Sargramostim Use in COVID-19 to Recover Patient Health

NCT04707664 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: PTX-001-003
• Study Type: interventional
• Target: 600
• Locations: 2 countries
• Sites: 37

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.

Conditions

Covid19; SARS-CoV Infection

Keywords

COVID-19; SARS-CoV2; sargramostim; Leukine; (recombinant human) GM-CSF; immune modulator; SCOPE

Outcome Measures

Primary Outcomes (1)

• Number of Participants With an Emergency Room Visit or Hospitalization, or Death by Day 28

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

  28 days

Secondary Outcomes (4)

• Disease Progression Based on NIAID Score

  Day 28 and Day 60

• Time to Disease Progression Based on NIAID Score

  Day 28 and Day 60

• Change From Baseline in Overall Symptom Scores

  Day 7, 14, and 28

• Number of Participants With Adverse Events

  60 days

Study Arms (2)

Sargramostim Arm

EXPERIMENTAL

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

Drug: Sargramostim

Placebo Arm

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

Sargramostim DRUG

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.

Also known as: Leukine, GM-CSF

Sargramostim Arm

Placebo DRUG

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.

Placebo Arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.
• At higher risk for progression to more severe COVID-19
• Age ≥ 60 years
• 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:

You may not qualify if:

• 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

Sponsors & Collaborators

• Partner Therapeutics, Inc.: lead
• United States Department of Defense: collaborator

Study Sites (37)

West Valley Research Clinic, LLC

Phoenix, Arizona, 85031, United States

Location

Applied Research Center of Arkansas

Little Rock, Arkansas, 72212, United States

Location

Hope Clinical Research

Canoga Park, California, 91303, United States

Location

Benchmark Research

Colton, California, 92324, United States

Location

Synergy Healthcare

Bradenton, Florida, 34208, United States

Location

Invesclinic US, LLC.

Fort Lauderdale, Florida, 33308, United States

Location

Indago Research & Health Center, Inc. (Subject Visits Only)

Hialeah, Florida, 33012, United States

Location

Encore Medical Research

Hollywood, Florida, 33021, United States

Location

IMIC Inc.

Palmetto Bay, Florida, 33157, United States

Location

Encore Medical Research of Weston

Weston, Florida, 33331, United States

Location

Gwinnett Research Institute, LLC

Buford, Georgia, 30519, United States

Location

Paramount Research Solutions

College Park, Georgia, 30349, United States

Location

TidalHealth Peninsula Regional, Inc.

Salisbury, Maryland, 21801, United States

Location

Revive Research Institute, Inc.

Farmington Hills, Michigan, 48334, United States

Location

Revival Research Institute, LLC.

Sterling Heights, Michigan, 48312, United States

Location

Olive Branch Family Medical Center

Olive Branch, Mississippi, 38654, United States

Location

Great Plains Health

North Platte, Nebraska, 69101, United States

Location

Excel Clinical Research

Las Vegas, Nevada, 89109, United States

Location

Richmond University Medical Center

Staten Island, New York, 10310, United States

Location

Monroe Biomedical Research

Monroe, North Carolina, 28112, United States

Location

Hometown Urgent Care and Research

Cincinnati, Ohio, 45215, United States

Location

Hometown Urgent Care and Research

Columbus, Ohio, 43214, United States

Location

Urgent Care Specialists, LLC DBA Hometown Urgent Care and Research

Dayton, Ohio, 45424, United States

Location

TruCare Internal Medicine and Infectious Disease

DuBois, Pennsylvania, 15801, United States

Location

Urgent Care Clinical Trials @ AFC Urgent Care - Easley

Easley, South Carolina, 29640, United States

Location

University Diabetes & Endocrine Consultants

Chattanooga, Tennessee, 37411, United States

Location

UCCT @ City Doc Urgent Care-McKinney

Dallas, Texas, 75204, United States

Location

Invesclinic US LLC

Edinburg, Texas, 78539, United States

Location

Dorrington Medical Associates

Houston, Texas, 77030, United States

Location

Encore Imaging & Medical Research

Houston, Texas, 77065, United States

Location

SMS Clinical Research, LLC

Mesquite, Texas, 75149, United States

Location

Novotrial Research Group

Pearland, Texas, 77584, United States

Location

Sun Research Institute

San Antonio, Texas, 78215, United States

Location

University of Utah Health

Salt Lake City, Utah, 84108, United States

Location

Centro de Investigaciones Medicas Mar del Plata

Mar del Plata, Buenos Aires, 7600, Argentina

Location

Instituto Medico de la Fundacion Estudios Clinicos

Rosario, Santa Fe Province, 2000, Argentina

Location

Sanatorio Santa Barbara

Buenos Aires, C1414DTK, Argentina

Location

MeSH Terms

Conditions

COVID-19; Severe Acute Respiratory Syndrome

Interventions

sargramostim; Granulocyte-Macrophage Colony-Stimulating Factor

Condition Hierarchy (Ancestors)

Pneumonia, Viral; Pneumonia; Respiratory Tract Infections; Infections; Virus Diseases; Coronavirus Infections; Coronaviridae Infections; Nidovirales Infections; RNA Virus Infections; Lung Diseases; Respiratory Tract Diseases

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Results Point of Contact

• Title: Medical Information
• Organization: Partner Therapeutics, Inc.

PartnerTx@medcomminc.com

1-888-479-5385

Study Officials

• Fiona Garner, PhD

  Partner Therapeutics, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Double blind
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: January 11, 2021
• First Posted: January 13, 2021
• Study Start: April 27, 2021
• Primary Completion: December 28, 2021
• Study Completion: January 31, 2022
• Last Updated: February 6, 2023
• Results First Posted: February 6, 2023

Record last verified: 2022-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (34); AR (3)