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

January 24, 2010
May 21, 2014
January 2010
May 2012   (final data collection date for primary outcome measure)
  • 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 ]
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 ]
Complete list of historical versions of study NCT01056341 on ClinicalTrials.gov Archive Site
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
-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 ]
Not Provided
Not Provided
 
Study to Demonstrate the Efficacy and Safety of Propranolol Oral Solution in Infants With Proliferating Infantile Hemangiomas Requiring Systemic Therapy
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).

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.

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.

Interventional
Phase 2
Phase 3
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Infantile Hemangioma
  • Drug: Propranolol
    Propranolol (1 or 3 mg/kg/day for 3 or 6 months)
  • Drug: Placebo
    Treatment with placebo for 6 months
  • Experimental: Propranolol oral solution
    Intervention: Drug: Propranolol
  • Placebo Comparator: Placebo
    Intervention: Drug: Placebo

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
510
November 2013
May 2012   (final data collection date for primary outcome measure)

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
Both
35 Days to 150 Days
No
Contact information is only displayed when the study is recruiting subjects
United States,   Australia,   Canada,   Czech Republic,   France,   Germany,   Hungary,   Italy,   Lithuania,   Mexico,   New Zealand,   Peru,   Poland,   Romania,   Russian Federation,   Spain
 
NCT01056341
V00400 SB 201 Study
Yes
Pierre Fabre Dermatology
Pierre Fabre Dermatology
Not Provided
Study Chair: Christine Labreze, MD Hopital de Bordeaux
Pierre Fabre Dermatology
May 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP