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A Phase 2 Trial of Ponatinib in Participants With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor (GIST)

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ClinicalTrials.gov Identifier: NCT01874665
Recruitment Status : Completed
First Posted : June 11, 2013
Results First Posted : May 17, 2016
Last Update Posted : May 18, 2018
Sponsor:
Information provided by (Responsible Party):
Takeda ( Ariad Pharmaceuticals )

Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: GIST
Intervention: Drug: Ponatinib

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Cohort A Participants with KIT exon 11-mutant GIST ponatinib: 45 milligram (mg), taken orally once-daily.
Cohort B Participants with gastrointestinal stromal tumors (GIST) that lack KIT exon 11 mutations (Cohort B) ponatinib: 45 mg, taken orally once-daily.

Participant Flow:   Overall Study
    Cohort A   Cohort B
STARTED   30   15 
COMPLETED   0   0 
NOT COMPLETED   30   15 
Adverse Event                6                2 
Withdrawal by Subject                4                1 
Physician Decision                3                0 
Clinical Progressive Disease                4                3 
Study Terminated by Sponsor                0                2 
Documented Progressive Disease                11                7 
Other                2                0 



  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.
The intention to treat (ITT) population included all participants who received any dose of ponatinib in the study.

Reporting Groups
  Description
Cohort A Participants with KIT exon 11-mutant GIST ponatinib: 45 mg, taken orally once-daily.
Cohort B Participants with GIST that lack KIT exon 11 mutations (Cohort B) ponatinib: 45 mg, taken orally once-daily.
Total Total of all reporting groups

Baseline Measures
   Cohort A   Cohort B   Total 
Overall Participants Analyzed 
[Units: Participants]
 30   15   45 
Age [1] 
[Units: Years]
Mean (Standard Deviation)
 59.3  (10.37)   53.9  (16.06)   57.5  (12.63) 
[1] The ITT population included all participants who received any dose of ponatinib in the study.
Sex: Female, Male [1] 
[Units: Participants]
Count of Participants
     
Female      11  36.7%      8  53.3%      19  42.2% 
Male      19  63.3%      7  46.7%      26  57.8% 
[1] The ITT population included all participants who received any dose of ponatinib in the study.
Ethnicity (NIH/OMB) [1] 
[Units: Participants]
Count of Participants
     
Hispanic or Latino      1   3.3%      0   0.0%      1   2.2% 
Not Hispanic or Latino      29  96.7%      15 100.0%      44  97.8% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0% 
[1] The ITT population included all participants who received any dose of ponatinib in the study.
Race (NIH/OMB) [1] 
[Units: Participants]
Count of Participants
     
American Indian or Alaska Native      0   0.0%      0   0.0%      0   0.0% 
Asian      0   0.0%      0   0.0%      0   0.0% 
Native Hawaiian or Other Pacific Islander      0   0.0%      0   0.0%      0   0.0% 
Black or African American      0   0.0%      0   0.0%      0   0.0% 
White      29  96.7%      15 100.0%      44  97.8% 
More than one race      0   0.0%      0   0.0%      0   0.0% 
Unknown or Not Reported      1   3.3%      0   0.0%      1   2.2% 
[1] The ITT population included all participants who received any dose of ponatinib in the study.
Region of Enrollment [1] 
[Units: Participants]
Count of Participants
     
United States   30   15   45 
[1] The ITT population included all participants who received any dose of ponatinib in the study.
Weight [1] 
[Units: Kilogram (kg)]
Mean (Standard Deviation)
 80.12  (20.699)   75.10  (13.917)   78.45  (18.702) 
[1] The ITT population included all participants who received any dose of ponatinib in the study.
Height [1] 
[Units: Centimeter (cm)]
Mean (Standard Deviation)
 171.20  (7.839)   171.28  (7.149)   171.23  (7.534) 
[1] The ITT population included all participants who received any dose of ponatinib in the study.


  Outcome Measures

1.  Primary:   Clinical Benefit Rate (CBR) in Cohort A   [ Time Frame: 16 weeks after first dose ]

2.  Secondary:   Clinical Benefit Rate (CBR) in Cohort B   [ Time Frame: 16 weeks after first dose ]

3.  Secondary:   Progression-free Survival (PFS)   [ Time Frame: From date of enrollment until the end of the study or disease progression or death due to any cause, whichever came first, assessed up to 3 years ]

4.  Secondary:   Percentage of Participants With Objective Response Rate (ORR)   [ Time Frame: From date of enrollment until discontinuation or the end of the study, whichever came first, assessed up to 3 years ]

5.  Secondary:   Overall Survival (OS)   [ Time Frame: From first dose of drug until the end of the study or death, whichever came first, assessed up to 3 years ]

6.  Secondary:   Number of Participants With Physical Examination   [ Time Frame: From date of enrollment until the End-of-Treatment, assessed up to 3 years ]

7.  Secondary:   Number of Participants With Treatment-emergent Adverse Events (TEAEs) Related to Vital Sign Measurements   [ Time Frame: From date of enrollment until the End-of-Treatment, assessed up to 3 years ]

8.  Secondary:   Number of Participants With Worst Shift From Baseline Values to Post-baseline Values in Laboratory Parameters   [ Time Frame: From date of enrollment until the End-of-Treatment, assessed up to 3 years ]

9.  Secondary:   Number of Participants With TEAEs Related to Electrocardiogram (ECG) Findings   [ Time Frame: From date of enrollment until the End-of-Treatment, assessed up to 3 years ]

10.  Secondary:   Number of Participants With TEAEs Related to Echocardiography Parameter   [ Time Frame: From date of enrollment until the End-of-Treatment, assessed up to 3 years ]

11.  Secondary:   Number of Participants Reporting One or More TEAEs and Serious Adverse Event (SAE)   [ Time Frame: From date of enrollment until the End-of-Treatment, assessed up to 3 years ]

12.  Secondary:   Cmax, SS: Maximum Observed Plasma Concentration at Steady State for Ponatinib   [ Time Frame: Pre-dose and at multiple timepoints (up to 1 month) post-dose ]


  Serious Adverse Events


  Other Adverse Events


  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
Study is ongoing. Uncontrolled Study. Small participant population. Non-randomized.


  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: Medical Director
Organization: Ariad Pharmaceuticals
phone: +1-844-662-8532
e-mail: globaloncologymedinfo@takeda.com



Responsible Party: Takeda ( Ariad Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01874665     History of Changes
Other Study ID Numbers: AP24534-12-202
First Submitted: May 29, 2013
First Posted: June 11, 2013
Results First Submitted: March 14, 2016
Results First Posted: May 17, 2016
Last Update Posted: May 18, 2018