DDrare: Database of Drug Development for Rare Diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
229	肺胞蛋白症（自己免疫性又は先天性）	2

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	JPRN-JMA-IIA00013		16/08/2007	A Phase II Study of Inhaled GM-CSF in Patients With Idiopathic Pulmonary Alveolar Proteinosis (iPAP)	A Phase II Study of Inhaled GM-CSF in Patients With Idiopathic Pulmonary Alveolar Proteinosis (iPAP)	Idiopathic pulmonary alveolar proteinosis	Intervention type:DRUG. Intervention1:Granulocyte-macrophage colony stimulating factor (GM-CSF), Dose form:INJECTION, Route of administration:INHALATIONAL, intended dose regimen:Weeks 1-12: 6 cycles of inhaled GM-CSF 125 mcg twice daily on days 1 through 8 of a 14-day Weeks 13-24: 6 cycles of inhaled GM-CSF 125 mcg once daily on days 1 through 4 of a 14-day cycle.	Koh Nakata, M.D., Ph.D.Bioscience Medical Research Center (BMRC), Niigata University Medical & Dental Hospital	Toshihiro NUKIWA, M.D., Ph.D., Department of Respiratory Medicine, Tohoku University Medical SchoolYoshikazu INOUE, M.D., Ph.D., National Hospital Organization Kinki-Chuo Chest Medical CenterToshinori TAKADA M.D., Ph.D., Department of Internal Medicine, Niigata University Medical and Dental Hospital	Completed	>=16 YEARS	<=80 YEARS	BOTH	40	Phase 2	Japan

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

JPRN-JMA-IIA00013

16/08/2007

A Phase II Study of Inhaled GM-CSF in Patients With Idiopathic Pulmonary Alveolar Proteinosis (iPAP)

Idiopathic pulmonary alveolar proteinosis

Intervention type:DRUG. Intervention1:Granulocyte-macrophage colony stimulating factor (GM-CSF), Dose form:INJECTION, Route of administration:INHALATIONAL, intended dose regimen:Weeks 1-12: 6 cycles of inhaled GM-CSF 125 mcg twice daily on days 1 through 8 of a 14-day
Weeks 13-24: 6 cycles of inhaled GM-CSF 125 mcg once daily on days 1 through 4 of a 14-day cycle.

Koh Nakata, M.D., Ph.D.Bioscience Medical Research Center (BMRC), Niigata University Medical & Dental Hospital

Toshihiro NUKIWA, M.D., Ph.D., Department of Respiratory Medicine, Tohoku University Medical SchoolYoshikazu INOUE, M.D., Ph.D., National Hospital Organization Kinki-Chuo Chest Medical CenterToshinori TAKADA M.D., Ph.D., Department of Internal Medicine, Niigata University Medical and Dental Hospital

Completed

>=16 YEARS

<=80 YEARS

BOTH

Phase 2

Japan