Drugs / RP-L301
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT04105166 | Jul 2020 → Jun 2025 | pyruvate kinase deficiency of red cells | Rocket Pharmaceuticals Inc. | Completed | No outcome recorded |
| Phase 21 trial | ||||||
| Phase 2 | NCT06422351 | Apr 2026 → Jan 2029 | pyruvate kinase deficiency of red cells | Rocket Pharmaceuticals Inc. | Suspended | No outcome recorded |
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | RP-L301 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04105166 ↗ |
| Action | Restore | “with the goal of correcting the hematological manifestations of the disease” NCT04105166 ↗ |
| Route | Intravenous | “will be infused intravenously” NCT04105166 ↗ |
| Target | PKLR | “carrying a correct copy of the deficient PKLR (Pyruvate Kinase L/R) gene” NCT06422351 ↗ |