Nadolol for Proliferating Infantile Hemangiomas

• ClinicalTrials.gov Identifier: NCT01010308
• Recruitment Status: Completed
• First Posted: November 10, 2009
• Last Update Posted: August 2, 2013
• Sponsor: The Hospital for Sick Children
• Information provided by (Responsible Party): Elena Pope, The Hospital for Sick Children

Tracking Information

• First Submitted Date: November 8, 2009
• First Posted Date: November 10, 2009
• Last Update Posted Date: August 2, 2013
• Study Start Date: November 2009
• Actual Primary Completion Date: May 2011 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: November 9, 2009): Proportion of subjects with at least 75% improvement in the extent of the hemangioma [ Time Frame: Baseline, 6months ]
• Original Primary Outcome Measures: Same as current
• Change History: Complete list of historical versions of study NCT01010308 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures (submitted: November 9, 2009)

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

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

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

Brief Summary

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: 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.
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Non-Randomized; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Infantile Hemangioma

Intervention

Drug: Nadolol

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.

Study Arms

• 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

  Intervention: Drug: Nadolol
• 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.

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: August 1, 2013): 20
• Original Estimated Enrollment (submitted: November 9, 2009): 30
• Actual Study Completion Date: February 2012
• Actual Primary Completion Date: May 2011 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 1 Month to 1 Year (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Canada

Administrative Information

• NCT Number: NCT01010308
• Other Study ID Numbers: 1000014079
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Elena Pope, The Hospital for Sick Children
• Study Sponsor: The Hospital for Sick Children
• Collaborators: Not Provided

Investigators

• Principal Investigator: Elena Pope, MD; The Hospital for Sick Children

• PRS Account: The Hospital for Sick Children
• Verification Date: August 2013