Clinical Trial: Trial of Pazopanib in Patients With Solitary Fibrous Tumor and Extraskeletal Myxoid Chondrosarcoma

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase II Open-Label Trial of Pazopanib Administered as a Single Agent in Patients With Unresectable or Metastatic Solitary Fibrous Tumor (SFT) and Extraskeletal Myxoid C

Brief Summary: Phase II, open-label, non-randomized, international multicenter clinical trial with two strata (SFT and EMC). 8 sites in Spain, 5 sites in Italy and 5 sites in France. Patients will receive oral pazopanib at 800 mg once daily continuously. Patients will continue to receive treatment until there is evidence of progressive disease, unacceptable toxicity, non-compliance, withdrawn consent or investigator decision. The main goal is to determine the objective response rate (ORR) (confirmed complete response [CR] and partial response [PR]) in patients with unresectable, locally advanced or metastatic solitary fibrous tumor and extraskeletal myxoid chondrosarcoma, using Choi and RECIST 1.1 criteria respectively.

Detailed Summary:

To estimate the sample size for stratum 1 (SFT), a Simon's optimal 2-stage phase II design has been used, having considered the published response rate based on Choi criteria in SFT patients which correspond to 40% in monotherapy. For a design with P0=0.40, P1=0.60; α=0.1 and β=0.1. At the first stage, 18 patients should be enrolled into the study, if there are fewer than 8 responses (7 or less) the trial will be terminated and it will be concluded that pazopanib is not sufficiently active. At the second stage, another 28 patients (total 46 patients) would be enrolled into the study. To reject the null hypothesis for the SFT stratum 23 responses or more (Choi criteria), out of the 46 patients, are needed.

To estimate the simple size for stratum 2 (EMC), a Simon's optimal 2-stage phase II design has been used, having considered the very scarce published information on response rate based on RECIST criteria. For a design with P0= 0.05, P1= 0.25, α=0.1 and β=0.1. At the first stage, 9 patients should be enrolled into the study, if there are not responses the trial will be terminated and it will be concluded that pazopanib is not sufficiently active. If there is at least 1 response in this first stage, the trial will be continued and at the second stage, another 15 patients (total 24 patients) would be enrolled into the study. To reject the null hypothesis for the EMC stratum 3 responses or more (RECIST criteria), out of the 24 patients, are needed.

For variables that follow binomial distributions (e.g. response rate) frequency and percentages will be calculated, together with their corresponding exact 95% confidence intervals. For time-to-event variables (e.g. PFS or OS) Kaplan-Meier estimations will be used. To analyze the reduction of risk and the influence of other variables on time-to-event variables C
Sponsor: Grupo Espanol de Investigacion en Sarcomas

Current Primary Outcome: Objective response rate (ORR) [ Time Frame: 48 weeks ]

Objective response rate (ORR) (confirmed complete response [CR] and partial response [PR]), measured using Choi and RECIST 1.1 criteria. Response criteria will be based on the baseline identification of target lesions and follow-up until tumor progression.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Efficacy [ Time Frame: 48 weeks ]
    Efficacy measured by the progression-free survival (PFS) rate assessed by median time
  • Overall survival (OS) [ Time Frame: 72 weeks ]
    Overall survival (OS) measured since treatment start date until date of death, whichever the cause
  • Clinical benefit rate (CBR) [ Time Frame: 48 weeks ]
    Clinical benefit rate (CBR). Patients who have reached CR, PR or SD during 6 months or more, presenting clinical improvement of symptoms, will be considered as having experienced clinical benefit.
  • Long term safety profile of pazopanib [ Time Frame: 48 weeks ]
    Long term safety profile of pazopanib, through assessment of adverse event type, incidence, severity, time of appearance, related causes, as well as physical explorations and laboratory tests. Toxicity will be graded and tabulated by using NCI-CTCAE 4.0.


Original Secondary Outcome: Same as current

Information By: Grupo Espanol de Investigacion en Sarcomas

Dates:
Date Received: February 14, 2014
Date Started: June 2014
Date Completion: June 2018
Last Updated: October 3, 2014
Last Verified: October 2014