Study to Demonstrate the Efficacy and Safety of Propranolol Oral Solution in Infants With Proliferating Infantile Hemangiomas Requiring Systemic Therapy
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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). |
- 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 ]
- -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 ]
| Enrollment: | 460 |
| Study Start Date: | January 2010 |
| Estimated Study Completion Date: | December 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 |
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
Show 71 Study Locations| 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 |
| Last Updated: | March 28, 2013 |
| 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 Propranolol Neoplasms, Vascular Tissue Neoplasms by Histologic Type Neoplasms 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 May 22, 2013