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Pulmonary alveolar proteinosis
MONDO:0001437A rare lung disorder characterized by the filling of the pulmonary alveoli with proteinaceous material which stains positive with periodic acid-Schiff stain. It may be idiopathic or secondary due to hematologic malignancies or the inhalation of mineral dusts. Signs and symptoms include dyspnea, cough and low grade fever.
Also known as: PAP, pulmonary alveolar proteinosis
13 clinical trials for this condition and its sub-types, 9 tagged with Pulmonary alveolar proteinosis itself.
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Sub-types of Pulmonary alveolar proteinosis
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Hereditary pulmonary alveolar proteinosis 3 trials · 6 incl. sub-types
8 sub-types
- Severe early-onset pulmonary alveolar proteinosis due to MARS deficiency 3 trials
- SFTPC-related interstitial lung disease 0 trials
- Chronic respiratory distress with surfactant metabolism deficiency 0 trials
- Interstitial lung disease due to ABCA3 deficiency 0 trials
- Surfactant metabolism dysfunction, pulmonary, 1 0 trials
- Surfactant metabolism dysfunction, pulmonary, 2 0 trials
- Surfactant metabolism dysfunction, pulmonary, 4 0 trials
- Surfactant metabolism dysfunction, pulmonary, 5 0 trials
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New hope for rare lung disease: investigational drug now available through expanded access
Disease control Expanded accessThis program offers adults with autoimmune pulmonary alveolar proteinosis (aPAP) access to an investigational inhaled medication called molgramostim. aPAP is a rare lung condition where surfactant builds up, making breathing difficult. The drug aims to help the lungs clear this b…
Sponsor: Savara Inc. • Aim: Disease control
Last updated Jul 30, 2026 00:00 UTC
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New hope for kids with rare lung condition: inhaled protein therapy in trial
Disease control Recruiting nowThis study tests an inhaled drug called molgramostim in children aged 6 to 18 with autoimmune pulmonary alveolar proteinosis (aPAP), a rare disease where protein builds up in the lungs and makes breathing hard. All 5 participants will take the drug daily via a nebulizer for 12 mo…
Phase 3 • Sponsor: Savara Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:05 UTC
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First human trial of Gene-Edited lung cells for rare breathing disorder
Disease control Recruiting nowThis study tests a new treatment for hereditary pulmonary alveolar proteinosis (hPAP), a rare lung disease where breathing becomes difficult. The therapy involves taking a patient's own lung cells, adding a working gene, and putting them back into the lungs. Only 3 people will jo…
Phase 1/2 • Sponsor: Children's Hospital Medical Center, Cincinnati • Aim: Disease control
Last updated Jun 27, 2026 08:05 UTC
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Scientists hunt for hidden genes behind pituitary tumors
Knowledge-focused Recruiting nowThis study aims to find new genes that cause pituitary tumors, including those that lead to acromegaly (gigantism) and prolactinomas. Researchers will collect blood and tissue samples from up to 10,000 people with these tumors and their family members. By analyzing DNA, they hope…
Sponsor: Barts & The London NHS Trust • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:35 UTC
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New national registry aims to unlock mysteries of rare lung disease
Knowledge-focused Recruiting nowThis study creates a national registry for people with pulmonary alveolar proteinosis (PAP), a rare lung condition. Researchers will collect data and blood samples to improve diagnosis, understand how the disease progresses, and develop better ways to measure its severity. Up to …
Sponsor: Children's Hospital Medical Center, Cincinnati • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:02 UTC