A Phase II/III Study of Sargramostim

• ClinicalTrials.gov Identifier: NCT04642950
• Recruitment Status: Completed
• First Posted: November 24, 2020
• Last Update Posted: November 3, 2021
• Sponsor: Nobelpharma
• Information provided by (Responsible Party): Nobelpharma

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
COVID-19	Drug: Sargramostim; Drug: Placebo	Phase 2; Phase 3

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 70 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: A Phase II/III Study of Sargramostim in Patients With Coronavirus Disease-2019 (COVID-19)
• Actual Study Start Date: December 17, 2020
• Actual Primary Completion Date: October 25, 2021
• Actual Study Completion Date: October 25, 2021

Arms and Interventions

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 Name: 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.

Outcome Measures

Primary Outcome Measures :

1. 2-rank improvement on a 7-point ordinal scale [ Time Frame: Period until Day 28 (including the case after discharge). ]
   Number of days to achieve at least 2-rank improvement on a 7-point ordinal scale from baseline until Day 28.

Secondary Outcome Measures :

1. Changes from baseline in alveolar-arterial oxygen partial pressure gradient (A-aDO) [ Time Frame: Period until Day 28 (including the case after discharge). ]
   Changes from baseline in alveolar-arterial oxygen partial pressure gradient (A-aDO) on Day 5 and at the end of administration.
2. Number of days until discharge from baseline [ Time Frame: Period until Day 28 (including the case after discharge). ]
   Number of days until discharge from baseline (days of shifting to Category 7 on a 7-point ordinal scale).
3. Proportion of subjects whose category has shifted to Category 1 or 2 [ Time Frame: Period until Day 28 (including the case after discharge). ]
   Proportion of subjects whose category has shifted to Category 1 or 2 on a 7-point ordinal scale from baseline until Day 28

Eligibility Criteria

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

Criteria

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.

Contacts and Locations

Locations

Japan

IUHW Narita Hospital

Narita City, Chiba, Japan

Kanagawa Cardiovascular and Respiratory Center

Yokohama City, Kanagawa, Japan

Japanese Red Cross Society Saitama Red Cross Hospital

Saitama City, Saitama, Japan

St. Luke's International Hospital

Chuo-ku, Tokyo, Japan

Mishuku Hospital

Meguro-ku, Tokyo, Japan

Japanese Red Cross Medical Center

Shibuya-ku, Tokyo, Japan

Center Hospital of the National Center for Global Health and Medicine

Shinjuku-Ku, Tokyo, Japan

Sponsors and Collaborators

Nobelpharma

More Information

• Responsible Party: Nobelpharma
• ClinicalTrials.gov Identifier: NCT04642950
• Other Study ID Numbers: NPC-26-1
• First Posted: November 24, 2020
• Last Update Posted: November 3, 2021
• Last Verified: November 2021

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

COVID-19; 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