Granulocyte Macrophage-Colony Stimulating Factor (GM-CSF) Inhalation to Improve Host Defense and Pulmonary Barrier Restoration

• Status: Active, not recruiting
• Trial end date: 2022-07-31
• Target enrollment: 0
• Participant gender: All

Summary

This trial evaluates efficacy and safety of inhaled molgramostim (rhGM-CSF) in 45 patients with pneumonia associated acute respiratory distress syndrome (ARDS). A third of the patients will receive 150 mcg inhaled molgramostim, another third 450 mcg and the remaining third will receive inhaled placebo for 3 days. The patients will be followed for 28 days.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Savara Inc.; University of Giessen
• Treatments: Molgramostim; Sargramostim

Criteria

Inclusion Criteria:

1. Signed informed consent form by the patient or a legal representative according to local regulations

2. Man or woman 18 to 75 years of age, inclusive

3. Women who have been post-menopausal for more than 1 year or women of childbearing potential period using a highly efficient method of contraception (i.e. a method with less than 1% failure rate such as combined hormonal contraception, progesterone-only hormonal contraception, intrauterine device, intrauterine hormone-releasing system, bilateral tube occlusion, vasectomized partner, sexual abstinence) during dosing and hospitalisation. Women must have a negative serum or urine pregnancy test before the first dose of study medication and must not be lactating.

4. Diagnosis of pneumonia-associated ARDS, where the underlying condition is Community-Acquired Pneumonia (CAP) or Hospital-Acquired Pneumonia (HAP) in patients not on invasive ventilation upon diagnosis of HAP.

5. Diagnosis of ARDS according to the Berlin ARDS definition.

6. Requirement for positive pressure ventilation (non-invasive or via endotracheal tube) for more than 72 hours in total with inspiratory oxygen concentration (FiO2) ≥ 50% (or less when on additional ECMO therapy) not longer than 14 days

Exclusion Criteria:

1. Receiving vasopressors of >100 µg/min

2. History of liver cirrhosis Child Pugh C, chronic hemodialysis (before severe pneumonia/ARDS), lung cancer

3. Malignancy with expected survival time of less than 6 months

4. History of or listing for lung transplantation

5. Highly immunosuppressive therapy or anti-malignant combination chemotherapy within 3 weeks prior to first dose of study drug

6. Any anti-malignant chemotherapy within 24 hours prior to first dose of study drug

7. AIDS or known history of HIV infection

8. Pregnancy

9. Autoimmune thrombocytopenia, myelodysplastic syndromes with > 20% marrow blast cells

10. History or presence of hypersensitivity or idiosyncratic reaction to molgramostim or to related compounds (i.e., Growgen®, Leucomax®, Leukine™, Topleucon™)

11. Participation in another clinical trial within 90 days prior to the first dose of study drug