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	NCT02702180 (ClinicalTrials.gov)	May 9, 2016	28/2/2016	Efficacy and Safety of Inhaled Molgramostim (rhGM-CSF) in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)	A Randomised, Double-Blind, Placebo-Controlled Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary AlveoLAr Proteinosis Patients IMPALA	Autoimmune Pulmonary Alveolar Proteinosis	Drug: molgramostim;Drug: placebo	Savara Inc.	NULL	Completed	18 Years	75 Years	All	139	Phase 2;Phase 3	United States;Australia;Denmark;France;Germany;Greece;Israel;Italy;Japan;Korea, Republic of;Netherlands;Portugal;Russian Federation;Slovakia;Spain;Switzerland;Turkey;United Kingdom;Poland;Romania
2	NCT02840708 (ClinicalTrials.gov)	May 2016	13/7/2016	SK-1401 (rhGM-CSF Agent for Inhalation) GM-CSF Inhalation Pharmacokinetic Study	SK-1401 (rhGM-CSF Agent for Inhalation) GM-CSF Inhalation Pharmacokinetic Study	Pulmonary Alveolar Proteinosis, Autoimmune	Drug: Sargramostim	Niigata University Medical & Dental Hospital	NULL	Completed	20 Years	80 Years	All	14	Phase 1	Japan
3	NCT02468908 (ClinicalTrials.gov)	May 2015	3/6/2015	Inhaled Molgramostim (rhGM-CSF) in Healthy Adult Subjects	A Randomized, Double-Blind, Placebo-Controlled, Single-Centre, Single Ascending Dose and Multiple Ascending Dose Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Molgramostim When Administered by Inhalation to Healthy Adult Subjects	Pulmonary Alveolar Proteinosis;Bronchiectasis;Cystic Fibrosis;Acute Respiratory Distress Syndrome	Drug: Molgramostim;Drug: Placebo	Savara Inc.	Celerion	Completed	18 Years	55 Years	All	42	Phase 1	United Kingdom
4	NCT01983657 (ClinicalTrials.gov)	January 2012	7/11/2013	Study of Subcutaneous Injection of Low-dose rhGM-CSF +/- WLL in PAP.	Study of Subcutaneous Injection of Low-dose Recombinant Granulocyte Macrophage-Colony Stimulating Factor (rhGM-CSF) +/- Whole Lung Lavage(WLL) in Pulmonary Alveolar Proteinosis.	Pulmonary Alveolar Proteinosis	Drug: rhGM-CSF;Procedure: Whole Lung Lavage(WLL)	Shanghai Pulmonary Hospital, Shanghai, China	NULL	Recruiting	17 Years	80 Years	Both	40	Phase 2	China

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

NCT02702180
(ClinicalTrials.gov)

May 9, 2016

28/2/2016

Efficacy and Safety of Inhaled Molgramostim (rhGM-CSF) in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

A Randomised, Double-Blind, Placebo-Controlled Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary AlveoLAr Proteinosis Patients IMPALA

Autoimmune Pulmonary Alveolar Proteinosis

Drug: molgramostim;Drug: placebo

Savara Inc.

NULL

Completed

18 Years

75 Years

All

139

Phase 2;Phase 3

United States;Australia;Denmark;France;Germany;Greece;Israel;Italy;Japan;Korea, Republic of;Netherlands;Portugal;Russian Federation;Slovakia;Spain;Switzerland;Turkey;United Kingdom;Poland;Romania

NCT02840708
(ClinicalTrials.gov)

May 2016

13/7/2016

SK-1401 (rhGM-CSF Agent for Inhalation) GM-CSF Inhalation Pharmacokinetic Study

Pulmonary Alveolar Proteinosis, Autoimmune

Drug: Sargramostim

Niigata University Medical & Dental Hospital

NULL

Completed

20 Years

80 Years

All

Phase 1

Japan

NCT02468908
(ClinicalTrials.gov)

May 2015

3/6/2015

Inhaled Molgramostim (rhGM-CSF) in Healthy Adult Subjects

A Randomized, Double-Blind, Placebo-Controlled, Single-Centre, Single Ascending Dose and Multiple Ascending Dose Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Molgramostim When Administered by Inhalation to Healthy Adult Subjects

Pulmonary Alveolar Proteinosis;Bronchiectasis;Cystic Fibrosis;Acute Respiratory Distress Syndrome

Drug: Molgramostim;Drug: Placebo

Savara Inc.

Celerion

Completed

18 Years

55 Years

All

Phase 1

United Kingdom

NCT01983657
(ClinicalTrials.gov)

January 2012

7/11/2013

Study of Subcutaneous Injection of Low-dose rhGM-CSF +/- WLL in PAP.

Study of Subcutaneous Injection of Low-dose Recombinant Granulocyte Macrophage-Colony Stimulating Factor (rhGM-CSF) +/- Whole Lung Lavage(WLL) in Pulmonary Alveolar Proteinosis.

Pulmonary Alveolar Proteinosis

Drug: rhGM-CSF;Procedure: Whole Lung Lavage(WLL)

Shanghai Pulmonary Hospital, Shanghai, China

NULL

Recruiting

17 Years

80 Years

Both

Phase 2

China