DDrare: Database of Drug Development for Rare Diseases

229. 肺胞蛋白症（自己免疫性又は先天性）
［臨床試験数：35，薬物数：30（DrugBank：8），標的遺伝子数：3，標的パスウェイ数：12］

Searched query = "Autoimmune pulmonary alveolar proteinosis", "Congenital pulmonary alveolar proteinosis", "Hereditary pulmonary alveolar proteinosis", "Pulmonary alveolar proteinosis", "PAP"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = Device: Centricyte 1000; Diagnostic Test: Abdominal and liver ultrasound.; Diagnostic Test: Brain MRI; Diagnostic Test: Methionine/homocysteine Dosage; Diagnostic Test: Thoracic CT scan; GM-CSF; GM-CSF (granulocyte-macrophage colony-stimulating factor, sargramostim); GM-CSF [Leukine (Sargramostim)]; GM-CSF inhalation therapy; Granulocyte-macrophage colony stimulating factor (GM-CSF); Homocysteine; Leukine; Liberase Enzyme (Roche); MOLGRAMOSTIM; Methionine; Molgramostim; Molgramostim 300 mcg nebuliser solution; Molgramostim Nebuliser solution 300 mcg; Pioglitazone; Procedure: IV Deployment Of cSVF In Sterile Normal Saline IV Solution; Procedure: Microcannula Harvest Adipose Derived tissue stromal vascular fraction (tSVF); Procedure: WLL; Procedure: Whole Lung Lavage(WLL); RhGM-CSF; RhGm-CSF; Rituximab; Sargramostim; Sterile Normal Saline for Intravenous Use; Vitamin B12; Vitamin B12, B9, B6, C supplementation

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT00901511 (ClinicalTrials.gov)	July 2009	11/5/2009	Whole Lung Lavage (WLL)/Inhaled Granulocyte-macrophage Colony-stimulating Factor (GM-CSF) in Autoimmune Pulmonary Alveolar Proteinosis (PAP)	Whole Lung Lavage Followed by Inhaled Sargramostim in the Treatment of Autoimmune Pulmonary Alveolar Proteinosis	Pulmonary Alveolar Proteinosis	Drug: GM-CSF [Leukine (Sargramostim)];Procedure: WLL	IRCCS Policlinico S. Matteo	Agenzia Italiana del Farmaco	Enrolling by invitation	18 Years	N/A	Both	18	Phase 2;Phase 3	Italy

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

NCT00901511
(ClinicalTrials.gov)

July 2009

11/5/2009

Whole Lung Lavage (WLL)/Inhaled Granulocyte-macrophage Colony-stimulating Factor (GM-CSF) in Autoimmune Pulmonary Alveolar Proteinosis (PAP)

Whole Lung Lavage Followed by Inhaled Sargramostim in the Treatment of Autoimmune Pulmonary Alveolar Proteinosis

Pulmonary Alveolar Proteinosis

Drug: GM-CSF [Leukine (Sargramostim)];Procedure: WLL

IRCCS Policlinico S. Matteo

Agenzia Italiana del Farmaco

Enrolling by invitation

18 Years

N/A

Both

Phase 2;Phase 3

Italy