Trial Outcomes & Findings for Precision Medicine for Patients With Identified Actionable Mutations (NCT NCT04111107)

Last Updated: 2024-07-03

Results Overview

Estimation of progression-free ratio defined as the duration of time from start of treatment to the time of progression divided by the duration of time from the last treatment received pre-trial to the time of progression on that treatment. The median progression-free ratio will be estimated with the range and a two-sided Wilcoxon Signed Rank test will be calculated to see if the progression free survival ratio is different from 1.0. This trial is powered to detect differences in the progression-free ratio for those with actionable mutations identified by NGS results and then treated with a targeted therapy. A hypothesized PFS ratio larger than 1.3 would suggest that the targeted therapy is doing better than the previous treatment received (not targeted), and we assume a null hypothesis PFS ratio of 1.0 (no difference).

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

45 participants

Primary outcome timeframe

From the start of treatment to the time of progression, death, or date of last contact, assessed up to 2 years

Results posted on

2024-07-03

Participant Flow

Study participants were recruited between April 2020 and September 2022.

Participant milestones

Participant milestones
Measure	Drug Administration Based on Next Gen Sequencing Report Investigators will select the first drug listed in the tumor analysis report for the first mutation listed in the tumor analysis report. However, If the subject has a medical contraindication to the first listed drug (according to the drug label) or the first listed drug cannot be obtained for the patient, the study team will select the next drug presented by the tumor sequencing report. Patients receive targeted therapy based on next generation sequencing report. Cycles repeat every 2, 4, or 6 weeks in the absence of disease progression or unacceptable toxicity. Investigational Agent: Treatment will be administered on an inpatient or outpatient basis based on the type of medication selected. The investigatory will administer the drug as directed in the FDA approved label. Supportive Care Regimens: Supportive care regimens will vary depending on the type of drug that will be administered at the treating investigator's discretion. Next Gen Sequencing Report: 1) Next Gen Sequencing report obtained as Standard of Care within the past 12 months of enrollment date will be used. If more than one report exists in this time period, the most recent report will be used.
Overall Study STARTED	45
Overall Study COMPLETED	34
Overall Study NOT COMPLETED	11

Reasons for withdrawal

Reasons for withdrawal
Measure	Drug Administration Based on Next Gen Sequencing Report Investigators will select the first drug listed in the tumor analysis report for the first mutation listed in the tumor analysis report. However, If the subject has a medical contraindication to the first listed drug (according to the drug label) or the first listed drug cannot be obtained for the patient, the study team will select the next drug presented by the tumor sequencing report. Patients receive targeted therapy based on next generation sequencing report. Cycles repeat every 2, 4, or 6 weeks in the absence of disease progression or unacceptable toxicity. Investigational Agent: Treatment will be administered on an inpatient or outpatient basis based on the type of medication selected. The investigatory will administer the drug as directed in the FDA approved label. Supportive Care Regimens: Supportive care regimens will vary depending on the type of drug that will be administered at the treating investigator's discretion. Next Gen Sequencing Report: 1) Next Gen Sequencing report obtained as Standard of Care within the past 12 months of enrollment date will be used. If more than one report exists in this time period, the most recent report will be used.
Overall Study Withdrawal by Subject	6
Overall Study Declining Health	5

Baseline Characteristics

Precision Medicine for Patients With Identified Actionable Mutations

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Drug Administration Based on Next Gen Sequencing Report n=45 Participants Investigators will select the first drug listed in the tumor analysis report for the first mutation listed in the tumor analysis report. However, If the subject has a medical contraindication to the first listed drug (according to the drug label) or the first listed drug cannot be obtained for the patient, the study team will select the next drug presented by the tumor sequencing report. Patients receive targeted therapy based on next generation sequencing report. Cycles repeat every 2, 4, or 6 weeks in the absence of disease progression or unacceptable toxicity. Investigational Agent: Treatment will be administered on an inpatient or outpatient basis based on the type of medication selected. The investigatory will administer the drug as directed in the FDA approved label. Supportive Care Regimens: Supportive care regimens will vary depending on the type of drug that will be administered at the treating investigator's discretion. Next Gen Sequencing Report: 1) Next Gen Sequencing report obtained as Standard of Care within the past 12 months of enrollment date will be used. If more than one report exists in this time period, the most recent report will be used.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	17 Participants n=5 Participants
Age, Categorical >=65 years	28 Participants n=5 Participants
Age, Continuous	66 years STANDARD_DEVIATION 12.3 • n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants
Sex: Female, Male Male	31 Participants n=5 Participants
Race/Ethnicity, Customized Caucasian/White	36 Participants n=5 Participants
Race/Ethnicity, Customized African American/Black	8 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic/Latino	1 Participants n=5 Participants
Region of Enrollment United States	45 Participants n=5 Participants
Disease Site Lung	24 Participants n=5 Participants
Disease Site Head and Neck	13 Participants n=5 Participants
Disease Site Other	8 Participants n=5 Participants

PRIMARY outcome

Timeframe: From the start of treatment to the time of progression, death, or date of last contact, assessed up to 2 years

Population: For various reasons, 27 patients were not evaluable for the primary outcome. Five patients had no prior treatment but did have an actionable mutation to qualify them for inclusion in the study. Another ten patients were enrolled but never started treatment under this protocol. Nine patients started treatment but died prior to testing for best response and for progression. Two patients died with stable disease and one patient is alive with stable disease and no sign of progression .

Outcome measures

Outcome measures
Measure	Drug Administration Based on Next Gen Sequencing Report n=18 Participants Investigators will select the first drug listed in the tumor analysis report for the first mutation listed in the tumor analysis report. However, If the subject has a medical contraindication to the first listed drug (according to the drug label) or the first listed drug cannot be obtained for the patient, the study team will select the next drug presented by the tumor sequencing report. Patients receive targeted therapy based on next generation sequencing report. Cycles repeat every 2, 4, or 6 weeks in the absence of disease progression or unacceptable toxicity. Investigational Agent: Treatment will be administered on an inpatient or outpatient basis based on the type of medication selected. The investigatory will administer the drug as directed in the FDA approved label. Supportive Care Regimens: Supportive care regimens will vary depending on the type of drug that will be administered at the treating investigator's discretion. Next Gen Sequencing Report: 1) Next Gen Sequencing report obtained as Standard of Care within the past 12 months of enrollment date will be used. If more than one report exists in this time period, the most recent report will be used.
Progression Free Survival Ratio	0.73 ratio Interval 0.26 to 6.67

SECONDARY outcome

Timeframe: From the start of treatment to date of death or date of last contact, up to 2 years

Population: All enrolled participants, regardless of length of treatment.

Overall survival will be displayed using Kaplan-Meier curves with median survival times and 95% confidence intervals.

Outcome measures

Outcome measures
Measure	Drug Administration Based on Next Gen Sequencing Report n=45 Participants Investigators will select the first drug listed in the tumor analysis report for the first mutation listed in the tumor analysis report. However, If the subject has a medical contraindication to the first listed drug (according to the drug label) or the first listed drug cannot be obtained for the patient, the study team will select the next drug presented by the tumor sequencing report. Patients receive targeted therapy based on next generation sequencing report. Cycles repeat every 2, 4, or 6 weeks in the absence of disease progression or unacceptable toxicity. Investigational Agent: Treatment will be administered on an inpatient or outpatient basis based on the type of medication selected. The investigatory will administer the drug as directed in the FDA approved label. Supportive Care Regimens: Supportive care regimens will vary depending on the type of drug that will be administered at the treating investigator's discretion. Next Gen Sequencing Report: 1) Next Gen Sequencing report obtained as Standard of Care within the past 12 months of enrollment date will be used. If more than one report exists in this time period, the most recent report will be used.
Overall Survival	6.2 months Interval 4.3 to 8.8

SECONDARY outcome

Timeframe: Up to 30 days after treatment ends, up to 33 months

Population: Eleven patients did not begin treatment under this protocol.

Adverse events will be summarized in incidence tables by type for all patients who received at least one cycle of treatment.

Outcome measures

Outcome measures
Measure	Drug Administration Based on Next Gen Sequencing Report n=34 Participants Investigators will select the first drug listed in the tumor analysis report for the first mutation listed in the tumor analysis report. However, If the subject has a medical contraindication to the first listed drug (according to the drug label) or the first listed drug cannot be obtained for the patient, the study team will select the next drug presented by the tumor sequencing report. Patients receive targeted therapy based on next generation sequencing report. Cycles repeat every 2, 4, or 6 weeks in the absence of disease progression or unacceptable toxicity. Investigational Agent: Treatment will be administered on an inpatient or outpatient basis based on the type of medication selected. The investigatory will administer the drug as directed in the FDA approved label. Supportive Care Regimens: Supportive care regimens will vary depending on the type of drug that will be administered at the treating investigator's discretion. Next Gen Sequencing Report: 1) Next Gen Sequencing report obtained as Standard of Care within the past 12 months of enrollment date will be used. If more than one report exists in this time period, the most recent report will be used.
Number of Participants With Adverse Events	34 Participants

SECONDARY outcome

Timeframe: Up to 30 days after treatment ends, up to 33 months

Population: Eleven patients did not receive study treatment (due to patient withdrawal or declining health) and ten patients died prior to evaluation for response.

Response rate will be estimated for all patients with corresponding 95% confidence intervals. Complete response is the disappearance of all target lesions and normalization of tumor marker level. Any pathological lymph nodes must have reduction in short axis to less than 10 mm. Partial response is at least 30% decrease in the sum of diameters of target lesions. Progressive disease is greater than 20% increase and a minimum 5 mm increase over the nadir. Stable disease is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study.

Outcome measures

Outcome measures
Measure	Drug Administration Based on Next Gen Sequencing Report n=24 Participants Investigators will select the first drug listed in the tumor analysis report for the first mutation listed in the tumor analysis report. However, If the subject has a medical contraindication to the first listed drug (according to the drug label) or the first listed drug cannot be obtained for the patient, the study team will select the next drug presented by the tumor sequencing report. Patients receive targeted therapy based on next generation sequencing report. Cycles repeat every 2, 4, or 6 weeks in the absence of disease progression or unacceptable toxicity. Investigational Agent: Treatment will be administered on an inpatient or outpatient basis based on the type of medication selected. The investigatory will administer the drug as directed in the FDA approved label. Supportive Care Regimens: Supportive care regimens will vary depending on the type of drug that will be administered at the treating investigator's discretion. Next Gen Sequencing Report: 1) Next Gen Sequencing report obtained as Standard of Care within the past 12 months of enrollment date will be used. If more than one report exists in this time period, the most recent report will be used.
Response Rate Partial Response	1 Participants
Response Rate Stable Disease	10 Participants
Response Rate Progressive Disease	13 Participants

Adverse Events

Drug Administration Based on Next Gen Sequencing Report

Serious events: 18 serious events

Other events: 34 other events

Deaths: 32 deaths

Serious adverse events

Serious adverse events
Measure	Drug Administration Based on Next Gen Sequencing Report n=34 participants at risk Investigators will select the first drug listed in the tumor analysis report for the first mutation listed in the tumor analysis report. However, If the subject has a medical contraindication to the first listed drug (according to the drug label) or the first listed drug cannot be obtained for the patient, the study team will select the next drug presented by the tumor sequencing report. Patients receive targeted therapy based on next generation sequencing report. Cycles repeat every 2, 4, or 6 weeks in the absence of disease progression or unacceptable toxicity. Investigational Agent: Treatment will be administered on an inpatient or outpatient basis based on the type of medication selected. The investigatory will administer the drug as directed in the FDA approved label. Supportive Care Regimens: Supportive care regimens will vary depending on the type of drug that will be administered at the treating investigator's discretion. Next Gen Sequencing Report: 1) Next Gen Sequencing report obtained as Standard of Care within the past 12 months of enrollment date will be used. If more than one report exists in this time period, the most recent report will be used.
Blood and lymphatic system disorders Anemia	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Cardiac disorders Pericardial effusion	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Eye disorders Retinal detachment	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Gastrointestinal disorders Abdominal pain	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Gastrointestinal disorders Duodenal hemorrhage	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Gastrointestinal disorders Dysphagia	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Gastrointestinal disorders Nausea	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Gastrointestinal disorders Vomiting	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
General disorders Chills	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
General disorders Disease Progression	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
General disorders Fatigue	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
General disorders Fever	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Hepatobiliary disorders Portal vein thrombosis	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Infections and infestations Lung infection	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Infections and infestations Sepsis	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Injury, poisoning and procedural complications Fall	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Investigations CPK increased	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Investigations Weight loss	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Metabolism and nutrition disorders Dehydration	11.8% 4/34 • Number of events 4 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Metabolism and nutrition disorders Hyponatremia	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Musculoskeletal and connective tissue disorders Back pain	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Musculoskeletal and connective tissue disorders Generalized muscle weakness	8.8% 3/34 • Number of events 3 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Musculoskeletal and connective tissue disorders Pain in extremity	2.9% 1/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Nervous system disorders Cognitive disturbance	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Nervous system disorders Edema cerebral	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Nervous system disorders Headache	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Nervous system disorders Lethargy	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Nervous system disorders Recurrent laryngeal nerve palsy	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Nervous system disorders Seizure	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Renal and urinary disorders Acute kidney injury	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Renal and urinary disorders Urinary tract obstruction	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Respiratory, thoracic and mediastinal disorders Dyspnea	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Respiratory, thoracic and mediastinal disorders Pleural effusion	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Respiratory, thoracic and mediastinal disorders Laryngeal obstruction	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Respiratory, thoracic and mediastinal disorders Pleuritic pain	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Respiratory, thoracic and mediastinal disorders Productive cough	2.9% 1/34 • Number of events 1 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.

Other adverse events

Other adverse events
Measure	Drug Administration Based on Next Gen Sequencing Report n=34 participants at risk Investigators will select the first drug listed in the tumor analysis report for the first mutation listed in the tumor analysis report. However, If the subject has a medical contraindication to the first listed drug (according to the drug label) or the first listed drug cannot be obtained for the patient, the study team will select the next drug presented by the tumor sequencing report. Patients receive targeted therapy based on next generation sequencing report. Cycles repeat every 2, 4, or 6 weeks in the absence of disease progression or unacceptable toxicity. Investigational Agent: Treatment will be administered on an inpatient or outpatient basis based on the type of medication selected. The investigatory will administer the drug as directed in the FDA approved label. Supportive Care Regimens: Supportive care regimens will vary depending on the type of drug that will be administered at the treating investigator's discretion. Next Gen Sequencing Report: 1) Next Gen Sequencing report obtained as Standard of Care within the past 12 months of enrollment date will be used. If more than one report exists in this time period, the most recent report will be used.
Blood and lymphatic system disorders Anemia	38.2% 13/34 • Number of events 34 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Cardiac disorders Heart Failure	5.9% 2/34 • Number of events 3 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Cardiac disorders Sinus tachycardia	20.6% 7/34 • Number of events 9 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Eye disorders Vision decreased	11.8% 4/34 • Number of events 4 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Gastrointestinal disorders Abdominal pain	17.6% 6/34 • Number of events 7 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Gastrointestinal disorders Constipation	20.6% 7/34 • Number of events 7 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Gastrointestinal disorders Diarrhea	32.4% 11/34 • Number of events 12 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Gastrointestinal disorders Dysphagia	8.8% 3/34 • Number of events 4 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Gastrointestinal disorders Gastroesophageal reflux disease	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Gastrointestinal disorders Mucositis oral	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Gastrointestinal disorders Nausea	35.3% 12/34 • Number of events 14 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Gastrointestinal disorders Rectal- hemorrhage	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Gastrointestinal disorders Vomiting	38.2% 13/34 • Number of events 14 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
General disorders Chills	14.7% 5/34 • Number of events 5 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
General disorders Edema limbs	17.6% 6/34 • Number of events 7 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
General disorders Fatigue	55.9% 19/34 • Number of events 23 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
General disorders Fever	11.8% 4/34 • Number of events 6 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
General disorders Malaise	17.6% 6/34 • Number of events 6 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
General disorders Pain	11.8% 4/34 • Number of events 5 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Infections and infestations Lung infection	8.8% 3/34 • Number of events 3 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Infections and infestations Otitis media	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Infections and infestations Papulopustular rash	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Infections and infestations Pustular rash	5.9% 2/34 • Number of events 4 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Infections and infestations Upper respiratory infection	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Injury, poisoning and procedural complications Fall	17.6% 6/34 • Number of events 7 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Investigations Alanine aminotransferase increased	20.6% 7/34 • Number of events 9 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Investigations Alkaline phosphatase increased	44.1% 15/34 • Number of events 24 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Investigations Aspartate aminotransferase increased	26.5% 9/34 • Number of events 12 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Investigations CPK increased	8.8% 3/34 • Number of events 5 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Investigations Creatinine increased	11.8% 4/34 • Number of events 6 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Investigations Lymphocyte count decreased	64.7% 22/34 • Number of events 40 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Investigations Neutrophil count decreased	23.5% 8/34 • Number of events 14 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Investigations Platelet count decreased	41.2% 14/34 • Number of events 30 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Investigations Weight loss	8.8% 3/34 • Number of events 3 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Investigations White blood cell decreased	44.1% 15/34 • Number of events 28 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Metabolism and nutrition disorders Anorexia	20.6% 7/34 • Number of events 8 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Metabolism and nutrition disorders Dehydration	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Metabolism and nutrition disorders Hyperglycemia	41.2% 14/34 • Number of events 21 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Metabolism and nutrition disorders Hypoalbuminemia	50.0% 17/34 • Number of events 28 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Metabolism and nutrition disorders Hypocalcemia	44.1% 15/34 • Number of events 32 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Metabolism and nutrition disorders Hypokalemia	14.7% 5/34 • Number of events 6 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Metabolism and nutrition disorders Hypomagnesemia	29.4% 10/34 • Number of events 11 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Metabolism and nutrition disorders Hyponatremia	55.9% 19/34 • Number of events 34 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Musculoskeletal and connective tissue disorders Back pain	8.8% 3/34 • Number of events 3 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Musculoskeletal and connective tissue disorders Chest wall pain	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Musculoskeletal and connective tissue disorders Generalized muscle weakness	20.6% 7/34 • Number of events 7 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Musculoskeletal and connective tissue disorders Muscle cramps	8.8% 3/34 • Number of events 3 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Musculoskeletal and connective tissue disorders Muscle weakness lower limb	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Musculoskeletal and connective tissue disorders Neck pain	8.8% 3/34 • Number of events 3 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Musculoskeletal and connective tissue disorders Pain in extremity	14.7% 5/34 • Number of events 7 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Nervous system disorders Cognitive disturbance	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Nervous system disorders Dizziness	8.8% 3/34 • Number of events 4 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Nervous system disorders Dysgeusia	11.8% 4/34 • Number of events 4 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Nervous system disorders Headache	17.6% 6/34 • Number of events 10 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Nervous system disorders Lethargy	11.8% 4/34 • Number of events 4 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Nervous system disorders Memory impairment	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Nervous system disorders Peripheral sensory neuropathy	8.8% 3/34 • Number of events 3 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Nervous system disorders Tremor	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Psychiatric disorders Confusion	11.8% 4/34 • Number of events 4 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Renal and urinary disorders Urinary retention	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Respiratory, thoracic and mediastinal disorders Cough	14.7% 5/34 • Number of events 5 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Respiratory, thoracic and mediastinal disorders Dyspnea	20.6% 7/34 • Number of events 7 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Respiratory, thoracic and mediastinal disorders Hoarseness	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Respiratory, thoracic and mediastinal disorders Nasal congestion	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Respiratory, thoracic and mediastinal disorders Pneumonitis	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Respiratory, thoracic and mediastinal disorders Productive cough	8.8% 3/34 • Number of events 3 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Respiratory, thoracic and mediastinal disorders Sore throat	8.8% 3/34 • Number of events 3 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Skin and subcutaneous tissue disorders Pruritis	8.8% 3/34 • Number of events 3 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Skin and subcutaneous tissue disorders Rash maculo-papular	11.8% 4/34 • Number of events 5 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Vascular disorders Hypertension	23.5% 8/34 • Number of events 13 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.
Vascular disorders Hypotension	5.9% 2/34 • Number of events 2 • Participants were followed for adverse events a minimum of 30 days after date of last study drug administration up to 33 months.

Additional Information

Stefan Grant, MD

Tulane University

Phone: 504-988-5800

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place