Trial Outcomes & Findings for Ponatinib in Advanced NSCLC w/ RET Translocations (NCT NCT01813734)
NCT ID: NCT01813734
Last Updated: 2019-12-24
Results Overview
The number of participants that achieved either a partial or complete response assessed using Response Evaluation Criteria in Solid Tumors (RECIST v 1.1) * Complete Response (CR): Disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \< 10 mm. * Partial Response (PR): At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
9 participants
Primary outcome timeframe
From the start of treatment until disease progression or death, up to approximately 2 years
Results posted on
2019-12-24
Participant Flow
Participant milestones
| Measure |
Ponatinib Treatment Arm
Ponatinib 30 mg PO daily
Ponatinib: 28 day cycle
|
|---|---|
|
Overall Study
STARTED
|
9
|
|
Overall Study
COMPLETED
|
9
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Ponatinib in Advanced NSCLC w/ RET Translocations
Baseline characteristics by cohort
| Measure |
Ponatinib Treatment Arm
n=9 Participants
Ponatinib 30 mg PO daily
Ponatinib: 28 day cycle
|
|---|---|
|
Age, Continuous
|
58 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
7 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
9 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From the start of treatment until disease progression or death, up to approximately 2 yearsThe number of participants that achieved either a partial or complete response assessed using Response Evaluation Criteria in Solid Tumors (RECIST v 1.1) * Complete Response (CR): Disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \< 10 mm. * Partial Response (PR): At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters.
Outcome measures
| Measure |
Ponatinib Treatment Arm
n=9 Participants
Ponatinib 30 mg PO daily
Ponatinib: 28 day cycle
|
|---|---|
|
Overall Response Rate
|
0 Participants
|
SECONDARY outcome
Timeframe: From the start of treatment until disease progression or death, up to approximately 2 yearsThe number of participants that achieved either a partial or complete response or stable disease, assessed using Response Evaluation Criteria in Solid Tumors (RECIST v 1.1) * Complete Response (CR): Disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \< 10 mm. * Partial Response (PR): At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. * Stable Disease (SD): Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease, taking as reference the smallest sum diameters while on study.
Outcome measures
| Measure |
Ponatinib Treatment Arm
n=9 Participants
Ponatinib 30 mg PO daily
Ponatinib: 28 day cycle
|
|---|---|
|
Disease Control Rate
|
5 Participants
|
SECONDARY outcome
Timeframe: From study entry until disease progression or death, median duration of 3.8 monthsThe duration of time from study entry until disease progression (assessed using RECIST 1.1.) or death. Progressive Disease (PD): At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study with at least a 5 mm absolute increase in the sum of all lesions. The appearance of one or more new lesions denotes disease progression.
Outcome measures
| Measure |
Ponatinib Treatment Arm
n=9 Participants
Ponatinib 30 mg PO daily
Ponatinib: 28 day cycle
|
|---|---|
|
Median Progression-Free Survival
|
3.8 Months
Interval 1.83 to 5.3
|
SECONDARY outcome
Timeframe: 1 yearThe number of participants surviving one year after study entry
Outcome measures
| Measure |
Ponatinib Treatment Arm
n=9 Participants
Ponatinib 30 mg PO daily
Ponatinib: 28 day cycle
|
|---|---|
|
1 Year Overall Survival Rate
|
5 Participants
|
SECONDARY outcome
Timeframe: From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)The number of participants with grade 3 plus adverse events as assessed using Common Toxicology Criteria for Adverse Events (CTCAE 4) that were deemed to be possibly, probably, or definitely related to study treatment.
Outcome measures
| Measure |
Ponatinib Treatment Arm
n=9 Participants
Ponatinib 30 mg PO daily
Ponatinib: 28 day cycle
|
|---|---|
|
Number of Participants With Adverse Events
|
2 Participants
|
Adverse Events
Ponatinib Treatment Arm
Serious events: 2 serious events
Other events: 9 other events
Deaths: 4 deaths
Serious adverse events
| Measure |
Ponatinib Treatment Arm
n=9 participants at risk
Ponatinib 30 mg PO daily
Ponatinib: 28 day cycle
|
|---|---|
|
Skin and subcutaneous tissue disorders
Dry skin
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
Other adverse events
| Measure |
Ponatinib Treatment Arm
n=9 participants at risk
Ponatinib 30 mg PO daily
Ponatinib: 28 day cycle
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
11.1%
1/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Blood and lymphatic system disorders
Lymph node pain
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Cardiac disorders
Pericardial tamponade
|
11.1%
1/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
General disorders
Chills
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
General disorders
Edema limbs
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
General disorders
Fatigue
|
33.3%
3/9 • Number of events 4 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
General disorders
Gait disturbance
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
General disorders
Non-cardiac chest pain
|
55.6%
5/9 • Number of events 6 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
General disorders
Pain
|
44.4%
4/9 • Number of events 11 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
22.2%
2/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
22.2%
2/9 • Number of events 3 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify
|
44.4%
4/9 • Number of events 7 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Endocrine disorders
Hypothyroidism
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Endocrine disorders
Endocrine disorders - Other, specify
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Gastrointestinal disorders
Abdominal pain
|
22.2%
2/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Gastrointestinal disorders
Anal hemorrhage
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Gastrointestinal disorders
Bloating
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Gastrointestinal disorders
Constipation
|
66.7%
6/9 • Number of events 10 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Gastrointestinal disorders
Diarrhea
|
66.7%
6/9 • Number of events 7 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Gastrointestinal disorders
Gastritis
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
22.2%
2/9 • Number of events 4 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Gastrointestinal disorders
Gastrointestinal pain
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Gastrointestinal disorders
Nausea
|
33.3%
3/9 • Number of events 3 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Gastrointestinal disorders
Vomiting
|
22.2%
2/9 • Number of events 3 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, specify
|
33.3%
3/9 • Number of events 4 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Infections and infestations
Bronchial infection
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Infections and infestations
Upper respiratory infection
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Infections and infestations
Infections and infestations - Other, specify
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Investigations
Alanine aminotransferase increased
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Investigations
Alkaline phosphatase increased
|
11.1%
1/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Investigations
Aspartate aminotransferase increased
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Investigations
Cholesterol high
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Investigations
Electrocardiogram QT corrected interval prolonged
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Investigations
Lymphocyte count decreased
|
11.1%
1/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Investigations
Weight loss
|
22.2%
2/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Metabolism and nutrition disorders
Anorexia
|
22.2%
2/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
11.1%
1/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
22.2%
2/9 • Number of events 3 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Metabolism and nutrition disorders
Metabolism and nutrition disorders - Other, specify
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
44.4%
4/9 • Number of events 7 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Musculoskeletal and connective tissue disorders
Chest wall pain
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
11.1%
1/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal deformity
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
11.1%
1/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Nervous system disorders
Dizziness
|
22.2%
2/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Nervous system disorders
Headache
|
22.2%
2/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Nervous system disorders
Paresthesia
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
22.2%
2/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Nervous system disorders
Somnolence
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Nervous system disorders
Nervous system disorders - Other, specify
|
11.1%
1/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Eye disorders
Blurred vision
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Eye disorders
Cataract
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Eye disorders
Dry eye
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Eye disorders
Floaters
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Eye disorders
Eye disorders - Other, specify
|
22.2%
2/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Psychiatric disorders
Anxiety
|
33.3%
3/9 • Number of events 4 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Psychiatric disorders
Depression
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Psychiatric disorders
Insomnia
|
22.2%
2/9 • Number of events 3 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Respiratory, thoracic and mediastinal disorders
Allergic rhinitis
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
44.4%
4/9 • Number of events 5 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
44.4%
4/9 • Number of events 6 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
22.2%
2/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Respiratory, thoracic and mediastinal disorders
Postnasal drip
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
22.2%
2/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Renal and urinary disorders
Urinary frequency
|
22.2%
2/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Reproductive system and breast disorders
Reproductive system and breast disorders - Other, specify
|
11.1%
1/9 • Number of events 1 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Vascular disorders
Flushing
|
22.2%
2/9 • Number of events 2 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
|
Vascular disorders
Hypertension
|
55.6%
5/9 • Number of events 5 • From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place