Inhaled Molgramostim for Autoimmune Pulmonary Alveolar Proteinosis
(IMPALA-2 Trial)

Recruiting in Palo Alto (17 mi)

+66 other locations

Overseen byBruce Trapnell, Prof

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Savara Inc.

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

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

Eligibility Criteria

Adults diagnosed with autoimmune pulmonary alveolar proteinosis (aPAP) who can perform certain physical tests without supplemental oxygen, have a history of PAP confirmed by specific lung tests, and are not on recent treatments like WLL or GM-CSF. They must be able to consent and follow trial procedures. Pregnant women, those with severe medical conditions or allergies to the treatment components, and individuals with other serious lung diseases are excluded.

Inclusion Criteria

Your lung function test shows that your ability to transfer oxygen is 70% or lower than expected.

Your oxygen level is above 85% when measured without using extra oxygen for 15 minutes.

I am either male or female.

+9 more

Exclusion Criteria

I haven't taken any experimental drugs recently.

I need strong medication (more than 10 mg/day of prednisolone) for my autoimmune disease.

I have not had plasmapheresis in the last 6 weeks.

+16 more

Participant Groups

The study is testing an inhaled solution called Molgramostim against a placebo in people with aPAP. Participants will use their assigned treatment daily for 48 weeks; afterwards, all subjects receive Molgramostim for another 96 weeks. The goal is to see if this drug helps improve lung function compared to no active treatment.

2Treatment groups

Experimental Treatment

Placebo Group

Group I: MolgramostimExperimental Treatment1 Intervention

Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks, followed by open-label treatment with molgramostim nebulizer solution 300 µg once daily for 96 weeks

Group II: PlaceboPlacebo Group2 Interventions

Double-blind treatment with placebo nebulizer solution once daily for 48 weeks, followed by open-label treatment with molgramostim nebulizer solution 300 µg once daily for 96 weeks