Nadolol for Proliferating Infantile Hemangiomas

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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Nadolol for Proliferating Infantile Hemangiomas: A Prospective Open Label Study With a Historical Control

Conditions
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Infantile Hemangioma

Intervention / Treatment
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* Drug: Nadolol


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Intervention Group 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. Historical Control Group 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 Angiogenesis Marker Control Group Infants aged 1 month to 1 year attending dermatology clinic Exclusion Criteria: Intervention Group Patients with PHACES syndrome (proven) or suspected PHACES (plaque like hemangioma awaiting imaging). Children with history of hypersensitivity to beta blockers Children with personal history or family history of a first degree relative with asthma Children with known renal impairment Children with known cardiac conditions which may predispose to heart blocks Personal history of hypoglycemia Children on medications that may interact with beta blockers Historical Control Group: No digital photography available documenting IHs progression Angiogenesis Marker Control Group: Children with IH Children on beta blocker or systemic corticosteroids

Ages Eligible for Study
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Minimum Age: 1 Month
Maximum Age: 1 Year

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Intervention Group:<br>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 | Drug: Nadolol<br>* 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.<br>|
| No Intervention: Historical control group<br>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<br>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. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of subjects with at least 75% improvement in the extent of the hemangioma |  | Baseline, 6months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The proportion of subjects with at least 50% improvement in the extent of the hemangiomas | The proportion of subjects with at least 50% improvement in the extent of the hemangiomas | Baseline and 6 months | 
| The percentage of patients with >75% improvement in the Nadolol group compared to a historical cohort of patients receiving propranolol. | The percentage of patients with >75% improvement in the Nadolol group compared to a historical cohort of patients receiving propranolol. | 6 months | 
| The correlation between the changes in the levels of angiogenesis markers and clinical response to treatment. | The correlation between the changes in the levels of angiogenesis markers and clinical response to treatment. | 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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infantile hemangioma, nadolol