DDrare: Database of Drug Development for Rare Diseases

Other: challenge agent: capsaicin (DrugBank: Capsaicin)

5 diseases

ID	Disease name (Link within this page)	Number of trials
2	Amyotrophic lateral sclerosis	0
6	Parkinson disease	0
19	Lysosomal storage disease	1
86	Pulmonary arterial hypertension	0
226	Interstitial cystitis with Hunners ulcer	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

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

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

1 / 784 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	NCT02533076 (ClinicalTrials.gov)	November 2014	14/7/2015	The Functional Consequences of the CTNS-deletion for the TRPV1-receptor in Cystinosis Patients	The Functional Consequences of the CTNS-deletion for the TRPV1-receptor in Cystinosis Patients	Cystinosis	Other: Challenge agent: capsaicin;Other: Challenge agent: placebo;Device: Mechanical stimulation with Von Frey filaments;Device: Temperature sensitivity measurement with Advanced Thermal Stimulation	Universitaire Ziekenhuizen Leuven	NULL	Completed	8 Years	N/A	Both	50	Phase 0	Belgium

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

226. Interstitial cystitis with Hunners ulcer [ 133 clinical trials, 151 drugs, (DrugBank: 46 drugs), 48 drug target genes, 137 drug target pathways]

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