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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pierre Fabre Dermatology
ClinicalTrials.gov Identifier:
NCT01056341
First received: January 24, 2010
Last updated: May 21, 2014
Last verified: May 2014
  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:
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Success/Failure Based on the Investigator Qualitative Assessment of Complete Resolution at W48. [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    Time to first sustained improvement based on centralized qualitative assessments of paired patient-visits


Enrollment: 510
Study Start Date: January 2010
Study Completion Date: November 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01056341

  Show 59 Study Locations
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
Results First Received: April 9, 2014
Last Updated: May 21, 2014
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 July 20, 2014