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Ponatinib Plus Chemotherapy in Acute Lymphoblastic Leukemia Patients

NCT05306301 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 32

Last updated 2026-03-06

No results posted yet for this study

Summary

Acute lymphoblastic leukemia (ALL) is the most frequent cancer in children, decreases in adolescence and adulthood, and a second peak can be recorded starting from the 6th decade of life. While the outcome in children is excellent, in the adolescent/adult population, the prognosis, though improved over the decades, it is still unsatisfactory and novel biologically-driven approaches are urgently needed. In this setting, thanks to the introduction of genome wide technologies, it was possible to recognize specific subset of ALL. Among those, the BCR/ABL1-like ALL are of extreme importance, since they are characterized by an unfavourable outcome and, on the other hand, can benefit of a targeted treatment, in particular with the pan-tyrosine kinase inhibitor ponatinib.

The primary objective is to evaluate the clinical response - in terms of MRD negativity - in patients with a BCR/ABL1-like profile, according to the BCR/ABL1-like predictor tool, treated with Ponatinib in combination with chemotherapy.

Conditions

  • Chemotherapy
  • Leukemia, Acute Lymphoblastic

Interventions

DRUG

Ponatinib

Ponatinib is a novel, synthetic, orally-active TKI discovered using a computational and structure based drug design approach. Ponatinib was specifically designed to inhibit all clinically relevant variants of BCR-ABL1, including the T315I mutant (15-17). In vitro assays have demonstrated that Ponatinib potently inhibits the kinase enzymatic activity of the T315I ABL kinase domain, as well as that of the native (unmutated) enzyme. In leukemia cell lines expressing these BCR-ABL1 variants, Ponatinib potently inhibited BCR-ABL1 signaling, leading to the reduction of cellular proliferation and induction of apoptosis. Ponatinib also inhibits the proliferation of cell lines expressing other major clinically-observed Imatinib-resistant mutants of BCR-ABL1.

Sponsors & Collaborators

  • Gruppo Italiano Malattie EMatologiche dell'Adulto

    lead OTHER

Principal Investigators

  • Sabina Chiaretti · Ematologia - Policlinico Umberto I di Roma

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-10-05
Primary Completion
2026-03-01
Completion
2028-07-01
FDA Drug
Yes

Countries

  • Italy

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05306301 on ClinicalTrials.gov