Trial Outcomes & Findings for Ponatinib for Squamous Cell Lung and Head and Neck Cancers (NCT NCT01761747)

Results Overview

Investigate the response rate of patients with previously treated lung or head and neck SCC to ponatinib as defined by the proportion of subjects with investigator-assessed confirmed complete response (CR) or partial response (PR). Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR."

Recruitment status

TERMINATED

Study phase

PHASE2/PHASE3

Target enrollment

3 participants

Primary outcome timeframe

2 years

Results posted on

2014-12-23

Participant Flow

Participant milestones

Participant milestones
Measure	Ponatinib Treatment Arm Ponatinib taken by mouth daily ponatinib
Overall Study STARTED	3
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Ponatinib for Squamous Cell Lung and Head and Neck Cancers

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ponatinib Treatment Arm n=3 Participants Ponatinib taken by mouth daily ponatinib
Age, Continuous	61 years n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Race/Ethnicity, Customized Caucasian	3 participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants
Disease	3 participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years

Investigate the response rate of patients with previously treated lung or head and neck SCC to ponatinib as defined by the proportion of subjects with investigator-assessed confirmed complete response (CR) or partial response (PR). Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR."

Outcome measures

Outcome measures
Measure	Ponatinib Treatment Arm n=3 Participants Ponatinib taken by mouth daily ponatinib
Response Rate of Patients With Lung or Head and Neck SCC Treated With Ponatinib	0 percentage of subjects with response

SECONDARY outcome

Timeframe: 2 years

Population: 2 participants had FGFR amplification, one with FGFR mutation

Test tumor DNA using molecular assays to measure the frequency of FGFR amplifications and mutations in study patients

Outcome measures

Outcome measures
Measure	Ponatinib Treatment Arm n=3 Participants Ponatinib taken by mouth daily ponatinib
Prevalence of Specific FGFR Amplifications/Mutations in the Study Population	3 participants

SECONDARY outcome

Timeframe: 2 years

Establish the progression-free survival of patients with SCC treated with ponatinib as defined by time to development of progression by RECIST criteria.

Outcome measures

Outcome measures
Measure	Ponatinib Treatment Arm n=2 Participants Ponatinib taken by mouth daily ponatinib
Progression-free Survival	44 days Interval 32.0 to 55.0

SECONDARY outcome

Timeframe: 2 years

Number of Participants with Adverse Events as a Measure of Safety and Tolerability

Outcome measures

Outcome measures
Measure	Ponatinib Treatment Arm n=3 Participants Ponatinib taken by mouth daily ponatinib
Define Toxicities of Ponatinib	2 participants

SECONDARY outcome

Timeframe: 2 years

Measure the overall survival time of patients treated with ponatinib

Outcome measures

Outcome measures
Measure	Ponatinib Treatment Arm n=3 Participants Ponatinib taken by mouth daily ponatinib
Overall Survival	276 days Interval 48.0 to 588.0

SECONDARY outcome

Timeframe: 2 years

Measure the disease control rate of patients treated with ponatinib

Outcome measures

Outcome measures
Measure	Ponatinib Treatment Arm n=2 Participants Ponatinib taken by mouth daily ponatinib
Disease Control	0 percentage of subjects

SECONDARY outcome

Timeframe: 2 years

Population: Too few subjects were enrolled on study to permit this outcome measure analysis.

For subjects with FGFR amplifications and for FGFR mutations we will ascertain the age, sex, disease stage, prior response to treatment and smoking history from past medical records and measure whether there are differences in these variables among subjects with amplification versus mutation.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Population: There were no observed responses on study so this outcome could not be determined.

Identify the response rate to ponatinib for FGFR specific FGFR amplifications/mutations.

Outcome measures

Outcome data not reported

Adverse Events

Ponatinib Treatment Arm

Serious events: 1 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Ponatinib Treatment Arm n=2 participants at risk Ponatinib taken by mouth daily ponatinib
Gastrointestinal disorders Pancreatits	50.0% 1/2 One individual withdrew consent the day after enrolling on study and was not dosed. Therefore only two individuals were evaluable for treatment-related toxicities.

Other adverse events

Other adverse events
Measure	Ponatinib Treatment Arm n=2 participants at risk Ponatinib taken by mouth daily ponatinib
General disorders Fatigue	100.0% 2/2 One individual withdrew consent the day after enrolling on study and was not dosed. Therefore only two individuals were evaluable for treatment-related toxicities.
Gastrointestinal disorders Diarrhea	50.0% 1/2 One individual withdrew consent the day after enrolling on study and was not dosed. Therefore only two individuals were evaluable for treatment-related toxicities.
Skin and subcutaneous tissue disorders Rash	50.0% 1/2 One individual withdrew consent the day after enrolling on study and was not dosed. Therefore only two individuals were evaluable for treatment-related toxicities.
Gastrointestinal disorders Mucositis	50.0% 1/2 One individual withdrew consent the day after enrolling on study and was not dosed. Therefore only two individuals were evaluable for treatment-related toxicities.
Musculoskeletal and connective tissue disorders Arthralgia	50.0% 1/2 One individual withdrew consent the day after enrolling on study and was not dosed. Therefore only two individuals were evaluable for treatment-related toxicities.
Musculoskeletal and connective tissue disorders Myalgia	50.0% 1/2 One individual withdrew consent the day after enrolling on study and was not dosed. Therefore only two individuals were evaluable for treatment-related toxicities.
Nervous system disorders Neuropathy	50.0% 1/2 One individual withdrew consent the day after enrolling on study and was not dosed. Therefore only two individuals were evaluable for treatment-related toxicities.
Gastrointestinal disorders Hepatitis	100.0% 2/2 One individual withdrew consent the day after enrolling on study and was not dosed. Therefore only two individuals were evaluable for treatment-related toxicities.

Additional Information

Dr. Peter Hammerman

Dana-Farber Cancer Institute

Phone: 617-632-6335

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place