Trial Outcomes & Findings for Sargramostim Use in COVID-19 to Recover Patient Health (NCT NCT04707664)

NCT ID: NCT04707664

Last Updated: 2023-02-06

Results Overview

Percentage of patients who experience any emergency room visit or hospitalization, or death

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

600 participants

Primary outcome timeframe

28 days

Results posted on

2023-02-06

Participant Flow

Participant milestones

Participant milestones
Measure	Sargramostim Arm Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: All patients randomized to the sargramostim treatment arm will be treated with 250 mcg inhaled sargramostim administered via a vibrating mesh nebulizer once daily for 5 days.	Placebo Arm Day 1 - 5: Placebo treatment in addition to standard of care for COVID-19 Placebo: All patients in the control arm will receive an equivalent volume of inhaled placebo diluent administered via a vibrating mesh nebulizer once daily for 5 days.
Overall Study STARTED	301	299
Overall Study Full Analysis Set	301	299
Overall Study Safety Population	297	290
Overall Study COMPLETED	272	263
Overall Study NOT COMPLETED	29	36

Reasons for withdrawal

Reasons for withdrawal
Measure	Sargramostim Arm Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: All patients randomized to the sargramostim treatment arm will be treated with 250 mcg inhaled sargramostim administered via a vibrating mesh nebulizer once daily for 5 days.	Placebo Arm Day 1 - 5: Placebo treatment in addition to standard of care for COVID-19 Placebo: All patients in the control arm will receive an equivalent volume of inhaled placebo diluent administered via a vibrating mesh nebulizer once daily for 5 days.
Overall Study Adverse Event	2	0
Overall Study Death	1	2
Overall Study Withdrawal by Subject	13	21
Overall Study Lost to Follow-up	11	13
Overall Study Not treated	2	0

Baseline Characteristics

BMI from 2 patients in the sargramostim arm and 2 patients in the placebo arm were not collected.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Sargramostim Arm n=301 Participants Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: All patients randomized to the sargramostim treatment arm will be treated with 250 mcg inhaled sargramostim administered via a vibrating mesh nebulizer once daily for 5 days.	Placebo Arm n=299 Participants Day 1 - 5: Placebo treatment in addition to standard of care for COVID-19 Placebo: All patients in the control arm will receive an equivalent volume of inhaled placebo diluent administered via a vibrating mesh nebulizer once daily for 5 days.	Total n=600 Participants Total of all reporting groups
Age, Continuous	43.1 years STANDARD_DEVIATION 15.38 • n=301 Participants	43.5 years STANDARD_DEVIATION 14.34 • n=299 Participants	43.3 years STANDARD_DEVIATION 14.86 • n=600 Participants
Sex/Gender, Customized Male	136 Participants n=301 Participants	138 Participants n=299 Participants	274 Participants n=600 Participants
Sex/Gender, Customized Female	165 Participants n=301 Participants	160 Participants n=299 Participants	325 Participants n=600 Participants
Sex/Gender, Customized Unknown	0 Participants n=301 Participants	1 Participants n=299 Participants	1 Participants n=600 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	207 Participants n=301 Participants	211 Participants n=299 Participants	418 Participants n=600 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	93 Participants n=301 Participants	87 Participants n=299 Participants	180 Participants n=600 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=301 Participants	1 Participants n=299 Participants	2 Participants n=600 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=301 Participants	2 Participants n=299 Participants	3 Participants n=600 Participants
Race (NIH/OMB) Asian	1 Participants n=301 Participants	4 Participants n=299 Participants	5 Participants n=600 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=301 Participants	1 Participants n=299 Participants	2 Participants n=600 Participants
Race (NIH/OMB) Black or African American	41 Participants n=301 Participants	29 Participants n=299 Participants	70 Participants n=600 Participants
Race (NIH/OMB) White	252 Participants n=301 Participants	258 Participants n=299 Participants	510 Participants n=600 Participants
Race (NIH/OMB) More than one race	4 Participants n=301 Participants	2 Participants n=299 Participants	6 Participants n=600 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=301 Participants	3 Participants n=299 Participants	4 Participants n=600 Participants
Region of Enrollment United States	301 participants n=301 Participants	299 participants n=299 Participants	600 participants n=600 Participants
Body mass index	32.3 kg/m^2 STANDARD_DEVIATION 6.15 • n=299 Participants • BMI from 2 patients in the sargramostim arm and 2 patients in the placebo arm were not collected.	32.6 kg/m^2 STANDARD_DEVIATION 6.03 • n=297 Participants • BMI from 2 patients in the sargramostim arm and 2 patients in the placebo arm were not collected.	32.5 kg/m^2 STANDARD_DEVIATION 6.08 • n=596 Participants • BMI from 2 patients in the sargramostim arm and 2 patients in the placebo arm were not collected.

PRIMARY outcome

Timeframe: 28 days

Percentage of patients who experience any emergency room visit or hospitalization, or death

Outcome measures

Outcome measures
Measure	Sargramostim Arm n=301 Participants Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: All patients randomized to the sargramostim treatment arm will be treated with 250 mcg inhaled sargramostim administered via a vibrating mesh nebulizer once daily for 5 days.	Placebo Arm n=299 Participants Day 1 - 5: Placebo treatment in addition to standard of care for COVID-19 Placebo: All patients in the control arm will receive an equivalent volume of inhaled placebo diluent administered via a vibrating mesh nebulizer once daily for 5 days.
Number of Participants With an Emergency Room Visit or Hospitalization, or Death by Day 28	21 Participants	16 Participants

SECONDARY outcome

Timeframe: Day 28 and Day 60

Population: The Full Analysis Set was used for analysis

Proportion of patients with any progression of disease as determined by a ≥ 2-point increase from baseline in the National Institute of Allergy and Infectious Disease (NIAID) Ordinal Scale up to Day 28, and Day 60. The NIAID Ordinal Scale is a 1-8 scale and is an assessment of clinical status on a given study day. A score of 1 indicates the patient is not hospitalized and has no limitations on activities. A score of 8 indicates the patient has died.

Outcome measures

Outcome measures
Measure	Sargramostim Arm n=301 Participants Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: All patients randomized to the sargramostim treatment arm will be treated with 250 mcg inhaled sargramostim administered via a vibrating mesh nebulizer once daily for 5 days.	Placebo Arm n=299 Participants Day 1 - 5: Placebo treatment in addition to standard of care for COVID-19 Placebo: All patients in the control arm will receive an equivalent volume of inhaled placebo diluent administered via a vibrating mesh nebulizer once daily for 5 days.
Disease Progression Based on NIAID Score By Day 28	14 Participants	14 Participants
Disease Progression Based on NIAID Score Day 29-60	0 Participants	0 Participants
Disease Progression Based on NIAID Score No COVID-19 progression	287 Participants	285 Participants

SECONDARY outcome

Timeframe: Day 28 and Day 60

Population: Full Analysis Set

Time to progression of disease as determined by a ≥ 2-point increase in the NIAID ordinal scale up to Day 28, and Day 60. The NIAID ordinal scale is a 1-8 scale and is an assessment of clinical status on a given study day. A score of 1 indicates the patient is not hospitalized and has no limitations on activities. A score of 8 indicates the patient has died.

Outcome measures

Outcome measures
Measure	Sargramostim Arm n=301 Participants Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: All patients randomized to the sargramostim treatment arm will be treated with 250 mcg inhaled sargramostim administered via a vibrating mesh nebulizer once daily for 5 days.	Placebo Arm n=299 Participants Day 1 - 5: Placebo treatment in addition to standard of care for COVID-19 Placebo: All patients in the control arm will receive an equivalent volume of inhaled placebo diluent administered via a vibrating mesh nebulizer once daily for 5 days.
Time to Disease Progression Based on NIAID Score	NA Days The median time to progression of COVID-19 could not be calculated based on the small numbers of patients that progressed.	NA Days The median time to progression of COVID-19 could not be calculated based on the small numbers of patients that progressed.

SECONDARY outcome

Timeframe: Day 7, 14, and 28

Population: The Full Analysis Set was used

Change from baseline in overall symptom score as measured by the Symptom Score Questionnaire on Day 7, Day 14 and Day 28. The overall symptom score is the sum of 14 individual symptom scores from Symptom Score Questionnaire. Individual symptom scores correspond to the following responses: None = 0, Mild = 1, Moderate = 2, Severe = 3; None = 0, 1-2 times = 1, 3-4 times = 2, 5 or more times = 3; Same as usual = 0, Less than usual = 1, No sense = 2. The lowest score could be 0, and the highest score could be 42. Patients with higher scores have more severe symptoms from COVID-19.

Outcome measures

Outcome measures
Measure	Sargramostim Arm n=301 Participants Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: All patients randomized to the sargramostim treatment arm will be treated with 250 mcg inhaled sargramostim administered via a vibrating mesh nebulizer once daily for 5 days.	Placebo Arm n=299 Participants Day 1 - 5: Placebo treatment in addition to standard of care for COVID-19 Placebo: All patients in the control arm will receive an equivalent volume of inhaled placebo diluent administered via a vibrating mesh nebulizer once daily for 5 days.
Change From Baseline in Overall Symptom Scores Day 7	-7.7 score on a scale Standard Deviation 7.02	-6.7 score on a scale Standard Deviation 7.33
Change From Baseline in Overall Symptom Scores Day 14	-11.7 score on a scale Standard Deviation 6.62	-10.6 score on a scale Standard Deviation 7.35
Change From Baseline in Overall Symptom Scores Day 28	-13.8 score on a scale Standard Deviation 6.69	-13.3 score on a scale Standard Deviation 7.19

SECONDARY outcome

Timeframe: 60 days

Population: Safety population

Adverse events up to Day 60

Outcome measures

Outcome measures
Measure	Sargramostim Arm n=297 Participants Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: All patients randomized to the sargramostim treatment arm will be treated with 250 mcg inhaled sargramostim administered via a vibrating mesh nebulizer once daily for 5 days.	Placebo Arm n=290 Participants Day 1 - 5: Placebo treatment in addition to standard of care for COVID-19 Placebo: All patients in the control arm will receive an equivalent volume of inhaled placebo diluent administered via a vibrating mesh nebulizer once daily for 5 days.
Number of Participants With Adverse Events	59 Participants	67 Participants

Adverse Events

Sargramostim Arm

Serious events: 13 serious events

Other events: 46 other events

Deaths: 1 deaths

Placebo Arm

Serious events: 11 serious events

Other events: 56 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	Sargramostim Arm n=297 participants at risk Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: All patients randomized to the sargramostim treatment arm will be treated with 250 mcg inhaled sargramostim administered via a vibrating mesh nebulizer once daily for 5 days.	Placebo Arm n=290 participants at risk Day 1 - 5: Placebo treatment in addition to standard of care for COVID-19 Placebo: All patients in the control arm will receive an equivalent volume of inhaled placebo diluent administered via a vibrating mesh nebulizer once daily for 5 days.
Cardiac disorders Acute myocardial infarction	0.34% 1/297 • Number of events 1 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.	0.00% 0/290 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.
Infections and infestations Pneumonia	1.3% 4/297 • Number of events 4 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.	1.0% 3/290 • Number of events 3 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.
Infections and infestations COVID-19 pneumonia	0.67% 2/297 • Number of events 2 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.	0.69% 2/290 • Number of events 2 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.
Infections and infestations Appendicitis	0.34% 1/297 • Number of events 1 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.	0.34% 1/290 • Number of events 1 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.
Infections and infestations COVID-19	0.00% 0/297 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.	0.34% 1/290 • Number of events 1 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.
Infections and infestations SARS-CoV-2 sepsis	0.00% 0/297 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.	0.34% 1/290 • Number of events 1 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.
Infections and infestations Sepsis	0.34% 1/297 • Number of events 1 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.	0.00% 0/290 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.
Injury, poisoning and procedural complications Exposure during pregnancy	0.61% 1/164 • Number of events 1 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.	0.00% 0/154 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.
Nervous system disorders Dizziness	0.00% 0/297 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.	0.34% 1/290 • Number of events 1 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.
Renal and urinary disorders Nephrolithiasis	0.00% 0/297 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.	0.34% 1/290 • Number of events 1 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.
Respiratory, thoracic and mediastinal disorders Hypoxia	1.0% 3/297 • Number of events 3 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.	0.00% 0/290 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.
Respiratory, thoracic and mediastinal disorders Acute respiratory failure	0.34% 1/297 • Number of events 1 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.	0.00% 0/290 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.
Vascular disorders Pulmonary embolism	0.00% 0/297 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.	0.34% 1/290 • Number of events 1 • Adverse events were collected up to 60 days Evaluation of safety and all-cause mortality was conducted with the Safety Population, having at least 1 dose of study treatment. Worsening of COVID-19 signs and symptoms were exempt from AE reporting, unless related to study treatment administration or judged to be more severe than expected.

Other adverse events

Additional Information

Medical Information

Partner Therapeutics, Inc.

Phone: 1-888-479-5385

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60