Clinical trial NCT00967226: Propranolol Versus Prednisolone for Treatment of Symptomatic Hemangiomas
Official title: Propranolol vs Prednisolone for Infant Hemangiomas-A Clinical and Molecular Study
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Hemangioma of Infancy
Keywords: hemangioma
Lead sponsor: Children's National Research Institute
Sponsor class: OTHER
First posted: 2009-08-27
Enrollment: 19
Sex: ALL
Minimum age (years): 2
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: Inclusion Criteria:

* infants with symptomatic hemangiomas

Exclusion Criteria:

* asthma
* diabetes
* hypertension
* hypotension
* hypoglycemia
* liver failure
* previous treatment for hemangiomas

Primary outcomes:
measure: Decrease in Size of Hemangioma (Length x Width) in Square mm
timeFrame: 4-5 months after initiating therapy
Secondary outcomes:
measure: Tolerability of Medication
timeFrame: enrollment until study close out or withdrawal up to 9 months
measure: Number of Serious Adverse Events (SAEs)
timeFrame: enrollment until study close out or withdrawal up to 9 months
measure: Growth and Development Adverse Events
timeFrame: enrollment to study withdrawal or close out up to 9 months
measure: Pulmonary/Respiratory Adverse Events
timeFrame: enrollment through study close out or withdrawal, up to 9 months
measure: Allergy/Immunology Adverse Events
timeFrame: enrollment through study closeout or study withdrawal up to 9 months
measure: Dermatologic Adverse Events
timeFrame: enrollment to study close out or withdrawal up to 9 months
measure: Endocrinologic Adverse Events
timeFrame: enrollment to close out or study withdrawal up to 9 months
measure: Gastrointestinal Adverse Events
timeFrame: enrollment to study withdrawal or study close out up to 9 months
measure: Infectious Adverse Events
timeFrame: enrollment to study withdrawal or close out up to 9 months
measure: Metabolic or Laboratory AEs
timeFrame: enrollment to study withdrawal or close out up to 9 months
measure: Vascular Adverse Events
timeFrame: enrollment to study withdrawal or close out up to 9 months
measure: Constitutional Adverse Events
timeFrame: enrollment to study close out or withdrawal up to 9 months
Other outcomes:
Locations:
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
Study officials:
affiliation: Children's Research Institute, Children's National Medical Center
name: Nancy M Bauman, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Perez RS, Mora PC, Rodriguez JD, Sanchez FR, de Torres Jde L. [Treatment of infantile hemangioma with propranolol]. An Pediatr (Barc). 2010 Feb;72(2):152-4. doi: 10.1016/j.anpedi.2009.05.019. Epub 2009 Jul 23. No abstract available. Spanish.
citation: Denoyelle F, Leboulanger N, Enjolras O, Harris R, Roger G, Garabedian EN. Role of Propranolol in the therapeutic strategy of infantile laryngotracheal hemangioma. Int J Pediatr Otorhinolaryngol. 2009 Aug;73(8):1168-72. doi: 10.1016/j.ijporl.2009.04.025. Epub 2009 May 29.
citation: Leaute-Labreze C, Dumas de la Roque E, Hubiche T, Boralevi F, Thambo JB, Taieb A. Propranolol for severe hemangiomas of infancy. N Engl J Med. 2008 Jun 12;358(24):2649-51. doi: 10.1056/NEJMc0708819. No abstract available.
citation: Bauman NM, McCarter RJ, Guzzetta PC, Shin JJ, Oh AK, Preciado DA, He J, Greene EA, Puttgen KB. Propranolol vs prednisolone for symptomatic proliferating infantile hemangiomas: a randomized clinical trial. JAMA Otolaryngol Head Neck Surg. 2014 Apr;140(4):323-30. doi: 10.1001/jamaoto.2013.6723.
citation: Patel NJ, Bauman NM. How should propranolol be initiated for infantile hemangiomas: inpatient versus outpatient? Laryngoscope. 2014 Jun;124(6):1279-81. doi: 10.1002/lary.24363. Epub 2013 Dec 17. No abstract available.