Disease · MONDO_0012579

autoimmune pulmonary alveolar proteinosis

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. autoimmune pulmonary alveolar proteinosis. Diseases, record MONDO_0012579. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0012579

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Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: autoimmune pulmonary alveolar proteinosis; Open Targets
Open Targets ID: MONDO_0012579; Open Targets
Other names: APAP, autoimmune PAP, iPAP, idiopathic PAP, idiopathic pulmonary alveolar proteinosis; Open Targets
MeSH: C567049; MeSH
Orphanet: ORPHA:747; Orphanet
Highest stage of its drugs: Approval, for 1 of its 4 drugs, from PMDA; Open Targets

Drugs4

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
SARGRAMOSTIM	CHEMBL1201670	Protein	Approval	PMDA	5	4	2	Drugs and targets
MOLGRAMOSTIM	CHEMBL2109134	Protein	Phase 3	ClinicalTrials.gov and TTD	7	6	4	Drugs and targets
REGRAMOSTIM	CHEMBL2107881	Protein	Phase 2	ClinicalTrials.gov	2	2	0	Drugs and targets
PIOGLITAZONE	CHEMBL595	Small molecule	Phase 1	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets4

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes	Source
CSF2RA	Granulocyte-macrophage colony-stimulating factor receptor	1	Granulocyte-macrophage colony-stimulating factor receptor agonist	Approval	https://conductscience.org/library/genes/1438	Drugs and targets
CSF2RB	Granulocyte-macrophage colony-stimulating factor receptor	1	Granulocyte-macrophage colony-stimulating factor receptor agonist	Approval	https://conductscience.org/library/genes/1439	Drugs and targets
CSF3R	Granulocyte colony-stimulating factor receptor	1	Granulocyte colony stimulating factor receptor agonist	Phase 2	https://conductscience.org/library/genes/1441	Drugs and targets
PPARG	Peroxisome proliferator-activated receptor gamma	1	Peroxisome proliferator-activated receptor gamma agonist	Phase 1	https://conductscience.org/library/genes/5468	Drugs and targets

Trials7

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 2 more are not shown, since the checks do not confirm them.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT04544293	Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)	Phase 3	164	2025-08-07	Autoimmune Pulmonary Alveolar Proteinosis	MOLGRAMOSTIM	Its record names the disease	ClinicalTrials.gov
NCT02702180	Efficacy and Safety of Inhaled Molgramostim (rhGM-CSF) in Autoimmune Pulmonary Alveolar Proteinosis	Phase 2	139	2022-04-15	Autoimmune Pulmonary Alveolar Proteinosis	MOLGRAMOSTIM	Its record names the disease	ClinicalTrials.gov
NCT03482752	Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis	Phase 3	60	2024-07-03	Autoimmune Pulmonary Alveolar Proteinosis	MOLGRAMOSTIM	Its record names the disease	ClinicalTrials.gov
NCT00901511	Inhaled GM-CSF Therapy of Autoimmune PAP	Phase 2	18	2023-06-26	Autoimmune Pulmonary Alveolar Proteinosis	SARGRAMOSTIM	Its record names the disease	ClinicalTrials.gov
NCT03006146	Evaluation of a Single Dose of Inhaled Sargramostim in Patients With Autoimmune Pulmonary Alveolar Proteinosis	Phase 1	10		Autoimmune Pulmonary Alveolar Proteinosis	SARGRAMOSTIM	Its record names the disease	ClinicalTrials.gov
NCT03231033	Pioglitazone Therapy of Autoimmune Pulmonary Alveolar Proteinosis Autoimmune Pulmonary Alveolar Proteinosis	Phase 1	3		Autoimmune Pulmonary Alveolar Proteinosis	PIOGLITAZONE	Its record names the disease	ClinicalTrials.gov
NCT06546098	Molgramostim Nebulizer Solution Expanded Access Program Protocol				Autoimmune Pulmonary Alveolar Proteinosis	MOLGRAMOSTIM	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials5

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

Comparison	Group 1, with the outcome	Group 2, with the outcome	Participants	Trial	Source
Molgramostim vs Placebo: deaths from any cause	0 of 81	0 of 83	164	NCT04544293	Trial comparisons
Molgramostim vs Placebo: serious adverse events	14 of 81	20 of 83	164	NCT04544293	Trial comparisons
GM-CSF Group vs Control Group: Number of Patients Requiring a Rescue WLL	1 of 9	7 of 9	18	NCT00901511	Trial comparisons
GM-CSF Group vs Control Group: deaths from any cause	0 of 9	0 of 9	18	NCT00901511	Trial comparisons
GM-CSF Group vs Control Group: serious adverse events	0 of 9	0 of 9	18	NCT00901511	Trial comparisons

Papers from its trials1

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it
40834301	DERIVED	1

Source PubMed

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

Sources 6

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
NLM's MeSHNLM's terms
Open TargetsCC0 1.0
PubMedNLM's terms

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
NLM's MeSHNLM's
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/MONDO_0012579

GET https://conductscience.org/api/diseases/v1/MONDO_0012579/drugs

API reference For AI assistants