DDrare: Database of Drug Development for Rare Diseases

Device: adalimumab delivered in physiolis autoinjector (DrugBank: Adalimumab)

16 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
19	ライソゾーム病	0
37	膿疱性乾癬（汎発型）	0
40	高安動脈炎	0
41	巨細胞性動脈炎	0
46	悪性関節リウマチ	1
56	ベーチェット病	0
84	サルコイドーシス	0
96	クローン病	0
97	潰瘍性大腸炎	0
151	ラスムッセン脳炎	0
160	先天性魚鱗癬	0
164	眼皮膚白皮症	0
222	一次性ネフローゼ症候群	0
226	間質性膀胱炎（ハンナ型）	0
269	化膿性無菌性関節炎・壊疽性膿皮症・アクネ症候群	0
271	強直性脊椎炎	0

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

37. 膿疱性乾癬（汎発型）　［臨床試験数：58，薬物数：50（DrugBank：18），標的遺伝子数：17，標的パスウェイ数：96］

Searched query = "Generalised pustular psoriasis", "Pustular psoriasis", "Herpetic impetigo", "Generalization of acrodermatitis continua", "Generalization of dermatitis continua of the extremities"
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 / 58 trial found

40. 高安動脈炎　［臨床試験数：22，薬物数：46（DrugBank：20），標的遺伝子数：22，標的パスウェイ数：111］

Searched query = "Takayasu arteritis", "Aortitis syndrome", "Pulseless 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 / 22 trial found

41. 巨細胞性動脈炎　［臨床試験数：108，薬物数：111（DrugBank：32），標的遺伝子数：33，標的パスウェイ数：121］

Searched query = "Giant cell arteritis", "Temporal arteritis"
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 / 108 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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

1 / 4,183 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	NCT01163617 (ClinicalTrials.gov)	May 2010	4/5/2010	The Usability and Injection Time of the Physiolis Syringe and Autoinjector in Rheumatoid Arthritis Patients	A Multicenter, Randomized, Open-Label Study of the Injection Time and Usability of the Physiolis Syringe and Autoinjector in Injection-Experienced Rheumatoid Arthritis Patients	Rheumatoid Arthritis	Device: Adalimumab delivered in current syringe;Device: Adalimumab delivered in Physiolis syringe;Device: Adalimumab delivered in current autoinjector;Device: Adalimumab delivered in Physiolis autoinjector	AbbVie (prior sponsor, Abbott)	NULL	Completed	18 Years	N/A	All	85	Phase 2	United States

56. ベーチェット病　［臨床試験数：68，薬物数：87（DrugBank：31），標的遺伝子数：36，標的パスウェイ数：113］

Searched query = "Behcet disease", "Behçet 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 / 68 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

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

151. ラスムッセン脳炎　［臨床試験数：2，薬物数：4（DrugBank：2），標的遺伝子数：6，標的パスウェイ数：84］

Searched query = "Rasmussen encephalitis"
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 trial found

160. 先天性魚鱗癬　［臨床試験数：32，薬物数：62（DrugBank：18），標的遺伝子数：15，標的パスウェイ数：107］

Searched query = "Congenital ichthyosis", "Keratinopathic ichthyosis", "Epidermolytic ichthyosis", "Harlequin ichthyosis", "Congenital Ichthyosiform Erythroderma", "Foliate ichthyosis", "Ichthyosis syndrome", "Netherton syndrome", "Sjogren-Larsson syndrome", "Sjögren-Larsson syndrome", "Keratitis-ichtyosis-deafness syndrome", "Dorfman-Chanarin syndrome", "Neutral lipid storage disease", "NLSD", "Multiple sulfatase deficiency", "Austin disease", "Recessive X-linked ichthyosis", "RXLI", "X-linked recessive ichthyosis", "Ichthyosis, brittle hair, impaired intelligence, decreased fertility and short stature", "IBID", "Trichothiodystrophy", "Follicular ichthyosis", "Congenital hemidysplasia, ichthyosiform erythroderma or nevus, and limb defects syndrome", "CHILD syndrome", "Conradi-Hunermann-Happle syndrome", "Conradi-Hünermann-Happle syndrome", "CHHS"
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 / 32 trial found

164. 眼皮膚白皮症　［臨床試験数：15，薬物数：57（DrugBank：34），標的遺伝子数：34，標的パスウェイ数：136］

Searched query = "Oculocutaneous albinism", "Hermansky-Pudlak syndrome", "Chediak-Higashi syndrome", "Griscelli 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 / 15 trial found

222. 一次性ネフローゼ症候群　［臨床試験数：234，薬物数：241（DrugBank：78），標的遺伝子数：59，標的パスウェイ数：185］

Searched query = "Primary nephrotic syndrome", "Minimal change nephrotic syndrome", "MCNS", "Membranous nephropathy", "Focal segmental glomerulosclerosis", "FSGS", "Membranoproliferative glomerulonephritis", "MPGN"
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 / 234 trial found

226. 間質性膀胱炎（ハンナ型）　［臨床試験数：133，薬物数：151（DrugBank：46），標的遺伝子数：48，標的パスウェイ数：137］

Searched query = "Interstitial cystitis with Hunners ulcer", "Interstitial cystitis"
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 / 133 trial found

269. 化膿性無菌性関節炎・壊疽性膿皮症・アクネ症候群　［臨床試験数：20，薬物数：26（DrugBank：10），標的遺伝子数：12，標的パスウェイ数：90］

Searched query = "Pyogenic arthritis", "Pyoderma gangrenosum", "Acne syndrome", "PAPA 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 / 20 trial found

271. 強直性脊椎炎　［臨床試験数：531，薬物数：343（DrugBank：66），標的遺伝子数：41，標的パスウェイ数：141］

Searched query = "Ankylosing spondylitis", "Spondylarthritis ankylopoietica"
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 / 531 trial found