Table 3.
Ongoing clinical trials for asciminib.
| Phase | Study title | Treatment | Target population | Primary endpoint |
|---|---|---|---|---|
| 3 First-line (NCT05456191) | A phase IIIb, open-label, randomized study of tolerability and efficacy of asciminib versus nilotinib in patients with newly diagnosed philadelphia chromosome-positive chronic myelogenous leukemia in chronic phase (ASC4START) | Asciminib 80 mg qd vs. nilotinib 300 mg bid | Newly diagnosed CP-CML | Time to discontinuation of study treatment due to an adverse event (TTDAE) |
| 3 First-line (NCT04971226) | A phase III, multicenter, open-label, randomized study of oral asciminib versus investigator selected TKI in patients with newly diagnosed philadelphia chromosome positive chronic myelogenous leukemia in chronic phase | Asciminib 80 mg qd vs. investigator-selected TKIs (imatinib, nilotinib, dasatinib, or bosutinib) | Newly diagnosed CP-CML | MMR at week 48 |
| 3b Third-line (NCT04948333) | A phase 3b, multicenter, open-label, treatment optimization study of oral asciminib in patients with chronic myelogenous leukemia in chronic phase (CML-CP) previously treated with 2 or more tyrosine kinase inhibitors | Asciminib 40 mg bid or 80 mg qd, with possible dose escalation upto 200 mg qd | CP-CML with warning or resistance criteria based on ELN 2020 recommendations | MMR rate at week 48 |
| 3b Third-line (NCT04666259) | An open label, multicenter phase IIIb study of asciminib (ABL001) monotherapy in previously treated patients with chronic myeloid leukemia in chronic phase (CML-CP) with and without T315I mutation | Asciminib 40 mg bid, 80 mg qd, or 200 mg bid | CP-CML failed ≥2 prior TKIs (no T315I) or 1 prior TKI (T315I) | Number of AE and SAE up to 24 weeks |
| 3 TFR (NCT05413915) | A phase 3, multicenter, randomized, open-label, trial evaluating the efficacy and safety of asciminib used in consolidation with imatinib vs. imatinib to achieve treatment-free remission in chronic phase-chronic myelogenous leukemia patients | Consolidation with asciminib 60 mg qd for 52 weeks added to imatinib vs. imatinib for 52 weeks followed by TFR | CP-CML in DMR (≥MR4) for at least 12 months with imatinib | EFS at 12 months |
| 2 First-line (NCT05143840) | Asciminib as initial therapy for patients with chronic myeloid leukemia in chronic phase | Asciminib 40 mg bid (nilotinib added for patients not achieving a response after 24 months of treatment) | Newly diagnosed CP-CML | MMR at week 24 |
| 2 Second-line (NCT05384587) | A phase II multicenter, open-label, single-arm dose escalation study of asciminib monotherapy in 2nd line chronic phase - chronic myelogenous leukemia (ASC2ESCALATE) | Asciminib 80 mg qd with possible dose escalation upto 200 mg bid | CP-CML failed 1L-TKI without T315I mutation | MMR at 12 months |
| 2 DMR (NCT03578367) | A phase 2, multicenter, open-label, randomized study of oral asciminib added to imatinib vs. continued imatinib versus switch to nilotinib in patients with CML-CP who have been previously treated with imatinib and have not achieved deep molecular response | Asciminib 60 mg qd+imatinib 400 mg qd vs. asciminib 40 mg qd+imatinib 400 mg qd vs. imatinib 400 mg qd vs. nilotinib 300 mg bid vs. asciminib 80 mg qd | CP-CML with BCR::ABL1 >0.01% and ≤0.1% receiving 1L-imatinib >12 months | MR4.5 at week 48 |
| 2 DMR (NCT04216563) | Phase II study of dual targeting of BCR::ABL1 by adding the allosteric inhibitor ABL001 in patients with chronic myeloid leukemia (CML) and minimal residual disease (MRD) while on therapy with tyrosine kinase inhibitors | Asciminib bid for up to 36 months while receiving standard-of-care dasatinib or nilotinib | CP-CML in CCyR but detectable BCR::ABL1 transcript on TKIs ≥24 months | Molecular response rate at 12 months |
| 2 TFR (NCT03874858) | A phase II, single-arm study of de-escalation and treatment-free remission in patients with chronic myeloid leukemia treated with nilotinib in first-line therapy followed by a second attempt after nilotinib and asciminib combination: DANTE Study | (Second stage) Asciminib 40 mg bid and nilotinib 300 mg bid for patients failed a first TFR attempt with nilotinib consolidation | CP-CML, failed the first TFR attempt, followed by nilotinib retreatment ≥1 yr and MR4 or better | TFR rate 48 weeks after the second TFR attempt |