medical University - Filatov Pediatric Hospital
🇷🇺Moscow, Russian Federation
Klinika Chirurgii i Urologii Dzieci i Mlodziezy Akademii Medycznej
🇵🇱Gdansk, Poland
Medical Pediatric Academy
🇷🇺St-Peterburg, Russian Federation
Vilnius University Children's Hospital
🇱🇹Vilnius, Lithuania
Clinica Internacional
🇵🇪Lima, Peru
Instituto Nacional de Salud del Niño
🇵🇪Lima, Peru
University Children's Hospital
🇵🇱Krakow, Poland
Department of Pediatric Surgery and Oncology
🇵🇱Lodz, Poland
Klinika Onkologii, Centrum Zdrowia Dziecka
🇵🇱Warszawa, Poland
Spitalul de Urgenta Copii, Louis Turcanu
🇷🇴Timisoara, Romania
Spitalul Clinic de Urgenta pentu Copii Sf. Maria
🇷🇴Iasi, Romania
Neonatal Intensive Care Department
🇷🇺St-Peterburg, Russian Federation
Hôpital Armand Trousseau
🇫🇷Paris, France
Hôpital Clocheville
🇫🇷Tours, France
Royal Children's Hospital
🇦🇺Melbourne, Australia
CHU St.Justine
🇨🇦Montreal, Canada
Hôpital Pellegrin-Enfants
🇫🇷Bordeaux, France
Hôpital Archet 2
🇫🇷Nice, France
State University of NY
🇺🇸Brooklyn, New York, United States
Sydney Children's Hospital
🇦🇺Randwick, Australia
Eastern Clinical Research Unit - Box Hill Hospital
🇦🇺Box Hill, Australia
Hôpital Necker Enfants malades
🇫🇷Paris, France
CHU Hôtel Dieu
🇫🇷Nantes, France
Hospital Sant Pau de Barcelona
🇪🇸Barcelona, Spain
Hôpital Femme Mère Enfant
🇫🇷Lyon, France
Kinderkrankenhaus Wilhelmstift
🇩🇪Hamburg, Germany
Hospital Universitario Virgen del Rocio
🇪🇸Sevilla, Spain
Hopital Robert Debre - Consultation de Dermatologie
🇫🇷Paris, France
Hospital La Paz
🇪🇸Madrid, Spain
Universitätsklinikum Freiburg
🇩🇪Freiburg, Germany
Hospital Infantil de Mexico Federico Gomez
🇲🇽Mexico CIty, Mexico
University of Bari
🇮🇹Bari, Italy
Hôpital des enfants
🇫🇷Toulouse, France
Waikato Clinical Research 2008 Ltd.
🇳🇿Hamilton, New Zealand
Clinica Dermatologica
🇮🇹Milano, Italy
Hospital Universitario de Valencia
🇪🇸Valencia, Spain
Servicio de Dermatologia del Hospital Infantil
🇪🇸A Coruna, Spain
Universitätsklinikum Schleswig-Holstein
🇩🇪Kiel, Germany
Hospital Universitario Infantil Niño Jesús
🇪🇸Madrid, Spain
Auckland Dermatology
🇳🇿Auckland, New Zealand
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
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