SCOPE: Sargramostim Use in COVID-19 to Recover Patient Health

Sponsor
Partner Therapeutics, Inc. (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT04707664
Collaborator
United States Department of Defense (U.S. Fed)
500
Enrollment
37
Locations
2
Arms
8.2
Anticipated Duration (Months)
13.5
Patients Per Site
1.7
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this research is to understand if the study drug, also called sargramostim or Leukine®, can help prevent the worsening of COVID-19 when the study drug is inhaled. This study will also help researchers understand if inhaled sargramostim can help prevent visits to the emergency room or hospitalization, or death.

Condition or DiseaseIntervention/TreatmentPhase
Phase 2

Detailed Description

This Phase 2b, multicenter, placebo-controlled, double-blind study will randomize approximately 500 adult patients who are symptomatic with mild or moderate COVID-19 (as defined in the FDA Guidance Document: Covid-19: Developing Drugs and Biological Products for Treatment or Prevention, May 2020) who are at high risk for progression to more severe disease. Patients will be randomized in a 1:1 ratio to inhaled sargramostim plus standard of care (SOC) or placebo plus SOC. Enrollment of patients who have completed a COVID-19 vaccination regimen or participated in a COVID-19 vaccine clinical trial will be capped at approximately 100 patients. All patients will be randomized to receive either 250 mcg of sargramostim or equivalent volume of placebo diluent. Treatment will be administered once daily for 5 days delivered via a vibrating mesh nebulizer. Patients will be followed for up to 60 days after start of treatment.

Sargramostim (Leukine) is a formulation of Granulocyte Macrophage Colony Stimulating Factor (GM-CSF), which is a critical cytokine for healthy pulmonary function. Detailed studies have shown that GM-CSF is necessary for alveolar macrophage (AM) maturation and maintenance. Although GM-CSF was discovered as a myelopoietic growth factor, it has diverse additional effects that both promote differentiation of myeloid precursors into neutrophils, monocytes, and dendritic cells and control function of mature myeloid cells. GM-CSF is also known to reverse immunoparalysis seen in sepsis, resulting in beneficial outcomes. In addition, GM-CSF prevents bacteremia in post influenza bacterial pneumonia through locally mediated improved lung antibacterial resistance and increased reactive oxygen species production by AMs. Pulmonary delivery of this GM-CSF has potential to reduce morbidity and mortality due to viral pneumonias, potentially including COVID-19.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
500 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Intervention Model Description:
This Phase 2b, multicenter, placebo-controlled, double-blind study will randomize approximately 500 adult patients who are symptomatic with mild or moderate COVID-19 (as defined in the FDA Guidance Document: Covid-19: Developing Drugs and Biological Products for Treatment or Prevention, May 2020) who are at high risk for progression to more severe disease. Patients will be randomized in a 1:1 ratio to inhaled sargramostim plus standard of care (SOC) or placebo plus SOC. Enrollment of patients who have completed a COVID-19 vaccination regimen or participated in a COVID-19 vaccine clinical trial will be capped at approximately 100 patients.This Phase 2b, multicenter, placebo-controlled, double-blind study will randomize approximately 500 adult patients who are symptomatic with mild or moderate COVID-19 (as defined in the FDA Guidance Document: Covid-19: Developing Drugs and Biological Products for Treatment or Prevention, May 2020) who are at high risk for progression to more severe disease. Patients will be randomized in a 1:1 ratio to inhaled sargramostim plus standard of care (SOC) or placebo plus SOC. Enrollment of patients who have completed a COVID-19 vaccination regimen or participated in a COVID-19 vaccine clinical trial will be capped at approximately 100 patients.
Masking:
Double (Participant, Investigator)
Masking Description:
Double blind
Primary Purpose:
Treatment
Official Title:
A Randomized Phase 2b Trial Evaluating Clinical Outcomes of Inhaled Sargramostim in High-risk Patients With Mild-moderate COVID-19
Actual Study Start Date :
Apr 27, 2021
Anticipated Primary Completion Date :
Dec 1, 2021
Anticipated Study Completion Date :
Jan 1, 2022

Arms and Interventions

ArmIntervention/Treatment
Experimental: Sargramostim Arm

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

Drug: 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.
Other Names:
  • Leukine
  • GM-CSF
  • Placebo Comparator: Placebo Arm

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

    Drug: 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.

    Outcome Measures

    Primary Outcome Measures

    1. Any emergency room visit or hospitalization, or death [28 days]

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

    Secondary Outcome Measures

    1. Disease Progression based on NIAID Score [28, 60 days]

      Proportion of patients with any progression of disease as determined by a ≥ 2-point increase from baseline in the NIAID ordinal scale up to Day 28, and Day 60

    2. Time to Disease Progression based on NIAID Score [28, 60 days]

      Time to progression of disease as determined by a ≥ 2-point increase in the NIAID ordinal scale up to Day 28, and Day 60

    3. Change from Baseline in Symptom Scores [28 days]

      Change from baseline in overall symptom score, and individual symptom scores, as measured by the Symptom Score Questionnaire up to Day 28

    4. Adverse Events [60 days]

      Adverse events up to Day 60

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:
    1. Patients with a positive laboratory diagnosis of SARS-CoV-2 infection by an antigen or a molecular test ≤5 days prior to randomization. The test should have been authorized by the relevant regulatory authority.

    2. Have one or more of the following mild or moderate COVID-19 symptoms for ≤5 days prior to randomization:

    3. Fever or chills

    4. New onset or worsening cough

    5. Sore throat

    6. Malaise or fatigue

    7. Headache

    8. Muscle pain (myalgias) or body aches

    9. Gastrointestinal symptoms (e.g., nausea, vomiting, diarrhea)

    10. New onset or worsening shortness of breath or difficulty breathing

    11. Nasal congestion or runny nose

    12. New loss of taste (ageusia) and/or smell (anosmia). Note: any of these symptoms (ageusia, anosmia) alone or in combination cannot be used as the SOLE qualifying symptoms for enrollment.

    13. At higher risk for progression to more severe COVID-19

    14. Age ≥ 60 years

    15. Age 18-59 years with a clinically stable medical history of at least 1 or more of the following conditions that could lead to severe COVID-19:

    • Chronic respiratory conditions such as asthma, chronic obstructive pulmonary disease (COPD), pulmonary fibrosis

    • Obesity with BMI ≥ 30 kg/m2

    • Cardiovascular disease

    • Sickle cell disease or thalassemia

    • Diabetes mellitus being managed with concomitant medications

    • Hypertension being managed with concomitant medications

    • Chronic kidney disease

    1. Oxygen saturation by pulse oximeter > 93% on room air. Note: at altitudes of >4000 feet above sea level, oxygen saturation by pulse oximeter > 91% on room air is permitted

    2. Negative pregnancy test (if woman of childbearing potential)

    3. Females of childbearing potential and males with female partners of childbearing potential must agree to use acceptable contraceptive methods from screening to Day 28

    4. The patient (or legally authorized decision maker) must give informed consent

    Exclusion Criteria:
    1. Hospitalized patients

    2. Patients who have received or are receiving other treatments that are not approved/authorized by the relevant regulatory authority for the treatment of patients with mild or moderate COVID-19 in an outpatient setting

    3. Patients enrolled in interventional clinical trials for other experimental therapies

    4. Patients on chronic oxygen supplementation due to cardiopulmonary or other conditions

    5. Patients with unstable comorbid conditions (e.g., decompensated congestive heart failure, COPD with exacerbation, current angina pectoris, uncontrolled diabetes mellitus, uncontrolled hypertension, uncontrolled asthma)

    6. Patients with severe pulmonary comorbid conditions, including systemic steroid-dependent asthma, systemic steroid-dependent COPD, oxygen-dependent COPD, lung transplant, or cystic fibrosis

    7. Patients who have received highly immunosuppressive therapy (to include systemic corticosteroids) or anti-cancer combination chemotherapy within 24 hours prior to first dose of study drug

    8. Patients with known or suspected intolerance or hypersensitivity to sargramostim, or any component of the product

    9. Patients who have previously experienced severe and unexplained side effects during aerosol delivery of any kind of medical product

    10. Pregnant or breastfeeding females

    11. Patients who, in the opinion of the Investigator, will not be able to comply with all the study procedures and visits as outlined in the schedule of events, including follow-up

    Contacts and Locations

    Locations

    SiteCityStateCountryPostal Code
    1West Valley Research Clinic, LLCPhoenixArizonaUnited States85031
    2Applied Research Center of ArkansasLittle RockArkansasUnited States72212
    3Hope Clinical ResearchCanoga ParkCaliforniaUnited States91303
    4Benchmark ResearchColtonCaliforniaUnited States92324
    5Synergy HealthcareBradentonFloridaUnited States34208
    6Invesclinic US, LLC.Fort LauderdaleFloridaUnited States33308
    7Indago Research & Health Center, Inc. (Subject Visits Only)HialeahFloridaUnited States33012
    8Encore Medical ResearchHollywoodFloridaUnited States33021
    9IMIC Inc.Palmetto BayFloridaUnited States33157
    10Encore Medical Research of WestonWestonFloridaUnited States33331
    11Gwinnett Research Institute, LLCBufordGeorgiaUnited States30519
    12Paramount Research SolutionsCollege ParkGeorgiaUnited States30349
    13TidalHealth Peninsula Regional, Inc.SalisburyMarylandUnited States21801
    14Revive Research Institute, Inc.Farmington HillsMichiganUnited States48334
    15Revival Research Institute, LLC.Sterling HeightsMichiganUnited States48312
    16Olive Branch Family Medical CenterOlive BranchMississippiUnited States38654
    17Great Plains HealthNorth PlatteNebraskaUnited States69101
    18Excel Clinical ResearchLas VegasNevadaUnited States89109
    19Richmond University Medical CenterStaten IslandNew YorkUnited States10310
    20Monroe Biomedical ResearchMonroeNorth CarolinaUnited States28112
    21Hometown Urgent Care and ResearchCincinnatiOhioUnited States45215
    22Hometown Urgent Care and ResearchColumbusOhioUnited States43214
    23Urgent Care Specialists, LLC DBA Hometown Urgent Care and ResearchDaytonOhioUnited States45424
    24TruCare Internal Medicine and Infectious DiseaseDuBoisPennsylvaniaUnited States15801
    25Urgent Care Clinical Trials @ AFC Urgent Care - EasleyEasleySouth CarolinaUnited States29640
    26University Diabetes & Endocrine ConsuttantsChattanoogaTennesseeUnited States37411
    27UCCT @ City Doc Urgent Care-McKinneyDallasTexasUnited States75204
    28Invesclinic US LLCEdinburgTexasUnited States78539
    29Dorrington Medical AssociatesHoustonTexasUnited States77030
    30Encore Imaging & Medical ResearchHoustonTexasUnited States77065
    31SMS Clinical Research, LLCMesquiteTexasUnited States75149
    32Novotrial Research GroupPearlandTexasUnited States77584
    33Sun Research InstituteSan AntonioTexasUnited States78215
    34University of Utah HealthSalt Lake CityUtahUnited States84108
    35Centro de Investigaciones Medicas Mar del PlataMar del PlataBuenos AiresArgentina7600
    36Instituto Medico de la Fundacion Estudios ClinicosRosarioSanta FeArgentina2000
    37Sanatorio Santa BarbaraBuenos AiresArgentinaC1414DTK

    Sponsors and Collaborators

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

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Partner Therapeutics, Inc.
    ClinicalTrials.gov Identifier:
    NCT04707664
    Other Study ID Numbers:
    • PTX-001-003
    First Posted:
    Jan 13, 2021
    Last Update Posted:
    Dec 3, 2021
    Last Verified:
    Dec 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    Undecided
    Plan to Share IPD:
    Undecided
    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

    No Results Posted as of Dec 3, 2021