DDrare: Database of Drug Development for Rare Diseases

Essential amino acid intake + leucine vs total aa supplement (DrugBank: Leucine)

10 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
5	進行性核上性麻痺	0
6	パーキンソン病	0
7	大脳皮質基底核変性症	0
19	ライソゾーム病	0
96	クローン病	0
97	潰瘍性大腸炎	0
127	前頭側頭葉変性症	0
283	後天性赤芽球癆	0
284	ダイアモンド・ブラックファン貧血	0
299	嚢胞性線維症	1

5. 進行性核上性麻痺　［臨床試験数：82，薬物数：107（DrugBank：36），標的遺伝子数：60，標的パスウェイ数：90］

Searched query = "Progressive supranuclear palsy", "PSP"
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 / 82 trial found

6. パーキンソン病　［臨床試験数：2,123，薬物数：2,046（DrugBank：324），標的遺伝子数：183，標的パスウェイ数：198］

Searched query = "Parkinson 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 / 2,123 trial found

7. 大脳皮質基底核変性症　［臨床試験数：16，薬物数：33（DrugBank：11），標的遺伝子数：6，標的パスウェイ数：13］

Searched query = "Corticobasal degeneration", "Corticobasal syndrome", "CBD"
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 / 16 trial found

19. ライソゾーム病　［臨床試験数：784，薬物数：673（DrugBank：101），標的遺伝子数：68，標的パスウェイ数：184］

Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
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 / 784 trial found

96. クローン病　［臨床試験数：2,209，薬物数：1,276（DrugBank：240），標的遺伝子数：166，標的パスウェイ数：210］

Searched query = "Crohn disease", "Terminal ileitis"
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 / 2,209 trial found

97. 潰瘍性大腸炎　［臨床試験数：2,269，薬物数：1,331（DrugBank：241），標的遺伝子数：114，標的パスウェイ数：181］

Searched query = "Ulcerative colitis"
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 / 2,269 trial found

127. 前頭側頭葉変性症　［臨床試験数：74，薬物数：83（DrugBank：28），標的遺伝子数：29，標的パスウェイ数：99］

Searched query = "Frontotemporal lobar degeneration", "Frontotemporal dementia", "Semantic dementia"
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 / 74 trial found

283. 後天性赤芽球癆　［臨床試験数：17，薬物数：36（DrugBank：19），標的遺伝子数：16，標的パスウェイ数：91］

Searched query = "Acquired pure red cell aplasia", "Pure red cell aplasia"
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 / 17 trial found

284. ダイアモンド・ブラックファン貧血　［臨床試験数：37，薬物数：110（DrugBank：34），標的遺伝子数：22，標的パスウェイ数：121］

Searched query = "Diamond-Blackfan anemia"
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 / 37 trial found

299. 嚢胞性線維症　［臨床試験数：1,592，薬物数：1,539（DrugBank：255），標的遺伝子数：81，標的パスウェイ数：162］

Searched query = "Cystic fibrosis"
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 / 1,592 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	NCT01172301 (ClinicalTrials.gov)	July 2008	27/7/2010	Leucine-enriched Essential Amino Acid Intake to Optimize Protein Anabolism in Children With Cystic Fibrosis	Leucine-enriched Essential Amino Acid Intake to Optimize Protein Anabolism in Children With Cystic Fibrosis	Cystic Fibrosis	Dietary Supplement: Essential amino acid intake + Leucine vs total AA supplement	Texas A&M University	Arkansas Children's Hospital Research Institute	Completed	10 Years	21 Years	Both	14	N/A	United States