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

Primary Purpose

Infantile Hemangioma

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Propranolol
Placebo
Sponsored by
Pierre Fabre Dermatology
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Infantile Hemangioma focused on measuring Infantile Hemangioma, Propranolol

Eligibility Criteria

35 Days - 150 Days (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of California
  • Lucile Packard Children's Hospital
  • Rady Children's Hospital
  • Miami Children's Hospital
  • Children's Memorial Hospital
  • Cardinal Glennon Children's Hospital
  • State University of NY
  • Oregon Health Sciences University
  • Dell Children's Medical center
  • Seattle Children's Hospital
  • Eastern Clinical Research Unit - Box Hill Hospital
  • Royal Children's Hospital
  • Sydney Children's Hospital
  • CHU St.Justine
  • The Hospital for Sick Children
  • Children Dermatology
  • Clinic of Dermatovenerology, University
  • Hôpital Pellegrin-Enfants
  • Hôpital Femme Mère Enfant
  • CHU Hôtel Dieu
  • Hôpital Archet 2
  • Hôpital Armand Trousseau
  • Hôpital Necker Enfants malades
  • Hopital Robert Debre - Consultation de Dermatologie
  • Hopital Nord-CHU St Etienne
  • Hôpital des enfants
  • Hôpital Clocheville
  • Universitätsklinikum Freiburg
  • Kinderkrankenhaus Wilhelmstift
  • Universitätsklinikum Schleswig-Holstein
  • Kinderchirurgische Klinik Ludwig-Maximilians-Universität
  • Heim Pál Gyermekkórház,
  • University of Bari
  • Clinica Dermatologica
  • Vilnius University Children's Hospital
  • Hospital Infantil de Mexico Federico Gomez
  • Auckland Dermatology
  • Waikato Clinical Research 2008 Ltd.
  • Clinica Internacional
  • Hospital Nacional Edgardo Rebagliati Martins
  • Instituto Nacional de Salud del Niño
  • Klinika Chirurgii i Urologii Dzieci i Mlodziezy Akademii Medycznej
  • University Children's Hospital
  • Department of Pediatric Surgery and Oncology
  • Klinika Onkologii, Centrum Zdrowia Dziecka
  • Spitalul Clinic Urgenta pentru Copii Grigore Alexandrescu
  • I.O.M.C Alfred Rusescu
  • Spitalul de Copii Dr. Victor Gomoiu
  • Spitalul Clinic de Urgenta pentu Copii Sf. Maria
  • Spitalul de Urgenta Copii, Louis Turcanu
  • Medical University - Filatov Pediatric Hospital
  • Medical Pediatric Academy
  • Neonatal Intensive Care Department
  • Servicio de Dermatologia del Hospital Infantil
  • Hospital Sant Pau de Barcelona
  • Hospital Universitario Infantil Niño Jesús
  • Hospital La Paz
  • Hospital Universitario Virgen del Rocio
  • Hospital Universitario de Valencia

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Propranolol oral solution

Placebo

Arm Description

Outcomes

Primary Outcome Measures

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.
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.

Secondary Outcome Measures

Success/Failure Based on the Investigator Qualitative Assessment of Complete Resolution at W48.
Time to first sustained improvement based on centralized qualitative assessments of paired patient-visits

Full Information

First Posted
January 24, 2010
Last Updated
November 12, 2015
Sponsor
Pierre Fabre Dermatology
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1. Study Identification

Unique Protocol Identification Number
NCT01056341
Brief Title
Study to Demonstrate the Efficacy and Safety of Propranolol Oral Solution in Infants With Proliferating Infantile Hemangiomas Requiring Systemic Therapy
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).
Study Type
Interventional

2. Study Status

Record Verification Date
November 2015
Overall Recruitment Status
Completed
Study Start Date
January 2010 (undefined)
Primary Completion Date
May 2012 (Actual)
Study Completion Date
November 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pierre Fabre Dermatology

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Infantile Hemangioma
Keywords
Infantile Hemangioma, Propranolol

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
512 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Propranolol oral solution
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
Propranolol
Intervention Description
Propranolol (1 or 3 mg/kg/day for 3 or 6 months)
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Treatment with placebo for 6 months
Primary Outcome Measure Information:
Title
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
Title
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
Secondary Outcome Measure Information:
Title
Success/Failure Based on the Investigator Qualitative Assessment of Complete Resolution at W48.
Description
Time to first sustained improvement based on centralized qualitative assessments of paired patient-visits
Time Frame
6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
35 Days
Maximum Age & Unit of Time
150 Days
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Christine Labreze, MD
Organizational Affiliation
Hopital de Bordeaux
Official's Role
Study Chair
Facility Information:
Facility Name
University of California
City
Irvine
State/Province
California
ZIP/Postal Code
92697-1385
Country
United States
Facility Name
Lucile Packard Children's Hospital
City
Redwood City
State/Province
California
ZIP/Postal Code
94063-5334
Country
United States
Facility Name
Rady Children's Hospital
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
Miami Children's Hospital
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
Facility Name
Children's Memorial Hospital
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60614
Country
United States
Facility Name
Cardinal Glennon Children's Hospital
City
St.Louis
State/Province
Missouri
ZIP/Postal Code
63104
Country
United States
Facility Name
State University of NY
City
Brooklyn
State/Province
New York
ZIP/Postal Code
11203
Country
United States
Facility Name
Oregon Health Sciences University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Dell Children's Medical center
City
Austin
State/Province
Texas
ZIP/Postal Code
78723
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
Eastern Clinical Research Unit - Box Hill Hospital
City
Box Hill
Country
Australia
Facility Name
Royal Children's Hospital
City
Melbourne
Country
Australia
Facility Name
Sydney Children's Hospital
City
Randwick
Country
Australia
Facility Name
CHU St.Justine
City
Montreal
ZIP/Postal Code
H3T 1C5
Country
Canada
Facility Name
The Hospital for Sick Children
City
Toronto
ZIP/Postal Code
M5G 1H4
Country
Canada
Facility Name
Children Dermatology
City
Brno
Country
Czech Republic
Facility Name
Clinic of Dermatovenerology, University
City
Prague
Country
Czech Republic
Facility Name
Hôpital Pellegrin-Enfants
City
Bordeaux
ZIP/Postal Code
33076
Country
France
Facility Name
Hôpital Femme Mère Enfant
City
Lyon
ZIP/Postal Code
69677
Country
France
Facility Name
CHU Hôtel Dieu
City
Nantes
ZIP/Postal Code
44093
Country
France
Facility Name
Hôpital Archet 2
City
Nice
ZIP/Postal Code
06202
Country
France
Facility Name
Hôpital Armand Trousseau
City
Paris
ZIP/Postal Code
75012
Country
France
Facility Name
Hôpital Necker Enfants malades
City
Paris
ZIP/Postal Code
75015
Country
France
Facility Name
Hopital Robert Debre - Consultation de Dermatologie
City
Paris
ZIP/Postal Code
75019
Country
France
Facility Name
Hopital Nord-CHU St Etienne
City
St-Etienne
ZIP/Postal Code
42055
Country
France
Facility Name
Hôpital des enfants
City
Toulouse
ZIP/Postal Code
31100
Country
France
Facility Name
Hôpital Clocheville
City
Tours
ZIP/Postal Code
37044
Country
France
Facility Name
Universitätsklinikum Freiburg
City
Freiburg
ZIP/Postal Code
D-79106
Country
Germany
Facility Name
Kinderkrankenhaus Wilhelmstift
City
Hamburg
ZIP/Postal Code
D-22149
Country
Germany
Facility Name
Universitätsklinikum Schleswig-Holstein
City
Kiel
ZIP/Postal Code
24105
Country
Germany
Facility Name
Kinderchirurgische Klinik Ludwig-Maximilians-Universität
City
München
ZIP/Postal Code
D-30337
Country
Germany
Facility Name
Heim Pál Gyermekkórház,
City
Budapest
Country
Hungary
Facility Name
University of Bari
City
Bari
ZIP/Postal Code
70124
Country
Italy
Facility Name
Clinica Dermatologica
City
Milano
ZIP/Postal Code
20122
Country
Italy
Facility Name
Vilnius University Children's Hospital
City
Vilnius
Country
Lithuania
Facility Name
Hospital Infantil de Mexico Federico Gomez
City
Mexico CIty
Country
Mexico
Facility Name
Auckland Dermatology
City
Auckland
Country
New Zealand
Facility Name
Waikato Clinical Research 2008 Ltd.
City
Hamilton
Country
New Zealand
Facility Name
Clinica Internacional
City
Lima
Country
Peru
Facility Name
Hospital Nacional Edgardo Rebagliati Martins
City
Lima
Country
Peru
Facility Name
Instituto Nacional de Salud del Niño
City
Lima
Country
Peru
Facility Name
Klinika Chirurgii i Urologii Dzieci i Mlodziezy Akademii Medycznej
City
Gdansk
Country
Poland
Facility Name
University Children's Hospital
City
Krakow
Country
Poland
Facility Name
Department of Pediatric Surgery and Oncology
City
Lodz
Country
Poland
Facility Name
Klinika Onkologii, Centrum Zdrowia Dziecka
City
Warszawa
Country
Poland
Facility Name
Spitalul Clinic Urgenta pentru Copii Grigore Alexandrescu
City
Bucharest
ZIP/Postal Code
011743
Country
Romania
Facility Name
I.O.M.C Alfred Rusescu
City
Bucharest
ZIP/Postal Code
020395
Country
Romania
Facility Name
Spitalul de Copii Dr. Victor Gomoiu
City
Bucharest
ZIP/Postal Code
022102
Country
Romania
Facility Name
Spitalul Clinic de Urgenta pentu Copii Sf. Maria
City
Iasi
ZIP/Postal Code
700309
Country
Romania
Facility Name
Spitalul de Urgenta Copii, Louis Turcanu
City
Timisoara
ZIP/Postal Code
300011
Country
Romania
Facility Name
Medical University - Filatov Pediatric Hospital
City
Moscow
Country
Russian Federation
Facility Name
Medical Pediatric Academy
City
St-Peterburg
Country
Russian Federation
Facility Name
Neonatal Intensive Care Department
City
St-Peterburg
Country
Russian Federation
Facility Name
Servicio de Dermatologia del Hospital Infantil
City
A Coruna
ZIP/Postal Code
15006
Country
Spain
Facility Name
Hospital Sant Pau de Barcelona
City
Barcelona
ZIP/Postal Code
08025
Country
Spain
Facility Name
Hospital Universitario Infantil Niño Jesús
City
Madrid
ZIP/Postal Code
28009
Country
Spain
Facility Name
Hospital La Paz
City
Madrid
ZIP/Postal Code
28056
Country
Spain
Facility Name
Hospital Universitario Virgen del Rocio
City
Sevilla
ZIP/Postal Code
41013
Country
Spain
Facility Name
Hospital Universitario de Valencia
City
Valencia
ZIP/Postal Code
15006
Country
Spain

12. IPD Sharing Statement

Citations:
PubMed Identifier
18550886
Citation
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.
Results Reference
background
PubMed Identifier
19706583
Citation
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.
Results Reference
background
PubMed Identifier
25693013
Citation
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.
Results Reference
result

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

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