Carfilzomib in Combination With Cyclophosphamide and Etoposide for Children (POE14-01)
This study evaluates the use of carfilzomib in combination with cyclophosphamide and etoposide for children with relapsed/refractory solid tumors or leukemia. The medications cyclophosphamide and etoposide are standard drugs often used together for the treatment of cancer in children with solid tumors or leukemia.
Carfilzomib is FDA (Food and Drug Administration) approved in the United States for adults with multiple myeloma (a type of cancer). However, this drug is not approved for the disease being treated in this study. Since carfilzomib has not yet been used in this setting to treat this condition, the investigators must first find the best dose to give. The investigators are looking for the highest dose of carfilzomib that can be given safely. Therefore, not all children taking part in this study will receive the same dose of the study drug in the first part of the trial.
|Relapsed Solid Tumors Refractory Solid Tumors Leukemias||Drug: Carfilzomib Drug: Cyclophosphamide Drug: Etoposide||Phase 1|
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||Phase I Study of Carfilzomib in Combination With Cyclophosphamide and Etoposide for Children With Relapsed and Refractory Solid Tumors and Leukemias|
- To determine the DLTs and MTD of carfilzomib given in combination with cyclophosphamide and etoposide in pediatric patients with relapsed/refractory leukemias and solid tumors [ Time Frame: Screening to 2 years ]
- Collect information on all adverse events that occur with this regimen [ Time Frame: Screening to 2 years ]
- Determine patient response rate (CR, PR, SD, PD) with this regimen [ Time Frame: Screening to 2 years ]
- Circulating plasma proteasome (cProt) levels post treatment [ Time Frame: 2 years ]
- Levels of proteasome activity [ Time Frame: 2 years ]
- Inhibition of proteasome activity by carfilzomib [ Time Frame: 2 years ]
- Proteasome inhibition in patient PBMCs before and during treatment by determination of the level of protein ubiquitination. [ Time Frame: 2 years ]
- In vitro sensitivity of patient leukemias and solid tumors to carfilzomib alone and in combination with study chemotherapeutic agents [ Time Frame: Screening to 2 years ]
- Alterations of proteasome activity in tumor samples [ Time Frame: 2 years ]
- Expression of actionable mutations [ Time Frame: 2 years ]
|Study Start Date:||July 2015|
|Estimated Study Completion Date:||September 2018|
|Estimated Primary Completion Date:||September 2018 (Final data collection date for primary outcome measure)|
Carfilzomib in combination with cyclophosphamide and etoposide
Carfilzomib in combination with cyclophosphamide and etoposide for children with relapsed and refractory solid tumors and leukemiasDrug: Cyclophosphamide Drug: Etoposide
The purpose of this study is to find out what effects, good and/or bad, treatment with a new combination of drugs, cyclophosphamide, etoposide, and carfilzomib has on cancer.
In part 1 of the trial, small groups of children will be enrolled in steps. The first group will be given a certain dose of carfilzomib. If these children do not have side effects which are too bad, the next small group of children enrolled will receive a higher dose. This increase in doses with groups of people will continue until we find the highest dose of the drug that can be given without causing severe or unmanageable side effects.
Part 2 of this study will enroll additional patients at the highest tolerable dose found in Part 1 in order to get more information on side effects and make sure the dose is tolerable.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02512926
|Contact: Jessica Boklan, MDemail@example.com|
|Contact: Aru Narendran, MD, PhDfirstname.lastname@example.org|
|United States, Arizona|
|Phoenix Children's Hospital||Recruiting|
|Phoenix, Arizona, United States, 85016|
|Contact: Lauree Deublein 602-933-5004 email@example.com|
|Contact: Sam Chimienti 602-933-0188 firstname.lastname@example.org|