DDrare: Database of Drug Development for Rare Diseases

L-citrulline and metformin & l-citrulline (DrugBank: Citrulline, L-Citrulline, Metformin)

21 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
2	筋萎縮性側索硬化症	0
6	パーキンソン病	0
13	多発性硬化症／視神経脊髄炎	0
46	悪性関節リウマチ	0
49	全身性エリテマトーデス	0
60	再生不良性貧血	0
67	多発性嚢胞腎	0
75	クッシング病	0
77	下垂体性成長ホルモン分泌亢進症	0
81	先天性副腎皮質酵素欠損症	0
86	肺動脈性肺高血圧症	0
113	筋ジストロフィー	1
127	前頭側頭葉変性症	0
158	結節性硬化症	0
206	脆弱X症候群	0
225	先天性腎性尿崩症	0
233	ウォルフラム症候群	0
265	脂肪萎縮症	0
285	ファンコニ貧血	0
299	嚢胞性線維症	0
301	黄斑ジストロフィー	0

2. 筋萎縮性側索硬化症　［臨床試験数：508，薬物数：530（DrugBank：146），標的遺伝子数：170，標的パスウェイ数：221］

Searched query = "Amyotrophic lateral sclerosis", "ALS"
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 / 508 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

13. 多発性硬化症／視神経脊髄炎　［臨床試験数：3,050，薬物数：2,147（DrugBank：348），標的遺伝子数：244，標的パスウェイ数：228］

Searched query = "Multiple sclerosis/Neuromyelitis optica", "Multiple sclerosis", "Neuromyelitis optica", "MS", "NMOSD", "Devic disease", "Balo concentric sclerosis", "Baló concentric sclerosis"
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 / 3,050 trial found

46. 悪性関節リウマチ　［臨床試験数：4,183，薬物数：2,538（DrugBank：401），標的遺伝子数：183，標的パスウェイ数：219］

Searched query = "Malignant rheumatoid arthritis", "Rheumatoid arthritis", "Rheumatoid arthritis with vasculitis"
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 / 4,183 trial found

49. 全身性エリテマトーデス　［臨床試験数：827，薬物数：638（DrugBank：168），標的遺伝子数：108，標的パスウェイ数：191］

Searched query = "Systemic lupus erythematosus", "SLE"
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 / 827 trial found

60. 再生不良性貧血　［臨床試験数：218，薬物数：362（DrugBank：81），標的遺伝子数：39，標的パスウェイ数：155］

Searched query = "Aplastic 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 / 218 trial found

67. 多発性嚢胞腎　［臨床試験数：186，薬物数：196（DrugBank：47），標的遺伝子数：35，標的パスウェイ数：146］

Searched query = "Polycystic kidney disease", "PKD", "PCKD", "Polycystic kidney", "ADPKD", "ARPKD"
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 / 186 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

77. 下垂体性成長ホルモン分泌亢進症　［臨床試験数：1，薬物数：1（DrugBank：1），標的遺伝子数：2，標的パスウェイ数：19］

Searched query = "Growth hormone secreting pituitary adenoma", "Pituitary growth hormone secretion hyperthyroidism"
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 / 1 trial found

81. 先天性副腎皮質酵素欠損症　［臨床試験数：65，薬物数：77（DrugBank：20），標的遺伝子数：11，標的パスウェイ数：65］

Searched query = "Congenital adrenal hyperplasia", "Congenital adrenal enzyme deficiency", "Congenial adrenal cortex enzyme deficiency", "Congenital Lipoid Adrenal Hyperplasia", "3β-Hydroxysteroid Dehydrogenase Deficiency", "21-Hydroxylase deficiency", "11β-Hydroxylase deficiency", "17α-Hydroxylase deficiency", "P450 oxidoreductase deficiency", "Aldosterone synthase deficiency"
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 / 65 trial found

86. 肺動脈性肺高血圧症　［臨床試験数：1,083，薬物数：667（DrugBank：122），標的遺伝子数：98，標的パスウェイ数：185］

Searched query = "Pulmonary arterial hypertension", "PAH", "IPAH", "HPAH", "Eisenmenger syndrome"
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 / 1,083 trial found

113. 筋ジストロフィー　［臨床試験数：567，薬物数：442（DrugBank：93），標的遺伝子数：55，標的パスウェイ数：151］

Searched query = "Muscular dystrophy", "Dystrophinopathies", "Myotilinopathy", "Laminopathy", "Caveolinopathy", "LGMD1C", "Desminopathy", "Sarcoglycanopathy", "α-dystroglycanopathy", "FCMD", "Walker-Warburg syndrome", "Muscle-eye-brain disease", "Myotonic dystrophy", "Integrin α7 deficient CMD", "Rigid spine syndrome"
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 / 567 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	NCT02018731 (ClinicalTrials.gov)	June 2013	17/12/2013	L-citrulline and Metformin in Becker's Muscular Dystrophy	Pilot Study to Assess the Efficacy of L-Citrulline and Metformin in Adults With Becker's Muscular Dystrophy	Becker's Muscular Dystrophy (BMD)	Drug: Metformin and Metformin & L-Citrulline;Drug: L-Citrulline and Metformin & L-Citrulline	University Hospital, Basel, Switzerland	NULL	Completed	18 Years	N/A	Both	20	Phase 2	Switzerland

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

158. 結節性硬化症　［臨床試験数：100，薬物数：62（DrugBank：16），標的遺伝子数：35，標的パスウェイ数：115］

Searched query = "Tuberous sclerosis", "Tuberous sclerosis complex"
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 / 100 trial found

206. 脆弱X症候群　［臨床試験数：98，薬物数：73（DrugBank：27），標的遺伝子数：46，標的パスウェイ数：57］

Searched query = "Fragile X syndrome"
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 / 98 trial found

225. 先天性腎性尿崩症　［臨床試験数：12，薬物数：45（DrugBank：17），標的遺伝子数：30，標的パスウェイ数：63］

Searched query = "Congenital nephrogenic diabetes insipidus", "Hereditary nephrogenic diabetes insipidus", "Nephrogenic diabetes insipidus"
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 / 12 trial found

233. ウォルフラム症候群　［臨床試験数：6，薬物数：12（DrugBank：7），標的遺伝子数：11，標的パスウェイ数：40］

Searched query = "Wolfram syndrome", "Diabetes Insipidus, Diabetes mellitus, optic atrophy, and deafness syndrome", "DIDMOAD syndrome"
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 / 6 trial found

265. 脂肪萎縮症　［臨床試験数：109，薬物数：164（DrugBank：59），標的遺伝子数：26，標的パスウェイ数：94］

Searched query = "Lipodystrophy", "Berardinelli-Seip syndrome", "Lawrence syndrome", "Barraquer-Simons syndrome"
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 / 109 trial found

285. ファンコニ貧血　［臨床試験数：56，薬物数：111（DrugBank：31），標的遺伝子数：30，標的パスウェイ数：151］

Searched query = "Fanconi 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 / 56 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: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 1,592 trial found

301. 黄斑ジストロフィー　［臨床試験数：41，薬物数：47（DrugBank：12），標的遺伝子数：12，標的パスウェイ数：66］

Searched query = "Macular dystrophy", "Best disease", "Stargardt disease", "Cone dystrophy", "Cone rod dystrophy", "X-linked juvenile retinoschisis", "Central areolar choroidal dystrophy"
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 / 41 trial found