Clinical Trial: Propranolol Versus Prednisolone for Treatment of Symptomatic Hemangiomas

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: Propranolol vs Prednisolone for Infant Hemangiomas-A Clinical and Molecular Study

Brief Summary: Hemangiomas are relatively common lesions in infants. Most go away spontaneously after one year of life and do not need treatment. Others require treatment because they cause significant symptoms such as pain, or difficulty with breathing, eating or ambulating. Steroids have classically been used to treat hemangiomas and help to shrink them in 1/3 - 2/3 of patients. Unfortunately, steroids have many side effects in babies so physicians have sought other ways to treat them. Recently, the use of propranolol, a heart medication, was serendipitously found to reduce the size of hemangiomas. It appears to have many fewer side effects than steroids but it is not yet known if it works as well as steroids. This study seeks to compare the effect and the side effects of propranolol versus steroids for treating hemangiomas that cause symptoms in infants.

Detailed Summary:

Infants with symptomatic hemangiomas will be enrolled. Magnetic resonance imaging will be completed before starting medication if the extent of the hemangioma is not evident on clinical examination alone. Infants will be randomized to receive either propranolol or steroids for 4-6 months. Hemangioma response will be measured and compared monthly as will tolerability of the medications. Additionally, urine specimens will be collected at each visit to determine if markers are present that can predict response to therapy.

Additionally, any hemangiomas that are excised will be examined for genetic markers to aid in predicting response to therapy.


Sponsor: Nancy Bauman

Current Primary Outcome: Decrease in Size of Hemangioma (Length x Width) in Square mm [ Time Frame: 4-5 months after initiating therapy ]

A priori primary outcome was proportional change in the total surface area as measured by lesion's outer margin length x width at baseline minus the same measure at 4 months with surrogate data used at 5 months if 4 months not available.


Original Primary Outcome: reduction in size of hemangioma [ Time Frame: 4-6 months ]

Current Secondary Outcome:

  • Tolerability of Medication [ Time Frame: enrollment until study close out or withdrawal up to 9 months ]
    All adverse events relating to medication tolerability including: adrenal crisis, growth/development, constitutional (dehydration), allergy/immunology, dermatologic, endocrine, GI, infection, metabolism/labs, pulmonary, vascular.
  • Number of Serious Adverse Events (SAEs) [ Time Frame: enrollment until study close out or withdrawal up to 9 months ]
    Number of serious adverse events experienced by the participants in each treatment arm within the categories adrenal crisis, growth/development, constitutional. Serious adverse events are defined as events that result in death, require either inpatient hospitalization or the prolongation of hospitalization, are life-threatening, result in a persistent or significant disability/incapacity, or result in a congenital anomaly/birth defect. Other important medical events, based upon appropriate medical judgment, may also be considered Serious Adverse Events if a trial participant's health is at risk and intervention is required to prevent an outcome mentioned.
  • Growth and Development Adverse Events [ Time Frame: enrollment to study withdrawal or close out up to 9 months ]
    Number of Growth and Development AEs in each study arm
  • Pulmonary/Respiratory Adverse Events [ Time Frame: enrollment through study close out or withdrawal, up to 9 months ]
    Number of pulmonary/respiratory adverse events (CTCAE 22) in each study arm
  • Allergy/Immunology Adverse Events [ Time Frame: enrollment through study closeout or study withdrawal up to 9 months ]
    Number of allergy/immunology AE per study arm
  • Dermatologic Adverse Events [ Time Frame: enrollment to study close out or withdrawal up to 9 months ]
    Number of Dermatologic Adverse Events in each study arm.
  • Endocrinologic Adverse Events [ Time Frame: enrollment to close out or study withdrawal up to 9 months ]
    Number of Endocrinologic AEs (of which adrenal crisis does not overlap).
  • Gastrointestinal Adverse Events [ Time Frame: enrollment to study withdrawal or study close out up to 9 months ]
    Number of Gastrointestinal AEs in each arm
  • Infectious Adverse Events [ Time Frame: enrollment to study withdrawal or close out up to 9 months ]
    Number of infectious AEs in each study arm (i.e. conjunctivitis, thrush, fever)
  • Metabolic or Laboratory AEs [ Time Frame: enrollment to study withdrawal or close out up to 9 months ]
    Number of Metabolic or Laboratory AEs in each study arm.
  • Vascular Adverse Events [ Time Frame: enrollment to study withdrawal or close out up to 9 months ]
    Number of Vascular AEs in each study arm.
  • Constitutional Adverse Events [ Time Frame: enrollment to study close out or withdrawal up to 9 months ]
    Number of constitutional AEs in each study arm.


Original Secondary Outcome: Tolerability of medication [ Time Frame: 4-6 months ]

Information By: Children's Research Institute

Dates:
Date Received: August 26, 2009
Date Started: July 2009
Date Completion:
Last Updated: January 27, 2016
Last Verified: January 2016