Clinical Trial of Vincristine vs. Prednisolone for Treatment of Complicated Hemangiomas
The goal of this study is to determine the safety and efficacy of Prednisolone and Vincristine for treatment of large, complicated infantile hemangiomas. The diagnostic, therapeutic and response criteria experimentally determined in this study will be used as a framework for future infantile hemangioma studies.
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase II, Randomized, Clinical Trial Assessing Efficacy And Safety Of Oral Prednisolone vs Intravenous Vincristine In The Treatment Of Infantile|
- Decrease in size of hemangioma by MRI and clinical exam [ Time Frame: Initial visit, 6 weeks, 12 weeks ] [ Designated as safety issue: Yes ]A data and Safety Monitoring Board will be reviewing the data from this research throughout the study. We will tell you about new information from this or other studies that may affect your child's health, welfare, or willingness to stay in the study.
- Toxicity to medications [ Time Frame: Initial visit, 2, 4, 6, 10 and 12 weeks of therapy ] [ Designated as safety issue: Yes ]Your child will have blood draws and be seen regularily in the clinic. This study is also monitored by Data and Safety monitoring Board and you will be notified of new information affect safety of the study.
|Study Start Date:||November 2007|
|Estimated Study Completion Date:||January 2014|
|Estimated Primary Completion Date:||December 2013 (Final data collection date for primary outcome measure)|
Vincristine is a drug that has been used to treat cancers in children (including infants). It has been effective in treating a small number of infants with hemangiomas, most of whom failed previous therapies including steroids. Vincristine must be administered into a vein. Given the encouraging response data and documented safety record, Vincristine is a good choice for a clinical trial treating infants with complicated hemangiomas.
Vincristine (0.05 mg/kg/dose) will be administered into a vein (PICC line) every week for 12 weeks. If assigned to receive Vincristine, a PICC line will be placed by a doctor who is a specialist in this procedure, an interventional radiologist. This will require sedation and when possible, will be coordinated with sedation for the MRI.
Other Name: VINCRISTINE SULFATE (Oncovin®, VCR, LCR) NSC #67574 (042006)
Active Comparator: 2
The standard treatment for hemangioma at most centers is oral steroids (Prednisolone). Prednisolone has been used to stop the growth of infantile hemangiomas that are life threatening, that could harm important functions, or are likely to result in severe disfigurement (scarring) without treatment.
Prednisolone given at 3 mg/kg/day by mouth for 12 week
Other Name: PREDNISOLONE SODIUM PHOSPHATE SYRUP/SOLUTION 15mg/5 cc (Orapred®)
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.
|United States, Wisconsin|
|Medical College of Wisconsin/Children's Hospital of Wisconsin|
|Milwaukee, Wisconsin, United States, 53226|
|Principal Investigator:||Beth Drolet, MD||Medical College of Wisconsin|
|Principal Investigator:||Michael Kelly, MD, PhD||Medical College of Wisconsin|