Clinical trial NCT01010308: Nadolol for Proliferating Infantile Hemangiomas
Official title: Nadolol for Proliferating Infantile Hemangiomas: A Prospective Open Label Study With a Historical Control
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Infantile Hemangioma
Keywords: infantile hemangioma
nadolol
Lead sponsor: The Hospital for Sick Children
Sponsor class: OTHER
First posted: 2009-11-10
Enrollment: 20
Sex: ALL
Minimum age (years): 1
Maximum age (years): 1
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Proportion of subjects with at least 75% improvement in the extent of the hemangioma
timeFrame: Baseline, 6months
Secondary outcomes:
measure: The proportion of subjects with at least 50% improvement in the extent of the hemangiomas
timeFrame: Baseline and 6 months
measure: The percentage of patients with >75% improvement in the Nadolol group compared to a historical cohort of patients receiving propranolol.
timeFrame: 6 months
measure: The correlation between the changes in the levels of angiogenesis markers and clinical response to treatment.
timeFrame: 6 months
Other outcomes:
Locations:
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
Study officials:
affiliation: The Hospital for Sick Children
name: Elena Pope, MD
role: PRINCIPAL_INVESTIGATOR
References: