Approval Probability
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QR-1123 · 1 trial · 8 indications
Incidence and severity of ocular adverse events scored based on CTCAC in the study and fellow eye
Incidence and severity of non-ocular adverse events scored based on CTCAC in the study and fellow eye
| Arm | Type | Description |
|---|---|---|
| QR-1123 Single dose - dose level 1 | EXPERIMENTAL | Open label Single dose cohort: dose level 1 |
| QR-1123 Single dose - dose level 2 | EXPERIMENTAL | Open label Single dose cohort: dose level |
| QR-1123 Single dose - dose level 3 | EXPERIMENTAL | Open label Single dose cohort: dose level 3 |
| QR-1123 Single dose - dose level 4 | EXPERIMENTAL | Open label Single dose cohort: dose level 4 |
| QR-1123 Single dose - dose level 5 | EXPERIMENTAL | Open label Single dose cohort: dose level 5 |
| Repeat dose cohort 1 | EXPERIMENTAL | Double-masked, randomized, sham controlled, Repeat dose cohort. Dose levels will be determined following DMC review of obtained safety and efficacy data. |
| Name | Type | Description |
|---|---|---|
| QR-1123 | DRUG | unilateral IVT injection |
| Sham procedure | OTHER | Sham procedures (i.e. no penetration of the globe) closely mimic the active injection and serve to mask subjects to treatment assignment |
Main Inclusion Criteria: 1. Male or female, ≥ 18 years of age. 2. Clinical presentation consistent with adRP, based on ophthalmic examinations. 3. Impairment on VF in the opinion of the Investigator, as determined by perimetry. 4. A molecular diagnosis of autosomal dominant form of RP with the P23H...
QR-1123 is an investigational small molecule being developed for the treatment of Autosomal Dominant Retinitis Pigmentosa, a hereditary retinal disease that causes progressive vision loss. It is specifically being studied in patients with the P23H mutation in the RHO gene. The drug is administered to adults aged 18 years and older.
QR-1123 targets the RHO gene, which encodes rhodopsin, a light-sensitive protein in the retina. The drug is designed to address the P23H mutation in the RHO gene, which is a cause of Autosomal Dominant Retinitis Pigmentosa. By targeting this specific genetic mutation, QR-1123 aims to treat the underlying cause of the disease.
QR-1123 is being developed by ProQR Therapeutics N.V., a biopharmaceutical company listed on the NASDAQ under the ticker symbol PRQR. The company is conducting clinical research on this investigational drug for the treatment of Autosomal Dominant Retinitis Pigmentosa.
QR-1123 is currently in Phase 1 clinical development. It is an investigational drug that has not yet been approved by regulatory authorities. The ongoing Phase 1 study is evaluating the safety and tolerability of QR-1123 in patients with Autosomal Dominant Retinitis Pigmentosa.
QR-1123 is being studied in a Phase 1 clinical trial registered as NCT04123626. This study is evaluating the safety and tolerability of QR-1123 in subjects with Autosomal Dominant Retinitis Pigmentosa due to the P23H mutation in the RHO gene. The trial is active but not recruiting, with an enrollment of 11 participants in the United States.
QR-1123 is a small molecule, not a gene therapy. It is being developed as an investigational treatment for Autosomal Dominant Retinitis Pigmentosa caused by the P23H mutation in the RHO gene. The drug is currently in Phase 1 clinical trials to assess its safety and tolerability.