Table 3:
Ongoing clinical trials in ET and PV patients
| Drug | Phase | NCT | Patient characteristics | Intervention |
|---|---|---|---|---|
| Essential thrombocythemia | ||||
| Interferon | III | NCT04285086 | ET patients with resistance or intolerance to HU | Ropeginterferon alfa-2b vs anagrelide |
| II | NCT00452023 | ET and PV | Peg-IFN alfa-2a | |
| IV | NCT04226950 | Children (<18 years) with ET | Peg-IFN alfa-2b vs. IFN alfa | |
| Ruxolitinib | II | NCT03123588 | ET patients with resistance or intolerance to HU | Ruxolitinib + placebo vs anagrelide + placebo |
| II/III | NCT02962388 | ET patients with resistance or intolerance to HU | Ruxolitinib vs best available therapy (anagrelide, IFN alfa/peg-IFN alfa) | |
| II | NCT02577926 | High-risk ET or PV (>60 years or prior thromboembolic event) | Ruxolitinib vs BAT | |
| Navitoclax (BCL2 inhibitor) | I | NCT04041050 | Myeloproliferative neoplasms | Navitoclax alone or in combination with ruxolitinib (only after progression to myelofibrosis) |
| Givinostat (HDAC inhibitor) | II | NCT01761968 | JAK2 V617F-positive Myeloproliferative Neoplasms | Givinostat |
| IMG-7289 (LSD1 inhibitor) | II | NCT04081220 | ET patients with resistance or intolerance to hydroxyurea | LSD1 inhibitor IMG-7289 |
| II | NCT04262141 | ET and PV patients who failed at least one line of therapy | LSD1 inhibitor IMG-7289 | |
| II | NCT04254978 | ET and PV patients who failed at least one line of therapy | LSD1 inhibitor IMG-7289 | |
| Peptide vaccines | I | NCT03566446 | CALR-mutant myeloproliferative neoplasms | CALR Exon 9 mutant peptide vaccine |
| I/II | NCT04051307 | ET and PV | PD-L1 and Aginase1 peptide vaccines | |
| Polycythemia vera | ||||
| Interferon | II | NCT03003325 | Low-risk PV (i.e. age 18–60 years; no prior cardiovascular PV-related event) | Aspirin + phlebotomy +/− ropeginterferon alfa-2b |
| II | NCT04182100 | PV patients with limitations of standard therapy | Aspirin + phlebotomy + ropeginterferon alfa-2b | |
| Ruxolitinib | III | NCT04116502 | High risk PV (WBC >11 × 109/L + age >60 years, prior thrombosis or hemorrhage, or platelet count >1000 × 109/L) | Ruxolitinib vs hydroxyurea or IFN |
| KRT-232 (MDM2 inhibitor) | II | NCT03669965 | Part A: PV patients with/without splenomegaly; resistant or intolerant to HU or prior IFN Part B: PV patients with splenomegaly; resistant or intolerant to HU |
Part A: KRT-232 Part B: KRT-232 or ruxoltinib |
| PTG-300 (hepcidin mimetic) | II | NCT04057040 | Phlebotomy-dependent PV patients | PTG-300 vs placebo |
BAT – best available therapy; BCL2 – B-cell lymphoma 2; CALR – calreticulin; ET – essential thrombocythemia; HDAC – histone deacetylase; HU – hydroxyurea; IFN – interferon; JAK2 – janus kinase 2; LSD1 – lysine-specific demethylase 1A; PD-L1 – programmed death ligand-1; PEG – pegylated; PHR – partial hematologic remission; PV – polycythemia vera