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

NCT07356960 — Not yet recruiting, 103 participants, Gruppo Italiano Malattie EMatologiche dell'Adulto

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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+)

Registry	NCT07356960, acronym PONA4ALL Ph+
Status	Not yet recruiting
Study type	Observational
Enrollment	103 (Estimated)
Sponsor	Gruppo Italiano Malattie EMatologiche dell'Adulto (Other)
Conditions	Acute Lymphobkastic Leukemia, Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia
Dates	start 2026-04-02 (estimated), primary completion 2029-04-30 (estimated), completion 2029-06-30 (estimated), first posted 2026-01-21, last updated 2026-01-21

-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.

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+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.

+Arms and interventions

Arm	Type	Interventions	Description
Patients treated with ponatinib in first line			Patients treated with ponatinib as first line, alone or in combination with other antileukaemic agents, under Law 648/96.

+Outcome measures

Primary outcomes

Efficacy of ponatinib in terms of complete molecular response (CMR) achievement [3 months]: Rate of patients treated with ponatinb in first-line who achieve complete molecular response (CMR) after induction

+Eligibility

Sex: All, ages: 18 Years to any

Inclusion Criteria:

1. Patient is > 18 years old;
2. Patient was treated in first-line with ponatinib monotherapy or in association with chemotherapy or immunotherapy;
3. Patient received ponatinib under the regulations of Law 648/96;
4. \- Patient is alive and have at least 18 months of retrospective observation available from the start of ponatinib treatment, OR

   \- has died/lost to follow-up at any time after treatment initiation, regardless of the length of available follow-up;
5. Signed informed consent, if applicable.

Exclusion Criteria:

* None