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	NCT03887169 (ClinicalTrials.gov)	September 16, 2019	12/3/2019	Administration of Methionine in Patients With Pulmonary Alveolar Proteinosis by Mutation of the MARS Gene.	Oral or Enteral Administration of Methionine in Patients With Pulmonary Alveolar Proteinosis by Mutation of the MARS Gene.	Pulmonary Alveolar Proteinosis;Mutation Ala393Thr of the MARS Gene;mutationSer567Leu of the MARS Gene	Drug: Methionine;Drug: Vitamin B12 , B9, B6, C supplementation;Diagnostic Test: Methionine/homocysteine Dosage;Diagnostic Test: Thoracic CT scan;Diagnostic Test: Abdominal and liver ultrasound.;Diagnostic Test: Brain MRI	Assistance Publique - Hôpitaux de Paris	NULL	Completed	N/A	18 Years	All	3	Phase 1;Phase 2	France

No.

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Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

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size

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NCT03887169
(ClinicalTrials.gov)

September 16, 2019

12/3/2019

Administration of Methionine in Patients With Pulmonary Alveolar Proteinosis by Mutation of the MARS Gene.

Oral or Enteral Administration of Methionine in Patients With Pulmonary Alveolar Proteinosis by Mutation of the MARS Gene.

Pulmonary Alveolar Proteinosis;Mutation Ala393Thr of the MARS Gene;mutationSer567Leu of the MARS Gene

Drug: Methionine;Drug: Vitamin B12 , B9, B6, C supplementation;Diagnostic Test: Methionine/homocysteine Dosage;Diagnostic Test: Thoracic CT scan;Diagnostic Test: Abdominal and liver ultrasound.;Diagnostic Test: Brain MRI

Assistance Publique - Hôpitaux de Paris

NULL

Completed

N/A

18 Years

All

Phase 1;Phase 2

France