Study to Demonstrate the Efficacy and Safety of Propranolol Oral Solution in Infants With Proliferating Infantile Hemangiomas Requiring Systemic Therapy
This study is ongoing, but not recruiting participants.
Sponsor:
Pierre Fabre Dermatology
Information provided by (Responsible Party):
Pierre Fabre Dermatology
ClinicalTrials.gov Identifier:
NCT01056341
First received: January 24, 2010
Last updated: March 28, 2013
Last verified: March 2013
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Purpose
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 | Intervention | Phase |
|---|---|---|
|
Infantile Hemangioma |
Drug: Propranolol Drug: Placebo |
Phase 2 Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, 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). |
Resource links provided by NLM:
Further study details as provided by Pierre Fabre Dermatology:
Primary Outcome Measures:
- Complete/nearly complete resolution of the target IH at W24 compared to baseline based on the intra-patient blinded centralised independent qualitative assessments of W24 photographs. [ Time Frame: 6 months ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- -Success/failure based on the investigator qualitative assessment of complete resolution at W48. -Time to first sustained improvement based on centralised qualitative assessments of paired patient-visits [ Time Frame: 6 months ] [ Designated as safety issue: No ]
| Enrollment: | 460 |
| Study Start Date: | January 2010 |
| Estimated Study Completion Date: | December 2013 |
| Primary Completion Date: | May 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| 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
|
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.
Eligibility| Ages Eligible for Study: | 35 Days to 150 Days |
| Genders Eligible for Study: | Both |
| 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01056341
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Hide Study LocationsLocations
| 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, Georgia | |
| Emory University | |
| Atlanta, Georgia, United States, 30322 | |
| United States, Illinois | |
| Children's Memorial Hospital | |
| Chicago, Illinois, United States, 60614 | |
| United States, Maryland | |
| John Hopkins University School of Medicine | |
| Baltimore, Maryland, United States, 21287 | |
| United States, Massachusetts | |
| Children's Hospital Boston | |
| Boston, Massachusetts, United States, 02115 | |
| 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, Ohio | |
| Cincinnati Children's Hospital Medical Center | |
| Cincinnati, Ohio, United States, 45229 | |
| United States, Oregon | |
| Oregon Health Sciences University | |
| Portland, Oregon, United States, 97239 | |
| United States, Pennsylvania | |
| Children's Hospital of Philadelphia | |
| Philadelphia, Pennsylvania, United States, 19104 | |
| 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 | |
| Australia | |
| Eastern Clinical Research Unit - Box Hill Hospital | |
| Box Hill, Australia | |
| Royal Children's Hospital | |
| Melbourne, Australia | |
| Sydney Children's Hospital | |
| Randwick, Australia | |
| Canada | |
| 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 | |
| France | |
| Hôpital Pellegrin-Enfants | |
| Bordeaux, France, 33076 | |
| Hopital Claude Huriez | |
| Lille, France, 59037 | |
| Hôpital Femme Mère Enfant | |
| Lyon, France, 69677 | |
| CHU Hôtel Dieu | |
| Nantes, France, 44093 | |
| Hôpital Archet 2 | |
| Nice, France, 06202 | |
| Hopital Robert Debre - Consultation de Dermatologie | |
| Paris, France, 75019 | |
| Hôpital Armand Trousseau | |
| Paris, France, 75012 | |
| Hôpital Necker Enfants malades | |
| Paris, France, 75015 | |
| Hopital Nord-CHU St Etienne | |
| St-Etienne, France, 42055 | |
| Hôpital des enfants | |
| Toulouse, France, 31100 | |
| Hôpital Clocheville | |
| Tours, France, 37044 | |
| Germany | |
| 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 | |
| Hungary | |
| Heim Pál Gyermekkórház, | |
| Budapest, Hungary | |
| Italy | |
| University of Bari | |
| Bari, Italy, 70124 | |
| Clinica Dermatologica | |
| Milano, Italy, 20122 | |
| Ospedale Pediatrico Bambino Gesu | |
| Roma, Italy, 00165 | |
| Lithuania | |
| Vilnius University Children's Hospital | |
| Vilnius, Lithuania | |
| Mexico | |
| Hospital General de Mexico | |
| Mexico City, Mexico | |
| Instituto Nacional de Pediatria | |
| Mexico City, Mexico | |
| Hospital Infantil de Mexico Federico Gomez | |
| Mexico CIty, Mexico | |
| Instituto Dermatologico de Jalisco | |
| Zapopan, Mexico | |
| New Zealand | |
| Auckland Dermatology | |
| Auckland, New Zealand | |
| Waikato Clinical Research 2008 Ltd. | |
| Hamilton, New Zealand | |
| Peru | |
| Clinica Internacional | |
| Lima, Peru | |
| Instituto Nacional de Salud del Niño | |
| Lima, Peru | |
| Hospital Nacional Edgardo Rebagliati Martins | |
| Lima, Peru | |
| Poland | |
| 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 | |
| Romania | |
| I.O.M.C Alfred Rusescu | |
| Bucharest, Romania, 020395 | |
| Spitalul de Copii Dr. Victor Gomoiu | |
| Bucharest, Romania, 022102 | |
| Spitalul Clinic Urgenta pentru Copii Grigore Alexandrescu | |
| Bucharest, Romania, 011743 | |
| 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 | |
| Neonatal Intensive Care Department | |
| St-Peterburg, Russian Federation | |
| Medical Pediatric Academy | |
| St-Peterburg, Russian Federation | |
| GUZ Voronezh Regional Child Clinical Hospital | |
| Voronezh, Russian Federation | |
| Spain | |
| Servicio de Dermatologia del Hospital Infantil | |
| A Coruna, Spain, 15006 | |
| Hospital Sant Pau de Barcelona | |
| Barcelona, Spain, 08025 | |
| Hospital San Juan de Dios Barcelona | |
| Esplugues de Llobregat, Spain, 08950 | |
| Hospital La Paz | |
| Madrid, Spain, 28056 | |
| Hospital Universitario Infantil Niño Jesús | |
| Madrid, Spain, 28009 | |
| Hospital Universitario Virgen del Rocio | |
| Sevilla, Spain, 41013 | |
| Hospital Universitario de Valencia | |
| Valencia, Spain, 15006 | |
Sponsors and Collaborators
Pierre Fabre Dermatology
Investigators
| Study Chair: | Christine Labreze, MD | Hopital de Bordeaux |
More Information
Publications:
| Responsible Party: | Pierre Fabre Dermatology |
| ClinicalTrials.gov Identifier: | NCT01056341 History of Changes |
| Other Study ID Numbers: | V00400 SB 201 Study |
| Study First Received: | January 24, 2010 |
| Last Updated: | March 28, 2013 |
| Health Authority: | United States: Food and Drug Administration Canada: Health Canada France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) Germany: Federal Institute for Drugs and Medical Devices Italy: Ministry of Health Spain: Ministry of Health Romania: National Medicines Agency Peru: Instituto Nacional de Salud Mexico: Federal Commission for Sanitary Risks Protection New Zealand: Ministry of Health Australia: Department of Health and Ageing Therapeutic Goods Administration Poland: Ministry of Health Czech Republic: State Institute for Drug Control Hungary: National Institute of Pharmacy Lithuania: State Medicine Control Agency - Ministry of Health Russia: Ministry of Health of the Russian Federation |
Keywords provided by Pierre Fabre Dermatology:
|
Infantile Hemangioma Propranolol |
Additional relevant MeSH terms:
|
Hemangioma Hemangioma, Capillary Neoplasms, Vascular Tissue Neoplasms by Histologic Type Neoplasms Propranolol Anti-Arrhythmia Agents Cardiovascular Agents Therapeutic Uses |
Pharmacologic Actions Antihypertensive Agents Adrenergic beta-Antagonists Adrenergic Antagonists Adrenergic Agents Neurotransmitter Agents Molecular Mechanisms of Pharmacological Action Physiological Effects of Drugs Vasodilator Agents |
ClinicalTrials.gov processed this record on June 18, 2013