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

NCT ID: NCT01056341

Last Updated: 2015-12-10

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 512 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-01-31
• Study Completion Date: 2013-11-30

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.

Conditions

Infantile Hemangioma

Keywords

Infantile Hemangioma; Propranolol

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Propranolol oral solution

Group Type: EXPERIMENTAL

Propranolol

Intervention Type: DRUG

Propranolol (1 or 3 mg/kg/day for 3 or 6 months)

Placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Treatment with placebo for 6 months

Interventions

Propranolol

Propranolol (1 or 3 mg/kg/day for 3 or 6 months)

Intervention Type: DRUG

Placebo

Treatment with placebo for 6 months

Intervention Type: DRUG

Eligibility 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

• Minimum Eligible Age: 35 Days
• Maximum Eligible Age: 150 Days
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Pierre Fabre Dermatology

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Christine Labreze, MD

Role: STUDY_CHAIR

Hopital de Bordeaux

Locations

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

Cardinal Glennon Children's Hospital

St Louis, Missouri, United States

State University of NY

Brooklyn, New York, 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

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

The Hospital for Sick Children

Toronto, , Canada

Children Dermatology

Brno, , Czechia

Clinic of Dermatovenerology, University

Prague, , Czechia

Hôpital Pellegrin-Enfants

Bordeaux, , France

Hôpital Femme Mère Enfant

Lyon, , France

CHU Hôtel Dieu

Nantes, , France

Hôpital Archet 2

Nice, , France

Hôpital Armand Trousseau

Paris, , France

Hôpital Necker Enfants malades

Paris, , France

Hopital Robert Debre - Consultation de Dermatologie

Paris, , France

Hopital Nord-CHU St Etienne

Saint-Etienne, , France

Hôpital des enfants

Toulouse, , France

Hôpital Clocheville

Tours, , France

Universitätsklinikum Freiburg

Freiburg im Breisgau, , Germany

Kinderkrankenhaus Wilhelmstift

Hamburg, , Germany

Universitätsklinikum Schleswig-Holstein

Kiel, , Germany

Kinderchirurgische Klinik Ludwig-Maximilians-Universität

München, , Germany

Heim Pál Gyermekkórház,

Budapest, , Hungary

University of Bari

Bari, , Italy

Clinica Dermatologica

Milan, , Italy

Vilnius University Children's Hospital

Vilnius, , Lithuania

Hospital Infantil de Mexico Federico Gomez

Mexico City, , Mexico

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

Warsaw, , Poland

Spitalul Clinic Urgenta pentru Copii Grigore Alexandrescu

Bucharest, , Romania

I.O.M.C Alfred Rusescu

Bucharest, , Romania

Spitalul de Copii Dr. Victor Gomoiu

Bucharest, , Romania

Spitalul Clinic de Urgenta pentu Copii Sf. Maria

Iași, , Romania

Spitalul de Urgenta Copii, Louis Turcanu

Timișoara, , Romania

Medical University - Filatov Pediatric Hospital

Moscow, , Russia

Medical Pediatric Academy

St-Peterburg, , Russia

Neonatal Intensive Care Department

St-Peterburg, , Russia

Servicio de Dermatologia del Hospital Infantil

A Coruña, , Spain

Hospital Sant Pau de Barcelona

Barcelona, , Spain

Hospital Universitario Infantil Niño Jesús

Madrid, , Spain

Hospital La Paz

Madrid, , Spain

Hospital Universitario Virgen del Rocio

Seville, , Spain

Hospital Universitario de Valencia

Valencia, , Spain

Countries

United States; Australia; Canada; Czechia; France; Germany; Hungary; Italy; Lithuania; Mexico; New Zealand; Peru; Poland; Romania; Russia; Spain

References

Leaute-Labreze C, Dumas de la Roque E, Hubiche T, Boralevi F, Thambo JB, Taieb A. Propranolol for severe hemangiomas of infancy. N Engl J Med. 2008 Jun 12;358(24):2649-51. doi: 10.1056/NEJMc0708819. No abstract available.

Reference Type: BACKGROUND

PMID: 18550886 (View on PubMed)

Sans V, de la Roque ED, Berge J, Grenier N, Boralevi F, Mazereeuw-Hautier J, Lipsker D, Dupuis E, Ezzedine K, Vergnes P, Taieb A, Leaute-Labreze C. Propranolol for severe infantile hemangiomas: follow-up report. Pediatrics. 2009 Sep;124(3):e423-31. doi: 10.1542/peds.2008-3458. Epub 2009 Aug 10.

Reference Type: BACKGROUND

PMID: 19706583 (View on PubMed)

Leaute-Labreze C, Hoeger P, Mazereeuw-Hautier J, Guibaud L, Baselga E, Posiunas G, Phillips RJ, Caceres H, Lopez Gutierrez JC, Ballona R, Friedlander SF, Powell J, Perek D, Metz B, Barbarot S, Maruani A, Szalai ZZ, Krol A, Boccara O, Foelster-Holst R, Febrer Bosch MI, Su J, Buckova H, Torrelo A, Cambazard F, Grantzow R, Wargon O, Wyrzykowski D, Roessler J, Bernabeu-Wittel J, Valencia AM, Przewratil P, Glick S, Pope E, Birchall N, Benjamin L, Mancini AJ, Vabres P, Souteyrand P, Frieden IJ, Berul CI, Mehta CR, Prey S, Boralevi F, Morgan CC, Heritier S, Delarue A, Voisard JJ. A randomized, controlled trial of oral propranolol in infantile hemangioma. N Engl J Med. 2015 Feb 19;372(8):735-46. doi: 10.1056/NEJMoa1404710.

Reference Type: RESULT

PMID: 25693013 (View on PubMed)

Other Identifiers

V00400 SB 201 Study

Identifier Type: -

Identifier Source: org_study_id