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NCT Number: NCT06119269

Ponatinib in Chronic Myeloid Leukemia Patients in Chronic Phase

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.

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Key information

Conditions

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

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

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

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About this study

The study protocol is a non-interventional, retrospective observational study which involves the collection of data from medical records relating to the plasma dosages of ponatinib performed as per clinical practice, all carried out at the pharmacokinetics laboratory of the Unit of Clinical Pharmacology and Pharmacogenetics of Azienda Ospedaliero Universitaria Pisana (AOUP). These data will be collected in CRF and will be correlated to efficacy data (extent of molecular response) and toxicity of ponatinib (haematological, extra-haematological adverse events, together with adverse event severity). Real-life experience (dosage adjustments made by clinicians based on plasma dosage) will be used to propose an algorithm that can help in choosing the correct dosage of ponatinib.

Classical statistical analyses (for descriptive and comparison aims) will be used according to their appropriateness. Moreover, factors affecting the pharmacokinetics (PK) of ponatinib will be investigated by nonlinear mixed effect modeling adopting the MONOLIX 2021R2 suite. Final model will be used as parte of the dosing algorithm.

Notably, all clinical and laboratory data will be anonymized to protect patients' privacy, and harvested in a database that will be used to perform pharmacometric and statistical analyses.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Patients will be enrolled according the following inclusion criteria:

  • 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

Exclusion criteria

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

The co-administration of drugs other than ponatinib will not be considered an exclusion criterium, but all of the drugs will be registered (together with daily doses and duration of treatment) as possible factors of ponatinib PK variability.

Treatment and study plan

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

Primary outcomes

  1. Ponatinib plasma concentrations

    Time frame: 2 year

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

Secondary outcomes

  1. Molecular response

    Time frame: Up to 2 year

    Plasma levels of BCR-ABl transcript

  2. Occurrence of adverse events/adverse drug reactions

    Time frame: Up to 2 year

    Adverse events/Adverse drug reactions observed and registered during ponatinib administration

Study contacts

Contact information is provided by the study sponsor or research team.

Claudia Baratè, MD

CONTACT

[email protected]

+39 050 992111

Sara Galimberti, PhD

CONTACT

[email protected]

+39 050 993111 ext. 3488

Sponsors and collaborators

Lead sponsor

University of Pisa

Other

Registry information

Official study title

Ponatinib in cHronic myelOid LEukemia patieNts In Chronic phaSe: the PHOENICS Protocol

Acronym: PHOENICS

Important dates

Study start
2023
Primary completion
2025
Study completion
2025
First posted
Nov 7, 2023
Registry last updated
Jun 5, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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