Trial Outcomes & Findings for Trial of Ponatinib in Patients With Bevacizumab-Refractory Glioblastoma (NCT NCT02478164)

Last Updated: 2018-07-24

Results Overview

PFS3 is the proportion of patients remaining alive and progression-free at 3-months from study entry. Progressive disease was established based on Response Assessment in Neuro-Oncology (RANO) criteria. RANO criteria has 5 potential categories: Complete Response (CR), Partial Response (PR), Stable Disease (SD), Progressive disease (PD) and Unknown. CR: disappearance of all enhancing lesions, stable or improved non-enhancing lesions, and stable or improved clinically. PR: \>= 50% decrease in sum of perpendicular diameters of all measurable enhancing lesions, no progression of non-measurable disease, stable or improved non-enhancing lesions, and stable or improved clinically. PD: \>25% increase in sum of perpendicular diameters of all measurable enhancing lesions, significant increase of non-enhancing lesions, any new lesions, clear clinical deterioration, failure to return for evaluation due to death or deteriorating condition. SD: does not qualify for CR,PR or PD.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

17 participants

Primary outcome timeframe

3 months

Results posted on

2018-07-24

Participant Flow

Study began enrolling on 7/13/2015 and enrolled the last participant on 6/13/2017. Enrollment was stopped during a planned interim analysis due to futility. 17 participants were enrolled, but only 15 went on to receive ponatinib study treatment, as 2 participants withdrew after enrolling and before beginning ponatinib.

Participant milestones

Participant milestones
Measure	Ponatinib Drug will be administered once daily per cycle through oral ingestion.
Overall Study STARTED	15
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	15

Reasons for withdrawal

Reasons for withdrawal
Measure	Ponatinib Drug will be administered once daily per cycle through oral ingestion.
Overall Study Lack of Efficacy	13
Overall Study Adverse Event	1
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Trial of Ponatinib in Patients With Bevacizumab-Refractory Glioblastoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ponatinib n=15 Participants Drug will be administered once daily per cycle through oral ingestion.
Age, Continuous	62 years n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	11 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	13 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	13 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Initial Glioma Diagnosis Anaplastic Oligodendroglioma	1 Participants n=5 Participants
Initial Glioma Diagnosis Astrocytoma	1 Participants n=5 Participants
Initial Glioma Diagnosis Oligodendroglioma	1 Participants n=5 Participants
Initial Glioma Diagnosis Glioblastoma Multiforme	12 Participants n=5 Participants
Current Tumor Diagnosis	15 Participants n=5 Participants
Number of Prior Relapses Second relapse	10 Participants n=5 Participants
Number of Prior Relapses Third relapse	4 Participants n=5 Participants
Number of Prior Relapses Fourth relapse	1 Participants n=5 Participants
Number of Prior Relapses First relapse	0 Participants n=5 Participants
Baseline Karnofsky performance status (KPS) 100	0 Participants n=5 Participants
Baseline Karnofsky performance status (KPS) 90	4 Participants n=5 Participants
Baseline Karnofsky performance status (KPS) 80	8 Participants n=5 Participants
Baseline Karnofsky performance status (KPS) 70	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: 3 months

Outcome measures

Outcome measures
Measure	Ponatinib n=15 Participants Drug will be administered once daily per cycle through oral ingestion.
3-Month Progression-Free Survival (PFS3)	0 Participants

SECONDARY outcome

Timeframe: Disease was assessed radiographically for response every 8 weeks, assessed up to 24 weeks.

Radiographic response was established based on Response Assessment in Neuro-Oncology (RANO) criteria with 5 potential categories: Complete Response (CR), Partial Response (PR), Stable Disease (SD), Progressive disease (PD) and Unknown status.

Outcome measures

Outcome measures
Measure	Ponatinib n=15 Participants Drug will be administered once daily per cycle through oral ingestion.
Best Radiographic Response Complete Response	0 Participants
Best Radiographic Response Partial Response	0 Participants
Best Radiographic Response Stable Disease	2 Participants
Best Radiographic Response Progressive Disease	10 Participants
Best Radiographic Response Unknown	3 Participants

SECONDARY outcome

Timeframe: 2 years

OS based on Kaplan-Meier is defined as the time from study entry to death or date last known alive.

Outcome measures

Outcome measures
Measure	Ponatinib n=15 Participants Drug will be administered once daily per cycle through oral ingestion.
Overall Survival (OS)	98 days Interval 56.0 to 257.0

SECONDARY outcome

Timeframe: 3 months

PFS based on Kaplan-Meier is defined as the time from study entry to the earliest documentation of disease progression or death. Progressive disease was established based on Response Assessment in Neuro-Oncology (RANO) criteria.

Outcome measures

Outcome measures
Measure	Ponatinib n=15 Participants Drug will be administered once daily per cycle through oral ingestion.
Progression-Free Survival (PFS)	28 days Interval 27.0 to 30.0

Adverse Events

Ponatinib

Serious events: 2 serious events

Other events: 15 other events

Deaths: 4 deaths

Serious adverse events

Serious adverse events
Measure	Ponatinib n=15 participants at risk Drug will be administered once daily per cycle through oral ingestion.
Nervous system disorders Intracranial hemorrhage	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Skin and subcutaneous tissue disorders Bullous dermatitis	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.

Other adverse events

Other adverse events
Measure	Ponatinib n=15 participants at risk Drug will be administered once daily per cycle through oral ingestion.
Blood and lymphatic system disorders Anemia	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Gastrointestinal disorders Abdominal Pain	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Gastrointestinal disorders Constipation	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Gastrointestinal disorders Diarrhea	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Gastrointestinal disorders Gastroesophageal reflux disease	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Gastrointestinal disorders Mucositis Oral	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Gastrointestinal disorders Pancreatitis	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
General disorders Fatigue	20.0% 3/15 • Number of events 3 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Investigations Alanine aminotransferase increased	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Investigations Alkaline phosphatase increased	13.3% 2/15 • Number of events 2 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Investigations Aspartate aminotransferase increased	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Investigations CD4 lymphocytes decreased	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Investigations GGT increased	13.3% 2/15 • Number of events 2 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Investigations Lipase increased	13.3% 2/15 • Number of events 2 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Investigations Lymphocyte count decreased	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Investigations Platelet count decreased	26.7% 4/15 • Number of events 4 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Investigations Serum amylase increased	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Metabolism and nutrition disorders Anorexia	13.3% 2/15 • Number of events 2 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Metabolism and nutrition disorders Hypoalbuminemia	13.3% 2/15 • Number of events 2 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Metabolism and nutrition disorders Hypocalcemia	13.3% 2/15 • Number of events 2 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Metabolism and nutrition disorders Hyponatremia	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Musculoskeletal and connective tissue disorders Arthralgia	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Musculoskeletal and connective tissue disorders Generalized muscle weakness	13.3% 2/15 • Number of events 2 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Musculoskeletal and connective tissue disorders Myalgia	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Vascular disorders Hypertension	13.3% 2/15 • Number of events 2 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Gastrointestinal disorders Nausea	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Investigations Weight loss	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Skin and subcutaneous tissue disorders Dry Skin	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Skin and subcutaneous tissue disorders Rash acneiform	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Skin and subcutaneous tissue disorders Rash maculo-papular	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Metabolism and nutrition disorders Other - Mucosal lesions	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Skin and subcutaneous tissue disorders Other - rash	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.
Nervous system disorders Dysgeusia	6.7% 1/15 • Number of events 1 • Assessed each treatment cycle (1 cycle = 28 days) from time of first dose and up to day 30 post-treatment. Treatment duration in cycles was a median (range) of 1 (0-3). Assessed an average of 60 days. Maximum grade toxicity by type was first calculated. Serious AEs were defined as events with ponatinib study treatment-attribution of at least possibly and causing a hospitalization, deemed serious by the treating investigator or grade 5 events per CTCAE v4. Other AEs were defined as events with ponatinib study treatment-attribution of at least possibly and not requiring a hospitalization or deemed non-serious by treating investigator.

Additional Information

Eudocia Q. Lee, MD, MPH

Dana-Farber Cancer Institute

Phone: 617-632-2166

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place