NCT03780257View on ClinicalTrials.gov

Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

20

Participants

Timeline

Start Date

March 6, 2019

Primary Completion Date

October 14, 2021

Study Completion Date

October 14, 2021

Conditions
Retinitis PigmentosaUsher Syndrome Type 2Deaf BlindRetinal DiseaseEye DiseasesEye Diseases, HereditaryEye Disorders CongenitalVision Disorders
Interventions
DRUG

QR-421a

RNA antisense oligonucleotide for intravitreal injection

OTHER

Sham-procedure (dose cohort 1&2 only)

Sham-procedure (no experimental drug administered)

Trial Locations (7)

34295

Hôpital Gui de Chauliac - CHRU de Montpellier - Maladies Sensorielles Génétique, Montpellier

48105

University of Michigan, Kellogg Eye Center, Ann Arbor

75012

Centre de maladies rares CHNO des Quinze Vingts, Paris

75231

Retina Foundation of the Southwest, Dallas

97239

Casey Eye Institute, Oregon Health & Science University, Portland

02114

Center for Clinical Research Operations, Massachusetts Eye and Ear, Boston

H4A 3J1

Centre for Innovative Medicine, Department of Paediatric Surgery, Montreal Children's Hospital at the McGill University Health Centre, Montreal

Sponsors

Lead Sponsor

ProQR Therapeutics
All Listed Sponsors
lead

ProQR Therapeutics

INDUSTRY

NCT03780257 - Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene | Biotech Hunter | Biotech Hunter