Ponatinib in Chronic Myeloid Leukemia Patients in Chronic Phase

NCT06119269 | Recruiting DMC

• 1 other identifier: 23707
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 5

Brief Summary

The goal of this retrospective observational study is to evaluate any possible association between plasma concentrations of ponatinib and its pharmacodynamics (efficacy/tolerability) in patients affected by chronic myeloid leukemia in chronic phase (CML-CP). In particular, the aims of the study will be:

• primary aim: to investigate the relationships (if any) between plasma concentrations and activity/toxicity of ponatinib in a population of CML-CP patients enrolled in several Italian hematological centers;
• secondary aim: to set up an algorithm aimed at helping physicians to improve drug dosing based on several variables (i.e., plasma drug concentrations, tolerability, molecular response to therapy). The study will enroll CML-CP patients who were exposed to ponatinib as second, third or fourth line of chemotherapy.

Conditions

Chronic Myeloid Leukemia, Chronic Phase

Keywords

Chronic myeloid leukemia, chronic phase; Ponatinib; Therapeutic drug monitoring; Efficacy; Tolerability

Outcome Measures

Primary Outcomes (1)

• Ponatinib plasma concentrations

  Plasma concentrations of ponatinib included in the therapeutic range (i.e., \>21 nM)

  2 year

Secondary Outcomes (2)

• Molecular response

  Up to 2 year

• Occurrence of adverse events/adverse drug reactions

  Up to 2 year

Interventions

Therapeutic drug monitoring DIAGNOSTIC_TEST

At each follow up visit, plasma concentrations of ponatinib will be measured and compared with therapeutic range (according to a lower concentration equal to 21 nM)

Molecular Response DIAGNOSTIC_TEST

BCR-ABl transcript levels will be assessed to evaluate molecular response to ponatinib

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

Patients diagnosed with CML-CP who received ponatinib as 2nd-4th chemotherapy line will be enrolled according to inclusion/exclusion criteria listed.

You may qualify if:

• Subjects ≥18 years old affected by CML
• Patients being treated with ponatinib at a dose of 45, 30 or 15 mg/day for more than 14 days
• Patients who have provided informed consent to the study

You may not qualify if:

• Subjects \<18 years old
• Patients who did not provide an informed consent to the study

Sponsors & Collaborators

• University of Pisa: lead

Study Sites (5)

University of Cagliari - Businco Hospital - Unit of Hematology and Bone Marrow Transplant

Cagliari, 09124, Italy

RECRUITING

Policlinico Milano

Milan, 20122, Italy

RECRUITING

University of Naples Federico II - Unit of Hematology

Napoli, 80131, Italy

RECRUITING

Santa Chiara University Hospital

Pisa, 56126, Italy

RECRUITING

Ospedale S. Eugenio ASL 2 Roma

Roma, 00144, Italy

RECRUITING

Related Publications (1)

• Galimberti S, Abruzzese E, Luci G, Barate C, Luciano L, Iurlo A, Caocci G, Morganti R, Stefanelli F, Di Paolo A. A New Algorithm Integrating Molecular Response, Toxicity, and Plasma Level Measures for Ponatinib Dose Choice in Patients Affected by Chronic Myeloid Leukemia. Pharmaceutics. 2024 Mar 11;16(3):383. doi: 10.3390/pharmaceutics16030383.

  PMID: 38543276 RESULT

MeSH Terms

Conditions

Leukemia, Myeloid, Chronic-Phase

Interventions

Drug Monitoring

Condition Hierarchy (Ancestors)

Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Monitoring, Physiologic; Diagnostic Techniques and Procedures; Diagnosis

Study Officials

• Sara Galimberti, PhD

  University of Pisa - Dept. Clinical and Experimental Medicine

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Sara Galimberti, PhD

CONTACT

+39 050 993111; sara.galimberti@unipi.it

Claudia Baratè, MD

CONTACT

+39 050 992111; claudia.barate@gmail.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Pharmacology

Study Record Dates

• First Submitted: October 18, 2023
• First Posted: November 7, 2023
• Study Start: August 2, 2023
• Primary Completion: December 31, 2025
• Study Completion: December 31, 2025
• Last Updated: June 5, 2025

Record last verified: 2025-06

Locations

IT (5)