Now Available: Final Rule for FDAAA 801 and NIH Policy on Clinical Trial Reporting

Safety and Efficacy of Cabazitaxel in Pediatric Patients With Refractory Solid Tumors Including Central Nervous System Tumors

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Sanofi
ClinicalTrials.gov Identifier:
NCT01751308
First received: December 13, 2012
Last updated: June 28, 2016
Last verified: June 2016
Results First Received: June 28, 2016  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Malignant Solid Tumor - Malignant Nervous System Neoplasm
Intervention: Drug: Cabazitaxel (XRP6258)

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Participants were enrolled at 12 centers between February 2013 and March 2015.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Phase I was a dose escalation part of Cabazitaxel to determine maximum tolerated dose (MTD). Phase 2 was efficacy and safety evaluation of Cabazitaxel at the MTD, determined in Phase 1.

Reporting Groups
  Description
Phase 1: Cabazitaxel 20 mg/m^2 Cabazitaxel 20 mg/m^2 intravenous (IV) infusion on Day 1 of each 21-day cycle until disease progression (DP) or discontinuation due to adverse events (AE) or death (from any cause).
Phase 1: Cabazitaxel 25 mg/m^2 Cabazitaxel 25 mg/m^2 IV infusion on Day 1 of each 21-day cycle until DP or discontinuation due to AE or death (from any cause).
Phase 1: Cabazitaxel 30 mg/m^2 Cabazitaxel 30 mg/m^2 IV infusion on Day 1 of each 21-day cycle until DP or discontinuation due to AE or death (from any cause).
Phase 1: Cabazitaxel 35 mg/m^2 Cabazitaxel 35 mg/m^2 IV infusion on Day 1 of each 21-day cycle until DP or discontinuation due to AE or death (from any cause).
Phase 2: Cabazitaxel 30 mg/m^2 Cabazitaxel at the MTD as determined in phase 1 (30 mg/m^2) IV infusion on Day 1 of each 21-day cycle until DP or discontinuation due to AE or death (from any cause).

Participant Flow for 2 periods

Period 1:   Phase 1
    Phase 1: Cabazitaxel 20 mg/m^2     Phase 1: Cabazitaxel 25 mg/m^2     Phase 1: Cabazitaxel 30 mg/m^2     Phase 1: Cabazitaxel 35 mg/m^2     Phase 2: Cabazitaxel 30 mg/m^2  
STARTED     6     3     7     7     0 [1]
COMPLETED     6 [2]   3 [2]   7 [2]   7 [2]   0  
NOT COMPLETED     0     0     0     0     0  
[1] Phase 1 and Phase 2 were separate populations.
[2] DP, AE and death were considered as completed (defined in protocol)

Period 2:   Phase 2
    Phase 1: Cabazitaxel 20 mg/m^2     Phase 1: Cabazitaxel 25 mg/m^2     Phase 1: Cabazitaxel 30 mg/m^2     Phase 1: Cabazitaxel 35 mg/m^2     Phase 2: Cabazitaxel 30 mg/m^2  
STARTED     0     0     0     0     16 [1]
COMPLETED     0     0     0     0     16 [2]
NOT COMPLETED     0     0     0     0     0  
[1] Phase 1 and Phase 2 were separate populations.
[2] DP, AE and death were considered as completed (defined in protocol)



  Baseline Characteristics
  Hide Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
All treated (AT)/safety population was defined as all registered participants who actually received at least 1 dose or part of a dose of the investigational medicinal product (IMP).

Reporting Groups
  Description
Phase 1: Cabazitaxel 20 mg/m^2 Cabazitaxel 20 mg/m^2 IV infusion on Day 1 of each 21-day cycle until DP or discontinuation due to AE or death (from any cause).
Phase 1: Cabazitaxel 25 mg/m^2 Cabazitaxel 25 mg/m^2 IV infusion on Day 1 of each 21-day cycle until DP or discontinuation due to AE or death (from any cause).
Phase 1: Cabazitaxel 30 mg/m^2 Cabazitaxel 30 mg/m^2 IV infusion on Day 1 of each 21-day cycle until DP or discontinuation due to AE or death (from any cause).
Phase 1: Cabazitaxel 35 mg/m^2 Cabazitaxel 35 mg/m^2 IV infusion on Day 1 of each 21-day cycle until DP or discontinuation due to AE or death (from any cause).
Phase 2: Cabazitaxel 30 mg/m^2 Cabazitaxel at the maximum tolerated dose (MTD) as determined in phase 1 (30 mg/m^2) IV infusion on Day 1 of each 21-day cycle until DP or discontinuation due to AE or death (from any cause).
Total Total of all reporting groups

Baseline Measures
    Phase 1: Cabazitaxel 20 mg/m^2     Phase 1: Cabazitaxel 25 mg/m^2     Phase 1: Cabazitaxel 30 mg/m^2     Phase 1: Cabazitaxel 35 mg/m^2     Phase 2: Cabazitaxel 30 mg/m^2     Total  
Number of Participants  
[units: participants]
  6     3     7     7     16     39  
Age, Customized  
[units: participants]
           
2-4 years     0     0     2     0     2     4  
5-6 years     2     1     0     1     4     8  
7-11 years     1     1     3     5     4     14  
12-18 years     3     1     2     1     6     13  
Gender  
[units: participants]
           
Female     5     0     1     2     8     16  
Male     1     3     6     5     8     23  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Phase 1: Maximum Tolerated Dose of Cabazitaxel   [ Time Frame: Cycle 1 (21 days) ]

2.  Primary:   Phase 2: Percentage of Participants With Objective Response (OR)   [ Time Frame: Baseline, every 9 weeks until DP or death due to any cause (maximum duration: 12.1 weeks) ]

3.  Primary:   Phase 2: Duration of Response (DOR)   [ Time Frame: Baseline, every 9 weeks until DP or death due to any cause (maximum duration: 12.1 weeks) ]

4.  Secondary:   Phase 1 and 2: Number of Participants With Treatment Emergent Adverse Events (TEAEs)   [ Time Frame: Baseline up to DP or death due to any cause (maximum duration: 112.1 weeks for Phase 1 and 12.1 weeks for Phase 2) ]

5.  Secondary:   Phase 1: Number of Participants With Objective Response   [ Time Frame: Baseline, every 9 weeks until DP or death due to any cause (maximum duration: 112.1 weeks) ]

6.  Secondary:   Phase 1 and 2: Pharmacokinetics (PK) Parameter of Cabazitaxel: Area Under the Plasma Concentration (AUC) Versus Time Curve   [ Time Frame: Day 1 of Cycle 1: 5 minutes before EOI up to 71 hours after the EOI ]

7.  Secondary:   Phase 1 and 2: PK Parameter of Cabazitaxel: Total Plasma Clearance (CL)   [ Time Frame: Day 1 of Cycle 1: 5 minutes before EOI up to 71 hours after the EOI ]

8.  Secondary:   Phase 1 and 2: PK Parameter of Cabazitaxel: Volume of Distribution at Steady State (Vss)   [ Time Frame: Day 1 of Cycle 1: 5 minutes before EOI up to 71 hours after the EOI ]

9.  Secondary:   Phase 1 and 2: PK Parameter of Cabazitaxel: Maximum Plasma Concentration Observed (Cmax)   [ Time Frame: Day 1 of Cycle 1: 5 minutes before EOI up to 71 hours after the EOI ]

10.  Secondary:   Phase 2: Progression Free Survival (PFS)   [ Time Frame: Baseline, every 9 weeks until DP or death due to any cause (maximum duration: 12.1 weeks) ]

11.  Secondary:   Phase 2: Overall Survival (OS)   [ Time Frame: Baseline up to death or study cut-off (maximum duration: 12.1 weeks) ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
  Hide More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Trial Transparency Team
Organization: Sanofi
e-mail: Contact-US@sanofi.com



Responsible Party: Sanofi
ClinicalTrials.gov Identifier: NCT01751308     History of Changes
Other Study ID Numbers: TED12689
U1111-1128-5704 ( Other Identifier: UTN )
Study First Received: December 13, 2012
Results First Received: June 28, 2016
Last Updated: June 28, 2016
Health Authority: United States: Food and Drug Administration