DDrare: Database of Drug Development for Rare Diseases

Oxycodone/acetaminophen (DrugBank: Acetaminophen, Oxycodone)

16 diseases

ID	Disease name (Link within this page)	Number of trials
3	Spinal muscular atrophy	0
6	Parkinson disease	0
11	Myasthenia	0
13	Multiple sclerosis/Neuromyelitis optica	0
34	Neurofibromatosis	0
35	Pemphigus	0
46	Malignant rheumatoid arthritis	0
49	Systemic lupus erythematosus	0
51	Scleroderma	0
70	Spinal stenosis	0
86	Pulmonary arterial hypertension	0
96	Crohn disease	0
222	Primary nephrotic syndrome	0
231	Alpha-1-antitrypsin deficiency	1
256	Muscle glycogenosis	0
271	Ankylosing spondylitis	0

3. Spinal muscular atrophy [ 179 clinical trials, 102 drugs, (DrugBank: 26 drugs), 52 drug target genes, 78 drug target pathways]

Searched query = "Spinal muscular atrophy", "Myelopathic muscular atrophy", "SMA I", "Werdnig-Hoffman disease", "SMA II", "Dubowitz disease", "SMA III", "Kugelberg-Welander disease", "SMA IV"
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 / 179 trial found

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

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

35. Pemphigus [ 87 clinical trials, 114 drugs, (DrugBank: 37 drugs), 17 drug target genes, 158 drug target pathways]

Searched query = "Pemphigus"
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 / 87 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

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

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

70. Spinal stenosis [ 91 clinical trials, 195 drugs, (DrugBank: 54 drugs), 60 drug target genes, 85 drug target pathways]

Searched query = "Spinal stenosis", "Extensive spinal canal stenosis"
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 / 91 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

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

222. Primary nephrotic syndrome [ 234 clinical trials, 241 drugs, (DrugBank: 78 drugs), 59 drug target genes, 185 drug target pathways]

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

231. Alpha-1-antitrypsin deficiency [ 83 clinical trials, 89 drugs, (DrugBank: 16 drugs), 35 drug target genes, 46 drug target pathways]

Searched query = "Alpha-1-antitrypsin deficiency", "AATD"
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 / 83 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	NCT01810458 (ClinicalTrials.gov)	October 2013	6/3/2013	Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD)	Clinical Predictors and Epigenetic Markers for Liver Fibrosis in Alpha-1 Antitrypsin Deficiency	Liver Fibrosis;Alpha-1 Antitrypsin Deficiency;AAT Deficiency;AATD	Device: Abdominal ultrasound;Procedure: History and physical;Procedure: Intravenous catheter;Procedure: Blood draw;Other: Liver questionnaire;Procedure: Liver Biopsy;Drug: Midazolam;Drug: Fentanyl;Drug: Lidocaine;Drug: Acetaminophen;Drug: Lorazepam;Drug: Oxycodone/Acetaminophen;Drug: Ondansetron	University of Florida	NULL	Completed	18 Years	70 Years	All	109		United States

256. Muscle glycogenosis [ 161 clinical trials, 114 drugs, (DrugBank: 23 drugs), 26 drug target genes, 106 drug target pathways]

Searched query = "Muscle glycogenosis", "Muscular glycogenosis", "Muscle glycogen storage disease", "Muscular glycogen storage disease", "Glycogen storage disease type 0", "GSD0", "Glycogen synthase deficiency", "Glycogen storage disease type II", "GSDII", "Pompe disease", "Alpha-1,4-glucosidase acid deficiency", "Glycogen storage disease type III", "GSDIII", "Cori disease", "Glycogen debranching enzyme deficiency", "Glycogen storage disease type IV", "GSDIV", "Andersen disease", "Glycogen-branching enzyme deficiency", "GBED", "Glycogen storage disease type V", "GSDV", "McArdle disease", "Muscle phosphorylase deficiency", "Muscular phosphorylase deficiency", "Glycogen storage disease type VII", "GSDVII", "Tarui disease", "Phosphofructokinase deficiency", "PFK deficiency", "Glycogen storage disease type IX", "GSDIX", "Phosphorylase kinase deficiency", "Phosphoglycerate kinase deficiency", "PGK deficiency", "Glycogen storage disease type X", "GSDX", "Phosphoglycerate mutase deficiency", "Glycogen storage diseass type XI", "GSDXI", "Kanno disease", "Lactate dehydrogenase deficiency", "Glycogen storage diseass type XII", "GSDXII", "Aldolase A deficiency", "Glycogen storage diseass type XIII", "GSDXIII", "Beta-enolase deficiency", "Glycogen storage diseass type XIV", "GSDXIV", "Phosphoglucomutase deficiency", "Glycogen storage diseass type XV", "GSDXV", "Glycogenin 1 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 / 161 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: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 531 trial found