RecruitingNCT02461615
Respiratory
Pulmonary Alveolar Proteinosis
Also known as PAP, alveolar proteinosis, GM-CSF deficiency PAP
Pulmonary alveolar proteinosis is characterised by the accumulation of surfactant-derived lipoproteinaceous material in the alveoli due to impaired clearance by alveolar macrophages. The autoimmune form, caused by neutralising anti-GM-CSF a
ORPHA:747 ↗Gene CSF2RAGene CSF2RB (autoimmune)Prevalence Approximately 1–2 per millionOnset Adult (autoimmune form); neonatal (congenital form)
4
studies recruiting now
as of 7 Sept 2026
32
studies registered in total
as of 7 Sept 2026
4
countries with a recruiting site
as of 7 Sept 2026
29 May 2024
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 3NCT06431776
Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).
RecruitingPhase 1 / Phase 2NCT05761899
Safety and Efficacy of PMT Therapy of hPAP
RecruitingNCT02852928
European Management Platform for Childhood Interstitial Lung Diseases - chILD-EU Register and Biobank
Showing the 4 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
Search all Pulmonary Alveolar Proteinosis studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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About Pulmonary Alveolar Proteinosis
Pulmonary alveolar proteinosis is characterised by the accumulation of surfactant-derived lipoproteinaceous material in the alveoli due to impaired clearance by alveolar macrophages. The autoimmune form, caused by neutralising anti-GM-CSF autoantibodies, is the most common subtype in adults, while hereditary forms result from mutations in GM-CSF receptor genes. Whole lung lavage remains the standard of care, and GM-CSF supplementation is an emerging therapeutic strategy for autoimmune PAP.
Common clinical features
Progressive dyspnoea on exertionDry or mildly productive coughFatigueHypoxaemia at rest or on exertionCrazy-paving pattern on HRCTIncreased susceptibility to opportunistic infectionsWeight loss in advanced disease
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
8 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Phase 2Rituximab (Blitzima)
Phase 2Sargramostim (Leukine)
Phase 2Regramostim
Phase 1/2Methionine
Phase 1/2Folic Acid (Bio science)
Phase 1/2Ascorbic Acid (Ascor)
Phase 1/2Pyridoxine (Pyridoxine component of vitaped)
Phase 1/2Cyanocobalamin (Behepan)
Before you apply
Things trial teams commonly ask about for Pulmonary Alveolar Proteinosis. Not eligibility rules; those are set by each study.
- Anti-GM-CSF antibody titre is a key diagnostic and eligibility marker for autoimmune PAP trials; ensure serology is documented at a recognised laboratory.
- Some GM-CSF therapy trials exclude patients who have undergone whole lung lavage within a defined period; check the wash-out requirement before applying.
- Genetic testing to exclude hereditary PAP (CSF2RA/CSF2RB mutations) may be required before enrolment in autoimmune-specific studies.
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).