Trial Outcomes & Findings for Netupitant and Palonosetron Hydrochloride in Preventing Chronic Nausea and Vomiting in Patients With Cancer (NCT NCT03040726)

NCT ID: NCT03040726

Last Updated: 2023-10-17

Results Overview

Average intensity of nausea over the past 24 hours was assessed daily using a validated numeric rating scale from 0 to 10, where 0= none and 10= worse possible nausea. The total score ranged from 0-10. We measured the within-group change in nausea intensity from day 5 to day 15. Wilcoxon rank sum test was used for analysis.

Recruitment status

COMPLETED

Study phase

PHASE2/PHASE3

Target enrollment

53 participants

Primary outcome timeframe

Day 5 and Day 15

Results posted on

2023-10-17

Participant Flow

Cancer patients with age \>=18, chronic nausea over the past 4 weeks, an average nausea numeric rating scale (NRS) \>=4/10 over the past 5 days were recruited from the Supportive Care, Gastrointestinal Medical Oncology, Breast Medical Oncology, and the Gastroenterology, Hepatology and Nutrition clinics at the University of Texas MD Anderson Cancer Center.

A total of 53 participants were enrolled but 46 were randomized. 7 participants were not randomized for various reasons that included "Change in cancer treatment" (n=3), "Developed renal failure" (n=1), "Developed bowel obstruction" (n=1) and "Other" (n=2)

Participant milestones

Participant milestones
Measure
Blinded Intervention gp.
Took one Netupitant/Palonosetron (NEPA) capsule every 5 days for 2 doses
Blinded Placebo gp.
Took one Placebo capsule every 5 days for 2 doses
Overall Study
STARTED
31
15
Overall Study
Started Placebo run-in Phase
31
15
Overall Study
Started Blinded Phase
22
11
Overall Study
COMPLETED
21
9
Overall Study
NOT COMPLETED
10
6

Reasons for withdrawal

Reasons for withdrawal
Measure
Blinded Intervention gp.
Took one Netupitant/Palonosetron (NEPA) capsule every 5 days for 2 doses
Blinded Placebo gp.
Took one Placebo capsule every 5 days for 2 doses
Overall Study
Reasons for not completing Placebo run in phase phase; Drop Out
4
0
Overall Study
Reasons for not completing Placebo run in phase phase; withdrawal
1
1
Overall Study
Reasons for not completing Placebo run in phase: Nausea NRS score >4
4
3
Overall Study
Reasons for not completing Blinded phase: Hospitalization
0
2
Overall Study
Reasons for not completing Blinded phase : Withdrawal
1
0

Baseline Characteristics

Netupitant and Palonosetron Hydrochloride in Preventing Chronic Nausea and Vomiting in Patients With Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Blinded Intervention gp.
n=31 Participants
Took one Netupitant/Palonosetron (NEPA) capsule every 5 days for 2 doses
Blinded Placebo gp.
n=15 Participants
Took Placebo capsule every 5 days for 2 doses
Total
n=46 Participants
Total of all reporting groups
Age, Continuous
56.2 years
n=5 Participants
52.7 years
n=7 Participants
55 years
n=5 Participants
Sex: Female, Male
Female
23 Participants
n=5 Participants
7 Participants
n=7 Participants
30 Participants
n=5 Participants
Sex: Female, Male
Male
8 Participants
n=5 Participants
8 Participants
n=7 Participants
16 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
3 Participants
n=5 Participants
4 Participants
n=7 Participants
7 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
28 Participants
n=5 Participants
11 Participants
n=7 Participants
39 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
9 Participants
n=5 Participants
3 Participants
n=7 Participants
12 Participants
n=5 Participants
Race (NIH/OMB)
White
21 Participants
n=5 Participants
12 Participants
n=7 Participants
33 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Region of Enrollment
United States
31 Participants
n=5 Participants
15 Participants
n=7 Participants
46 Participants
n=5 Participants
Cancer Stage
Metastatic
15 Participants
n=5 Participants
9 Participants
n=7 Participants
24 Participants
n=5 Participants
Cancer Stage
Locally advanced
15 Participants
n=5 Participants
6 Participants
n=7 Participants
21 Participants
n=5 Participants
Cancer Stage
Recurrent or persistent
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants
Cancer Type
Breast
4 Participants
n=5 Participants
1 Participants
n=7 Participants
5 Participants
n=5 Participants
Cancer Type
Gastrointestinal
21 Participants
n=5 Participants
12 Participants
n=7 Participants
33 Participants
n=5 Participants
Cancer Type
Genitourinary
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants
Cancer Type
Respiratory
3 Participants
n=5 Participants
0 Participants
n=7 Participants
3 Participants
n=5 Participants
Cancer Type
Hematology
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants
Cancer Type
Gynaecological
0 Participants
n=5 Participants
1 Participants
n=7 Participants
1 Participants
n=5 Participants
Cancer Type
Other
1 Participants
n=5 Participants
1 Participants
n=7 Participants
2 Participants
n=5 Participants

PRIMARY outcome

Timeframe: Day 5 and Day 15

Population: Those who completed Day 15

Average intensity of nausea over the past 24 hours was assessed daily using a validated numeric rating scale from 0 to 10, where 0= none and 10= worse possible nausea. The total score ranged from 0-10. We measured the within-group change in nausea intensity from day 5 to day 15. Wilcoxon rank sum test was used for analysis.

Outcome measures

Outcome measures
Measure
Blinded Intervention Group
n=21 Participants
Netupitant/Palonosetron (NEPA) capsule every 5 days for 2 doses
Blinded Placebo Group
n=10 Participants
Placebo capsule every 5 days for 2 doses
Change in Nausea Numerical Rating Scale (NRS) Between Day 5 and Day 15
-2.0 score on a scale
Interval -3.1 to -0.8
-2.3 score on a scale
Interval -3.9 to -0.7

SECONDARY outcome

Timeframe: Baseline and Day 15

Population: Those who completed Day 15

FLIE is a questionnaire validated to assess the impact of chemotherapy induced nausea and vomiting on patient's function and quality of life over the past 5 days. It consists of 18 items, with 9 items on nausea and 9 items on vomiting. Each question was rated using a Numerical Rating Scale (NRS) from 1 to 7. The total Nausea sub-score ranges from 9 to 63, where a higher score indicates higher quality of life. We measured the change of nausea sub-score between baseline 5 to day 15. Wilcoxon rank sum test was used for analysis.

Outcome measures

Outcome measures
Measure
Blinded Intervention Group
n=21 Participants
Netupitant/Palonosetron (NEPA) capsule every 5 days for 2 doses
Blinded Placebo Group
n=9 Participants
Placebo capsule every 5 days for 2 doses
Functional Living Index Emesis (FLIE): Nausea Sub-score
-16.6 score on a scale
Interval -22.6 to -10.5
-18 score on a scale
Interval -22.0 to -14.0

SECONDARY outcome

Timeframe: Baseline and Day 15

Population: Those who completed Day 15

FLIE is a questionnaire validated to assess the impact of chemotherapy induced nausea and vomiting on patient's function and quality of life over the past 5 days. It consists of 18 items, with 9 items on nausea and 9 items on vomiting. Each question was rated using a Numerical Rating Scale (NRS) from 1 to 7. The total Vomiting sub-score ranges from 9 to 63, where a higher score indicates higher quality of life. We measured the change of vomiting sub-score from baseline to day 15.Wilcoxon rank sum test was used for analysis.

Outcome measures

Outcome measures
Measure
Blinded Intervention Group
n=21 Participants
Netupitant/Palonosetron (NEPA) capsule every 5 days for 2 doses
Blinded Placebo Group
n=9 Participants
Placebo capsule every 5 days for 2 doses
Functional Living Index Emesis (FLIE): Vomiting Sub-score
-12.5 score on a scale
Interval -20.8 to -4.2
-9.7 score on a scale
Interval -18.2 to -1.2

SECONDARY outcome

Timeframe: Day 5 and Day 15

Population: Those who completed Day 15

Index of Nausea, Vomiting, and Retching, which consists of 8 items asking about the patient's experience regarding nausea and vomiting over the past 12 hours. Each item included a 5-point Likert scale (0-4 points) with descriptive words. Total experience score ranged from 0-32 with higher score indicates more nausea/vomiting. We measured the change in score between day 5 and day 15. Wilcoxon rank sum test was used for analysis.

Outcome measures

Outcome measures
Measure
Blinded Intervention Group
n=21 Participants
Netupitant/Palonosetron (NEPA) capsule every 5 days for 2 doses
Blinded Placebo Group
n=10 Participants
Placebo capsule every 5 days for 2 doses
Index of Nausea, Vomiting and Retching: Total Experience Score
-2.3 score on a scale
Interval -4.4 to -0.3
-2.5 score on a scale
Interval -6.9 to 1.9

Adverse Events

Blinded Intervention gp.

Serious events: 1 serious events
Other events: 10 other events
Deaths: 3 deaths

Placebo gp.

Serious events: 3 serious events
Other events: 9 other events
Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure
Blinded Intervention gp.
n=30 participants at risk
Took one Netupitant/Palonosetron (NEPA) capsule every 5 days for 2 doses
Placebo gp.
n=43 participants at risk
Took one Placebo capsule every 5 days for 2 doses
Gastrointestinal disorders
Dehydration
3.3%
1/30 • Study Day 15
Adverse events were assessed within 15 study days
0.00%
0/43 • Study Day 15
Adverse events were assessed within 15 study days
Gastrointestinal disorders
Hepatic failure
3.3%
1/30 • Study Day 15
Adverse events were assessed within 15 study days
0.00%
0/43 • Study Day 15
Adverse events were assessed within 15 study days
Gastrointestinal disorders
Abdominal pain
0.00%
0/30 • Study Day 15
Adverse events were assessed within 15 study days
2.3%
1/43 • Study Day 15
Adverse events were assessed within 15 study days
Renal and urinary disorders
Acute Kidney imjury
0.00%
0/30 • Study Day 15
Adverse events were assessed within 15 study days
2.3%
1/43 • Study Day 15
Adverse events were assessed within 15 study days
General disorders
Death NOS
0.00%
0/30 • Study Day 15
Adverse events were assessed within 15 study days
4.7%
2/43 • Study Day 15
Adverse events were assessed within 15 study days

Other adverse events

Other adverse events
Measure
Blinded Intervention gp.
n=30 participants at risk
Took one Netupitant/Palonosetron (NEPA) capsule every 5 days for 2 doses
Placebo gp.
n=43 participants at risk
Took one Placebo capsule every 5 days for 2 doses
Respiratory, thoracic and mediastinal disorders
Cough
3.3%
1/30 • Study Day 15
Adverse events were assessed within 15 study days
0.00%
0/43 • Study Day 15
Adverse events were assessed within 15 study days
Gastrointestinal disorders
Abdominal Pain
3.3%
1/30 • Study Day 15
Adverse events were assessed within 15 study days
0.00%
0/43 • Study Day 15
Adverse events were assessed within 15 study days
Musculoskeletal and connective tissue disorders
Back pain
3.3%
1/30 • Study Day 15
Adverse events were assessed within 15 study days
2.3%
1/43 • Study Day 15
Adverse events were assessed within 15 study days
Nervous system disorders
Dizziness
0.00%
0/30 • Study Day 15
Adverse events were assessed within 15 study days
2.3%
1/43 • Study Day 15
Adverse events were assessed within 15 study days
Nervous system disorders
Headache
10.0%
3/30 • Study Day 15
Adverse events were assessed within 15 study days
7.0%
3/43 • Study Day 15
Adverse events were assessed within 15 study days
Gastrointestinal disorders
Dyspepsia
0.00%
0/30 • Study Day 15
Adverse events were assessed within 15 study days
2.3%
1/43 • Study Day 15
Adverse events were assessed within 15 study days
Gastrointestinal disorders
Stomach pain
0.00%
0/30 • Study Day 15
Adverse events were assessed within 15 study days
2.3%
1/43 • Study Day 15
Adverse events were assessed within 15 study days
Gastrointestinal disorders
Constipation
3.3%
1/30 • Study Day 15
Adverse events were assessed within 15 study days
2.3%
1/43 • Study Day 15
Adverse events were assessed within 15 study days
General disorders
Edema face
0.00%
0/30 • Study Day 15
Adverse events were assessed within 15 study days
2.3%
1/43 • Study Day 15
Adverse events were assessed within 15 study days
General disorders
Edema legs
6.7%
2/30 • Study Day 15
Adverse events were assessed within 15 study days
0.00%
0/43 • Study Day 15
Adverse events were assessed within 15 study days
General disorders
Fatigue
6.7%
2/30 • Study Day 15
Adverse events were assessed within 15 study days
0.00%
0/43 • Study Day 15
Adverse events were assessed within 15 study days
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder - Other, specify
3.3%
1/30 • Study Day 15
Adverse events were assessed within 15 study days
0.00%
0/43 • Study Day 15
Adverse events were assessed within 15 study days
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, specify
3.3%
1/30 • Study Day 15
Adverse events were assessed within 15 study days
0.00%
0/43 • Study Day 15
Adverse events were assessed within 15 study days
Respiratory, thoracic and mediastinal disorders
Dyspnea
3.3%
1/30 • Study Day 15
Adverse events were assessed within 15 study days
0.00%
0/43 • Study Day 15
Adverse events were assessed within 15 study days

Additional Information

Dr. David Hui

The University of Texas MD Anderson Cancer Center

Phone: (713) 792-6258

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place