Chronic Myeloid Leukemia, Chronic PhasePHOENICSNCT06119269
Ponatinib in Chronic Myeloid Leukemia Patients in Chronic Phase
An observational study of Chronic Myeloid Leukemia, Chronic Phase, run by University of Pisa at 5 sites in Italy.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Myeloid Leukemia, Chronic Phase
- What is being tested?
- Therapeutic drug monitoring and Molecular Response
- How many people
- 100 participants
- Where
- 5 sitesItaly
- Study dates
- 2 Aug 2023to 31 Dec 2025
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients diagnosed with CML-CP who received ponatinib as 2nd-4th chemotherapy line will be enrolled according to inclusion/exclusion criteria listed.
Who may be able to join
None listed.
Who cannot join
- 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.
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
Italy5 sites, 5 recruiting
- Recruiting
University of Cagliari - Businco Hospital - Unit of Hematology and Bone Marrow Transplant
Cagliari, 09124
Giovanni Caocci, PhD, +39 070 5296 ext. 2065, giovanni.caocci@unica.it
- Recruiting
Policlinico Milano
Milan, 20122
Alessandra Iurlo, MD, +39 02 5503.1 ext. 3422, alessandra.iurlo@policlinico.mi.it
- Recruiting
University of Naples Federico II - Unit of Hematology
Naples, 80131
Luigia Luciano, MD, +39 081 746 ext. 2165, lulucian@unina.it
- Recruiting
Santa Chiara University Hospital
Pisa, 56126
Sara Galimberti, PhD, +39 050 993111 ext. 3488, sara.galimberti@unipi.it
Claudia Baratè, MD, +39 050 992111, claudia.barate@gmail.com
- Recruiting
Ospedale S. Eugenio ASL 2 Roma
Roma, 00144
Elisabetta Abruzzese, MD, +39 06 51001, elisabetta.abruzzese@uniroma2.it
About this study
Official title: Ponatinib in cHronic myelOid LEukemia patieNts In Chronic phaSe: the PHOENICS Protocol
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:
Read more
- 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.
Read the detailed description
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.
- Conditions
- Chronic Myeloid Leukemia, Chronic Phase
- Being tested
- Therapeutic drug monitoring, Molecular Response
- Keywords
- Chronic myeloid leukemia, chronic phase, Ponatinib, Therapeutic drug monitoring, Efficacy, Tolerability
- Medical subject headings
- Leukemia, Myeloid, Chronic-Phase
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Therapeutic drug monitoringDiagnostic 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 ResponseDiagnostic test
BCR-ABl transcript levels will be assessed to evaluate molecular response to ponatinib
What is being measured
The primary outcome is the main question the study is designed to answer.
Ponatinib plasma concentrations
Plasma concentrations of ponatinib included in the therapeutic range (i.e., >21 nM)
Measured: 2 year
Secondary outcomes (2)
Molecular response
Plasma levels of BCR-ABl transcript
Measured: Up to 2 year
Occurrence of adverse events/adverse drug reactions
Adverse events/Adverse drug reactions observed and registered during ponatinib administration
Measured: Up to 2 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2023
- Study start2 Aug 2023
- Primary completion31 Dec 2025 (estimated)
- Study completion31 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Chronic Myeloid Leukemia, Chronic PhaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Pisa
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Antonello Di Paolo, M.D., Ph.D., University of Pisa
- Investigators
- Sara Galimberti, PhD, Principal investigator, University of Pisa - Dept. Clinical and Experimental Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06119269
- Sponsor's protocol ID
- 23707
- Acronym
- PHOENICS
- Status last verified
- Jun 2025
- First submitted
- 18 Oct 2023
- Last updated
- 5 Jun 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
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.
PubMed 38543276
Study at a glance
From the registry record, last updated by the sponsor on 5 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ponatinib in cHronic myelOid LEukemia patieNts In Chronic phaSe: the PHOENICS Protocol
- ClinicalTrials.gov ID
- NCT06119269
- Acronym / sponsor ID
- PHOENICS / 23707
- Conditions
- Chronic Myeloid Leukemia, Chronic Phase
- Keywords
- Chronic myeloid leukemia, chronic phase, Ponatinib, Therapeutic drug monitoring, Efficacy, Tolerability
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- University of PisaUniversity, hospital or other
- Study start
- 2 Aug 2023 (actual)
- Primary completion
- 31 Dec 2025 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 7 Nov 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients diagnosed with CML-CP who received ponatinib as 2nd-4th chemotherapy line will be enrolled according to inclusion/exclusion criteria listed.
Key inclusion criteria (0 listed)
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Therapeutic drug monitoringAt 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) | Diagnostic test | – |
| Molecular ResponseBCR-ABl transcript levels will be assessed to evaluate molecular response to ponatinib | Diagnostic test | – |
Primary outcome
Ponatinib plasma concentrations
Time frame: 2 year
Plasma concentrations of ponatinib included in the therapeutic range (i.e., >21 nM)
2 secondary outcomes
Molecular response
Time frame: Up to 2 year
Occurrence of adverse events/adverse drug reactions
Time frame: Up to 2 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jun 2025.
- Results publication: PubMed 38543276Galimberti 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.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Sara Galimberti, PhD principal investigator
University of Pisa - Dept. Clinical and Experimental Medicine
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Chronic Myeloid Leukemia, Chronic Phase, run by University of Pisa at 5 sites in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 0 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sara Galimberti, PhD (+39 050 993111 ext. 3488, sara.galimberti@unipi.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Ponatinib plasma concentrations (time frame: 2 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06119269, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jun 2025.