DDrare: Database of Drug Development for Rare Diseases

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.

Drug = Blood draw for the laboratory assessment; Lentiviral vector containing MYO7A gene; Lentiviral vector containing the human MY07A gene; NPI-001; NT-501; Other: Sham-procedure (dose cohort 1&2 only); Procedure: Sham; QR-421a; SAR421869; UshStat; UshStat (SAR421869)

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

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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

Phase 1;Phase 2

United States;Belgium;Canada;France