Propranolol Versus Prednisolone for Treatment of Symptomatic Hemangiomas
- Registration Number
- NCT00967226
- Lead Sponsor
- Nancy Bauman
- 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 Description
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.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 19
- infants with symptomatic hemangiomas
- asthma
- diabetes
- hypertension
- hypotension
- hypoglycemia
- liver failure
- previous treatment for hemangiomas
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description propranolol for treatment of hemangiomas propranolol Assessing efficacy and tolerability of propranolol in management of symptomatic hemangiomas Prednisolone Prednisolone Assessing efficacy and tolerability of prednisolone in management of symptomatic hemangiomas and comparing to propranolol.
- Primary Outcome Measures
Name Time Method Decrease in Size of Hemangioma (Length x Width) in Square mm 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.
- Secondary Outcome Measures
Name Time Method Tolerability of Medication 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) 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.
Pulmonary/Respiratory Adverse Events 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 enrollment through study closeout or study withdrawal up to 9 months Number of allergy/immunology AE per study arm
Dermatologic Adverse Events enrollment to study close out or withdrawal up to 9 months Number of Dermatologic Adverse Events in each study arm.
Endocrinologic Adverse Events 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 enrollment to study withdrawal or study close out up to 9 months Number of Gastrointestinal AEs in each arm
Infectious Adverse Events 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 enrollment to study withdrawal or close out up to 9 months Number of Metabolic or Laboratory AEs in each study arm.
Vascular Adverse Events enrollment to study withdrawal or close out up to 9 months Number of Vascular AEs in each study arm.
Constitutional Adverse Events enrollment to study close out or withdrawal up to 9 months Number of constitutional AEs in each study arm.
Growth and Development Adverse Events enrollment to study withdrawal or close out up to 9 months Number of Growth and Development AEs in each study arm
Trial Locations
- Locations (1)
Children's National Medical Center
🇺🇸Washington, District of Columbia, United States
Children's National Medical Center🇺🇸Washington, District of Columbia, United States