Ongoing phase 3 trials in JAKi-naïve patients with MF
| Agent Mechanism of action . | Clinical trial characteristics . | Key inclusion criteria . | Primary end point . |
|---|---|---|---|
| Pelabresib (PELA) BET inhibitor | MANIFEST-2 NCT04603495 PELA + RUX vs PBO + RUX (estimated n = 400) PELA 125 mg QD, RUX 10 or 15 mg BID (based on PLT count) | DIPSS Int/high-risk symptomatic MF PLT ≥100 × 109/L Spleen volume ≥450 cm3 | SVR35 at 24 wk |
| Navitoclax (NAVI) BCL-XL/BCL-2 inhibitor | TRANSFORM-1 NCT04472598 NAVI + RUX vs PBO + RUX (estimated n = 230) NAVI 100 or 200 mg QD, RUX 15 or 20 mg BID (both based on PLT count) | DIPSS+ Int-2/high-risk symptomatic MF PLT ≥100 × 109/L Spleen length ≥5 cm below LCM or volume ≥450 cm3 | SVR35 at 24 wk |
| Parsaclisib (PARS) PI3Kδ inhibitor | LIMBER-313 NCT04551066 PARS + RUX vs PBO + RUX (estimated n = 440) PARS 5 mg QD, RUX 5 or 15 mg BID (based on PLT count) | DIPSS Int/high-risk symptomatic MF PLT ≥50 × 109/L Spleen length ≥5 cm below LCM | SVR35 at 24 wk |
| Agent Mechanism of action . | Clinical trial characteristics . | Key inclusion criteria . | Primary end point . |
|---|---|---|---|
| Pelabresib (PELA) BET inhibitor | MANIFEST-2 NCT04603495 PELA + RUX vs PBO + RUX (estimated n = 400) PELA 125 mg QD, RUX 10 or 15 mg BID (based on PLT count) | DIPSS Int/high-risk symptomatic MF PLT ≥100 × 109/L Spleen volume ≥450 cm3 | SVR35 at 24 wk |
| Navitoclax (NAVI) BCL-XL/BCL-2 inhibitor | TRANSFORM-1 NCT04472598 NAVI + RUX vs PBO + RUX (estimated n = 230) NAVI 100 or 200 mg QD, RUX 15 or 20 mg BID (both based on PLT count) | DIPSS+ Int-2/high-risk symptomatic MF PLT ≥100 × 109/L Spleen length ≥5 cm below LCM or volume ≥450 cm3 | SVR35 at 24 wk |
| Parsaclisib (PARS) PI3Kδ inhibitor | LIMBER-313 NCT04551066 PARS + RUX vs PBO + RUX (estimated n = 440) PARS 5 mg QD, RUX 5 or 15 mg BID (based on PLT count) | DIPSS Int/high-risk symptomatic MF PLT ≥50 × 109/L Spleen length ≥5 cm below LCM | SVR35 at 24 wk |
BID, twice daily; DIPSS, Dynamic International Prognostic Scoring System; LCM, left costal margin; MF, myelofibrosis; PBO, placebo; PLT, platelets; QD, once daily; SVR35, spleen volume reduction of 35%.