DDrare: Database of Drug Development for Rare Diseases

Other: sham-procedure (dose cohort 1&2 only) (DrugBank: -)

2 diseases

ID	Disease name (Link within this page)	Number of trials
90	Retinitis pigmentosa	1
303	Usher syndrome	1

90. Retinitis pigmentosa [ 103 clinical trials, 158 drugs, (DrugBank: 42 drugs), 52 drug target genes, 107 drug target pathways]

Searched query = "Retinitis pigmentosa", "Rod dystrophy", "Rod-Cone Dystrophy"
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 / 103 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	NCT03780257 (ClinicalTrials.gov)	March 6, 2019	17/12/2018	Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene	A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene	Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision Disorders	Drug: QR-421a;Other: Sham-procedure (dose cohort 1&2 only)	ProQR Therapeutics	NULL	Recruiting	18 Years	N/A	All	18	Phase 1;Phase 2	United States;Belgium;Canada;France

303. Usher syndrome [ 6 clinical trials, 11 drugs, (DrugBank: 0), 0 drug target gene, 0 drug target pathway]

Searched query = "Usher syndrome"
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 / 6 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	NCT03780257 (ClinicalTrials.gov)	March 6, 2019	17/12/2018	Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene	A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene	Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision Disorders	Drug: QR-421a;Other: Sham-procedure (dose cohort 1&2 only)	ProQR Therapeutics	NULL	Recruiting	18 Years	N/A	All	18	Phase 1;Phase 2	United States;Belgium;Canada;France