NCT07356960

Evaluation of the Efficacy of Ponatinib in Ph+ ALL in the Real-world

Retrospective, Observational, Study on the Evaluation of the Real-world Outcome of Philadelphia-positive Acute Lymphoblastic Leukaemia Patients Treated With Ponatinib as First-line Treatment Under the Italian Law 648/96 (PONA4ALL Ph+)

• Status: Not Yet Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 103 (estimated)
• Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The goal of this retrospective observational study is to learn about the efficacy of ponatinib in Philadelphia-positive ALL (Ph+ ALL) patients in a real-world setting. The main goal of the study is to assess the rate of complete molecular response (CMR) induced by ponatinib in patients treated under the regulations of Law 648/96, outside clinical trials. Patients who were treated with ponatinib as part of their regular medical care and completed the follow-up period will be included in the study.

Detailed description

This is a non-interventional, multicenter, strictly retrospective study including Ph+ ALL patients treated with ponatinib, alone or in combination with other antileukaemic agents, under Law 648/96. According to AIFA guideline 425-2024, all patients who received ponatinib as first-line treatment under Law 648/96 are eligible for inclusion provided that, at the time of enrolment, they are alive and have at least 18 months of retrospective observation available from the start of ponatinib treatment, or have died/lost to follow-up at any time after treatment initiation, regardless of the length of available follow-up.

Conditions

• Acute Lymphobkastic Leukemia
• Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia

Timeline

• Start date: 2026-04-02
• Primary completion: 2029-04-30
• Completion: 2029-06-30
• First posted: 2026-01-21
• Last updated: 2026-01-21