DDrare: Database of Drug Development for Rare Diseases

Diagnostic test: methionine/homocysteine dosage (DrugBank: Methionine, Homocysteine)

7 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
34	神経線維腫症	0
75	クッシング病	0
84	サルコイドーシス	0
93	原発性胆汁性胆管炎［原発性胆汁性肝硬変 (~2017.3)］	0
229	肺胞蛋白症（自己免疫性又は先天性）	1
298	遺伝性膵炎	0
300	IgG4関連疾患	0

34. 神経線維腫症　［臨床試験数：120，薬物数：182（DrugBank：72），標的遺伝子数：84，標的パスウェイ数：194］

Searched query = "Neurofibromatosis", "NF1", "von Recklinghausen disease", "NF2"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 120 trial found

75. クッシング病　［臨床試験数：191，薬物数：172（DrugBank：48），標的遺伝子数：61，標的パスウェイ数：121］

Searched query = "Cushing disease", "Cushing"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 191 trial found

84. サルコイドーシス　［臨床試験数：143，薬物数：221（DrugBank：79），標的遺伝子数：82，標的パスウェイ数：165］

Searched query = "Sarcoidosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 143 trial found

93. 原発性胆汁性胆管炎［原発性胆汁性肝硬変 (~2017.3)］　［臨床試験数：230，薬物数：215（DrugBank：51），標的遺伝子数：34，標的パスウェイ数：107］

Searched query = "Primary biliary cholangitis", "Primary biliary cirrhosis", "PBC"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 230 trial found

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.

1 / 35 trial found

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

298. 遺伝性膵炎　［臨床試験数：93，薬物数：168（DrugBank：51），標的遺伝子数：48，標的パスウェイ数：134］

Searched query = "Hereditary pancreatitis", "Chronic pancreatitis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 93 trial found

300. IgG4関連疾患　［臨床試験数：31，薬物数：33（DrugBank：16），標的遺伝子数：11，標的パスウェイ数：81］

Searched query = "IgG4-related disease", "Autoimmune pancreatitis", "IgG4-related sclerosing cholangitis", "IgG4-related lacrimal gland, orbital, and salivary gland lesions", "IgG4-related kidney disease"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 31 trial found