DDrare: Database of Drug Development for Rare Diseases

Other: 0.9ml sodium chloride (DrugBank: Sodium chloride, Chloride)

20 diseases

ID	Disease name (Link within this page)	Number of trials
6	Parkinson disease	0
11	Myasthenia	0
13	Multiple sclerosis/Neuromyelitis optica	0
14	Chronic inflammatory demyelinating polyneuropathy	0
34	Neurofibromatosis	0
46	Malignant rheumatoid arthritis	0
50	Dermatomyositis	0
51	Scleroderma	0
53	Sjogren syndrome	0
60	Aplastic anemia	0
65	Primary immunodeficiency	0
78	Hypopituitarism	0
96	Crohn disease	0
168	Ehlers-Danlos syndrome	0
193	Prader-Willi syndrome	0
227	Osler disease	0
228	Bronchiolitis obliterans	0
271	Ankylosing spondylitis	1
288	Autoimmune acquired coagulation factor deficiency [Autoimmune Hemorrhaphilia XIII (~Mar 2017)]	0
299	Cystic fibrosis	0

6. Parkinson disease [ 2,123 clinical trials, 2,046 drugs, (DrugBank: 324 drugs), 183 drug target genes, 198 drug target pathways]

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

11. Myasthenia [ 226 clinical trials, 172 drugs, (DrugBank: 45 drugs), 43 drug target genes, 118 drug target pathways]

Searched query = "Myasthenia", "MG"
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 / 226 trial found

13. Multiple sclerosis/Neuromyelitis optica [ 3,050 clinical trials, 2,147 drugs, (DrugBank: 348 drugs), 244 drug target genes, 228 drug target pathways]

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

14. Chronic inflammatory demyelinating polyneuropathy [ 145 clinical trials, 139 drugs, (DrugBank: 23 drugs), 12 drug target genes, 21 drug target pathways]

Searched query = "Chronic inflammatory demyelinating polyneuropathy", "Chronic inflammatory demyelinating poly (radiculo) neuropathy", "Multifocal motor neuropathy", "CIDP"
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 / 145 trial found

34. Neurofibromatosis [ 120 clinical trials, 182 drugs, (DrugBank: 72 drugs), 84 drug target genes, 194 drug target pathways]

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

46. Malignant rheumatoid arthritis [ 4,183 clinical trials, 2,538 drugs, (DrugBank: 401 drugs), 183 drug target genes, 219 drug target pathways]

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

50. Dermatomyositis [ 157 clinical trials, 207 drugs, (DrugBank: 76 drugs), 47 drug target genes, 142 drug target pathways]

Searched query = "Dermatomyositis", "Polymyositis"
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 / 157 trial found

51. Scleroderma [ 466 clinical trials, 536 drugs, (DrugBank: 142 drugs), 110 drug target genes, 210 drug target pathways]

Searched query = "Scleroderma", "Systemic sclerosis", "SSc", "dcSSc", "lcSSc"
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 / 466 trial found

53. Sjogren syndrome [ 234 clinical trials, 270 drugs, (DrugBank: 85 drugs), 49 drug target genes, 174 drug target pathways]

Searched query = "Sjogren syndrome", "Sjögren 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 / 234 trial found

60. Aplastic anemia [ 218 clinical trials, 362 drugs, (DrugBank: 81 drugs), 39 drug target genes, 155 drug target pathways]

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

65. Primary immunodeficiency [ 413 clinical trials, 581 drugs, (DrugBank: 97 drugs), 68 drug target genes, 202 drug target pathways]

Searched query = "Primary immunodeficiency", "X-SCID", "Reticular dysgenesis", "Adenosine deaminase deficiency", "Omenn syndrome", "Purine nucleoside phosphorylase deficiency", "CD8 deficiency", "ZAP-70 deficiency", "MHC class I deficiency", "MHC class II deficiency", "Combined immunodeficiency", "Wiskott-Aldrich syndrome", "Telangiectasia ataxia", "Nijmegen breakage syndrome", "Bloom syndrome", "Immunodeficiency, centromere region instability, facial anomalies syndrome", "ICF syndrome", "PMS2 deficiency", "Radiosensitivity, immunodeficiency, dysmorphic features, and learning difficulties syndrome", "RIDDLE syndrome", "Schimke syndrome", "Netherton syndrome", "Thymic hypoplasia", "DiGeorge syndrome", "22q11.2 deletion syndrome", "Hyper-IgE syndrome", "Hepatic venoocclusive immunodeficiency", "Immunodeficiency with central hepatic vein atresia", "Dyskeratosis congenita", "X-linked agammaglobulinaemia", "Common variable immunodeficiency", "Hyper-IgM syndrome", "Isolated IgG subclass deficiency", "Selective IgA deficiency", "Specific antibody production deficiency", "Infant transient hypogammaglobulinemia", "Chédiak-Higashi syndrome", "Chediak-Higashi syndrome", "X-linked lymphoproliferative syndrome", "SAP deficiency", "SH2D1A/SLAM-associated protein deficiency", "XIAP deficiency", "X-linked inhibitor of apoptosis deficiency", "Autoimmune lymphoproliferative syndrome", "ALPS", "Familial hemophagocytic syndrome", "Perforin deficiency", "Munc13-4 deficiency", "Syntaxin 11 deficiency", "Munc18-2 deficiency", "Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy", "APECED", "Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome", "IPEX syndrome", "CD25 deficiency", "ITCH deficiency", "Primary phagocytic dysfunction", "Severe congenital neutropenia", "Cyclic neutropenia", "Hermanskyi-Pudlak syndrome type 2", "Hermanskyi-Pudlak syndrome 2", "Griscelli syndrome type 2", "Griscelli syndrome 2", "p14 deficiency", "Warts, hypogammaglobulinemia, infections, myelokathexis syndrome", "WHIM syndrome", "Glycogen storage disease type Ib", "Leukocyte adhesion deficiency", "Shwachman-Diamond syndrome", "Chronic granulomatous disease", "Myeloperoxidase deficiency", "Mendelian susceptibility to mycobacterial disease", "MSMD", "Anhidrotic ectodermal dysplasia with immunodeficiency", "EDA-ID", "Interleukin-1 receptor-associated kinase-4 deficiency", "IRAK4 deficiency", "IMyD88 deficiency", "Chronic mucocutaneous candidiasis", "Epidermodysplasia verruciformis", "Herpes simplex encephalitis", "Caspase recruitment domain family member 9 deficiency", "CARD9 deficiency", "Trypanosomiasis", "Congenital complement deficiency", "C1q deficiency", "CC1r deficiency", "CC1s deficiency", "CC2 deficiency", "CC3 deficiency", "CC4 deficiency", "CC5 deficiency", "CC6 deficiency", "CC7 deficiency", "CC8 deficiency", "CC9 deficiency", "Factor D deficiency", "Properdin deficiency", "Factor I deficiency", "Factor H deficiency", "MASP1 deficiency", "3MC syndrome", "Mannose-binding protein-associated serine protease 2 deficiency", "MASP2 deficiency", "FCN3", "Hereditary angioedema type 1", "Hereditary angioedema type I", "C1 inhibitor deficiency type 1", "C1 inhibitor deficiency type I", "Hereditary angioedema type 2", "Hereditary angioedema type II", "C1 inhibitor deficiency type 2", "C1 inhibitor deficiency type II", "Hereditary angioedema type 3", "Hereditary angioedema type III", "C1 inhibitor deficiency type 3", "C1 inhibitor deficiency type III"
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 / 413 trial found

78. Hypopituitarism [ 462 clinical trials, 346 drugs, (DrugBank: 45 drugs), 41 drug target genes, 80 drug target pathways]

Searched query = "Hypopituitarism", "Anterior pituitary hypothyroidism", "Syndrome of abnormal secretion of gonadotropin", "Hyposecretion of gonadotropins", "Adrenocorticotropic hormone deficiency", "ACTH deficiency", "Thyroid-stimulating hormone deficiency", "TSH deficiency", "Growth hormone deficiency", "GH deficiency", "GHD", "CGHD", "AGHD", "Prolactin deficiency", "PRL 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 / 462 trial found

96. Crohn disease [ 2,209 clinical trials, 1,276 drugs, (DrugBank: 240 drugs), 166 drug target genes, 210 drug target pathways]

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

168. Ehlers-Danlos syndrome [ 7 clinical trials, 16 drugs, (DrugBank: 8 drugs), 9 drug target genes, 54 drug target pathways]

Searched query = "Ehlers-Danlos syndrome", "EDS", "cEDS", "hEDS", "clEDS", "vEDS", "kEDS", "aEDS", "dEDS", "DDEDS"
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 / 7 trial found

193. Prader-Willi syndrome [ 95 clinical trials, 104 drugs, (DrugBank: 27 drugs), 50 drug target genes, 63 drug target pathways]

Searched query = "Prader-Willi 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 / 95 trial found

227. Osler disease [ 49 clinical trials, 69 drugs, (DrugBank: 21 drugs), 23 drug target genes, 132 drug target pathways]

Searched query = "Osler disease", "Hereditary hemorrhagic telangiectasia", "Osler-Weber-Rendu 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 / 49 trial found

228. Bronchiolitis obliterans [ 92 clinical trials, 125 drugs, (DrugBank: 34 drugs), 33 drug target genes, 152 drug target pathways]

Searched query = "Bronchiolitis obliterans", "Obliterating bronchiolitis"
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 / 92 trial found

271. Ankylosing spondylitis [ 531 clinical trials, 343 drugs, (DrugBank: 66 drugs), 41 drug target genes, 141 drug target pathways]

Searched query = "Ankylosing spondylitis", "Spondylarthritis ankylopoietica"
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 / 531 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	NCT01212653 (ClinicalTrials.gov)	October 2010	30/9/2010	Effect of Anti-TNF (Alpha) Treatment on Vascular Stiffness in Ankylosing Spondylitis (AS)	Effect of Anti-TNF(Alpha)Treatment on Circulating Endothelial Progenitor Cells (EPCs) and Vascular Stiffness in AS	Ankylosing Spondylitis(AS)	Drug: Simponi;Other: 0.9ml sodium chloride	Chinese University of Hong Kong	NULL	Completed	18 Years	75 Years	Both	50	Phase 4	China

288. Autoimmune acquired coagulation factor deficiency [Autoimmune Hemorrhaphilia XIII (~Mar 2017)] [ 189 clinical trials, 219 drugs, (DrugBank: 29 drugs), 18 drug target genes, 26 drug target pathways]

Searched query = "Autoimmune acquired coagulation factor deficiency", "Coagulation factor deficiency", "Factor XIII deficiency", "Factor VIII deficiency", "Acquired hemophilia A", "von Willebrand Disease", "Factor V 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 / 189 trial found

299. Cystic fibrosis [ 1,592 clinical trials, 1,539 drugs, (DrugBank: 255 drugs), 81 drug target genes, 162 drug target pathways]

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