Cardiovascular Assessment of Ponatinib as Third Line Treatment in Chronic Phase Chronic Myeloid Leukemia (CarPAs)
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ClinicalTrials.gov Identifier: NCT04709731 |
Recruitment Status :
Not yet recruiting
First Posted : January 14, 2021
Last Update Posted : February 3, 2021
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Condition or disease | Intervention/treatment | Phase |
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Chronic Myeloid Leukemia (CML) | Drug: Ponatinib 15mg QD Drug: Ponatinib 30mg QD | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 50 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Cardiovascular Assessment of Ponatinib as Third Line Treatment Option in Chronic Phase Chronic Myeloid Leukemia After Failure of Imatinib and Bosutinib (CarPAs) |
Estimated Study Start Date : | February 1, 2021 |
Estimated Primary Completion Date : | October 31, 2024 |
Estimated Study Completion Date : | April 1, 2025 |

Arm | Intervention/treatment |
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Experimental: Total Patients
Intolerant Group Ponatinib 15 mg tablet, taken orally once daily (QD) Resistant Group Ponatinib 30 mg tablet, taken orally once daily (QD) The dose will be reduced to 15mg once daily (QD) as soon as a Complete Cytogenetic Response will be obtained. In those patients showing Major Molecular Response or better, the dose could be further reduced to 15MG every other day (EOD), due to the prolonged half-life of the drug.
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Drug: Ponatinib 15mg QD
Ponatinib 15 mg tablet, taken orally once daily
Other Names:
Drug: Ponatinib 30mg QD Ponatinib 30 mg tablet, taken orally once daily
Other Names:
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- Exposure adjusted Rate of Arterial Occlusive Events and Serious Arterial Occlusive Events at 1 year after study treatment initiation of each patient [ Time Frame: 1 year ]Exposure adjusted Rate of Arterial Occlusive Events (AOE) and Serious AOE (SOE) at 1 year after study treatment initiation of each patient

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Ages Eligible for Study: | 18 Years to 99 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Signed and dated Informed Consent approved by Local Ethical Committee before any protocol-specific screening procedures.
- CML diagnosis, Chronic Phase (CP), treated with imatinib and bosutinib. Previous treatment with dasatinib or nilotinib will not be allowed.
- Resistant or intolerant to imatinib and/or bosutinib.
- Able to take oral therapy.
- Female or male, 18 years of age or older.
- ECOG performance status 0-2.
- Minimum life expectancy of 3 months or more.
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Adequate organ function as defined by the following criteria:
- Serum aspartate transaminase (AST) and serum alanine transaminase (ALT) ≤ 2.5 x upper limit of normal (ULN) or AST and ALT ≤ 5 x ULN if liver function abnormalities are due to underlying malignancy
- Total serum bilirubin ≤ 1.5 x ULN (except patients with documented Gilbert's syndrome)
- Creatinine ≤ 1.5 x ULN
- Prothrombin time (PT) < 1.5 × ULN
- Lipase ≤ 1.5 × ULN for institution
- Amylase ≤ 1.5 × ULN for institution
- Normal QTcF interval on screening electrocardiogram (ECG) evaluation, defined as QTcF of ≤ 450 ms in males or ≤ 470 ms in females.
- Willingness and ability to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures.
- Female and male patients who are of childbearing potential must agree to use an effective form of contraception (2 forms of contraception) with their partners throughout participation in this study and for at least 90 days after the last dose of treatment.
- For females of childbearing potential, a negative pregnancy test must be documented prior to enrolment.
Exclusion Criteria:
- Current treatment on another therapeutic clinical trial.
- Received TKI therapy within 7 days prior to receiving the first dose of ponatinib, or have not recovered (> grade 1 by NCI CTCAE, v. 4.0) from AEs (except alopecia) due to agents previously administered.
- Underwent autologous or allogeneic stem cell transplant < 60 days prior to receiving the first dose of ponatinib; any evidence of on-going graft versus-host disease (GVHD), or GVHD requiring immunosuppressive therapy.
- Take medications that are known to be associated with Torsades de Pointes.
- Require concurrent treatment with immunosuppressive agents, other than corticosteroids prescribed for a short course of therapy.
- Have previously been treated with ponatinib.
- Have active central nervous system (CNS) disease as evidenced by cytology or pathology. In the absence of clinical CNS disease, lumbar puncture is not required. History itself of CNS involvement is not exclusionary if CNS has been cleared with a documented negative lumbar puncture.
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Have significant or active cardiovascular disease, specifically including, but not restricted to:
- Myocardial infarction within 3 months prior to first dose of ponatinib,
- History of clinically significant atrial arrhythmia or any ventricular arrhythmia,
- Unstable angina within 3 months prior to first dose of ponatinib,
- Congestive heart failure within 3 months prior to first dose of ponatinib.
- Have a significant bleeding disorder unrelated to CML or Ph+ ALL.
- Have a history of pancreatitis or alcohol abuse.
- Have uncontrolled hypertriglyceridemia (triglycerides > 450 mg/dL).
- Have malabsorption syndrome or other gastrointestinal illness that could affect absorption of orally administered ponatinib.
- Have been diagnosed with another primary malignancy within the past 3 years (except for non-melanoma skin cancer or cervical cancer in situ, or controlled prostate cancer, which are allowed within 3 years).
- Pregnancy or breastfeeding.
- Underwent major surgery (with the exception of minor surgical procedures, such as catheter placement or BM biopsy) within 14 days prior to first dose of ponatinib.
- Have ongoing or active infection (including known history of human immunodeficiency virus [HIV], hepatitis B virus [HBV], or hepatitis C virus [HCV]). Testing for these viruses is not required in the absence of history.
- Other severe acute or chronic medical or psychiatric conditions, or laboratory abnormalities that would impart, in the judgment of the investigator and/or sponsor, excess risk associated with study participation or study drug administration.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04709731
Contact: Michaela De Palo | 0283427930 | michaela.depalo@galseq.com, studiclinici@galseq.com | |
Contact: Nicoletta Re | 0258103979 | aip.info@libero.it, re.nicoletta@outlook.com |
Italy | |
Presidio Ospedaliero "Oncologico Businco" - Cagliari (CA) | |
Cagliari, Italy, 09121 | |
Contact: Giovanni Caocci, Prof. giovanni.caocci@unica.it | |
Principal Investigator: Giovanni Caocci | |
AOU "Policlinico Vittorio Emanuele" - Catania (CT) | |
Catania, Italy, 95124 | |
Contact: Fabio Stagno, Dr. fsematol@tiscali.it | |
Principal Investigator: Fabio Stagno | |
Ospedale San Gerardo - Monza (MB) | |
Milano, Italy, 20090 | |
Contact: Carlo Gambacorti, Prof. carlo.gambacorti@unimib.it | |
Principal Investigator: Carlo Gambacorti Passerini | |
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico - Milano (MI) | |
Milano, Italy, 20122 | |
Contact: Alessandra Iurlo, Dr.ssa alessandra.iurlo@policlinico.mi.it | |
Principal Investigator: Alessandra Iurlo | |
Azienda Ospedaliera Universitaria "Federico II" - Napoli (NA) | |
Napoli, Italy, 80131 | |
Contact: Luigia Luciano, Dr.ssa lulucian@unina.it | |
Principal Investigator: Luigia Luciano | |
Fondazione IRCCS Policlinico San Matteo - Pavia (PV) | |
Pavia, Italy, 27100 | |
Contact: Chiara Elena, Dr.ssa chiara.elena1@gmail.com | |
Principal Investigator: Chiara Elena | |
Grande Ospedale Metropolitano "Bianchi-Melacrino-Morelli" - Reggio Calabria (RC) | |
Reggio Calabria, Italy, 89133 | |
Contact: Bruno Martino, Dr. ematologiarc@alice.it | |
Principal Investigator: Bruno Martino | |
AUSL Reggio Emilia (RE) | |
Reggio Emilia, Italy, 42122 | |
Contact: Isabella Capodanno, Dr.ssa capodanno.isabella@ausl.re.it | |
Principal Investigator: Isabella Capodanno | |
ASL Roma 2 "Ospedale S. Eugenio" - Roma (RM) | |
Roma, Italy, 00144 | |
Contact: Elisabetta Abruzzese, Dr.ssa elisabetta.abruzzese@uniroma2.it | |
Principal Investigator: Elisabetta Abruzzese | |
AOU Policlinico Umberto I "Università La Sapienza" - Roma (RM) | |
Roma, Italy, 00161 | |
Contact: Roberto Latagliata, Dr. latagliata@bce.uniroma1.it | |
Principal Investigator: Roberto Latagliata | |
Azienda Ospedaliero-Universitaria Senese - Siena (SI) | |
Siena, Italy, 53100 | |
Contact: Monica Bocchia, Prof.ssa bocchia@unisi.it | |
Principal Investigator: Monica Bocchia | |
AOU Città della Salute e della Scienza - Torino (TO) | |
Torino, Italy, 10126 | |
Contact: Patrizia Pregno, Dr.ssa ppregno@cittadellasalute.to.it | |
Principal Investigator: Patrizia Pregno | |
AOU Integrata Verona "Ospedale Borgo Roma" - Verona (VN) | |
Verona, Italy, 37134 | |
Contact: Massimiliano Bonifacio, Dr. massimiliano.bonifacio@univr.it | |
Principal Investigator: Massimiliano Bonifacio |
Principal Investigator: | Carlo Gambacorti | Ospedale San Gerardo - Monza (MI) |
Responsible Party: | Associazione Italiana Pazienti Leucemia Mieloide Cronica |
ClinicalTrials.gov Identifier: | NCT04709731 |
Other Study ID Numbers: |
AssociazioneIPLMC 2018-001334-18 ( EudraCT Number ) |
First Posted: | January 14, 2021 Key Record Dates |
Last Update Posted: | February 3, 2021 |
Last Verified: | January 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Chronic Myeloid Leukemia Chronic Phase Ponatinib BCR-ABL Positive (BCR-ABL+) |
Philadelphia Chromosome Positive (Ph+) Imatinib and/or Bosutinib Intolerance Imatinib and/or Bosutinib Resistance |
Leukemia Leukemia, Myeloid Leukemia, Myelogenous, Chronic, BCR-ABL Positive Leukemia, Myeloid, Chronic-Phase Neoplasms by Histologic Type Neoplasms Myeloproliferative Disorders |
Bone Marrow Diseases Hematologic Diseases Ponatinib Antineoplastic Agents Protein Kinase Inhibitors Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action |