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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Experimental drug shows promise for rare immune disorders
Disease control CompletedThis early-phase study tested the safety of rituximab, a drug that targets faulty white blood cells, in 7 adults with rare immune diseases like pulmonary alveolar proteinosis and severe candidiasis. Participants received several doses over 6 months and were monitored for 18 month…
Phase 1 • Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Disease control
Last updated Sep 18, 2026 00:00 UTC
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Could a simple amino acid ease a devastating lung condition?
Disease control CompletedThis study tested whether giving methionine (an amino acid) by mouth or feeding tube is safe and tolerable for children with a severe, inherited lung disease called pulmonary alveolar proteinosis. The disease is caused by a faulty MARS gene that prevents cells from using methioni…
Phase 1/2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control
Last updated Jun 27, 2026 07:58 UTC
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Scientists investigate why rare lung disease develops in children
Knowledge-focused CompletedThis study looked at 20 children with a rare lung condition called pulmonary alveolar proteinosis (PAP) caused by changes in the MARS gene. Researchers wanted to understand if the lung cleaning cells (macrophages) are not working properly. They grew these cells in a lab from bloo…
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:04 UTC