Pulmonary Alveolar Proteinosis (PAP) Clinical Trials

The air sacs of the lungs are coated with surfactant, an oily film that keeps them from collapsing. Cleanup cells called alveolar macrophages constantly remove the used surfactant, but they only do that job when a signal protein called GM-CSF tells them to. In autoimmune PAP, the immune system makes antibodies that bind to GM-CSF and block it.

The most common symptoms of Pulmonary Alveolar Proteinosis include Shortness of breath that creeps in slowly, first with activity and later at rest, A cough that is dry or brings up whitish phlegm, Tiredness and a reduced ability to exercise, such as being winded on a flight of stairs, Bluish fingertips from low blood oxygen as the disease progresses, Fever, chest pain or blood-streaked phlegm, usually a sign of a lung infection on top of PAP, among others. About 6 to 7 people per million (NORD and the PAP Foundation); autoimmune PAP makes up 85% to 90% of cases, with hereditary, secondary and congenital forms each around 5%. Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Pulmonary Alveolar Proteinosis from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. There is no FDA-approved drug for PAP in the United States as of October 2026, so every medicine used for it is given off label or inside a trial. That may change on November 22, 2026, the FDA's target date for molgramostim inhalation solution, which Savara plans to sell under the name Molbreevi.

As of September 2026, Trial Friend's curated drug index does not include an FDA-approved treatment specific to Pulmonary Alveolar Proteinosis. The Treatments section of this page checks FDA drug labels live and lists any approved drug whose label names the condition, and the trials listed below cover the investigational options.

Pulmonary Alveolar Proteinosis is an acquired condition, not an inherited one. The common autoimmune form is not inherited. A rare hereditary form in children is caused by changes in the genes for the GM-CSF receptor and is diagnosed by genetic testing. About 6 to 7 people per million (NORD and the PAP Foundation); autoimmune PAP makes up 85% to 90% of cases, with hereditary, secondary and congenital forms each around 5%.

Eligibility for Pulmonary Alveolar Proteinosis clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Pulmonary Alveolar Proteinosis clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.