Trials / Terminated
TerminatedNCT05176717
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)
A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss
- Status
- Terminated
- Phase
- Phase 2 / Phase 3
- Study type
- Interventional
- Enrollment
- 5 (actual)
- Sponsor
- Laboratoires Thea · Industry
- Sex
- All
- Age
- 12 Years
- Healthy volunteers
- Not accepted
Summary
The purpose of this study is to evaluate the efficacy 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 with early to moderate vision loss.
Detailed description
The purpose of this study is to evaluate the efficacy 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 with early to moderate vision loss. The below dose levels of QR-421a will be evaluated with the loading dose administered at Day 1 and maintenance dose administered at Month 3 and every 6 months thereafter: 1. Loading dose of 180 μg, maintenance dose of 60 μg 2. Loading dose of 60 μg, maintenance dose of 60 μg Dose levels will include subjects randomized to sham-procedure or treatment with QR-421a. After the study eye has been treated for at least 12 months, treatment of the fellow eye and cross-over of subjects assigned to sham-procedure may be initiated in eligible eyes based on assessment of benefit-risk.
Conditions
- Retinitis Pigmentosa
- Usher Syndrome Type 2
- Deaf Blind
- Retinal Disease
- Eye Diseases
- Eye Diseases, Hereditary
- Eye Disorders Congenital
- Vision Disorders
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | QR-421a | RNA antisense oligonucleotide for intravitreal injection |
| OTHER | Sham-procedure | Sham-procedure (no experimental drug administered) |
Timeline
- Start date
- 2021-12-15
- Primary completion
- 2022-08-02
- Completion
- 2022-08-02
- First posted
- 2022-01-04
- Last updated
- 2024-08-07
- Results posted
- 2023-01-20
Locations
3 sites across 2 countries: United States, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT05176717. Inclusion in this directory is not an endorsement.