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	NCT01511068 (ClinicalTrials.gov)	August 2012	12/12/2011	Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)	Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)	Hereditary Pulmonary Alveolar Proteinosis	Drug: Leukine	Children's Hospital Medical Center, Cincinnati	Virginia Commonwealth University;Genzyme, a Sanofi Company	Completed	8 Years	N/A	All	2	Phase 2	United States
2	EUCTR2008-007086-23-IT (EUCTR)	12/12/2008	27/01/2009	Whole lung lavage followed by inhaled Sargramostim in the treatment of autoimmune pulmonary alveolar proteinosis. - WLL/inhaled GM-CSF in autoimmune PAP	Whole lung lavage followed by inhaled Sargramostim in the treatment of autoimmune pulmonary alveolar proteinosis. - WLL/inhaled GM-CSF in autoimmune PAP	Autoimmune PAP MedDRA version: 9.1;Level: LLT;Classification code 10037316;Term: Pulmonary alveolar proteinosis	Trade Name: Leukine INN or Proposed INN: Sargramostim	OSPEDALE POLICLINICO S. MATTEO	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes			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

NCT01511068
(ClinicalTrials.gov)

August 2012

12/12/2011

Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)

Hereditary Pulmonary Alveolar Proteinosis

Drug: Leukine

Children's Hospital Medical Center, Cincinnati

Virginia Commonwealth University;Genzyme, a Sanofi Company

Completed

8 Years

N/A

All

Phase 2

United States

EUCTR2008-007086-23-IT
(EUCTR)

12/12/2008

27/01/2009

Whole lung lavage followed by inhaled Sargramostim in the treatment of autoimmune pulmonary alveolar proteinosis. - WLL/inhaled GM-CSF in autoimmune PAP

Autoimmune PAP
MedDRA version: 9.1;Level: LLT;Classification code 10037316;Term: Pulmonary alveolar proteinosis

Trade Name: Leukine
INN or Proposed INN: Sargramostim

OSPEDALE POLICLINICO S. MATTEO

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Italy