Trial Outcomes & Findings for Ponatinib for Patients Whose Advanced Solid Tumor Cancer Has Activating Mutations Involving the Following Genes: FGFR1, FGFR2, FGFR3, FGFR4, RET, KIT. (NCT NCT02272998)

Last Updated: 2025-03-24

Results Overview

The proportion of responses for the purposes of the decision rule will be calculated out of all eligible patients who receive any treatment. Assuming the number of responses is binomially distributed, 95% binomial confidence intervals will also be calculated for the estimate of the proportion of responses. Response for tumors was assessed using the RECIST 1.1 criteria (using computed tomography \[CT\] scans), where response was defined as a partial or complete response.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

22 participants

Primary outcome timeframe

Up to 6 months

Results posted on

2025-03-24

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (Ponatinib Hydrochloride) Patients receive ponatinib hydrochloride PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. ponatinib hydrochloride: Given PO laboratory biomarker analysis: Correlative studies
Overall Study STARTED	22
Overall Study COMPLETED	22
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Ponatinib for Patients Whose Advanced Solid Tumor Cancer Has Activating Mutations Involving the Following Genes: FGFR1, FGFR2, FGFR3, FGFR4, RET, KIT.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Ponatinib Hydrochloride) n=22 Participants Patients receive ponatinib hydrochloride PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. ponatinib hydrochloride: Given PO laboratory biomarker analysis: Correlative studies
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	16 Participants n=5 Participants
Age, Categorical >=65 years	6 Participants n=5 Participants
Sex: Female, Male Female	11 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	22 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 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	1 Participants n=5 Participants
Race (NIH/OMB) White	15 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	6 Participants n=5 Participants
Region of Enrollment United States	22 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 6 months

Outcome measures

Outcome measures
Measure	Participants With Genomic Alterations in FGFR n=17 Participants Patients receive ponatinib hydrochloride PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. ponatinib hydrochloride: Given PO laboratory biomarker analysis: Correlative studies	Participants With Genomic Alterations in Rare Genomic Targets (KIT, PDGFRα, RET, ABL1, and FLT3) n=5 Participants Patients receive ponatinib hydrochloride PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. ponatinib hydrochloride: Given PO laboratory biomarker analysis: Correlative studies
Overall Response, Defined as the Number of Patients Who Achieve Any Response According to Disease Type in the First 6 Courses of Treatment	1 Participants	0 Participants

SECONDARY outcome

Timeframe: Up to 30 days after last dose of study drug, up to a total of 6 years

Frequency and severity of adverse events will be collected and summarized by descriptive statistics. The maximum grade for each type of toxicity will be recorded for each patient, and frequency tables will be reviewed to determine toxicity patterns for each of the cohorts as well as across cohorts. In addition, all adverse event data that is graded as 3, 4, or 5 will be reviewed and classified as either "unrelated" or "unlikely to be related" to study treatment in the event of an actual relationship developing.

Outcome measures

Outcome measures
Measure	Participants With Genomic Alterations in FGFR n=22 Participants Patients receive ponatinib hydrochloride PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. ponatinib hydrochloride: Given PO laboratory biomarker analysis: Correlative studies	Participants With Genomic Alterations in Rare Genomic Targets (KIT, PDGFRα, RET, ABL1, and FLT3) Patients receive ponatinib hydrochloride PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. ponatinib hydrochloride: Given PO laboratory biomarker analysis: Correlative studies
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Alkaline Phosphatase Increased, Grade 1	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Headache, Grade 1	13.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Abdominal Pain, Grade ≥3	13.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Lymphocyte Count Decreased, Grade 1	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Lymphocyte Count Decreased, Grade ≥3	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Anorexia, Grade 1	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Anorexia, Grade 2	9.1 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Alanine Aminotransferase Increased, Grade 1	13.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Pancreatitis, Grade 2	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Dyspnea, Grade ≥3	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Dry Skin, Grade 1	36.4 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Dry Skin, Grade 2	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Dry Skin, Grade ≥3	9.1 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Rash Maculo-papular, Grade 1	27.3 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Rash Maculo-papular, Grade 2	9.1 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Hypertension, Grade 1	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Hypertension, Grade 2	9.1 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Hypertension, Grade ≥3	13.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Fever, Grade 1	27.3 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Alkaline Phosphatase Increased, Grade 2	13.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Alkaline Phosphatase Increased, Grade ≥3	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Headache, Grade 2	9.1 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Nausea, Grade 1	9.1 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Nausea, Grade 2	9.1 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Lymphocyte Count Decreased, Grade 2	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Fatigue, Grade 1	9.1 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Fatigue, Grade 2	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Pancreatitis, Grade ≥3	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Dyspnea, Grade 2	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Dry Mouth, Grade 1	9.1 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Mucositis, Grade 1	9.1 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Diarrhea, Grade 1	9.1 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Aspartate Aminotransferase Increased, Grade 1	9.1 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Dysgeusia, Grade 1	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Dysgeusia, Grade 2	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Arthralgia, Grade 1	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Arthralgia, Grade 2	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Serum Amylase Increased, Grade ≥3	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Supraventricular Tachycardia, Grade ≥3	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Lipase Increased, Grade ≥3	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Palmar-plantar Erythrodysesthesia Syndrome, Grade 1	4.6 percentage of participants	—
Percentage of Participants With Adverse Events, Defined as Adverse Events That Are Classified as Either Possibly, Probably, or Definitely Related to Study Treatment Per National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 Thromboembolic Event, Grade 2	4.6 percentage of participants	—

SECONDARY outcome

Timeframe: Up to 30 days after last dose of study drug

Collected and summarized by descriptive statistics.

Outcome measures

Outcome measures
Measure	Participants With Genomic Alterations in FGFR n=22 Participants Patients receive ponatinib hydrochloride PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. ponatinib hydrochloride: Given PO laboratory biomarker analysis: Correlative studies	Participants With Genomic Alterations in Rare Genomic Targets (KIT, PDGFRα, RET, ABL1, and FLT3) Patients receive ponatinib hydrochloride PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. ponatinib hydrochloride: Given PO laboratory biomarker analysis: Correlative studies
Tolerability of the Regimen, Assessed by the Number of Patients Who Required Dose Modifications and/or Dose Delays	11 Participants	—

SECONDARY outcome

Timeframe: The time from treatment initiation to death, assessed up to 72 months

Kaplan-Meier curves will be used to estimate the survival distribution.

Outcome measures

Outcome measures
Measure	Participants With Genomic Alterations in FGFR n=22 Participants Patients receive ponatinib hydrochloride PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. ponatinib hydrochloride: Given PO laboratory biomarker analysis: Correlative studies	Participants With Genomic Alterations in Rare Genomic Targets (KIT, PDGFRα, RET, ABL1, and FLT3) Patients receive ponatinib hydrochloride PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. ponatinib hydrochloride: Given PO laboratory biomarker analysis: Correlative studies
Overall Survival	5.3 months Interval 0.0 to 72.0	—

SECONDARY outcome

Timeframe: The time from treatment initiation to progression or death, assessed up to 2 years

Kaplan-Meier curves will be used to estimate the survival distribution.

Outcome measures

Outcome measures
Measure	Participants With Genomic Alterations in FGFR n=22 Participants Patients receive ponatinib hydrochloride PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. ponatinib hydrochloride: Given PO laboratory biomarker analysis: Correlative studies	Participants With Genomic Alterations in Rare Genomic Targets (KIT, PDGFRα, RET, ABL1, and FLT3) Patients receive ponatinib hydrochloride PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. ponatinib hydrochloride: Given PO laboratory biomarker analysis: Correlative studies
Progression Free Survival	1.9 months Interval 0.0 to 19.7	—

SECONDARY outcome

Timeframe: 6 months

Calculated by the number of patients who have achieve a response and/or are progression-free and alive at 6 months divided by the total number of evaluable patients. Exact binomial 95% confidence intervals for CBR will be calculated.

Outcome measures

Outcome measures
Measure	Participants With Genomic Alterations in FGFR n=22 Participants Patients receive ponatinib hydrochloride PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. ponatinib hydrochloride: Given PO laboratory biomarker analysis: Correlative studies	Participants With Genomic Alterations in Rare Genomic Targets (KIT, PDGFRα, RET, ABL1, and FLT3) Patients receive ponatinib hydrochloride PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. ponatinib hydrochloride: Given PO laboratory biomarker analysis: Correlative studies
Clinical Benefit Rate (CBR)	31.8 percentage of participants	—

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 3 years (time of progression)

Correlative gene and protein markers will be summarized univariately in a quantitative manner and also summarized by clinical outcome group (e.g. response vs. no response). Graphical analyses will be largely used to assess potential patterns and relationships; e.g. side-by-side boxplots to assess differences in continuous marker levels between those with vs. without the clinical improvement (e.g. response vs. no response). Overall, hypothesis testing will largely be avoided given the sample size limitations.

Outcome measures

Outcome data not reported

Adverse Events

Treatment (Ponatinib Hydrochloride)

Serious events: 3 serious events

Other events: 22 other events

Deaths: 16 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Ponatinib Hydrochloride) n=22 participants at risk Patients receive ponatinib hydrochloride PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. ponatinib hydrochloride: Given PO laboratory biomarker analysis: Correlative studies
Gastrointestinal disorders Abdominal pain	4.5% 1/22 • Number of events 1 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Metabolism and nutrition disorders Anorexia	4.5% 1/22 • Number of events 1 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Gastrointestinal disorders Ascites	4.5% 1/22 • Number of events 1 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Vascular disorders Thromboembolic Event	4.5% 1/22 • Number of events 1 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.

Other adverse events

Other adverse events
Measure	Treatment (Ponatinib Hydrochloride) n=22 participants at risk Patients receive ponatinib hydrochloride PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. ponatinib hydrochloride: Given PO laboratory biomarker analysis: Correlative studies
Gastrointestinal disorders Abdominal cramping	9.1% 2/22 • Number of events 2 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Gastrointestinal disorders Abdominal Pain	36.4% 8/22 • Number of events 8 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Investigations Alanine aminotransferase increased	13.6% 3/22 • Number of events 3 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Investigations Alanine transaminase increased	9.1% 2/22 • Number of events 2 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Investigations Alkaline phosphatase increased	31.8% 7/22 • Number of events 7 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Skin and subcutaneous tissue disorders Alopecia	9.1% 2/22 • Number of events 2 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Blood and lymphatic system disorders Anemia	54.5% 12/22 • Number of events 12 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Metabolism and nutrition disorders Anorexia	54.5% 12/22 • Number of events 12 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Psychiatric disorders Anxiety	18.2% 4/22 • Number of events 4 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Musculoskeletal and connective tissue disorders Arthralgia	27.3% 6/22 • Number of events 6 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Musculoskeletal and connective tissue disorders Arthritis	9.1% 2/22 • Number of events 2 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Gastrointestinal disorders Ascites	13.6% 3/22 • Number of events 3 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Investigations Aspartate aminotransferase increased	18.2% 4/22 • Number of events 4 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Musculoskeletal and connective tissue disorders Back pain	40.9% 9/22 • Number of events 9 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Reproductive system and breast disorders Breast pain	9.1% 2/22 • Number of events 2 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
General disorders Non-cardiac chest pain	18.2% 4/22 • Number of events 4 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Psychiatric disorders Confusion	9.1% 2/22 • Number of events 2 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Gastrointestinal disorders Constipation	68.2% 15/22 • Number of events 15 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Respiratory, thoracic and mediastinal disorders Cough	31.8% 7/22 • Number of events 7 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Investigations Creatinine increased	13.6% 3/22 • Number of events 3 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Metabolism and nutrition disorders Dehydration	18.2% 4/22 • Number of events 4 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Psychiatric disorders Depression	40.9% 9/22 • Number of events 9 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Gastrointestinal disorders Diarrhea	40.9% 9/22 • Number of events 9 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Nervous system disorders Dizziness	13.6% 3/22 • Number of events 3 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Eye disorders Dry eye	9.1% 2/22 • Number of events 2 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Gastrointestinal disorders Dry mouth	27.3% 6/22 • Number of events 6 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Skin and subcutaneous tissue disorders Dry skin	68.2% 15/22 • Number of events 15 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Nervous system disorders Dysesthesia	13.6% 3/22 • Number of events 3 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Nervous system disorders Dysgeusia	9.1% 2/22 • Number of events 2 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Gastrointestinal disorders Dyspepsia	13.6% 3/22 • Number of events 3 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Respiratory, thoracic and mediastinal disorders Dyspnea	63.6% 14/22 • Number of events 14 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Renal and urinary disorders Dysuria	9.1% 2/22 • Number of events 2 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
General disorders Edema limbs	31.8% 7/22 • Number of events 7 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Respiratory, thoracic and mediastinal disorders Epistaxis	9.1% 2/22 • Number of events 2 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Eye disorders Eye disorders, other	22.7% 5/22 • Number of events 5 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
General disorders Fatigue	90.9% 20/22 • Number of events 20 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
General disorders Fever	40.9% 9/22 • Number of events 9 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Musculoskeletal and connective tissue disorders Flank pain	13.6% 3/22 • Number of events 3 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Vascular disorders Flushing	9.1% 2/22 • Number of events 2 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Gastrointestinal disorders Gastroesophageal reflux disease	18.2% 4/22 • Number of events 4 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Metabolism and nutrition disorders Glucose Intolerance	13.6% 3/22 • Number of events 3 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Nervous system disorders Headache	36.4% 8/22 • Number of events 8 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Respiratory, thoracic and mediastinal disorders Hoarseness	13.6% 3/22 • Number of events 3 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Metabolism and nutrition disorders Hyperglycemia	36.4% 8/22 • Number of events 8 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Vascular disorders Hypertension	40.9% 9/22 • Number of events 9 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Metabolism and nutrition disorders Hypoalbuminemia	22.7% 5/22 • Number of events 5 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Metabolism and nutrition disorders Hypokalemia	9.1% 2/22 • Number of events 2 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Metabolism and nutrition disorders Hypomagnesemia	9.1% 2/22 • Number of events 2 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Metabolism and nutrition disorders Hyponatremia	18.2% 4/22 • Number of events 4 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Psychiatric disorders Insomnia	31.8% 7/22 • Number of events 7 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Skin and subcutaneous tissue disorders Itching	9.1% 2/22 • Number of events 2 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Investigations Lymphocyte count decreased	31.8% 7/22 • Number of events 7 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Skin and subcutaneous tissue disorders Rash maculo-papular	40.9% 9/22 • Number of events 9 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Gastrointestinal disorders Mucositis oral	18.2% 4/22 • Number of events 4 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Musculoskeletal and connective tissue disorders Myalgia	18.2% 4/22 • Number of events 4 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Gastrointestinal disorders Nausea	59.1% 13/22 • Number of events 13 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Nervous system disorders Peripheral sensory neuropathy	27.3% 6/22 • Number of events 6 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Musculoskeletal and connective tissue disorders Pain in extremity	13.6% 3/22 • Number of events 3 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Investigations Pancreatic Enzymes Decreased	9.1% 2/22 • Number of events 2 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Gastrointestinal disorders Pancreatitis	13.6% 3/22 • Number of events 3 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Respiratory, thoracic and mediastinal disorders Postnasal drip	9.1% 2/22 • Number of events 2 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Respiratory, thoracic and mediastinal disorders Rhinorrhea	9.1% 2/22 • Number of events 2 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Infections and infestations Sepsis	9.1% 2/22 • Number of events 2 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Gastrointestinal disorders Vomiting	27.3% 6/22 • Number of events 6 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.
Investigations Weight Loss	45.5% 10/22 • Number of events 10 • Adverse event data was collected for each patient from the start of treatment with the investigational drug up to 30 days after ending treatment. The total time period in which adverse event data was collected for this study was 6 years.

Additional Information

Dr. Sameek Roychowdhury

The Ohio State University Comprehensive Cancer Center

Phone: 614-685-5842

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place