NCT04544293

Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

A Randomized, Double-blind, Placebo-controlled Clinical Trial of Once-daily Inhaled Molgramostim Nebulizer Solution in Adult Subjects With Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 164 (actual)
• Sponsor: Savara Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

160 subjects with autoimmune pulmonary alveolar proteinosis (aPAP) will be randomized to receive once daily treatment with inhaled molgramostim (MOL) or placebo (PBO) for 48 weeks. Subjects completing the 48-week placebo-controlled period will receive open-label treatment with once daily inhaled molgramostim for 96 weeks.

Detailed description

This is an interventional, randomized, double-blind, 2-arm, parallel groups, placebo-controlled, multi-center, phase 3 trial in adult subjects who are diagnosed with aPAP. An aPAP diagnosis should be confirmed by a Granulocyte-macrophage colony stimulating factor (GM-CSF) auto-antibody test result, and history of PAP based on either high resolution computed tomography, lung biopsy, or bronchoalveolar lavage cytology, should be available. The trial consists of a 6-week screening period, a 48-week randomized, double-blind treatment period, a 96-week open-label treatment period, and a conditional 4-week safety follow-up period. The maximum treatment duration will be 145 weeks and the maximum trial duration will be 156 weeks. During the trial, whole lung lavage will be allowed as rescue treatment in case of worsening of aPAP.

Conditions

• Autoimmune Pulmonary Alveolar Proteinosis

Interventions

Type	Name	Description
DRUG	Molgramostim	Molgramostim 300 µg nebulizer solution
DRUG	Placebo	Matching placebo nebulizer solution
DRUG	Molgramostim Open-label	Molgramostim 300 µg nebulizer solution

Timeline

• Start date: 2021-05-19
• Primary completion: 2023-11-30
• Completion: 2027-05-30
• First posted: 2020-09-10
• Last updated: 2025-08-07
• Results posted: 2025-08-07

Locations

54 sites across 18 countries: United States, Australia, Belgium, Canada, France, Germany, Greece, Ireland, Italy, Japan, Netherlands, Poland, Portugal, Romania, South Korea, Spain, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study