Sorafenib and Topotecan in Refractory/Recurrent Pediatric Malignancies
H. Lee Moffitt Cancer Center and Research Institute will be the Sunshine Project Coordinator, but will not be recruiting locally.
The purpose of this research study is to establish a dose of the combination of drugs, Topotecan and Sorafenib in children. This will be called the maximum tolerated dose. The chemotherapy in this study is a combination of Topotecan and Sorafenib. The investigators are trying to find the highest dose of Topotecan and Sorafenib that can be given safely to children with Refractory or Recurrent Pediatric Solid Malignancies. The investigators will do this by testing different doses of these drugs in different groups of children. The investigators will also study how the body processes these drugs.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase I, Traditional 3+3, Trial of PO Sorafenib and Topotecan in Refractory or Recurrent Pediatric Solid Malignancies|
- Maximum Tolerated Dose (MTD) [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]Establish the recommended phase II dose of the combination of topotecan and sorafenib in children. This will be the maximum tolerated dose.
- Time to Progression (TTP) [ Time Frame: 24 months ] [ Designated as safety issue: No ]Determine Time to Progression (TTP) for all patients, comparing TTP on study to TTP on previous regimen. This study will use the (RECIST 1.1) Response Evaluation Criteria in Solid Tumors from the NCI for assessment of radiographic response in patients with solid tumors and in order to determine if patients have met off study criteria, i.e. disease progression. Progressive Disease (PD): At least a 20% increase in the disease measurement, taking as reference the smallest disease measurement recorded since the start of treatment, or the appearance of one or more new lesions, or evidence of laboratory or clinical progression.
- The Number of Participants with Adverse Events as a Measure of Safety and Feasibility [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]Describe the toxicity as per NCI Common Toxicity Criteria, version 4.0 (CTCAE 4.0).
|Study Start Date:||January 2013|
|Estimated Study Completion Date:||November 2015|
|Estimated Primary Completion Date:||November 2015 (Final data collection date for primary outcome measure)|
Experimental: Combination Chemotherapy
Combination Chemotherapy: Topotecan and Sorafenib. Participants will receive the treatment in cycles. Every cycle is 28 days long. For the first cycle participants will get the chemotherapy drugs:
Topotecan will be given by mouth.
Other Name: Topoisomerase-I inhibitorDrug: Sorafenib
Sorafenib will be given by mouth.
Other Name: BAY 43-9006
This protocol will evaluate a combination of a targeted agent, sorafenib, and a cytotoxic agent with broad spectrum activity, topotecan, in relapsed and refractory childhood solid tumors.
Patients will be enrolled in a traditional 3+3 phase I design to receive increasing doses of topotecan daily x 5 days on weeks 1 and 2 and sorafenib twice daily by mouth continuously. The duration of each treatment cycle will last 28 days.
|Contact: Kathleen Manningfirstname.lastname@example.org|
|Contact: Damon Reed, M.D.||email@example.com|
|United States, California|
|Childrens Hospital Los Angeles||Recruiting|
|Los Angeles, California, United States, 90027|
|Contact: Trang Nguyen 323-361-7309 firstname.lastname@example.org|
|Principal Investigator: Leo Mascarenhas, M.D.|
|Sub-Investigator: Venkatramani Rajkumar, M.D.|
|United States, Delaware|
|Nemours/Alfred I. duPont Hospital for Children, Delaware||Recruiting|
|Wilmington, Delaware, United States, 19803|
|Contact: Debra J. Bertz 302-651-5757 email@example.com|
|Principal Investigator: Edward A. Kolb, M.D.|
|United States, Florida|
|University of Florida, Gainesville||Recruiting|
|Gainesville, Florida, United States, 32610|
|Contact: Ashley Bayne 352-265-0027 firstname.lastname@example.org|
|Principal Investigator: Joanne Lagmay, M.D.|
|Nemours Children's Clinic, Jacksonville||Recruiting|
|Jacksonville, Florida, United States, 32207|
|Contact: Ingrid Ingram 904-697-3985 email@example.com|
|Principal Investigator: Scott Bradfield, M.D.|
|University of Miami, Sylvester Comprehensive Cancer Center||Recruiting|
|Miami, Florida, United States, 33136|
|Contact: Myriam Zayas 305-243-7846 MZayas2@med.miami.edu|
|Principal Investigator: John M. Goldberg, M.D.|
|All Children's Hospital, St. Petersburg||Recruiting|
|St. Petersburg, Florida, United States, 33701|
|Contact: Ashley Repp 727-767-4784 firstname.lastname@example.org|
|Principal Investigator: Damon Reed, M.D.|
|United States, New York|
|Montefiore Medical Center, Children's Hospital at Montefiore||Recruiting|
|Bronx, New York, United States, 10467|
|Contact: Noam Zeffren 718-741-2356 email@example.com|
|Principal Investigator: Jonathan Gill, M.D.|
|Principal Investigator:||Damon Reed, M.D.||H. Lee Moffitt Cancer Center and Research Institute|
|Principal Investigator:||G. Douglas Letson, M.D.||H. Lee Moffitt Cancer Center and Research Institute|