Hemangioma of Infancy Clinical Trial
Official title:
Propranolol vs Prednisolone for Infant Hemangiomas-A Clinical and Molecular Study
| Verified date | January 2016 |
| Source | Children's Research Institute |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | United States: Institutional Review Board |
| Study type | Interventional |
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.
| Status | Terminated |
| Enrollment | 19 |
| Est. completion date | December 2014 |
| Est. primary completion date | January 2013 |
| Accepts healthy volunteers | No |
| Gender | Both |
| Age group | N/A to 6 Months |
| Eligibility |
Inclusion Criteria: - infants with symptomatic hemangiomas Exclusion Criteria: - asthma - diabetes - hypertension - hypotension - hypoglycemia - liver failure - previous treatment for hemangiomas |
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Single Blind (Outcomes Assessor), Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| United States | Children's National Medical Center | Washington | District of Columbia |
| Lead Sponsor | Collaborator |
|---|---|
| Nancy Bauman |
United States,
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. — View Citation
Léauté-Labrèze C, Dumas de la Roque E, Hubiche T, Boralevi F, Thambo JB, Taïeb A. Propranolol for severe hemangiomas of infancy. N Engl J Med. 2008 Jun 12;358(24):2649-51. doi: 10.1056/NEJMc0708819. — View Citation
Pérez RS, Mora PC, Rodríguez JD, Sánchez 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. Spanish. — View Citation
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Decrease in Size of Hemangioma (Length x Width) in Square mm | 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. | 4-5 months after initiating therapy | No |
| Secondary | Tolerability of Medication | All adverse events relating to medication tolerability including: adrenal crisis, growth/development, constitutional (dehydration), allergy/immunology, dermatologic, endocrine, GI, infection, metabolism/labs, pulmonary, vascular. | enrollment until study close out or withdrawal up to 9 months | Yes |
| Secondary | Number of Serious Adverse Events (SAEs) | 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. | enrollment until study close out or withdrawal up to 9 months | Yes |
| Secondary | Growth and Development Adverse Events | Number of Growth and Development AEs in each study arm | enrollment to study withdrawal or close out up to 9 months | No |
| Secondary | Pulmonary/Respiratory Adverse Events | Number of pulmonary/respiratory adverse events (CTCAE 22) in each study arm | enrollment through study close out or withdrawal, up to 9 months | Yes |
| Secondary | Allergy/Immunology Adverse Events | Number of allergy/immunology AE per study arm | enrollment through study closeout or study withdrawal up to 9 months | No |
| Secondary | Dermatologic Adverse Events | Number of Dermatologic Adverse Events in each study arm. | enrollment to study close out or withdrawal up to 9 months | No |
| Secondary | Endocrinologic Adverse Events | Number of Endocrinologic AEs (of which adrenal crisis does not overlap). | enrollment to close out or study withdrawal up to 9 months | No |
| Secondary | Gastrointestinal Adverse Events | Number of Gastrointestinal AEs in each arm | enrollment to study withdrawal or study close out up to 9 months | No |
| Secondary | Infectious Adverse Events | Number of infectious AEs in each study arm (i.e. conjunctivitis, thrush, fever) | enrollment to study withdrawal or close out up to 9 months | No |
| Secondary | Metabolic or Laboratory AEs | Number of Metabolic or Laboratory AEs in each study arm. | enrollment to study withdrawal or close out up to 9 months | No |
| Secondary | Vascular Adverse Events | Number of Vascular AEs in each study arm. | enrollment to study withdrawal or close out up to 9 months | No |
| Secondary | Constitutional Adverse Events | Number of constitutional AEs in each study arm. | enrollment to study close out or withdrawal up to 9 months | No |