Molgramostim for Pulmonary Alveolar Proteinosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests molgramostim as a treatment for autoimmune pulmonary alveolar proteinosis (aPAP) in children and teenagers. Researchers aim to determine if molgramostim improves breathing and activity levels and assess its safety. Participants will use molgramostim daily through a nebulizer for about a year and monitor their oxygen use. This trial may suit young individuals diagnosed with aPAP who experience lung function difficulties. As a Phase 3 trial, it represents the final step before FDA approval, allowing participants to contribute to a potentially groundbreaking treatment.
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.
Is there any evidence suggesting that molgramostim is likely to be safe for pediatric patients with aPAP?
Research has shown that molgramostim is generally well-tolerated by people with autoimmune pulmonary alveolar proteinosis (aPAP). One study found that the most common side effects were cough and a cold-like nose and throat infection, affecting about 11.9% of patients. Other side effects, such as respiratory infections, joint pain, and COVID-19, occurred less frequently.
Although the treatment is still under investigation, its late-stage trial status indicates some evidence of safety. This suggests it could be a manageable option for those considering joining the trial.12345Why do researchers think this study treatment might be promising for aPAP?
Unlike the standard treatments for pulmonary alveolar proteinosis, which often involve whole lung lavage or GM-CSF injections, molgramostim is administered as a daily nebulized therapy. This delivery method allows for direct targeting of the lungs, potentially increasing efficacy and reducing systemic side effects. Researchers are excited about molgramostim because it combines an innovative delivery system with the active ingredient GM-CSF, aiming to improve lung function more conveniently and comfortably for patients.
What evidence suggests that molgramostim might be an effective treatment for aPAP?
Research has shown that molgramostim, the treatment under study in this trial, can improve lung function in people with autoimmune pulmonary alveolar proteinosis (aPAP). In earlier studies, patients using molgramostim demonstrated better lung performance, measured by gas transfer efficiency, compared to those on a placebo. Specifically, lung function improved by 11.6% after 48 weeks of treatment. Additionally, molgramostim was well tolerated, with no serious side effects reported. These findings suggest that molgramostim could effectively treat people with aPAP.46789
Who Is on the Research Team?
Yasmine Wasfi, MD, Ph.D.
Principal Investigator
Savara Inc.
Are You a Good Fit for This Trial?
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive molgramostim once daily via nebulizer for 48 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Molgramostim
Find a Clinic Near You
Who Is Running the Clinical Trial?
Savara Inc.
Lead Sponsor