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Sargramostim Use in COVID-19 to Recover Patient Health (SCOPE)

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ClinicalTrials.gov Identifier: NCT04707664
Recruitment Status : Not yet recruiting
First Posted : January 13, 2021
Last Update Posted : January 29, 2021
Sponsor:
Collaborator:
United States Department of Defense
Information provided by (Responsible Party):
Partner Therapeutics, Inc.

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 and hospitalization related to COVID-19 or death.

Condition or disease Intervention/treatment Phase
Covid19 SARS-CoV Infection Drug: Sargramostim Drug: Placebo Phase 2

Detailed Description:

This Phase 2b, multicenter, placebo-controlled, double-blind study will randomize approximately 400 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. All patients on the sargramostim arm will be treated with 250 mcg inhaled sargramostim once daily for 5 days delivered via a vibrating mesh nebulizer. Patients on the placebo arm will receive an equivalent volume of inhaled placebo diluent once daily for 5 days delivered via a vibrating mesh nebulizer. Patients will be followed for up to 28 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 400 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: This Phase 2b, multicenter, placebo-controlled, double-blind study will randomize approximately 400 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.
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
Estimated Study Start Date : February 2021
Estimated Primary Completion Date : June 2021
Estimated Study Completion Date : June 2021

Resource links provided by the National Library of Medicine


Arm Intervention/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.




Primary Outcome Measures :
  1. COVID-19-related emergency room visit or COVID-19- related hospitalization, or death (any cause) [ Time Frame: 28 days ]
    Proportion of patients who experience COVID-19-related emergency room visit or COVID-19- related hospitalization, or death (any cause)


Secondary Outcome Measures :
  1. Adverse Events [ Time Frame: 28 days ]
    Adverse events during treatment period and up to Day 28

  2. Hospitalization [ Time Frame: 28 days ]
    Time to COVID-19-related emergency room visit or COVID-19-related hospitalization, or death (any cause)

  3. Time to progression based on NIAID ordinal scale [ Time Frame: 28 days ]
    Time to progression of disease as determined by a ≥ 2-point increase in the NIAID ordinal scale up to Day 28

  4. Progression based on NIAID ordinal scale [ Time Frame: 28 days ]
    Proportion of patients with progression of disease as determined by a ≥ 2-point increase in the NIAID ordinal scale up to Day 28

  5. Symptom improvement [ Time Frame: 7, 14, 28 days ]
    Proportion of patients with symptom improvement via a symptom questionnaire by days 7, 14, and 28

  6. Symptom resolution [ Time Frame: 7, 14, 28 days ]
    Proportion of patients with symptom resolution via a symptom questionnaire by days 7, 14, and 28



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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:

    1. Fever
    2. New onset or worsening cough
    3. Sore throat
    4. Malaise or fatigue
    5. Headache
    6. Muscle pain (myalgias)
    7. Gastrointestinal symptoms (e.g., nausea, vomiting, diarrhea)
    8. New onset or worsening shortness of breath (dyspnea)
    9. 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.
  3. At higher risk for progression to more severe COVID-19

    1. Age ≥ 60 years
    2. Age 18-59 years and having one or more of the following co-morbid conditions which are clinically stable in the opinion of the Investigator:

      • Chronic respiratory conditions such as asthma, chronic obstructive pulmonary disease (COPD), pulmonary fibrosis
      • Obesity with BMI ≥ 30 kg/m2
      • Cardiovascular disease
      • Sickle cell disease
      • Diabetes mellitus being managed with concomitant medications
      • Hypertension being managed with concomitant medications
  4. Oxygen saturation by pulse oximeter > 93% on room air
  5. Negative pregnancy test (if woman of childbearing potential)
  6. Females of childbearing potential and males with female partners of childbearing potential must agree to use acceptable contraceptive methods for the duration of the study (screening to Day 28)
  7. The patient must give informed consent

Exclusion Criteria:

  1. Hospitalized patients
  2. Patients who have received or are receiving other treatments to prevent progression of COVID-19 disease severity, including but not limited to:

    1. Monoclonal antibodies e.g., bamlanivimab, casirivimab plus imdevimab
    2. Antiviral agents e.g., remdesivir
    3. Other COVID-19 therapies used as part of the standard of care, e.g., convalescent plasma, dexamethasone
  3. Patients who have received a COVID-19 vaccine or participated in a clinical trial for a COVID19 vaccine
  4. Patients enrolled in interventional clinical trials for other experimental therapies
  5. Patients on chronic oxygen supplementation due to cardiopulmonary or other conditions
  6. Patients with unstable comorbid conditions (e.g., decompensated congestive heart failure (CHF), COPD with exacerbation, current angina pectoris, uncontrolled diabetes mellitus, uncontrolled hypertension, uncontrolled asthma)
  7. Patients with severe pulmonary comorbid conditions, including systemic steroid-dependent asthma, systemic steroid-dependent COPD, oxygen-dependent COPD, lung transplant, or cystic fibrosis
  8. 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
  9. Patients with known or suspected intolerance or hypersensitivity to sargramostim, or any component of the product
  10. Patients who have previously experienced severe and unexplained side effects during aerosol delivery of any kind of medical product
  11. Pregnant or breastfeeding females
  12. 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04707664


Contacts
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Contact: Sanjeev Ahuja, MD (781)819-4949 ext 1588 scopestudy@partnertx.com

Sponsors and Collaborators
Partner Therapeutics, Inc.
United States Department of Defense
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Responsible Party: Partner Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04707664    
Other Study ID Numbers: PTX-001-003
First Posted: January 13, 2021    Key Record Dates
Last Update Posted: January 29, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Partner Therapeutics, Inc.:
COVID-19
SARS-CoV2
sargramostim
Leukine
(recombinant human) GM-CSF
immune modulator
SCOPE
Additional relevant MeSH terms:
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Coronavirus Infections
Severe Acute Respiratory Syndrome
Coronaviridae Infections
Nidovirales Infections
RNA Virus Infections
Virus Diseases
Respiratory Tract Infections
Respiratory Tract Diseases
Sargramostim
Immunologic Factors
Physiological Effects of Drugs