Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)

PHASE2/PHASE3TerminatedINTERVENTIONAL
Enrollment

5

Participants

Timeline

Start Date

December 15, 2021

Primary Completion Date

August 2, 2022

Study Completion Date

August 2, 2022

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

Sham-procedure (no experimental drug administered)

Trial Locations (3)

53705

University of Wisconsin - Madison, Madison

75231

Retina Foundation of the Southwest, Dallas

Unknown

Moorfields Eye Hospital, London

Sponsors

Lead Sponsor

All Listed Sponsors
collaborator

Sepul Bio

INDUSTRY

lead

Laboratoires Thea

INDUSTRY