iLeukPulm: Study of Sargramostim in Patients With COVID-19

Sponsor
Partner Therapeutics, Inc. (Industry)
Overall Status
Completed
CT.gov ID
NCT04411680
Collaborator
United States Department of Defense (U.S. Fed)
123
11
2
9
11.2
1.2

Study Details

Study Description

Brief Summary

The purpose of this research is to find out if a drug (sargramostim) also known as Leukine® could help patient recover faster from COVID-19. Sargramostim may help the lungs recover from the effects of COVID-19, and this research study will help to find this out.

Condition or Disease Intervention/Treatment Phase
Phase 2

Detailed Description

This Phase 2 study is designed as a proof of concept study and will randomize 2:1 approximately 120 patients with COVID-19 associated acute hypoxemia: of which 80 patients will receive sargramostim plus standard of care, and 40 patients who will receive standard of care alone. The aim of the study is to determine if inhaled sargramostim, as an adjunct to institutional standard of care, improves clinical outcomes in patients with COVID-19-associated acute hypoxemia. All patients on the sargramostim arm will be treated with 125 mcg inhaled sargramostim twice daily for 5 days, in addition to institutional standard of care. If required, upon progression to an invasive mechanical ventilator, administration of sargramostim may be delivered by intravenous infusion to complete a total of 5 days (including days delivered via inhalation).

GM-CSF is a critical cytokine for healthy pulmonary function and is necessary for the maturation and maintenance of alveolar macrophages. Preclinical studies have shown GM-CSF confers resistance to influenza by enhancing innate immune mechanisms that depend on alveolar macrophages for their health and normal functioning. Clinical studies of sargramostim in patients with severe sepsis and respiratory dysfunction or acute respiratory distress syndrome have shown improvements in oxygenation and lung compliance.

Study Design

Study Type:
Interventional
Actual Enrollment :
123 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Intervention Model Description:
This Phase 2, randomized, open-label study will enroll approximately 120 patients with COVID-19 associated acute hypoxemia. It is a randomized 2:1 study to evaluate sargramostim treatment plus institutional standard of care compared to institutional standard of care alone.This Phase 2, randomized, open-label study will enroll approximately 120 patients with COVID-19 associated acute hypoxemia. It is a randomized 2:1 study to evaluate sargramostim treatment plus institutional standard of care compared to institutional standard of care alone.
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase 2 Trial Evaluating Sargramostim in Patients With COVID-19 Associated Acute Hypoxemia
Actual Study Start Date :
Aug 18, 2020
Actual Primary Completion Date :
Mar 2, 2021
Actual Study Completion Date :
May 19, 2021

Arms and Interventions

Arm Intervention/Treatment
Experimental: Sargramostim Arm

Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19

Drug: Sargramostim
Sargramostim is a glycosylated, yeast-derived recombinant human granulocyte-macrophage colony stimulating factor (rhu-GM-CSF). Dosage for inhaled sargramostim: 125 mcg twice a day. Dosage for intravenous sargramostim: 125mcg/m2/day given over a 4 hour period.
Other Names:
  • Leukine
  • GM-CSF
  • Drug: Standard of care
    Standard of care for COVID-19

    Active Comparator: Control Arm

    Standard of care for COVID-19

    Drug: Standard of care
    Standard of care for COVID-19

    Outcome Measures

    Primary Outcome Measures

    1. Change in Oxygenation Parameter of P(A-a)O2 Gradient by Day 6 [1-6 days]

      The P(A-a)O2 gradient is a measure of how well the oxygen moves from the lungs into the bloodstream. Patients with a high gradient have less oxygen in the bloodstream.

    2. Number of Patients Who Have Been Intubated by Day 14 [1-14 days]

    Secondary Outcome Measures

    1. Change in Ordinal Scale [1-28 days]

      The ordinal scale was used, where 0 is not hospitalized, no clinical or virological evidence of infection, and 8 is death.

    2. All Cause 28-day Mortality [1-28 days]

    3. Number of Patients With Treatment-related Adverse Events [1-58 days]

      Number of patients with side effects related to treatment with sargramostim. Patients on the control arm did not receive sargramostim.

    4. Survival Time of Patients [Day 1-90]

    5. Causes of Death [Day 1-90]

    6. Change From Baseline in P/F Ratio [From Day 1 up to Day 6]

      The P/F ratio is a simple way to assess severity of low blood oxygen. It's the ratio of PaO2 (arterial oxygen partial pressure obtained from an arterial blood gas) to FiO2 (fraction of inspired oxygen expressed as a decimal). A normal arterial blood is gas 80 to 100 mm Hg. Normal room air has 21% or 0.21 oxygen. So a "normal" P/F ratio is about 380-475. A high P/F ratio means more oxygen in the bloodstream and a low P/F ratio means less oxygen in the bloodstream. This outcome measure compares the improvement (increase) in P/F ratio between treatment arms.

    7. Number of Participants Wtih Nosocomial Infections [Day 1-90]

      Nosocomial infections also referred to as healthcare-associated infections (HAI), are infection(s) acquired during the process of receiving health care that was not present during the time of admission.

    8. Number of Patients Requiring Invasive Mechanical Ventilation [Day 1-90]

      The is the number of patients that needed to be placed on a ventilator.

    9. Duration of Invasive, Non-invasive and Supplemental Oxygen [Day 1-90]

      This endpoint includes duration of any/all types of respiratory support

    10. Duration of Hospitalization [Day 1-90]

    11. Time to Normalization of White Blood Cells and Lymphocytes [Day 1-90]

      Normalization of WBC and lymphocyte count is defined as whether WBC and lymphocyte count results are normal or below normal or above normal ranges at available visits.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:
    • Patients aged ≥ 18 years

    • Patients (or legally authorized decision maker) must provide informed consent

    • Test positive for SARS-CoV-2 virus by PCR

    • Admitted to hospital

    • Presence of acute hypoxemia defined as (either or both)

    • saturation below 93% on ≥ 2 L/min oxygen supplementation

    • PaO2/FiO2 below 350

    Exclusion Criteria:
    • Patients requiring invasive (mechanical ventilation) or non-invasive (CPAP, BiPAP for hypoxemia) ventilation or ECMO (Note: oxygen supplementation using high flow oxygen systems or low flow oxygen systems would not exclude patients from this study)

    • Intractable metabolic acidosis

    • Cardiogenic pulmonary edema

    • Hypotension requiring use of vasopressors

    • Hyperferritinemia (serum ferritin ≥2,000 mcg/L)

    • White blood cell count > 50,000/mm3

    • Participation in another interventional clinical trial for COVID-19 therapy

    • Highly immunosuppressive therapy or anti-cancer combination chemotherapy within 24 hours prior to first dose of sargramostim

    • Known or suspected intolerance or hypersensitivity to sargramostim, or any component of the product

    • Previous experience of severe and unexplained side effects during aerosol delivery of any kind of medical product

    • Presence of any preexisting illness that, in the opinion of the Investigator, would place the patient at an unreasonably increased risk through participation in this study

    • Pregnant or breastfeeding females

    • Severe or uncontrolled pulmonary comorbid conditions, including systemic steroid dependent asthma, systemic steroid dependent COPD, oxygen dependent COPD, lung transplant, known interstitial lung disease, or cystic fibrosis

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 St. Jude Medical Center Fullerton California United States 92835
    2 St. Joseph Hospital of Orange Orange California United States 92868
    3 California Pacific Medical Center - Van Ness Campus San Francisco California United States 94109
    4 TidalHealth Peninsula Regional, Inc Salisbury Maryland United States 21801
    5 University of Missouri Health Care Columbia Missouri United States 65212
    6 Great Plains Health North Platte Nebraska United States 69101
    7 Richmond University Medical Center Staten Island New York United States 10310
    8 Brody School of Medicine at East Carolina University Greenville North Carolina United States 27834
    9 University of Toledo Medical Center Toledo Ohio United States 43614
    10 Memorial Hermann Hospital Affiliated with the University of Texas Health Science Center at Houston, McGovern Medical School Houston Texas United States 77030
    11 University of Utah Health Salt Lake City Utah United States 84108

    Sponsors and Collaborators

    • Partner Therapeutics, Inc.
    • United States Department of Defense

    Investigators

    • Study Director: Medical Information, Partner Therapeutics, Inc.

    Study Documents (Full-Text)

    More Information

    Publications

    None provided.
    Responsible Party:
    Partner Therapeutics, Inc.
    ClinicalTrials.gov Identifier:
    NCT04411680
    Other Study ID Numbers:
    • PTX-001-002
    First Posted:
    Jun 2, 2020
    Last Update Posted:
    Feb 18, 2022
    Last Verified:
    Feb 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Partner Therapeutics, Inc.
    Additional relevant MeSH terms:

    Study Results

    Participant Flow

    Recruitment Details
    Pre-assignment Detail One participant was randomized to the sargramostim arm and then withdrew consent prior to treatment.
    Arm/Group Title Sargramostim Arm Control Arm
    Arm/Group Description Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: Sargramostim is a recombinant human granulocyte-macrophage colony stimulating factor (rhu-GM-CSF). Dosage for inhaled sargramostim: 125 mcg twice a day. Dosage for intravenous sargramostim: 125mcg/m2/day given over a 4 hour period. Standard of care: Standard of care for COVID-19 Standard of care for COVID-19 Standard of care: Standard of care for COVID-19
    Period Title: Treatment Period
    STARTED 78 44
    COMPLETED 49 36
    NOT COMPLETED 29 8
    Period Title: Treatment Period
    STARTED 78 44
    COMPLETED 60 30
    NOT COMPLETED 18 14

    Baseline Characteristics

    Arm/Group Title Sargramostim Arm Control Arm Total
    Arm/Group Description Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: Sargramostim is a recombinant human granulocyte-macrophage colony stimulating factor (rhu-GM-CSF). Dosage for inhaled sargramostim: 125 mcg twice a day. Dosage for intravenous sargramostim: 125mcg/m2/day given over a 4 hour period. Standard of care: Standard of care for COVID-19 Standard of care for COVID-19 Standard of care: Standard of care for COVID-19 Total of all reporting groups
    Overall Participants 78 44 122
    Age (Count of Participants)
    <=18 years
    0
    0%
    0
    0%
    0
    0%
    Between 18 and 65 years
    47
    60.3%
    30
    68.2%
    77
    63.1%
    >=65 years
    31
    39.7%
    14
    31.8%
    45
    36.9%
    Age (years) [Mean (Standard Deviation) ]
    Mean (Standard Deviation) [years]
    61.7
    (13.00)
    58.2
    (10.61)
    60.4
    (12.26)
    Sex: Female, Male (Count of Participants)
    Female
    43
    55.1%
    18
    40.9%
    61
    50%
    Male
    35
    44.9%
    26
    59.1%
    61
    50%
    Ethnicity (NIH/OMB) (Count of Participants)
    Hispanic or Latino
    24
    30.8%
    11
    25%
    35
    28.7%
    Not Hispanic or Latino
    53
    67.9%
    33
    75%
    86
    70.5%
    Unknown or Not Reported
    1
    1.3%
    0
    0%
    1
    0.8%
    Race (NIH/OMB) (Count of Participants)
    American Indian or Alaska Native
    2
    2.6%
    0
    0%
    2
    1.6%
    Asian
    2
    2.6%
    0
    0%
    2
    1.6%
    Native Hawaiian or Other Pacific Islander
    0
    0%
    1
    2.3%
    1
    0.8%
    Black or African American
    6
    7.7%
    1
    2.3%
    7
    5.7%
    White
    48
    61.5%
    33
    75%
    81
    66.4%
    More than one race
    0
    0%
    0
    0%
    0
    0%
    Unknown or Not Reported
    20
    25.6%
    9
    20.5%
    29
    23.8%
    Body Mass Index (kilograms/meter squared) [Mean (Standard Deviation) ]
    Mean (Standard Deviation) [kilograms/meter squared]
    34.86
    (8.988)
    35.44
    (9.647)
    35.07
    (9.200)

    Outcome Measures

    1. Primary Outcome
    Title Change in Oxygenation Parameter of P(A-a)O2 Gradient by Day 6
    Description The P(A-a)O2 gradient is a measure of how well the oxygen moves from the lungs into the bloodstream. Patients with a high gradient have less oxygen in the bloodstream.
    Time Frame 1-6 days

    Outcome Measure Data

    Analysis Population Description
    Patients analyzed if they had both a baseline and at least 1 post-baseline measurement.
    Arm/Group Title Sargramostim Arm Control Arm
    Arm/Group Description Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: Sargramostim is a recombinant human granulocyte-macrophage colony stimulating factor (rhu-GM-CSF). Dosage for inhaled sargramostim: 125 mcg twice a day. Dosage for intravenous sargramostim: 125mcg/m2/day given over a 4 hour period. Standard of care: Standard of care for COVID-19 Standard of care for COVID-19 Standard of care: Standard of care for COVID-19
    Measure Participants 63 33
    Least Squares Mean (Standard Error) [millimeters of mercury (mmHg)]
    -102.3
    (19.44)
    -30.5
    (26.88)
    2. Primary Outcome
    Title Number of Patients Who Have Been Intubated by Day 14
    Description
    Time Frame 1-14 days

    Outcome Measure Data

    Analysis Population Description
    [Not Specified]
    Arm/Group Title Sargramostim Arm Control Arm
    Arm/Group Description Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: Sargramostim is a recombinant human granulocyte-macrophage colony stimulating factor (rhu-GM-CSF). Dosage for inhaled sargramostim: 125 mcg twice a day. Dosage for intravenous sargramostim: 125mcg/m2/day given over a 4 hour period. Standard of care: Standard of care for COVID-19 Standard of care for COVID-19 Standard of care: Standard of care for COVID-19
    Measure Participants 78 44
    Count of Participants [Participants]
    9
    11.5%
    7
    15.9%
    3. Secondary Outcome
    Title Change in Ordinal Scale
    Description The ordinal scale was used, where 0 is not hospitalized, no clinical or virological evidence of infection, and 8 is death.
    Time Frame 1-28 days

    Outcome Measure Data

    Analysis Population Description
    Only 86 patients had follow up data at Day 28
    Arm/Group Title Sargramostim Arm Control Arm
    Arm/Group Description Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: Sargramostim is a recombinant human granulocyte-macrophage colony stimulating factor (rhu-GM-CSF). Dosage for inhaled sargramostim: 125 mcg twice a day. Dosage for intravenous sargramostim: 125mcg/m2/day given over a 4 hour period. Standard of care: Standard of care for COVID-19 Standard of care for COVID-19 Standard of care: Standard of care for COVID-19
    Measure Participants 61 25
    Mean (Standard Deviation) [score on a scale]
    -2.6
    (1.49)
    -2.5
    (1.48)
    4. Secondary Outcome
    Title All Cause 28-day Mortality
    Description
    Time Frame 1-28 days

    Outcome Measure Data

    Analysis Population Description
    [Not Specified]
    Arm/Group Title Sargramostim Arm Control Arm
    Arm/Group Description Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: Sargramostim is a recombinant human granulocyte-macrophage colony stimulating factor (rhu-GM-CSF). Dosage for inhaled sargramostim: 125 mcg twice a day. Dosage for intravenous sargramostim: 125mcg/m2/day given over a 4 hour period. Standard of care: Standard of care for COVID-19 Standard of care for COVID-19 Standard of care: Standard of care for COVID-19
    Measure Participants 78 44
    Count of Participants [Participants]
    9
    11.5%
    6
    13.6%
    5. Secondary Outcome
    Title Number of Patients With Treatment-related Adverse Events
    Description Number of patients with side effects related to treatment with sargramostim. Patients on the control arm did not receive sargramostim.
    Time Frame 1-58 days

    Outcome Measure Data

    Analysis Population Description
    [Not Specified]
    Arm/Group Title Sargramostim Arm Control Arm
    Arm/Group Description Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: Sargramostim is a recombinant human granulocyte-macrophage colony stimulating factor (rhu-GM-CSF). Dosage for inhaled sargramostim: 125 mcg twice a day. Dosage for intravenous sargramostim: 125mcg/m2/day given over a 4 hour period. Standard of care: Standard of care for COVID-19 Standard of care for COVID-19 Standard of care: Standard of care for COVID-19
    Measure Participants 78 44
    Count of Participants [Participants]
    5
    6.4%
    0
    0%
    6. Secondary Outcome
    Title Survival Time of Patients
    Description
    Time Frame Day 1-90

    Outcome Measure Data

    Analysis Population Description
    [Not Specified]
    Arm/Group Title Sargramostim Arm Control Arm
    Arm/Group Description Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: Sargramostim is a recombinant human granulocyte-macrophage colony stimulating factor (rhu-GM-CSF). Dosage for inhaled sargramostim: 125 mcg twice a day. Dosage for intravenous sargramostim: 125mcg/m2/day given over a 4 hour period. Standard of care: Standard of care for COVID-19 Standard of care for COVID-19 Standard of care: Standard of care for COVID-19
    Measure Participants 78 44
    Mean (Standard Deviation) [Days]
    76.0
    (27.05)
    72.6
    (28.56)
    7. Secondary Outcome
    Title Causes of Death
    Description
    Time Frame Day 1-90

    Outcome Measure Data

    Analysis Population Description
    Patients alive at Day 90 were not analyzed
    Arm/Group Title Sargramostim Arm Control Arm
    Arm/Group Description Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: Sargramostim is a recombinant human granulocyte-macrophage colony stimulating factor (rhu-GM-CSF). Dosage for inhaled sargramostim: 125 mcg twice a day. Dosage for intravenous sargramostim: 125mcg/m2/day given over a 4 hour period. Standard of care: Standard of care for COVID-19 Standard of care for COVID-19 Standard of care: Standard of care for COVID-19
    Measure Participants 11 8
    Acute respiratory distress syndrome
    4
    5.1%
    2
    4.5%
    Respiratory failure
    6
    7.7%
    3
    6.8%
    Cardiorespiratory arrest
    1
    1.3%
    2
    4.5%
    Septic shock
    0
    0%
    1
    2.3%
    8. Secondary Outcome
    Title Change From Baseline in P/F Ratio
    Description The P/F ratio is a simple way to assess severity of low blood oxygen. It's the ratio of PaO2 (arterial oxygen partial pressure obtained from an arterial blood gas) to FiO2 (fraction of inspired oxygen expressed as a decimal). A normal arterial blood is gas 80 to 100 mm Hg. Normal room air has 21% or 0.21 oxygen. So a "normal" P/F ratio is about 380-475. A high P/F ratio means more oxygen in the bloodstream and a low P/F ratio means less oxygen in the bloodstream. This outcome measure compares the improvement (increase) in P/F ratio between treatment arms.
    Time Frame From Day 1 up to Day 6

    Outcome Measure Data

    Analysis Population Description
    Patients were analyzed if they had both a baseline and at least 1 post-baseline measurement of P/F ratio.
    Arm/Group Title Sargramostim Arm Control Arm
    Arm/Group Description Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: Sargramostim is a recombinant human granulocyte-macrophage colony stimulating factor (rhu-GM-CSF). Dosage for inhaled sargramostim: 125 mcg twice a day. Dosage for intravenous sargramostim: 125mcg/m2/day given over a 4 hour period. Standard of care: Standard of care for COVID-19 Standard of care for COVID-19 Standard of care: Standard of care for COVID-19
    Measure Participants 63 33
    Mean (Standard Deviation) [ratio]
    65.70
    (97.964)
    47.24
    (99.942)
    9. Secondary Outcome
    Title Number of Participants Wtih Nosocomial Infections
    Description Nosocomial infections also referred to as healthcare-associated infections (HAI), are infection(s) acquired during the process of receiving health care that was not present during the time of admission.
    Time Frame Day 1-90

    Outcome Measure Data

    Analysis Population Description
    [Not Specified]
    Arm/Group Title Sargramostim Arm Control Arm
    Arm/Group Description Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: Sargramostim is a recombinant human granulocyte-macrophage colony stimulating factor (rhu-GM-CSF). Dosage for inhaled sargramostim: 125 mcg twice a day. Dosage for intravenous sargramostim: 125mcg/m2/day given over a 4 hour period. Standard of care: Standard of care for COVID-19 Standard of care for COVID-19 Standard of care: Standard of care for COVID-19
    Measure Participants 78 44
    Count of Participants [Participants]
    0
    0%
    0
    0%
    10. Secondary Outcome
    Title Number of Patients Requiring Invasive Mechanical Ventilation
    Description The is the number of patients that needed to be placed on a ventilator.
    Time Frame Day 1-90

    Outcome Measure Data

    Analysis Population Description
    [Not Specified]
    Arm/Group Title Sargramostim Arm Control Arm
    Arm/Group Description Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: Sargramostim is a recombinant human granulocyte-macrophage colony stimulating factor (rhu-GM-CSF). Dosage for inhaled sargramostim: 125 mcg twice a day. Dosage for intravenous sargramostim: 125mcg/m2/day given over a 4 hour period. Standard of care: Standard of care for COVID-19 Standard of care for COVID-19 Standard of care: Standard of care for COVID-19
    Measure Participants 78 44
    Count of Participants [Participants]
    12
    15.4%
    9
    20.5%
    11. Secondary Outcome
    Title Duration of Invasive, Non-invasive and Supplemental Oxygen
    Description This endpoint includes duration of any/all types of respiratory support
    Time Frame Day 1-90

    Outcome Measure Data

    Analysis Population Description
    [Not Specified]
    Arm/Group Title Sargramostim Arm Control Arm
    Arm/Group Description Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: Sargramostim is a recombinant human granulocyte-macrophage colony stimulating factor (rhu-GM-CSF). Dosage for inhaled sargramostim: 125 mcg twice a day. Dosage for intravenous sargramostim: 125mcg/m2/day given over a 4 hour period. Standard of care: Standard of care for COVID-19 Standard of care for COVID-19 Standard of care: Standard of care for COVID-19
    Measure Participants 78 44
    Mean (Standard Deviation) [Days]
    37.9
    (35.64)
    32.8
    (34.25)
    12. Secondary Outcome
    Title Duration of Hospitalization
    Description
    Time Frame Day 1-90

    Outcome Measure Data

    Analysis Population Description
    [Not Specified]
    Arm/Group Title Sargramostim Arm Control Arm
    Arm/Group Description Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: Sargramostim is a recombinant human granulocyte-macrophage colony stimulating factor (rhu-GM-CSF). Dosage for inhaled sargramostim: 125 mcg twice a day. Dosage for intravenous sargramostim: 125mcg/m2/day given over a 4 hour period. Standard of care: Standard of care for COVID-19 Standard of care for COVID-19 Standard of care: Standard of care for COVID-19
    Measure Participants 78 44
    Mean (Standard Deviation) [Days]
    12.1
    (9.39)
    11.8
    (8.63)
    13. Secondary Outcome
    Title Time to Normalization of White Blood Cells and Lymphocytes
    Description Normalization of WBC and lymphocyte count is defined as whether WBC and lymphocyte count results are normal or below normal or above normal ranges at available visits.
    Time Frame Day 1-90

    Outcome Measure Data

    Analysis Population Description
    Of 78 and 44 participants in the Sargramostim and Control Arms, respectively, only 28 (35.8%) and 17 (38.6%) had both baseline and post-baseline values available for evaluation at the Day 6 timepoint of interest.
    Arm/Group Title Sargramostim Arm Control Arm
    Arm/Group Description Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: Sargramostim is a recombinant human granulocyte-macrophage colony stimulating factor (rhu-GM-CSF). Dosage for inhaled sargramostim: 125 mcg twice a day. Dosage for intravenous sargramostim: 125mcg/m2/day given over a 4 hour period. Standard of care: Standard of care for COVID-19 Standard of care for COVID-19 Standard of care: Standard of care for COVID-19
    Measure Participants 28 17
    Mean (Standard Deviation) [Days]
    NA
    (NA)
    NA
    (NA)

    Adverse Events

    Time Frame Adverse Events: Day 1-58, All-Cause Mortality: Day 1-90
    Adverse Event Reporting Description
    Arm/Group Title Sargramostim Arm Control Arm
    Arm/Group Description Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19 Sargramostim: Sargramostim is a recombinant human granulocyte-macrophage colony stimulating factor (rhu-GM-CSF). Dosage for inhaled sargramostim: 125 mcg twice a day. Dosage for intravenous sargramostim: 125mcg/m2/day given over a 4 hour period. Standard of care: Standard of care for COVID-19 Standard of care for COVID-19 Standard of care: Standard of care for COVID-19
    All Cause Mortality
    Sargramostim Arm Control Arm
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 11/78 (14.1%) 8/44 (18.2%)
    Serious Adverse Events
    Sargramostim Arm Control Arm
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 15/78 (19.2%) 14/44 (31.8%)
    Cardiac disorders
    Atrial fibrillation 1/78 (1.3%) 1/44 (2.3%)
    Atrial flutter 0/78 (0%) 1/44 (2.3%)
    Cardio-respiratory arrest 1/78 (1.3%) 1/44 (2.3%)
    General disorders
    Cardiac death 0/78 (0%) 1/44 (2.3%)
    Infections and infestations
    Cellulitis 1/78 (1.3%) 0/44 (0%)
    Septic shock 0/78 (0%) 2/44 (4.5%)
    Investigations
    False positive investigation result 1/78 (1.3%) 0/44 (0%)
    Metabolism and nutrition disorders
    Hyperkalaemia 1/78 (1.3%) 1/44 (2.3%)
    Psychiatric disorders
    Alcohol withdrawal syndrome 0/78 (0%) 1/44 (2.3%)
    Renal and urinary disorders
    Acute kidney injury 1/78 (1.3%) 2/44 (4.5%)
    Anuria 0/78 (0%) 1/44 (2.3%)
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory distress syndrome 4/78 (5.1%) 3/44 (6.8%)
    Chronic obstructive pulmonary disease 1/78 (1.3%) 0/44 (0%)
    Hypoxia 0/78 (0%) 1/44 (2.3%)
    Pulmonary embolism 0/78 (0%) 2/44 (4.5%)
    Respiratory failure 8/78 (10.3%) 5/44 (11.4%)
    Other (Not Including Serious) Adverse Events
    Sargramostim Arm Control Arm
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 47/78 (60.3%) 30/44 (68.2%)
    Blood and lymphatic system disorders
    Anemia 5/78 (6.4%) 3/44 (6.8%)
    Gastrointestinal disorders
    Constipation 7/78 (9%) 3/44 (6.8%)
    Infections and infestations
    Septic shock 5/78 (6.4%) 1/44 (2.3%)
    Metabolism and nutrition disorders
    Hyperglycemia 8/78 (10.3%) 4/44 (9.1%)
    Hypokalaemia 7/78 (9%) 3/44 (6.8%)
    Psychiatric disorders
    Anxiety 4/78 (5.1%) 4/44 (9.1%)
    Insomnia 3/78 (3.8%) 4/44 (9.1%)
    Renal and urinary disorders
    Acute kidney injury 3/78 (3.8%) 4/44 (9.1%)
    Vascular disorders
    Hypotension 5/78 (6.4%) 4/44 (9.1%)

    Limitations/Caveats

    Limitations include lack of a blinded placebo control and reliance on ABG measurement for estimation of P(A-a)O2 gradient. Some clinical endpoints trial might have been affected by observer bias, availability of hospital or ICU resources, and/or hospital discharge. Missing ABG measurements may have resulted from decreased standard utilization of ABG in recent years, and hospital staff overload due to the COVID-19 pandemic may have led to absent follow-up assessment.

    More Information

    Certain Agreements

    Principal Investigators are NOT employed by the organization sponsoring the study.

    The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.

    Results Point of Contact

    Name/Title Medical Information
    Organization Partner Therapeutics, Inc.
    Phone 1-888-479-5385
    Email PartnerTx@medcomminc.com
    Responsible Party:
    Partner Therapeutics, Inc.
    ClinicalTrials.gov Identifier:
    NCT04411680
    Other Study ID Numbers:
    • PTX-001-002
    First Posted:
    Jun 2, 2020
    Last Update Posted:
    Feb 18, 2022
    Last Verified:
    Feb 1, 2022