Drugs / QR-421a
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial · 1 met primary | ||||||
| Phase 1/2 | NCT03780257 | Mar 2019 → Oct 2021 | Usher syndrome type 2, retinitis pigmentosa, vision disorder | ProQR Therapeutics | Completed | Met primary |
| Phase 21 trial | ||||||
| Phase 2 | NCT05085964 | Sep 2021 → Oct 2022 | Usher syndrome type 2, retinitis pigmentosa | Laboratoires Thea | Terminated | No outcome recorded |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT05176717 | Dec 2021 → Aug 2022 | Usher syndrome type 2, retinitis pigmentosa, vision disorder | Laboratoires Thea | Terminated | No outcome recorded Stop: Business |
News releases announcing trial results or a regulatory action · 6
| Date | Issuer | Release |
|---|---|---|
| 2021-11-18 | ProQR Therapeutics | Results ProQR Announces Highlights from Analyst Event proqr.com ↗
The Phase 1/2 Stellar trial demonstrated concordant benefit on multiple measures of vision, including visual acuity, visual fields, and the objective measure of optical coherence tomography (OCT) retinal imaging, after a single dose of QR-421a. |
| 2021-03-24 | ProQR Therapeutics | Results ProQR Announces Positive Results from Clinical Trial of QR-421a in Usher Syndrome and Plans to Start Pivotal Trials proqr.com ↗
In the trial, QR-421a demonstrated benefit on multiple measures of vision that moved in concordance, including visual acuity, visual fields, and optical coherence tomography (OCT) retinal imaging, after a single dose. |
| 2020-03-31 | ProQR Therapeutics | Results ProQR Announces Positive Findings From an Interim Analysis in the Phase 1/2 trial of QR-421a for Usher Syndrome and Provides Business Update proqr.com ↗
QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse events |
| 2020-01-30 | ProQR Therapeutics | Regulatory ProQR Receives Rare Pediatric Disease Designation from FDA for QR-421a proqr.com ↗ |
| 2019-01-02 | ProQR Therapeutics | Regulatory ProQR Receives Fast Track Designation from FDA for QR-421a for Usher Syndrome Type 2 proqr.com ↗
ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced that it received Fast Track designation from the Food and Drug Administration (FDA) for QR-421a. |
| 2018-12-04 | ProQR Therapeutics | Regulatory ProQR Announces Clearance of IND to Start Clinical Trial of QR-421a in Usher Syndrome Type 2 Patients proqr.com ↗ |
All press releases naming this drug 16 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | QR-421a | “QR-421a (ultevursen) administered via intravitreal (IVT) injection” NCT05085964 ↗ |
| Known as | ultevursen | “QR-421a (ultevursen) administered via intravitreal (IVT) injection” NCT05085964 ↗ |
| Modality | Antisense oligonucleotide | “RNA antisense oligonucleotide for intravitreal injection” NCT03780257 ↗ |
| Route | Intravitreal | “QR-421a administered via intravitreal injection (IVT)” NCT03780257 ↗ |