Ongoing clinical trials in PV
| Agent (mechanism of action) . | NCT (trial name if available) . | Phase . | Patient population . |
|---|---|---|---|
| Ruxolitinib (JAK1/2 inhibitor) | NCT04644211 | 2 | Low-risk PV (no history of thrombosis and age <60 y) |
| TGR-1202 (PI3Kδ inhibitor) + ruxolitinib | NCT02493530 | 1 | Patients with MF, MDS/MPN, or PV resistant to hydroxyurea |
| Ruxolitinib vs BAT | NCT02577926 (RUXO-BEAT) | 2 | High-risk PV or ET; no prior cytoreductive therapy permitted |
| Ruxolitinib in combination with either hydroxyurea or IFN-α | NCT04116502 (MITHRIDATE) | 3 | High-risk PV defined as WBC count >11 × 109/L + at least 1 of the following: age >60 y, prior thrombosis or hemorrhage, or platelet count >1000 × 109/L |
| ropeg-IFN-α2b | NCT05485948 | 2 | Patients with PV with intolerance or resistance to hydroxyurea |
| Phlebotomy + aspirin w/wo pegylated proline-IFN- α2b (AOP2014) | NCT03003325 (Low-PV) | 2 | Patients with low-risk PV; prior cytoreductive therapy was not permitted |
| ropeg-IFN-α2b | NCT04655092 | 3 | Long-term extension study in patients with PV previously treated with ropeg-IFN-α2b |
| ropeg-IFN-α2b (randomized between various dosing regimens) | NCT05481151 (ECLIPSE PV) | 3 | Patients with PV; no specifications regarding phlebotomy or prior cytoreductive therapy |
| Rusfertide (PTG-300-08; hepcidin mimetic) | NCT04767802 (PACIFIC) | 2 | Newly diagnosed PV with a baseline hematocrit level >48% |
| Rusfertide vs placebo | NCT05210790 (VERIFY) | 3 | Patients with PV who are phlebotomy dependent |
| Sapablursen (antisense oligonucleotide targeting transmembrane protease serine 6 (TMPRSS6) mRNA) | NCT05143957 | 2 | Patients with PV who are phlebotomy dependent |
| PPMX-T003 (monoclonal antibody against transferrin receptor 1) | NCT05074550 | 1 | Patients with PV who are phlebotomy dependent; cytoreductive therapy not permitted |
| IMG-7289 (LSD1 inhibitor) | NCT04262141 | 2 | Patients with ET or PV with intolerance or resistance to at least 1 line of prior therapy |
| Givinostat (HDAC inhibitor) | NCT01761968 | 2 | Long-term extension study in patients with JAK2 V617F-mutant MPNs previously treated with givinostat |
| Rivaroxaban or apixaban vs aspirin | NCT05198960 (AVAJAK) | 3 | Patients with high-risk PV or ET (aged >60 y or prior thrombotic event) with JAK2 V617F mutation |
| Agent (mechanism of action) . | NCT (trial name if available) . | Phase . | Patient population . |
|---|---|---|---|
| Ruxolitinib (JAK1/2 inhibitor) | NCT04644211 | 2 | Low-risk PV (no history of thrombosis and age <60 y) |
| TGR-1202 (PI3Kδ inhibitor) + ruxolitinib | NCT02493530 | 1 | Patients with MF, MDS/MPN, or PV resistant to hydroxyurea |
| Ruxolitinib vs BAT | NCT02577926 (RUXO-BEAT) | 2 | High-risk PV or ET; no prior cytoreductive therapy permitted |
| Ruxolitinib in combination with either hydroxyurea or IFN-α | NCT04116502 (MITHRIDATE) | 3 | High-risk PV defined as WBC count >11 × 109/L + at least 1 of the following: age >60 y, prior thrombosis or hemorrhage, or platelet count >1000 × 109/L |
| ropeg-IFN-α2b | NCT05485948 | 2 | Patients with PV with intolerance or resistance to hydroxyurea |
| Phlebotomy + aspirin w/wo pegylated proline-IFN- α2b (AOP2014) | NCT03003325 (Low-PV) | 2 | Patients with low-risk PV; prior cytoreductive therapy was not permitted |
| ropeg-IFN-α2b | NCT04655092 | 3 | Long-term extension study in patients with PV previously treated with ropeg-IFN-α2b |
| ropeg-IFN-α2b (randomized between various dosing regimens) | NCT05481151 (ECLIPSE PV) | 3 | Patients with PV; no specifications regarding phlebotomy or prior cytoreductive therapy |
| Rusfertide (PTG-300-08; hepcidin mimetic) | NCT04767802 (PACIFIC) | 2 | Newly diagnosed PV with a baseline hematocrit level >48% |
| Rusfertide vs placebo | NCT05210790 (VERIFY) | 3 | Patients with PV who are phlebotomy dependent |
| Sapablursen (antisense oligonucleotide targeting transmembrane protease serine 6 (TMPRSS6) mRNA) | NCT05143957 | 2 | Patients with PV who are phlebotomy dependent |
| PPMX-T003 (monoclonal antibody against transferrin receptor 1) | NCT05074550 | 1 | Patients with PV who are phlebotomy dependent; cytoreductive therapy not permitted |
| IMG-7289 (LSD1 inhibitor) | NCT04262141 | 2 | Patients with ET or PV with intolerance or resistance to at least 1 line of prior therapy |
| Givinostat (HDAC inhibitor) | NCT01761968 | 2 | Long-term extension study in patients with JAK2 V617F-mutant MPNs previously treated with givinostat |
| Rivaroxaban or apixaban vs aspirin | NCT05198960 (AVAJAK) | 3 | Patients with high-risk PV or ET (aged >60 y or prior thrombotic event) with JAK2 V617F mutation |
ET, essential thrombocythemia; HDAC, histone deacetylase; LSD1, lysine-specific demethylase 1; MDS, myelodysplastic syndrome; MF, myelofibrosis; mRNA, messenger RNA; NCT, National Clinical Trials number; WBC, white blood cell; w/wo, with or without.