A Phase II/III Study of Sargramostim

A Phase II/III Study of Sargramostim in Patients With Coronavirus Disease-2019 (COVID-19)

This is a randomized, placebo-controlled, double-blind, group comparison, multicenter study to evaluate the efficacy and safety of inhalation administration of sargramostim for 5 days, in principle (up to 10 days) as Add-on treatment to the standard treatment in COVID-19 patients.

Study Overview

• Status: Completed
• Conditions: COVID-19
• Intervention / Treatment: Drug: Sargramostim; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 70
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• Japan
  • Chiba
    • Narita City, Chiba, Japan
      • IUHW Narita Hospital
  • Kanagawa
    • Yokohama City, Kanagawa, Japan
      • Kanagawa Cardiovascular and Respiratory Center
  • Saitama
    • Saitama City, Saitama, Japan
      • Japanese Red Cross Society Saitama Red Cross Hospital
  • Tokyo
    • Chuo-ku, Tokyo, Japan
      • St. Luke's International Hospital
    • Meguro-ku, Tokyo, Japan
      • Mishuku Hospital
    • Shibuya-ku, Tokyo, Japan
      • Japanese Red Cross Medical Center
    • Shinjuku-Ku, Tokyo, Japan
      • Center Hospital of the National Center for Global Health and Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 20 years to 85 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

Japanese male or female subjects who have been confirmed to meet all the following criteria.

1. Hospitalized patients under treatment who were severe acute respiratory syndrome coronavirus 2 [SARS-CoV-2] positive by polymerase chain reaction (PCR) test.
2. Patients with clinically diagnosed pneumonia and a percutaneous oxygen saturation [SpO2] of 93% or less on breathing of room air at bed rest.
3. Patients for whom written informed consent has been obtained from those themselves or the legally acceptable representatives.
4. Patients aged 20 years or older and younger than 85 years at the time of obtaining informed consent.

Exclusion Criteria:

Subjects who meet any of the following criteria will be excluded. Unless otherwise stated, the following criteria refer to those at the time of screening.

1. Patients who have been participating in other intervention studies, such as studies on unapproved pharmacotherapy, within 90 days prior to screening.
2. Patients who have experienced off-label use of approved drugs (including those for COVID-19 treatment other than steroids as standard treatment) within 7 days prior to screening.
3. Patients who are not expected to survive longer than 24 hours after commencement of study drug administration.
4. Patients who are using invasive ventilator or extracorporeal membrane oxygenation (ECMO).
5. Patients who have a chronic respiratory disease requiring continuous home oxygen therapy or ventilator use.
6. Patients with an underlying condition that is considered very unlikely to withdraw ventilator (e.g., motor neuron disease, Duchenne muscular dystrophy, rapidly progressive interstitial pulmonary fibrosis).
7. Patients who have a disease including bronchial asthma, lower respiratory tract infections, and interstitial lung diseases that may affect the assessment of the clinical study, since before the symptom onset of COVID-19.
8. Patients who have a disease including leukemia and leukocytosis that causes leukocytosis.
9. Patients who have a chronic kidney disease requiring dialysis.
10. Patients who have severe liver failure (Child Pugh grade C).
11. Patients aged 80 years or older with any of heart failure, cerebrovascular disease, obesity (BMI 30 or higher), dyslipidemia, hypertension or diabetes.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: NPC-26; Sargramostim (125 μg) will be administered by inhalation twice daily for 5 days, in principle (up to 10 days) as Add-on treatment to the standard treatment.	Drug: Sargramostim; 250 μg/vial of sargramostim will be dissolved in 4 mL of physiological saline and 125 μg of which will be administered using inhaler twice daily in approximately 10-15 minutes.; Other Names: Leukine
Placebo Comparator: NP-26 Placebo; Physiological saline will be administered by inhalation twice daily for 5 days, in principle (up to 10 days) as Add-on treatment to the standard treatment.	Drug: Placebo; 2 mL of physiological saline will be administered using inhaler twice daily in approximately 10-15 minutes.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
2-rank Improvement on a 7-point Ordinal Scale	Number of days to achieve at least 2-rank improvement on a 7-point ordinal scale from baseline until Day 28.	Period until Day 28 (including the case after discharge).

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes From Baseline in Alveolar-arterial Oxygen Partial Pressure Gradient (A-aDO)	Changes from baseline in alveolar-arterial oxygen partial pressure gradient (A-aDO) on Day 5 and at the end of administration.	Period until Day 28 (including the case after discharge).
Number of Days Until Discharge From Baseline	Number of days until discharge from baseline (days of shifting to Category 7 on a 7-point ordinal scale).	Period until Day 28 (including the case after discharge).
Proportion of Subjects Whose Category Has Shifted to Category 1 or 2	Proportion of subjects whose category has shifted to Category 1 or 2 on a 7-point ordinal scale from baseline until Day 28	Period until Day 28 (including the case after discharge).

Collaborators and Investigators

• Sponsor: Nobelpharma

Study record dates

Study Major Dates

• Study Start (Actual): December 17, 2020
• Primary Completion (Actual): October 25, 2021
• Study Completion (Actual): October 25, 2021

Study Registration Dates

• First Submitted: November 19, 2020
• First Submitted That Met QC Criteria: November 19, 2020
• First Posted (Actual): November 24, 2020

Study Record Updates

• Last Update Posted (Actual): July 19, 2024
• Last Update Submitted That Met QC Criteria: February 6, 2024
• Last Verified: November 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Coronavirus Infections; Coronaviridae Infections; Nidovirales Infections; RNA Virus Infections; Virus Diseases; Infections; Respiratory Tract Infections; Respiratory Tract Diseases; Pneumonia, Viral; Pneumonia; Lung Diseases; COVID-19; Physiological Effects of Drugs; Immunologic Factors; Sargramostim
• Other Study ID Numbers: NPC-26-1

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No