Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene
Last updated on July 2021Recruitment
- Recruitment Status
- Recruiting
- Estimated Enrollment
- Same as current
Summary
- Conditions
- Deaf Blind
- Eye Diseases
- Eye Diseases Hereditary
- Eye Disorders Congenital
- Retinal Disease
- Retinitis Pigmentosa
- Usher Syndrome Type 2
- Vision Disorders
- Type
- Interventional
- Phase
- Phase 1Phase 2
- Design
- Allocation: RandomizedIntervention Model: Sequential AssignmentMasking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)Primary Purpose: Treatment
Participation Requirements
- Age
- Between 18 years and 125 years
- Gender
- Both males and females
Description
The purpose of this study is to evaluate the safety and tolerability of QR-421a administered via IVT in subjects with RP due to mutations in exon 13 of the USH2A gene. Subjects will receive one single IVT injection of QR-421a or sham-procedure in one eye (subject's worse eye) and will be followed up...
The purpose of this study is to evaluate the safety and tolerability of QR-421a administered via IVT in subjects with RP due to mutations in exon 13 of the USH2A gene. Subjects will receive one single IVT injection of QR-421a or sham-procedure in one eye (subject's worse eye) and will be followed up for 24 months. Three dose levels of QR-421a will be evaluated: 50, 100, and 200 µg. Additional dose levels (eg, 25 or 400 µg) may be evaluated based on ongoing safety and efficacy data monitoring. Initial dose cohorts will include subjects randomized to sham-procedure or treatment with QR-421a. Additional subjects may be allocated to treatment with QR-421a in subsequent or initial dose cohorts.
Tracking Information
- NCT #
- NCT03780257
- Collaborators
- Not Provided
- Investigators
- Study Director: ProQR Medical Monitor ProQR Therapeutics Study Director: ProQR Clinical Trial Manager ProQR Therapeutics
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