Clinical Trial: To Find a Safe Dose and Show Early Clinical Activity of Weekly Nab-paclitaxel in Pediatric Patients With Recurrent/ Refractory Solid Tumors
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Phase 1/2, Multicenter, Open-label, Dose-finding Study to Assess the Safety, Tolerability, and Preliminary Efficacy of Weekly Nab-paclitaxel in Pediatric Patients With Recurrent or Refractory Solid
Brief Summary: The purpose of this study is to find the safe dose of nab-paclitaxel in children with solid tumors, and to see if it works to treat these solid tumors in children and young adults (≤ 21 years old). After the final dose has been chosen, patients will be enrolled according to the specific solid tumor type, (neuroblastoma, rhabdomyosarcoma, or Ewing's sarcoma), to see how nab-paclitaxel works in treating these tumors.
Detailed Summary: ABI-007-PST-001 is a Phase 1 / 2, multicenter, open-label, dose-finding study to assess the safety , tolerability, and preliminary efficacy of weekly nab-paclitaxel in pediatric patients with recurrent or refractory solid tumors (excluding brain tumors). The Phase 1 portion of the study, with a dose escalation design, ended and the recommended Phase 2 dose (RP2D) was determined as 240 mg/m2 IV in patients weighing > 10 kg and 11.5 mg/kg in patients weighing ≤ 10 kg on Days 1, 8 and 15 of a 28-day cycle . The Phase 2 portion of the study will enroll additional patients at the RP2D into one of three solid tumor groups [Neuroblastomas, Rhabdomyosarcomas, Ewing's Sarcomas]. Both phases of the study are open-label and conducted at multiple centers.
Sponsor: Celgene
Current Primary Outcome:
- Incidence of Dose Limiting Toxicities (DLT) (Phase 1) [ Time Frame: Approximately 98 days ]Number of patients with a DLT
- Incidence of adverse events (Phase 1) [ Time Frame: Up to 2 years ]Number of patients with adverse events
- Overall response rate (ORR) (Phase 2) [ Time Frame: Up to 2 years ]Number of patients with complete response (CR) or partial response (PR) based on investigator assessment of response using RECIST 1.1 criteria. In the neuroblastoma group the ORR will be determined by RECIST and/or the Curie Scale (MIBG response)
Original Primary Outcome:
- Incidence of Dose Limiting Toxicities (DLT) (Phase 1) [ Time Frame: Approximately 98 days ]Number of patients with a DLT
- Incidence of adverse events (Phase 1) [ Time Frame: Up to 2 years ]Number of patients with adverse events
- Overall response rate (ORR) (Phase 2) [ Time Frame: Up to 1 year ]Number of patients with Complete response (CR) or partial response (PR) based on investigator assessment of response using RECIST 1.1 guidelines
Current Secondary Outcome:
- Pharmacokinetics (PK) - Cmax (Phase 1) [ Time Frame: Days 1 through 4 ]Maximum observed concentration in blood plasma
- Pharmacokinetics (PK) - AUC (Phase 1) [ Time Frame: Days 1 through 4 ]Area under the plasma concentration-time curve
- Pharmacokinetics (PK) - Clearance (Phase 1) [ Time Frame: Days 1 through 4 ]Measurement of renal clearance from the body
- Pharmacokinetics (PK) - Vss (Phase 1) [ Time Frame: Days 1 through 4 ]Volume of distribution
- Overall Response Rate (ORR) (Phase 1) [ Time Frame: Up to 2 year ]Number of patients with complete response (CR) or partial response (PR) based on investigator assessment of response using RECIST 1.1 guidelines
- Duration of Response (DOR) [ Time Frame: Up to 2 years ]Time from complete response (CR) or partial response (PR) [whichever is first recorded] until the date that progression-free survival event is documented
- Disease Control Rate (DCR) [ Time Frame: Up to 2 years ]The percentage of patients with a confirmed objective CR or PR, or stable disease for at least 16 weeks
- Progression-free survival (PFS) [ Time Frame: Up to 3 years ]Time from the start of treatment until the first progression or death by any cause, according to RECIST 1.1 guidelines. In the neuroblastoma group the PFS will be determined by RECIST and/or the Curie scale (MIBG response).
- 1-year survival [ Time Frame: Up to approximately 2 years ]Time from the start of treatment until death by any cause
- Safety [ Time Frame: Up to 2 years ]Number of participants with adverse events
- Pharmacokinetics (PK) -Cmax (Phase 2) [ Time Frame: Days 1 through 4 ]Maximum observed concentration in blood
- Pharmacokinetics (PK) - AUC (Phase 2) [ Time Frame: Days 1 through 4 ]Area under the plasma concentration-time curve
- Pharmacokinetics (PK) - Clearance (Phase 2) [ Time Frame: Days 1 through 4 ]Measurement of renal clearance from the body
- Pharmacokinetics (PK) - Vss (Phase 2) [ Time Frame: Days 1 through 4 ]Volume of distribution from both Phase 1 and Phase 2
Original Secondary Outcome:
- Pharmacokinetics (PK) - Cmax (Phase 1) [ Time Frame: Days 1 through 4 ]Maximum observed concentration in blood plasma
- Pharmacokinetics (PK) - AUC (Phase 1) [ Time Frame: Days 1 through 4 ]Area under the plasma concentration-time curve
- Pharmacokinetics (PK) - Clearance (Phase 1) [ Time Frame: Days 1 through 4 ]Measurement of renal clearance from the body
- Pharmacokinetics (PK) - Vss (Phase 1) [ Time Frame: Days 1 through 4 ]Volume of distribution
- Overall Response Rate (ORR) (Phase 1) [ Time Frame: Up to 1 year ]Number of patients with complete response (CR) or partial response (PR) based on investigator assessment of response using RECIST 1.1 guidelines
- Duration of Response (DOR) [ Time Frame: Up to 2 years ]Time from complete response (CR) or partial response (PR) [whichever is first recorded] until the date that progression-free survival event is documented
- Disease Control Rate (DCR) [ Time Frame: Up to 2 years ]The percentage of patients with a confirmed objective CR or PR, or stable disease for at least 16 weeks
- Progression-free survival (PFS) [ Time Frame: Up to 2 years ]Time from the start of treatment until the first progression or death by any cause, according to RECIST 1.1 guidelines
- 1-year survival [ Time Frame: Up to approximately 2 years ]Time from the start of treatment until death by any cause
- Safety [ Time Frame: Up to 2 years ]Number of participants with adverse events
- Pharmacokinetics (PK) -Cmax (Phase 2) [ Time Frame: Days 1 through 4 ]Maximum observed concentration in blood
- Pharmacokinetics (PK) - AUC (Phase 2) [ Time Frame: Days 1 through 4 ]Area under the plasma concentration-time curve
- Pharmacokinetics (PK) - Clearance (Phase 2) [ Time Frame: Days 1 through 4 ]Measurement of renal clearance from the body
- Pharmacokinetics (PK) - Vss (Phase 2) [ Time Frame: Days 1 through 4 ]Volume of distribution from both Phase 1 and Phase 2
Information By: Celgene
Dates:
Date Received: September 27, 2013
Date Started: December 4, 2013
Date Completion: February 10, 2020
Last Updated: March 2, 2017
Last Verified: March 2017
