Molgramostim for Pulmonary Alveolar Proteinosis

Overseen By Raymond D Pratt, MD

Age: < 65

Sex: Any

Trial Phase: Phase 3

Sponsor: Savara Inc.

Must be taking: Molgramostim

Disqualifiers: Hereditary PAP, Metabolic disorder, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 12

Duration: 48 weeks

5 Participants Needed

Trial Summary

Do I need to stop my current medications for this trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What data supports the effectiveness of the drug Molgramostim for treating Pulmonary Alveolar Proteinosis?

Research shows that inhaled GM-CSF, like Molgramostim, can help improve symptoms and reduce the need for whole lung lavage (a demanding procedure) in patients with autoimmune pulmonary alveolar proteinosis. A case study of a 14-year-old successfully treated with Molgramostim after whole lung lavage suggests it can be an effective alternative treatment. ^{1 2 3 4 5}

Is molgramostim safe for humans?

Molgramostim has been studied for safety in different conditions, including severe sepsis and pulmonary alveolar proteinosis. In these studies, no adverse events were considered drug-related, suggesting it is generally safe for human use. ^{1 2 6 7 8}

How does the drug molgramostim differ from other treatments for pulmonary alveolar proteinosis?

Molgramostim is unique because it is an inhaled form of recombinant human GM-CSF, allowing patients to be treated at home, unlike the traditional whole lung lavage, which is invasive and requires hospitalization. This drug offers a less invasive option and has shown promise in improving symptoms and radiographic findings, although its effectiveness on lung function tests is still being evaluated. ^{1 2 5 9 10}

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.

Research Team

Yasmine Wasfi, MD, Ph.D.

Principal Investigator

Savara Inc.

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.

Exclusion Criteria

I had a lung lavage treatment within the last month.

I have a genetic or acquired lung condition affecting surfactant.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Treatment

Participants receive molgramostim once daily via nebulizer for 48 weeks

48 weeks

Clinic visits approximately every 12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Molgramostim

Trial Overview 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.

Participant Groups

Group I: molgramostim Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

Savara Inc.

Lead Sponsor

Trials

11

Recruited

750+

Findings from Research

A young man with pulmonary alveolar proteinosis achieved complete remission after being treated with granulocyte-macrophage colony stimulating factor (GM-CSF), suggesting it as a promising new treatment option.

This case highlights the potential of GM-CSF as an alternative to the traditional whole lung lavage treatment for this rare condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pulmonary alveolar proteinosis: a complete response to GM-CSF therapy. Barraclough, RM., Gillies, AJ.[2019]

References

1. England pubmed.ncbi.nlm.nih.gov

Nebulised sargramostim in pulmonary alveolar proteinosis. [2022]

2. England pubmed.ncbi.nlm.nih.gov

Autoimmune pulmonary alveolar proteinosis in an adolescent successfully treated with inhaled rhGM-CSF (molgramostim). [2020]

3. France pubmed.ncbi.nlm.nih.gov

[Alveolar proteinosis. Prolonged favorable course after total pulmonary lavage]. [2016]

4. England pubmed.ncbi.nlm.nih.gov

Inhaled GM-CSF reduces the need for whole lung lavage and improves gas exchange in autoimmune PAP patients. [2023]

5. England pubmed.ncbi.nlm.nih.gov

Pulmonary alveolar proteinosis: a complete response to GM-CSF therapy. [2019]

6. England pubmed.ncbi.nlm.nih.gov

Safety and efficacy of molgramostim as an adjunctive therapy in critically ill patients with severe sepsis. [2019]

7. England pubmed.ncbi.nlm.nih.gov

Inhaled granulocyte-macrophage colony stimulating factor for mild-to-moderate autoimmune pulmonary alveolar proteinosis - a six month phase II randomized study with 24 months of follow-up. [2021]

8. United States pubmed.ncbi.nlm.nih.gov

Filgrastim in patients with pneumonia and severe sepsis or septic shock. [2020]

9. Spain pubmed.ncbi.nlm.nih.gov

[Therapeutic efficacy of GM-CSF in pulmonary alveolar proteinosis]. [2019]

10. United States pubmed.ncbi.nlm.nih.gov

Inhaled GM-CSF for Pulmonary Alveolar Proteinosis. [2021]