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

NCT07356960 | Not Yet Recruiting

• 1 other identifier: ALL3125
• Study Type: observational
• Target: 103
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Acute Lymphobkastic Leukemia; Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Efficacy of ponatinib in terms of complete molecular response (CMR) achievement

  Rate of patients treated with ponatinb in first-line who achieve complete molecular response (CMR) after induction

  3 months

Study Arms (1)

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.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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.

You may qualify if:

• Patient is \> 18 years old;
• Patient was treated in first-line with ponatinib monotherapy or in association with chemotherapy or immunotherapy;
• Patient received ponatinib under the regulations of Law 648/96;
• \- 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;
• Signed informed consent, if applicable.

You may not qualify if:

• None

Sponsors & Collaborators

• Gruppo Italiano Malattie EMatologiche dell'Adulto: lead

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Condition Hierarchy (Ancestors)

Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Central Study Contacts

Paola Fazi

CONTACT

0039 0670390528; p.fazi@gimema.it

Enrico Crea

CONTACT

0039 0670390514; e.crea@gimema.it

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 12, 2026
• First Posted: January 21, 2026
• Study Start: April 2, 2026
• Primary Completion (Estimated): April 30, 2029
• Study Completion (Estimated): June 30, 2029
• Last Updated: January 21, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share