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

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT07356960

Acronym

PONA4ALL Ph+

Enrollment

103

Registered

2026-01-21

Start date

2026-04-02

Completion date

2029-06-30

Last updated

2026-01-21

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Acute Lymphobkastic Leukemia, Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia

Keywords

ponatinib, real-world, philadelphia-positive acute lymphoblastic leukemia, Ph+ ALL, adult

Brief 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.

Interventions

None listed

Study design

Observational model

CASE_ONLY

Time perspective

RETROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
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

Contacts

CONTACTPaola Fazi

p.fazi@gimema.it0039 0670390528

CONTACTEnrico Crea

e.crea@gimema.it0039 0670390514

Outcome results