Table 1.
Ponatinib’s preclinical trials on CML and Ph+ ALL patients
| Phase | Clinical trial study | Patient criteria | Status | Outcome | Trial number |
|---|---|---|---|---|---|
| I | Safety Study of AP24534 to Treat Chronic Myelogenous Leukemia (CML) and Other Haematological Malignancies | Patients with resistant/refractory hematologic malignancies (n=81) | Completed | • Significant and durable responses were observed in heavily pretreated chronic-phase CML patients regardless of mutation status, and ponatinib was generally well tolerated • 72% of patients showed a major cytogenic response |
NCT0066092072 |
| II | Ponatinib Ph-Positive Acute Lymphoblastic Leukemia [ALL] and CML Evaluation (PACE) trial | Chronic-phase CML, accelerated-phase CML, blast-phase CML, or Ph+ ALL with resistance to or unacceptable side effects from dasatinib or nilotinib or with the T315I mutation (n=449) | Results obtained and ongoing | • Ponatinib had significant antileukemic activity across categories of disease stage and mutation status • 83% of patients showed a major cytogenic response |
NCT0120744019,73 |
| II | Ponatinib in Participants With Resistant Chronic Phase Chronic Myeloid Leukemia (CP-CML) to Characterize the Efficacy and Safety of a Range of Doses (OPTIC) | Patients with resistant CML in chronic phase and have received at least two prior TKI therapies or acquiring T315I mutation after receiving any number of prior TKI therapies (estimated n=276) | Recruiting | • Ongoing | NCT02467270 |
| N/a | Post-Marketing Surveillance of Safety and Effectiveness of Iclusig Tablets in Korean Patients with CML or Ph+ ALL under the “Risk Management Plan” | Observational study for patients with CML or Ph+ ALL (estimated n=50) | Recruiting | • Ongoing | NCT03709017 |
| N/a | Retrospective Evaluation of CML Patients in the National Compassionate Program | CML patients who are Ph+ and have received either dasatinib or nilotinib and are resistant or intolerant to the drugs or have developed the T315I mutation (estimated n=80) | Recruiting | • Ongoing | NCT02448095 |
| N/a | Iclusig (Ponatinib) in Clinical Practice for the Treatment of Chronic Myeloid Leukemia or Ph+ Acute Lymphoblastic Leukemia in Belgium | CML or Ph+ ALL patients who are resistant or intolerant to dasatinib or nilotinib, and for whom subsequent treatment with imatinib is not clinically appropriate, or who have the T315I mutation (estimated n=125) | Recruiting | • Ongoing | NCT03678454 |
| II | Activity and Risk Profile of Ponatinib in Chronic Phase Patients with Chronic Myeloid Leukemia Resistant to Imatinib | Patients with CML who are in a chronic phase and are resistant to imatinib (estimated n=78) | Recruiting | • Ongoing | NCT02398825 |
| N/a | An Observational Registry to Evaluate the Incidence of and Risk Factors for Vascular Occlusive Events Associated with Iclusig | Safety profile of patients with CML or Ph+ ALL treated with Iclusig (ponatinib) in routine clinical practice in the USA (estimated n=300) | Recruiting | • Ongoing | NCT02455024 |
| III | A Study Comparing Ponatinib and Nilotinib in Patients With Chronic Myeloid Leukemia (OPTIC-2L) | Chronic-phase CML patients who are resistant to imatinib and have received no other TKI (n=44) | Active, not recruiting | • Ongoing | NCT02627677 |
| II | Ponatinib for Chronic Myeloid Leukemia Evaluation and Ph+ Acute Lymphoblastic Leukemia | Patients with CML or Ph+ ALL who are resistant or intolerant to either dasatinib or nilotinib or have the T315I mutation (n=449) | Active, not recruiting | • Ongoing | NCT01207440 |
Abbreviations: ALL, acute lymphoblastic leukemia; CML, chronic myeloid leukemia; Ph+, Philadelphia chromosome-positive; TKI, tyrosine kinase inhibitor.