Sargramostim Use in COVID-19 to Recover Patient Health (SCOPE)
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| ClinicalTrials.gov Identifier: NCT04707664 |
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Recruitment Status :
Completed
First Posted : January 13, 2021
Results First Posted : February 6, 2023
Last Update Posted : February 6, 2023
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Covid19 SARS-CoV Infection | Drug: Sargramostim Drug: Placebo | Phase 2 |
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 Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 600 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. |
| 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 : | April 27, 2021 |
| Actual Primary Completion Date : | December 28, 2021 |
| Actual Study Completion Date : | January 31, 2022 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: Sargramostim Arm
Day 1 - 5: Sargramostim treatment in addition to standard of care for COVID-19
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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:
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Placebo Comparator: Placebo Arm
Day 1 - 5: Placebo treatment in addition to standard of care for COVID-19
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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. |
- Number of Participants With an Emergency Room Visit or Hospitalization, or Death by Day 28 [ Time Frame: 28 days ]Percentage of patients who experience any emergency room visit or hospitalization, or death
- Disease Progression Based on NIAID Score [ Time Frame: Day 28 and Day 60 ]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.
- Time to Disease Progression Based on NIAID Score [ Time Frame: Day 28 and Day 60 ]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.
- Change From Baseline in Overall Symptom Scores [ Time Frame: Day 7, 14, and 28 ]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.
- Number of Participants With Adverse Events [ Time Frame: 60 days ]Adverse events up to Day 60
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.
| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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.
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Have one or more of the following mild or moderate COVID-19 symptoms for ≤5 days prior to randomization:
- Fever or chills
- New onset or worsening cough
- Sore throat
- Malaise or fatigue
- Headache
- Muscle pain (myalgias) or body aches
- Gastrointestinal symptoms (e.g., nausea, vomiting, diarrhea)
- New onset or worsening shortness of breath or difficulty breathing
- Nasal congestion or runny nose
- 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.
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At higher risk for progression to more severe COVID-19
- Age ≥ 60 years
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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
- 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
- Negative pregnancy test (if woman of childbearing potential)
- Females of childbearing potential and males with female partners of childbearing potential must agree to use acceptable contraceptive methods from screening to Day 28
- The patient (or legally authorized decision maker) must give informed consent
Exclusion Criteria:
- Hospitalized patients
- 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
- Patients enrolled in interventional clinical trials for other experimental therapies
- Patients on chronic oxygen supplementation due to cardiopulmonary or other conditions
- Patients with unstable comorbid conditions (e.g., decompensated congestive heart failure, COPD with exacerbation, current angina pectoris, uncontrolled diabetes mellitus, uncontrolled hypertension, uncontrolled asthma)
- Patients with severe pulmonary comorbid conditions, including systemic steroid-dependent asthma, systemic steroid-dependent COPD, oxygen-dependent COPD, lung transplant, or cystic fibrosis
- 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
- Patients with known or suspected intolerance or hypersensitivity to sargramostim, or any component of the product
- Patients who have previously experienced severe and unexplained side effects during aerosol delivery of any kind of medical product
- Pregnant or breastfeeding females
- 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
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
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| Study Director: | Fiona Garner, PhD | Partner Therapeutics, Inc. |
Documents provided by Partner Therapeutics, Inc.:
| Responsible Party: | Partner Therapeutics, Inc. |
| ClinicalTrials.gov Identifier: | NCT04707664 |
| Other Study ID Numbers: |
PTX-001-003 |
| First Posted: | January 13, 2021 Key Record Dates |
| Results First Posted: | February 6, 2023 |
| Last Update Posted: | February 6, 2023 |
| Last Verified: | December 2022 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
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COVID-19 SARS-CoV2 sargramostim Leukine |
(recombinant human) GM-CSF immune modulator SCOPE |
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COVID-19 Severe Acute Respiratory Syndrome Pneumonia, Viral Pneumonia Respiratory Tract Infections Infections Virus Diseases Coronavirus Infections |
Coronaviridae Infections Nidovirales Infections RNA Virus Infections Lung Diseases Respiratory Tract Diseases Sargramostim Immunologic Factors Physiological Effects of Drugs |