Trial Outcomes & Findings for Dexamethasone in Controlling Dyspnea in Patients With Cancer (NCT NCT03367156)

NCT ID: NCT03367156

Last Updated: 2025-11-12

Results Overview

The average dyspnea intensity over the past 24 hours was assessed daily using a validated numeric rating scale from 0 to 10. The total score ranged from 0-10 where higher scores indicate worse dyspnea. The change in Dyspnea scores between Baseline and Day 7 were measured. Linear model analysis was used for analysis.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

135 participants

Primary outcome timeframe

Baseline and Day 7

Results posted on

2025-11-12

Participant Flow

Ambulatory patients with cancer age ≥18, average dyspnea intensity over the past week ≥4/10 in a 0-10 point numeric rating scale, Karnofsky performance status ≥30% and ability to communicate in English or Spanish were selected from MD Anderson Cancer Center and Lyndon B. Johnson General Hospital, Houston, Texas, United States.

A total of 135 participants were enrolled but 128 were randomized. 7 participants were not randomized for various reasons.

Participant milestones

Participant milestones
Measure
Dexamethasone
Participants were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Placebo
Placebo capsules were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Overall Study
STARTED
85
43
Overall Study
Completed 7 Days
76
38
Overall Study
Completed 14 Days
61
35
Overall Study
COMPLETED
61
35
Overall Study
NOT COMPLETED
24
8

Reasons for withdrawal

Reasons for withdrawal
Measure
Dexamethasone
Participants were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Placebo
Placebo capsules were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Overall Study
Lack of interest
3
1
Overall Study
Death
2
0
Overall Study
Lost to Follow-up
2
1
Overall Study
Became ineligible
1
2
Overall Study
Hospitalization
1
0
Overall Study
Concerned about Treatment
11
3
Overall Study
Started new treatment
4
1

Baseline Characteristics

Dexamethasone in Controlling Dyspnea in Patients With Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Dexamethasone
n=85 Participants
Participants were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Placebo
n=43 Participants
Placebo capsules were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Total
n=128 Participants
Total of all reporting groups
Age, Continuous
66 years
n=10 Participants
63 years
n=10 Participants
65 years
n=20 Participants
Sex: Female, Male
Female
50 Participants
n=10 Participants
24 Participants
n=10 Participants
74 Participants
n=20 Participants
Sex: Female, Male
Male
35 Participants
n=10 Participants
19 Participants
n=10 Participants
54 Participants
n=20 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
11 Participants
n=10 Participants
10 Participants
n=10 Participants
21 Participants
n=20 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
74 Participants
n=10 Participants
33 Participants
n=10 Participants
107 Participants
n=20 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=10 Participants
0 Participants
n=10 Participants
0 Participants
n=20 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=10 Participants
0 Participants
n=10 Participants
0 Participants
n=20 Participants
Race (NIH/OMB)
Asian
0 Participants
n=10 Participants
0 Participants
n=10 Participants
0 Participants
n=20 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=10 Participants
0 Participants
n=10 Participants
0 Participants
n=20 Participants
Race (NIH/OMB)
Black or African American
11 Participants
n=10 Participants
9 Participants
n=10 Participants
20 Participants
n=20 Participants
Race (NIH/OMB)
White
71 Participants
n=10 Participants
34 Participants
n=10 Participants
105 Participants
n=20 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=10 Participants
0 Participants
n=10 Participants
0 Participants
n=20 Participants
Race (NIH/OMB)
Unknown or Not Reported
3 Participants
n=10 Participants
0 Participants
n=10 Participants
3 Participants
n=20 Participants
Region of Enrollment
United States
85 participants
n=10 Participants
43 participants
n=10 Participants
128 participants
n=20 Participants
Cancer Stage
Stage I-II
18 Participants
n=10 Participants
15 Participants
n=10 Participants
33 Participants
n=20 Participants
Cancer Stage
Stage III
17 Participants
n=10 Participants
4 Participants
n=10 Participants
21 Participants
n=20 Participants
Cancer Stage
Stage IV
50 Participants
n=10 Participants
24 Participants
n=10 Participants
74 Participants
n=20 Participants
Cancer Type
Breast
5 Participants
n=10 Participants
2 Participants
n=10 Participants
7 Participants
n=20 Participants
Cancer Type
Gastrointestinal
6 Participants
n=10 Participants
7 Participants
n=10 Participants
13 Participants
n=20 Participants
Cancer Type
Genitourinary
2 Participants
n=10 Participants
4 Participants
n=10 Participants
6 Participants
n=20 Participants
Cancer Type
Respiratory
65 Participants
n=10 Participants
27 Participants
n=10 Participants
92 Participants
n=20 Participants
Cancer Type
Other
7 Participants
n=10 Participants
3 Participants
n=10 Participants
10 Participants
n=20 Participants
Comorbidity
COPD
32 Participants
n=10 Participants
12 Participants
n=10 Participants
44 Participants
n=20 Participants
Comorbidity
Asthma
6 Participants
n=10 Participants
5 Participants
n=10 Participants
11 Participants
n=20 Participants
Comorbidity
Bronchiectasis
1 Participants
n=10 Participants
1 Participants
n=10 Participants
2 Participants
n=20 Participants
Comorbidity
Heart Failure
5 Participants
n=10 Participants
3 Participants
n=10 Participants
8 Participants
n=20 Participants
Comorbidity
Pulmonary Embolism
2 Participants
n=10 Participants
1 Participants
n=10 Participants
3 Participants
n=20 Participants
Comorbidity
Other
5 Participants
n=10 Participants
1 Participants
n=10 Participants
6 Participants
n=20 Participants

PRIMARY outcome

Timeframe: Baseline and Day 7

Population: The Dyspnea Average Intensity Numeric score was missing for 4 participants in the Dexamethasone Arm and 1 participant in the Placebo Arm.

The average dyspnea intensity over the past 24 hours was assessed daily using a validated numeric rating scale from 0 to 10. The total score ranged from 0-10 where higher scores indicate worse dyspnea. The change in Dyspnea scores between Baseline and Day 7 were measured. Linear model analysis was used for analysis.

Outcome measures

Outcome measures
Measure
Dexamethasone
n=72 Participants
Participants were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Placebo
n=37 Participants
Placebo capsules were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Change in Dyspnea Numeric Score Over the Past 24 Hours for Baseline and Day 7 Average Intensity
-1.6 score on a scale
Interval -2.0 to -1.2
-1.6 score on a scale
Interval -2.3 to -0.9

SECONDARY outcome

Timeframe: Baseline and Day 14

Population: Participants who were on the study at day 14 and received assessment.

The average dyspnea intensity over the past 24 hours was assessed daily using a validated numeric rating scale from 0 to 10. The total score ranged from 0-10 where higher scores indicate worse dyspnea. The change in Dyspnea scores between Baseline and Day 14 were measured. Linear model analysis was used for analysis.

Outcome measures

Outcome measures
Measure
Dexamethasone
n=68 Participants
Participants were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Placebo
n=35 Participants
Placebo capsules were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Change in Dyspnea Numeric Score Over the Past 24 Hours for Baseline and Day 14 Average Intensity
-1.8 score on a scale
Interval -2.2 to -1.3
-1.9 score on a scale
Interval -2.6 to -1.2

SECONDARY outcome

Timeframe: Baseline and Day 7

Population: The Dyspnea Average Unpleasantness Numeric score was missing for 4 participants in the Dexamethasone Arm and 2 participant in the Placebo Arm.

The average dyspnea unpleasantness over the past 24 hours was assessed daily using a validated numeric rating scale from 0 to 10. The total score ranged from 0-10 where higher scores indicate worse dyspnea. The change in Dyspnea unpleasantness scores between Baseline and Day 7 were measured. Linear model analysis was used for analysis.

Outcome measures

Outcome measures
Measure
Dexamethasone
n=72 Participants
Participants were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Placebo
n=36 Participants
Placebo capsules were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Change in Dyspnea Numeric Score Over the Past 24 Hours for Baseline and Day 7 Average Unpleasantness
-1.8 score on a scale
Interval -2.3 to -1.2
-2.2 score on a scale
Interval -3.2 to -1.2

SECONDARY outcome

Timeframe: Baseline and Day 7

Population: The ESAS Dyspnea Numeric score was missing for 3 participants in the Dexamethasone Arm and 2 participant in the Placebo Arm.

ESAS (Edmonton Symptom Assessment Scale) is a validated scale ranging from 0 (not at all) to 10 (very much) used to assess 10 symptoms commonly experienced by cancer patients during the previous 24 hours: pain, fatigue, nausea, depression, anxiety, drowsiness, dyspnea, anorexia, sleep and feeling of well being. The change in ESAS fatigue score between Baseline and Day 7 were measured. Total ESAS fatigue score ranged from 0-10, with a higher score indicating higher fatigue. Linear model analysis was used for analysis.

Outcome measures

Outcome measures
Measure
Dexamethasone
n=73 Participants
Participants were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Placebo
n=36 Participants
Placebo capsules were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Change in Edmonton Symptom Assessment Scale (ESAS) Dyspnea Score Between Baseline and Day 7
-1 score on a scale
Interval -3.0 to -1.0
-1.5 score on a scale
Interval -3.0 to 0.0

SECONDARY outcome

Timeframe: Baseline and Day 7

Population: The EORTC QLQ-C30 Dyspnea score was missing for 4 participants in the Dexamethasone Arm and 2 participant in the Placebo Arm.

European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) is a well-validated quality-of-life assessment for patients with cancer, consisting of 30 questions that encompass three symptom scales (pain, fatigue, and nausea/vomiting) and six questions about single symptoms, as well as five functional scales (physical, cognitive, role, emotional, and social) and one scale assessing global health status/quality of life. Each single symptoms have four response categories (1=not at all, and 4=very much). The change in EORTC fatigue score between Baseline and Day 7 were measured. Total scores using a complex scoring procedures ranges from 0 to 100 with a higher scores indicating higher fatigue. Linear model analysis was used for analysis.

Outcome measures

Outcome measures
Measure
Dexamethasone
n=72 Participants
Participants were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Placebo
n=36 Participants
Placebo capsules were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Change in European Organization for Research and Treatment of Cancer-Quality of Life (EORTC QLQ-C30) Dyspnea Score Between Baseline and Day 7
-33.3 score on a scale
Interval -33.3 to 0.0
-33.3 score on a scale
Interval -33.3 to 0.0

SECONDARY outcome

Timeframe: Baseline and Day 14

Population: The Dyspnea Average Unpleasantness score was missing for 1 participant in the Placebo Arm. Participants who were on the study at day 14 and received assessment in the Dexamethasone Arm.

The average dyspnea unpleasantness over the past 24 hours was assessed daily using a validated numeric rating scale from 0 to 10. The total score ranged from 0-10 where higher scores indicate worse dyspnea. The change in Dyspnea unpleasantness scores between Baseline and Day 14 were measured. Linear model analysis was used for analysis.

Outcome measures

Outcome measures
Measure
Dexamethasone
n=68 Participants
Participants were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Placebo
n=34 Participants
Placebo capsules were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Change in Dyspnea Numeric Score Over the Past 24 Hours for Baseline and Day 14 Average Unpleasantness
-1.7 score on a scale
Interval -2.4 to -1.1
-2.3 score on a scale
Interval -3.4 to -1.3

SECONDARY outcome

Timeframe: Baseline and Day 14

Population: The ESAS Dyspnea score was missing for 3 participants in the Dexamethasone Arm and 2 participant in the Placebo Arm. Participants who were on the study at day 14 and received assessment in the Dexamethasone Arm.

ESAS (Edmonton Symptom Assessment Scale) is a validated scale ranging from 0 (not at all) to 10 (very much) used to assess 10 symptoms commonly experienced by cancer patients during the previous 24 hours: pain, fatigue, nausea, depression, anxiety, drowsiness, dyspnea, anorexia, sleep and feeling of well being. The change in ESAS fatigue score between Baseline and Day 14 were measured. Total ESAS fatigue score ranged from 0-10, with a higher score indicating higher fatigue. Linear model analysis was used for analysis.

Outcome measures

Outcome measures
Measure
Dexamethasone
n=65 Participants
Participants were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Placebo
n=33 Participants
Placebo capsules were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Change in Edmonton Symptom Assessment Scale (ESAS) Dyspnea Score Between Baseline and Day 14
-2 score on a scale
Interval -3.0 to 0.0
-1 score on a scale
Interval -4.0 to 0.0

SECONDARY outcome

Timeframe: Baseline and Day 14

Population: The EORTC Dyspnea score was missing for 2 participants in the Dexamethasone Arm and 3 participant in the Placebo Arm. Participants who were on the study at day 14 and received assessment in the Dexamethasone Arm.

European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) is a well-validated quality-of-life assessment for patients with cancer, consisting of 30 questions that encompass three symptom scales (pain, fatigue, and nausea/vomiting) and six questions about single symptoms, as well as five functional scales (physical, cognitive, role, emotional, and social) and one scale assessing global health status/quality of life. Each single symptoms have four response categories (1=not at all, and 4=very much). The change in EORTC fatigue score between Baseline and Day 14 were measured. Total scores using a complex scoring procedures ranges from 0 to 100 with a higher scores indicating higher fatigue. Linear model analysis was used for analysis.

Outcome measures

Outcome measures
Measure
Dexamethasone
n=66 Participants
Participants were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Placebo
n=32 Participants
Placebo capsules were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Change in European Organization for Research and Treatment of Cancer Quality of Life (EORTC) Dyspnea Score Between Baseline and Day 14
-33.3 score on a scale
Interval -33.3 to 0.0
-33.3 score on a scale
Interval -33.3 to 0.0

Adverse Events

Dexamethasone

Serious events: 24 serious events
Other events: 32 other events
Deaths: 2 deaths

Placebo

Serious events: 3 serious events
Other events: 7 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Dexamethasone
n=85 participants at risk
Participants were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Placebo
n=43 participants at risk
Placebo capsules were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Gastrointestinal disorders
Abdominal pain
2.4%
2/85 • Day 0 to Day 14
2.3%
1/43 • Day 0 to Day 14
Cardiac disorders
Cardiac Arrest
1.2%
1/85 • Day 0 to Day 14
0.00%
0/43 • Day 0 to Day 14
Renal and urinary disorders
Catheter related infection
1.2%
1/85 • Day 0 to Day 14
0.00%
0/43 • Day 0 to Day 14
Psychiatric disorders
Confusion
2.4%
2/85 • Day 0 to Day 14
0.00%
0/43 • Day 0 to Day 14
Gastrointestinal disorders
Dehydration
1.2%
1/85 • Day 0 to Day 14
0.00%
0/43 • Day 0 to Day 14
Psychiatric disorders
Depression
1.2%
1/85 • Day 0 to Day 14
0.00%
0/43 • Day 0 to Day 14
Musculoskeletal and connective tissue disorders
Fracture
1.2%
1/85 • Day 0 to Day 14
0.00%
0/43 • Day 0 to Day 14
Respiratory, thoracic and mediastinal disorders
Lung Infection
8.2%
7/85 • Day 0 to Day 14
4.7%
2/43 • Day 0 to Day 14
Cardiac disorders
Myocardial Infarction
1.2%
1/85 • Day 0 to Day 14
0.00%
0/43 • Day 0 to Day 14
Gastrointestinal disorders
Nausea
1.2%
1/85 • Day 0 to Day 14
0.00%
0/43 • Day 0 to Day 14
Psychiatric disorders
Pyschosis
1.2%
1/85 • Day 0 to Day 14
0.00%
0/43 • Day 0 to Day 14
Respiratory, thoracic and mediastinal disorders
Respiratory Distress
2.4%
2/85 • Day 0 to Day 14
0.00%
0/43 • Day 0 to Day 14
Cardiac disorders
Thromboembolic Event
2.4%
2/85 • Day 0 to Day 14
0.00%
0/43 • Day 0 to Day 14
Musculoskeletal and connective tissue disorders
Weakness
1.2%
1/85 • Day 0 to Day 14
0.00%
0/43 • Day 0 to Day 14

Other adverse events

Other adverse events
Measure
Dexamethasone
n=85 participants at risk
Participants were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Placebo
n=43 participants at risk
Placebo capsules were given 2 capsules (4mg each) by mouth twice daily (day1-day7), then 1 capsule twice daily for the next 7 days (day 8-day 14).
Respiratory, thoracic and mediastinal disorders
Respiratory Distress
2.4%
2/85 • Day 0 to Day 14
0.00%
0/43 • Day 0 to Day 14
Cardiac disorders
Myocardial Infarction
1.2%
1/85 • Day 0 to Day 14
2.3%
1/43 • Day 0 to Day 14
Nervous system disorders
Insomnia
8.2%
7/85 • Day 0 to Day 14
2.3%
1/43 • Day 0 to Day 14
Nervous system disorders
Pain
2.4%
2/85 • Day 0 to Day 14
2.3%
1/43 • Day 0 to Day 14
Nervous system disorders
Neuropsychiatric symptoms
3.5%
3/85 • Day 0 to Day 14
0.00%
0/43 • Day 0 to Day 14
Gastrointestinal disorders
Dyspepsia
2.4%
2/85 • Day 0 to Day 14
0.00%
0/43 • Day 0 to Day 14
General disorders
Infection
10.6%
9/85 • Day 0 to Day 14
7.0%
3/43 • Day 0 to Day 14
General disorders
Fatigue
3.5%
3/85 • Day 0 to Day 14
0.00%
0/43 • Day 0 to Day 14
Cardiac disorders
Hypertension
2.4%
2/85 • Day 0 to Day 14
0.00%
0/43 • Day 0 to Day 14
Gastrointestinal disorders
Nausea
1.2%
1/85 • Day 0 to Day 14
2.3%
1/43 • Day 0 to Day 14

Additional Information

Dr. David Hui- Professor, Palliative Care Medicine

UT MD Anderson Cancer Center

Phone: (713) 792-6258

Results disclosure agreements

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