DDrare: Database of Drug Development for Rare Diseases

Anakinra and peg stnf-r1 (DrugBank: Anakinra, sTNF-R1)

19 diseases

ID	Disease name (Link within this page)	Number of trials
2	Amyotrophic lateral sclerosis	0
13	Multiple sclerosis/Neuromyelitis optica	0
15	Inclusion body myositis	0
19	Lysosomal storage disease	0
28	Systemic amyloidosis	0
37	Generalised pustular psoriasis	0
41	Giant cell arteritis	0
46	Malignant rheumatoid arthritis	1
49	Systemic lupus erythematosus	0
50	Dermatomyositis	0
56	Behcet disease	0
65	Primary immunodeficiency	0
84	Sarcoidosis	0
86	Pulmonary arterial hypertension	0
97	Ulcerative colitis	0
106	Cryopyrin-associated periodic syndrome	0
107	Juvenile idiopathic arthritis [Systemic juvenile idiopathic arthritis (~Mar 2018)]	0
266	Familial mediterranean fever	0
299	Cystic fibrosis	0

2. Amyotrophic lateral sclerosis [ 508 clinical trials, 530 drugs, (DrugBank: 146 drugs), 170 drug target genes, 221 drug target pathways]

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

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

15. Inclusion body myositis [ 39 clinical trials, 32 drugs, (DrugBank: 11 drugs), 11 drug target genes, 119 drug target pathways]

Searched query = "Inclusion body myositis"
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 / 39 trial found

19. Lysosomal storage disease [ 784 clinical trials, 673 drugs, (DrugBank: 101 drugs), 68 drug target genes, 184 drug target pathways]

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

28. Systemic amyloidosis [ 212 clinical trials, 234 drugs, (DrugBank: 72 drugs), 54 drug target genes, 170 drug target pathways]

Searched query = "Systemic amyloidosis", "AL amyloidosis", "Immunoglobulin light chain amyloidosis", "Amyloid light-chain amyloidosis", "Familial amyloidosis", "Familial amyloid polyneuropathy", "Senile transthyretin amyloidosis", "Senile TTR amyloidosis"
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 / 212 trial found

37. Generalised pustular psoriasis [ 58 clinical trials, 50 drugs, (DrugBank: 18 drugs), 17 drug target genes, 96 drug target pathways]

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

41. Giant cell arteritis [ 108 clinical trials, 111 drugs, (DrugBank: 32 drugs), 33 drug target genes, 121 drug target pathways]

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. 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: 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	NCT00537667 (ClinicalTrials.gov)	August 2001	27/9/2007	The SPECTRA Study	A Single Centre, Open-Label, Randomised, Study to Evaluate the Effect of Anakinra (r-metHuIL-1ra) Alone and in Combination With PEGylated Recombinant Methionyl Human Soluble Tumour Necrosis Factor Receptor Type I (PEG sTNF-RI) on Synovial Inflammation in Subjects With Rheumatoid Arthritis.	Rheumatoid Arthritis	Drug: anakinra;Drug: anakinra and PEG sTNF-R1	Amgen	NULL	Terminated	18 Years	N/A	Both	22	Phase 2	NULL

49. Systemic lupus erythematosus [ 827 clinical trials, 638 drugs, (DrugBank: 168 drugs), 108 drug target genes, 191 drug target pathways]

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

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

56. Behcet disease [ 68 clinical trials, 87 drugs, (DrugBank: 31 drugs), 36 drug target genes, 113 drug target pathways]

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

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

84. Sarcoidosis [ 143 clinical trials, 221 drugs, (DrugBank: 79 drugs), 82 drug target genes, 165 drug target pathways]

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

86. Pulmonary arterial hypertension [ 1,083 clinical trials, 667 drugs, (DrugBank: 122 drugs), 98 drug target genes, 185 drug target pathways]

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

97. Ulcerative colitis [ 2,269 clinical trials, 1,331 drugs, (DrugBank: 241 drugs), 114 drug target genes, 181 drug target pathways]

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

106. Cryopyrin-associated periodic syndrome [ 39 clinical trials, 23 drugs, (DrugBank: 4 drugs), 4 drug target genes, 46 drug target pathways]

Searched query = "Cryopyrin-associated periodic syndrome", "Cryopyrin associated periodic fever syndrome", "Familial cold autoinflammatory syndrome", "FCAS", "Mucke-Wells syndrome", "MWS", "Chronic infantile neurologic cutaneous, and articular syndrome", "CINCA syndrome", "Neonatal onset multisystem inflammatory disease", "NOMID"
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 / 39 trial found

107. Juvenile idiopathic arthritis [Systemic juvenile idiopathic arthritis (~Mar 2018)] [ 151 clinical trials, 75 drugs, (DrugBank: 13 drugs), 16 drug target genes, 90 drug target pathways]

Searched query = "Juvenile idiopathic arthritis [Systemic juvenile idiopathic arthritis (~Mar 2018)]", "Systemic-onset juvenile idiopathic arthritis", "JIA", "SJIA"
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 / 151 trial found

266. Familial mediterranean fever [ 27 clinical trials, 23 drugs, (DrugBank: 6 drugs), 14 drug target genes, 57 drug target pathways]

Searched query = "Familial mediterranean fever"
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 / 27 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