A Phase II, Randomized, Clinical Trial Assessing Efficacy And Safety Of Oral Prednisolone vs Intravenous Vincristine In The Treatment Of Infantile
Infants with large hemangiomas are often treated systemically with oral steroids
(Prednisolone) to prevent complications. The best treatment for hemangiomas is not known and
there are no medications approved by the FDA for treatment of hemangiomas. Also, the best
method to measure the response of hemangioma to treatment is not known. Patients enrolling
on this study will be randomly assigned to receive either daily Prednisolone by mouth or
weekly Vincristine in a vein. Response to treatment will be monitored by clinical exams
every two weeks and by an MRI at study entry and six and twelve weeks later. Patients with
evidence of progressive disease (larger hemangiomas) on the week 6 MRI will be switched to
the other drug to complete a total of 12 weeks of therapy. Side effects of each medication
will be monitored closely determined from histories, physical exams, blood tests and other
studies as necessary. Participation in this study will last up to 12 weeks and follow up for
protocol.
Interventional
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Response of Hemangioma (IH) to Treatment
Response of IH not confined to the dermis will be coded using the following criteria: Progressive disease: >40% increase in volume by MRI, Partial response: >65% reduction in volume by MRI, Complete response: no visual or radiographic evidence of disease, Stable disease: none of the above or <40% increase or <65% decrease in volume by MRI. Response of superficial IH will be coded using the following criteria (based on RECIST): Progressive disease: >30% increase in IH size, Partial response: >30% reduction in size, Complete response: no evidence of disease, Stable disease: none of the above. Our first 3 patients showed limits to using MRI volume to measure IH size/response to therapy. Unlike other solid tumors, the superficial distribution of some IH made getting volume by MRI difficult, resulting in smaller tumor estimation compared to clinical assessment. Based on these observations, we amended the protocol to report response based on RECIST criteria instead of change in IH volume.
6 weeks
Yes
Beth Drolet, MD
Principal Investigator
Medical College of Wisconsin
United States: Food and Drug Administration
3429
NCT00555464
November 2007
December 2012
Name | Location |
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Medical College of Wisconsin/Children's Hospital of Wisconsin | Milwaukee, Wisconsin 53226 |