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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Ariad Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01874665
First received: May 29, 2013
Last updated: April 11, 2016
Last verified: March 2016
Results First Received: March 14, 2016  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: GIST
Intervention: Drug: ponatinib

  Participant Flow
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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

Patients with KIT exon 11-mutant GIST

ponatinib: 45 mg, taken orally once-daily

Cohort B

Patients with 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     26 [1]   13 [1]
NOT COMPLETED     4     2  
Treatment Ongoing                 4                 2  
[1] Completed = off treatment



  Baseline Characteristics
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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.
Intention To Treat Population

Reporting Groups
  Description
Cohort A

Patients with KIT exon 11-mutant GIST

ponatinib: 45 mg, taken orally once-daily

Cohort B

Patients 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  
Number of Participants  
[units: participants]
  30     15     45  
Age  
[units: participants]
     
<=18 years     0     0     0  
Between 18 and 65 years     16     10     26  
>=65 years     14     5     19  
Gender  
[units: participants]
     
Female     11     8     19  
Male     19     7     26  
Ethnicity (NIH/OMB)  
[units: participants]
     
Hispanic or Latino     1     0     1  
Not Hispanic or Latino     29     15     44  
Unknown or Not Reported     0     0     0  
Race (NIH/OMB)  
[units: participants]
     
American Indian or Alaska Native     0     0     0  
Asian     0     0     0  
Native Hawaiian or Other Pacific Islander     0     0     0  
Black or African American     0     0     0  
White     29     15     44  
More than one race     0     0     0  
Unknown or Not Reported     1     0     1  



  Outcome Measures
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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 ]
Results not yet reported.   Anticipated Reporting Date:   05/2017   Safety Issue:   No

4.  Secondary:   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 ]
Results not yet reported.   Anticipated Reporting Date:   05/2017   Safety Issue:   No

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 ]
Results not yet reported.   Anticipated Reporting Date:   05/2017   Safety Issue:   No

6.  Secondary:   Safety   [ Time Frame: From date of enrollment until the End-of-Treatment, assessed up to 3 years ]
Results not yet reported.   Anticipated Reporting Date:   05/2017   Safety Issue:   Yes

7.  Secondary:   Pharmacokinetic (PK) Parameters of Steady-state Plasma Concentration   [ Time Frame: Up to 1 month after the start of treatment ]
Results not yet reported.   Anticipated Reporting Date:   05/2017   Safety Issue:   No


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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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 patient population. Non-randomized.


  More Information
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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: David Kerstein, MD
Organization: ARIAD Pharmaceuticals, Inc.
phone: +1 (617) 494-0400
e-mail: David.Kerstein@ariad.com



Responsible Party: Ariad Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01874665     History of Changes
Other Study ID Numbers: AP24534-12-202
Study First Received: May 29, 2013
Results First Received: March 14, 2016
Last Updated: April 11, 2016
Health Authority: United States: Food and Drug Administration