Molgramostim for Pulmonary Alveolar Proteinosis

Recruiting in Palo Alto (17 mi)

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Savara Inc.

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?The goal of this open-label study is to study molgramostim as a treatment for autoimmune pulmonary alveolar proteinosis (aPAP) in pediatric patients between age 6 and 18. The main questions it aims to answer are: The effect of molgramostim on breathing tests and activity in pediatric patients with aPAP and the safety of molgramostim in pediatric patients with aPAP. This is an open-label study: all participants will receive treatment with molgramostim. Patients will: * Take molgramostim once daily via nebulizer every day for 12 months. * Visit the clinic approximately every 12 weeks for checkups and tests. * Keep a diary of any oxygen use.

Eligibility Criteria

This trial is for children aged 6-18 with autoimmune pulmonary alveolar proteinosis (aPAP), a rare lung disease. Participants must be able to use a nebulizer daily and visit the clinic every 12 weeks. Specific eligibility criteria are not provided, but typically include having the condition being studied and meeting certain health requirements.

Inclusion Criteria

I have been diagnosed with pulmonary alveolar proteinosis.

I am between 6 and 17 years old.

I tested positive for aPAP.

+1 more

Exclusion Criteria

I had a lung lavage treatment within the last month.

I have a genetic or acquired lung condition affecting surfactant.

Participant Groups

The study tests molgramostim, an inhaled medication delivered via nebulizer, as a treatment for pediatric aPAP. Over 12 months, all participants will receive this drug daily to assess its impact on breathing function and activity levels without comparing it to another drug or placebo.

1Treatment groups

Experimental Treatment

Group I: molgramostimExperimental Treatment1 Intervention

Molgramostim 300 mcg administered once daily via nebulizer for 48 weeks.