Phase 3
N=460
Study to Demonstrate the Efficacy and Safety of Propranolol Oral Solution in Infants With Proliferating Infantile Hemangiomas Requiring Systemic Therapy
Infantile Hemangioma
Bottom Line
View on ClinicalTrials.gov: NCT01056341 ↗Enrolled (actual)
460
Serious AEs
6.3%
Results posted
Jun 2014
Primary outcome: Primary: 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. — 8.0; 9.8; 37.5; 7.7 percentage of participants — p=< 0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Propranolol (Drug); Placebo (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Pierre Fabre Dermatology
- Primary completion
- May 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY 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. |
8.0; 9.8; 37.5; 7.7; 62.8 | < 0.0001 sig |
| PRIMARY 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. |
3.6; 60.4 | < 0.0001 sig |
| SECONDARY Success/Failure Based on the Investigator Qualitative Assessment of Complete Resolution at W48. |
— | — |
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.
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
Data sourced from ClinicalTrials.gov (NCT01056341). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.