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

NCT01056341 | Completed Phase 2 Results DMC

• 1 other identifier: V00400 SB 201 Study
• Study Type: interventional
• Target: 512
• Locations: 16 countries
• Sites: 59

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.

Conditions

Infantile Hemangioma

Keywords

Infantile Hemangioma; Propranolol

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (1)

• Success/Failure Based on the Investigator Qualitative Assessment of Complete Resolution at W48.

  6 months

Study Arms (2)

Propranolol oral solution

EXPERIMENTAL

Drug: Propranolol

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Propranolol DRUG

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

Propranolol oral solution

Placebo DRUG

Treatment with placebo for 6 months

Placebo

Eligibility Criteria

• Age: 35 Days - 150 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• \- 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

Sponsors & Collaborators

• Pierre Fabre Dermatology: lead

Study Sites (59)

University of California

Irvine, California, 92697-1385, United States

Location

Lucile Packard Children's Hospital

Redwood City, California, 94063-5334, United States

Location

Rady Children's Hospital

San Diego, California, 92123, United States

Location

Miami Children's Hospital

Miami, Florida, 33155, United States

Location

Children's Memorial Hospital

Chicago, Illinois, 60614, United States

Location

Cardinal Glennon Children's Hospital

St Louis, Missouri, 63104, United States

Location

State University of NY

Brooklyn, New York, 11203, United States

Location

Oregon Health Sciences University

Portland, Oregon, 97239, United States

Location

Dell Children's Medical center

Austin, Texas, 78723, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Eastern Clinical Research Unit - Box Hill Hospital

Box Hill, Australia

Location

Royal Children's Hospital

Melbourne, Australia

Location

Sydney Children's Hospital

Randwick, Australia

Location

CHU St.Justine

Montreal, H3T 1C5, Canada

Location

The Hospital for Sick Children

Toronto, M5G 1H4, Canada

Location

Children Dermatology

Brno, Czechia

Location

Clinic of Dermatovenerology, University

Prague, Czechia

Location

Hôpital Pellegrin-Enfants

Bordeaux, 33076, France

Location

Hôpital Femme Mère Enfant

Lyon, 69677, France

Location

CHU Hôtel Dieu

Nantes, 44093, France

Location

Hôpital Archet 2

Nice, 06202, France

Location

Hôpital Armand Trousseau

Paris, 75012, France

Location

Hôpital Necker Enfants malades

Paris, 75015, France

Location

Hopital Robert Debre - Consultation de Dermatologie

Paris, 75019, France

Location

Hopital Nord-CHU St Etienne

Saint-Etienne, 42055, France

Location

Hôpital des enfants

Toulouse, 31100, France

Location

Hôpital Clocheville

Tours, 37044, France

Location

Universitätsklinikum Freiburg

Freiburg im Breisgau, D-79106, Germany

Location

Kinderkrankenhaus Wilhelmstift

Hamburg, D-22149, Germany

Location

Universitätsklinikum Schleswig-Holstein

Kiel, 24105, Germany

Location

Kinderchirurgische Klinik Ludwig-Maximilians-Universität

München, D-30337, Germany

Location

Heim Pál Gyermekkórház,

Budapest, Hungary

Location

University of Bari

Bari, 70124, Italy

Location

Clinica Dermatologica

Milan, 20122, Italy

Location

Vilnius University Children's Hospital

Vilnius, Lithuania

Location

Hospital Infantil de Mexico Federico Gomez

Mexico City, Mexico

Location

Auckland Dermatology

Auckland, New Zealand

Location

Waikato Clinical Research 2008 Ltd.

Hamilton, New Zealand

Location

Clinica Internacional

Lima, Peru

Location

Hospital Nacional Edgardo Rebagliati Martins

Lima, Peru

Location

Instituto Nacional de Salud del Niño

Lima, Peru

Location

Klinika Chirurgii i Urologii Dzieci i Mlodziezy Akademii Medycznej

Gdansk, Poland

Location

University Children's Hospital

Krakow, Poland

Location

Department of Pediatric Surgery and Oncology

Lodz, Poland

Location

Klinika Onkologii, Centrum Zdrowia Dziecka

Warsaw, Poland

Location

Spitalul Clinic Urgenta pentru Copii Grigore Alexandrescu

Bucharest, 011743, Romania

Location

I.O.M.C Alfred Rusescu

Bucharest, 020395, Romania

Location

Spitalul de Copii Dr. Victor Gomoiu

Bucharest, 022102, Romania

Location

Spitalul Clinic de Urgenta pentu Copii Sf. Maria

Iași, 700309, Romania

Location

Spitalul de Urgenta Copii, Louis Turcanu

Timișoara, 300011, Romania

Location

Medical University - Filatov Pediatric Hospital

Moscow, Russia

Location

Medical Pediatric Academy

St-Peterburg, Russia

Location

Neonatal Intensive Care Department

St-Peterburg, Russia

Location

Servicio de Dermatologia del Hospital Infantil

A Coruña, 15006, Spain

Location

Hospital Sant Pau de Barcelona

Barcelona, 08025, Spain

Location

Hospital Universitario Infantil Niño Jesús

Madrid, 28009, Spain

Location

Hospital La Paz

Madrid, 28056, Spain

Location

Hospital Universitario Virgen del Rocio

Seville, 41013, Spain

Location

Hospital Universitario de Valencia

Valencia, 15006, Spain

Location

Related Publications (3)

• 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.

  PMID: 18550886 BACKGROUND

• 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.

  PMID: 19706583 BACKGROUND

• 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.

  PMID: 25693013 RESULT

MeSH Terms

Conditions

Hemangioma, Capillary

Interventions

Propranolol

Condition Hierarchy (Ancestors)

Hemangioma; Neoplasms, Vascular Tissue; Neoplasms by Histologic Type; Neoplasms

Intervention Hierarchy (Ancestors)

Phenoxypropanolamines; Propanolamines; Amino Alcohols; Alcohols; Organic Chemicals; Propanols; Amines; Naphthalenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Polycyclic Compounds

Results Point of Contact

• Title: Dr. Jean-Jacques VOISARD - General Manager -
• Organization: Pierre Fabre Dermatologie

jean.jacques.voisard@pierre-fabre.com

+33 5 63 58 88 00

Study Officials

• Christine Labreze, MD

  Hopital de Bordeaux

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 24, 2010
• First Posted: January 26, 2010
• Study Start: January 1, 2010
• Primary Completion: May 1, 2012
• Study Completion: November 1, 2013
• Last Updated: December 10, 2015
• Results First Posted: June 24, 2014

Record last verified: 2015-11

Locations

IT (2); HU (1); ES (6); PL (4); DE (4); ME (1); CA (2); PE (3); FR (10); LI (1); CZ (2); NZ (2); US (10); RO (5); RU (3); AU (3)