Dose Escalation Study of Sorafenib and Irinotecan Combination Therapy in Pediatric Patients With Solid Tumors
Rhabdomyosarcoma and Other Soft Tissue Sarcomas
Ewing's Sarcoma Family of Tumors
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase 1 Dose Escalation Study of Sorafenib and Irinotecan Combination Therapy in Pediatric Patients With Relapsed or Refractory Solid Tumors|
- Toxicity Profile [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]Determine the toxicity profile, dose-limiting toxicity (DLT) and maximum tolerated dose (MTD) of sorafenib, when administered in combination with oral irinotecan in children with relapsed or refractory solid tumors.
- Patient Related Outcomes [ Time Frame: 24 months ] [ Designated as safety issue: No ]
Demonstrate the feasibility of incorporating measurement of patient-related outcome into a four site phase 1 trial of sorafenib and irinotecan for children and adolescents.
Demonstrate feasibility: defined as 70% of participants will complete the 5 patient-reported outcomes (PROs) (pain, fatigue, worry, sadness and physical functioning) at both baseline (pre-treatment) and at the end of the first course and missing data will not be greater than 15% across the 5 PROs at either data point.
- Pharmacokinetic Profile [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]Describe the plasma pharmacokinetic profile of sorafenib and irinotecan when administered as combination therapy in children and adolescents.
- Disease Evaluation [ Time Frame: 24 months ] [ Designated as safety issue: No ]Evaluate disease response, based on criteria for measurable lesions (RECIST) and evaluable lesions, to guide further development in phase 2 studies.
- Integration of Patient Related Outcomes with other outcome measures [ Time Frame: 24 months ] [ Designated as safety issue: No ]Correlate and integrate the PRO scores with traditional endpoints of toxicity, pharmacokinetic profile, and tumor response associated with the sorafenib and irinotecan combination.
|Study Start Date:||December 2011|
|Study Completion Date:||January 2015|
|Primary Completion Date:||January 2015 (Final data collection date for primary outcome measure)|
Experimental: Combination Therapy
Three to 6 patient will be enrolled at each dose level and dose escalations will proceed in the absence of dose-limiting toxicity attributed to therapy, first with dose escalation of sorafenib and then, if tolerated, escalation of irinotecan.
sorafenib (50 and 200 mg tablets) orally twice daily, on a continuous schedule at a starting dose of 150mg/m2/dose.Drug: irinotecan
irinotecan (70 mg/m2/dose) orally, concurrently, once daily,starting at the beginning of the 21 day cycle,repeated every 21 days
Please refer to this study by its ClinicalTrials.gov identifier: NCT01518413
|United States, District of Columbia|
|Children's National Medical Center|
|Washington, District of Columbia, United States, 20010|
|United States, Maryland|
|National Cancer Institute|
|Bethesda, Maryland, United States, 20892|
|United States, Massachusetts|
|Children's Hospital Boston/Dana-Farber Cancer Institute|
|Boston, Massachusetts, United States, 02215|
|United States, Pennsylvania|
|The Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|Principal Investigator:||Holly Meany, MD||Children's Research Institute|