Clinical Trials Directory

Trials / Completed

CompletedNCT03780257

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

Status
Completed
Phase
Phase 1 / Phase 2
Study type
Interventional
Enrollment
20 (actual)
Sponsor
ProQR Therapeutics · Industry
Sex
All
Age
18 Years
Healthy volunteers
Not accepted

Summary

The purpose of this study is to evaluate the safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.

Detailed 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 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.

Conditions

Interventions

TypeNameDescription
DRUGQR-421aRNA antisense oligonucleotide for intravitreal injection
OTHERSham-procedure (dose cohort 1&2 only)Sham-procedure (no experimental drug administered)

Timeline

Start date
2019-03-06
Primary completion
2021-10-14
Completion
2021-10-14
First posted
2018-12-19
Last updated
2022-04-20

Locations

7 sites across 3 countries: United States, Canada, France

Regulatory

Source: ClinicalTrials.gov record NCT03780257. Inclusion in this directory is not an endorsement.