NCT02702180

Efficacy and Safety of Inhaled Molgramostim (rhGM-CSF) in Autoimmune Pulmonary Alveolar Proteinosis

A Randomised, Double-blind, Placebo-controlled Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Patients

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 139 (actual)
• Sponsor: Savara Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study evaluates inhaled molgramostim (recombinant human granulocyte macrophage-colony stimulating factor \[rhGM-CSF\]) in the treatment of autoimmune pulmonary alveolar proteinosis (aPAP) patients. A third of the patients will receive inhaled molgramostim once daily for 24 weeks, a third will receive inhaled molgramostim intermittently (7 days on, 7 days off) for 24 weeks and a third will receive inhaled matching placebo for 24 weeks.

Detailed description

The trial is a phase 2, randomized, double-blind, placebo-controlled multicentre clinical trial investigating efficacy and safety of inhaled molgramostim (rhGM-CSF) in patients with aPAP. The trial will include 2 periods; a double-blind treatment period consisting of up to 8 trial visits (Screening, Baseline, and at Weeks 4, 8,12, 16, 20 and 24 after randomisation) and a open-label follow-up period consisting of up to 5 trial visits (at Weeks 4, 12, 24, 36 and 48 post-treatment). In the double-blind treatment period, eligible subjects will be randomised to treatment for up to 24 weeks with either: 1) inhaled molgramostim (300 µg) once daily (MOL-OD), 2) inhaled molgramostim (300 µg) and matching placebo administered intermittently (7 days on and 7 days off) (MOL-INT) or 3) inhaled placebo once daily (PBO). During the follow-up period, all participants will receive inhaled molgramostim intermittently (7 days on, 7 days off). During the trial, whole lung lavage (WLL) may be applied as rescue therapy in case of significant clinical worsening.

Conditions

• Autoimmune Pulmonary Alveolar Proteinosis

Interventions

Type	Name	Description
DRUG	Molgramostim	300 mcg molgramostim (rhGM-CSF) nebulizer solution for inhalation
DRUG	Placebo	Placebo nebulizer solution for inhalation
DEVICE	PARI eFlow nebulizer system	PARI eFlow nebulizer system

Timeline

• Start date: 2016-03-21
• Primary completion: 2019-04-19
• Completion: 2019-09-27
• First posted: 2016-03-08
• Last updated: 2023-04-12
• Results posted: 2022-04-15

Locations

30 sites across 18 countries: United States, Australia, Denmark, France, Germany, Greece, Israel, Italy, Japan, Netherlands, Portugal, Russia, Slovakia, South Korea, Spain, Switzerland, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study
• FDA-regulated device study