Nadolol for Proliferating Infantile Hemangiomas
The purpose of this study is to explore the efficacy and safety of Nadolol in hemangiomas of infancy.
The secondary objective is to assess the feasibility of conducting a randomized controlled trial comparing nadolol with corticosteroids and propranolol.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Nadolol for Proliferating Infantile Hemangiomas: A Prospective Open Label Study With a Historical Control|
- Proportion of subjects with at least 75% improvement in the extent of the hemangioma [ Time Frame: Baseline, 6months ] [ Designated as safety issue: No ]
- The proportion of subjects with at least 50% improvement in the extent of the hemangiomas [ Time Frame: Baseline and 6 months ] [ Designated as safety issue: No ]
- The percentage of patients with >75% improvement in the Nadolol group compared to a historical cohort of patients receiving propranolol. [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- The correlation between the changes in the levels of angiogenesis markers and clinical response to treatment. [ Time Frame: 6 months ] [ Designated as safety issue: No ]
|Study Start Date:||November 2009|
|Study Completion Date:||February 2012|
|Primary Completion Date:||May 2011 (Final data collection date for primary outcome measure)|
Experimental: Intervention Group:
The patients in this study are infants aged 1 month to 1 year of age with head and neck hemangiomas currently causing /or with impending function loss (e.g. vision, airway obstruction, feeding, etc), or hemangiomas currently causing/or with potential for facial disfigurement
Infants aged 1 month to 1 year of age with head and neck hemangiomas that received treatment with systemic propranolol in the past 2 years as a control group
Nadolol will be administered orally at home starting at 0.5 mg/kg/day divided into 2 doses. Weekly, if BP and heart rate are acceptable, the dose will be increased by 0.5 mg/kg/day up to 2 mg/kg/day.
No Intervention: Historical control group
Ten infants (1-12 months of age) treated with propranolol will be identified from a Dermatology Database. Patients will be considered as controls if they were treated with propranolol before 1 year of age and had digital photography documentation of their hemangioma.
No Intervention: Angiogenesis marker control group
The angiogenesis marker control group will consist of 6 -10 patients seen in the Dermatology clinic for conditions other than IH and not receiving corticosteroids or beta blockers.
Systemic corticosteroids are currently the most frequent used medication for treatment of problematic infantile hemangiomas (IH's). Since June 2008, systemic propranolol has been an important addition to the therapeutic options for problematic IH, allowing decreased dependence on the systemic corticosteroids. So far, we have found excellent response with propranolol with minimal short-term side effects. Studies, which compared nadolol and propranolol in children with other conditions, suggest that nadolol is safer and more efficacious than propranolol. In addition, it has better dosing schedules and less central nervous system (CNS) penetration, making it suitable even for patients with suspected or proven PHACES syndrome.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01010308
|The Hospital for Sick Children|
|Toronto, Ontario, Canada|
|Principal Investigator:||Elena Pope, MD||The Hospital for Sick Children|