Drugs / ELUFORSEN
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT02564354 | Sep 2015 → Sep 2016 | cystic fibrosis | ProQR Therapeutics | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT02532764 | Jun 2015 → Sep 2017 | cystic fibrosis | ProQR Therapeutics | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 4
| Date | Issuer | Release |
|---|---|---|
| 2017-09-25 | ProQR Therapeutics | Results ProQR Announces Positive Top-Line Results from a Phase 1b Study of QR-010 in Subjects with Cystic Fibrosis proqr.com ↗ |
| 2017-06-08 | ProQR Therapeutics | Results ProQR to present QR-010 data at the European Cystic Fibrosis Society Conference and provides an update on the ongoing Phase 1b trial proqr.com ↗
The company also released preliminary data from the ongoing Phase 1b study, demonstrating safety and systemic uptake of QR-010 after a single dose through inhalation. |
| 2016-10-27 | ProQR Therapeutics | Results ProQR Announces that QR-010 Meets the Primary Endpoint in a Proof of Concept Study of Homozygous F508del Cystic Fibrosis Patients proqr.com ↗
The study met its primary endpoint in this cohort as measured by a change in total chloride response following 4 weeks of treatment with QR-010. |
| 2016-07-19 | ProQR Therapeutics | Regulatory ProQR Receives Fast Track Designation from FDA for QR-010 for Cystic Fibrosis proqr.com ↗ |
All press releases naming this drug 11 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | ELUFORSEN | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594587 ↗ |
| Known as | QR-010 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02564354 ↗ |
| Action | Restore | “restoration of CFTR function” NCT02564354 ↗ |
| Modality | Antisense oligonucleotide | “Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton” NCT02532764 ↗ |
| Route | Intranasal | “intranasal administration of QR-010” NCT02564354 ↗ |
| Target | CFTR | “increase the function of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)” NCT02564354 ↗ |