Ignite Creation Date:
2025-12-24 @ 10:47 PM
Ignite Modification Date:
2025-12-25 @ 8:17 PM
Study NCT ID:
NCT06119269
Status:
RECRUITING
Last Update Posted:
2025-06-05
First Post:
2023-10-18
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Ponatinib in Chronic Myeloid Leukemia Patients in Chronic Phase
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D015466', 'term': 'Leukemia, Myeloid, Chronic-Phase'}], 'ancestors': [{'id': 'D015464', 'term': 'Leukemia, Myelogenous, Chronic, BCR-ABL Positive'}, {'id': 'D007951', 'term': 'Leukemia, Myeloid'}, {'id': 'D007938', 'term': 'Leukemia'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D009196', 'term': 'Myeloproliferative Disorders'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D002908', 'term': 'Chronic Disease'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D016903', 'term': 'Drug Monitoring'}], 'ancestors': [{'id': 'D008991', 'term': 'Monitoring, Physiologic'}, {'id': 'D019937', 'term': 'Diagnostic Techniques and Procedures'}, {'id': 'D003933', 'term': 'Diagnosis'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'RETROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 100}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2023-08-02', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-06', 'completionDateStruct': {'date': '2025-12-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-06-02', 'studyFirstSubmitDate': '2023-10-18', 'studyFirstSubmitQcDate': '2023-11-03', 'lastUpdatePostDateStruct': {'date': '2025-06-05', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-11-07', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-12-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Ponatinib plasma concentrations', 'timeFrame': '2 year', 'description': 'Plasma concentrations of ponatinib included in the therapeutic range (i.e., \\>21 nM)'}], 'secondaryOutcomes': [{'measure': 'Molecular response', 'timeFrame': 'Up to 2 year', 'description': 'Plasma levels of BCR-ABl transcript'}, {'measure': 'Occurrence of adverse events/adverse drug reactions', 'timeFrame': 'Up to 2 year', 'description': 'Adverse events/Adverse drug reactions observed and registered during ponatinib administration'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Chronic myeloid leukemia, chronic phase', 'Ponatinib', 'Therapeutic drug monitoring', 'Efficacy', 'Tolerability'], 'conditions': ['Chronic Myeloid Leukemia, Chronic Phase']}, 'referencesModule': {'references': [{'pmid': '38543276', 'type': 'RESULT', 'citation': '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.'}]}, 'descriptionModule': {'briefSummary': '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).\n\nIn particular, the aims of the study will be:\n\n* 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;\n* 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).\n\nThe study will enroll CML-CP patients who were exposed to ponatinib as second, third or fourth line of chemotherapy.', 'detailedDescription': "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.\n\nClassical 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.\n\nNotably, 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."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Patients diagnosed with CML-CP who received ponatinib as 2nd-4th chemotherapy line will be enrolled according to inclusion/exclusion criteria listed.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Patients will be enrolled according the following inclusion criteria:\n\n* Subjects ≥18 years old affected by CML\n* Patients being treated with ponatinib at a dose of 45, 30 or 15 mg/day for more than 14 days\n* Patients who have provided informed consent to the study\n\nExclusion criteria:\n\n* Subjects \\<18 years old\n* Patients who did not provide an informed consent to the study\n\nThe 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.'}, 'identificationModule': {'nctId': 'NCT06119269', 'acronym': 'PHOENICS', 'briefTitle': 'Ponatinib in Chronic Myeloid Leukemia Patients in Chronic Phase', 'organization': {'class': 'OTHER', 'fullName': 'University of Pisa'}, 'officialTitle': 'Ponatinib in cHronic myelOid LEukemia patieNts In Chronic phaSe: the PHOENICS Protocol', 'orgStudyIdInfo': {'id': '23707'}}, 'armsInterventionsModule': {'interventions': [{'name': 'Therapeutic drug monitoring', 'type': 'DIAGNOSTIC_TEST', 'description': '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)'}, {'name': 'Molecular Response', 'type': 'DIAGNOSTIC_TEST', 'description': 'BCR-ABl transcript levels will be assessed to evaluate molecular response to ponatinib'}]}, 'contactsLocationsModule': {'locations': [{'zip': '09124', 'city': 'Cagliari', 'status': 'RECRUITING', 'country': 'Italy', 'contacts': [{'name': 'Giovanni Caocci, PhD', 'role': 'CONTACT', 'email': 'giovanni.caocci@unica.it', 'phone': '+39 070 5296', 'phoneExt': '2065'}], 'facility': 'University of Cagliari - Businco Hospital - Unit of Hematology and Bone Marrow Transplant', 'geoPoint': {'lat': 39.23054, 'lon': 9.11917}}, {'zip': '20122', 'city': 'Milan', 'status': 'RECRUITING', 'country': 'Italy', 'contacts': [{'name': 'Alessandra Iurlo, MD', 'role': 'CONTACT', 'email': 'alessandra.iurlo@policlinico.mi.it', 'phone': '+39 02 5503.1', 'phoneExt': '3422'}], 'facility': 'Policlinico Milano', 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'zip': '80131', 'city': 'Napoli', 'status': 'RECRUITING', 'country': 'Italy', 'contacts': [{'name': 'Luigia Luciano, MD', 'role': 'CONTACT', 'email': 'lulucian@unina.it', 'phone': '+39 081 746', 'phoneExt': '2165'}], 'facility': 'University of Naples Federico II - Unit of Hematology', 'geoPoint': {'lat': 40.87618, 'lon': 14.5195}}, {'zip': '56126', 'city': 'Pisa', 'status': 'RECRUITING', 'country': 'Italy', 'contacts': [{'name': 'Sara Galimberti, PhD', 'role': 'CONTACT', 'email': 'sara.galimberti@unipi.it', 'phone': '+39 050 993111', 'phoneExt': '3488'}, {'name': 'Claudia Baratè, MD', 'role': 'CONTACT', 'email': 'claudia.barate@gmail.com', 'phone': '+39 050 992111'}, {'name': 'Federica Ricci, MD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Marinunzia Franciosa, MD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Ignazio Santo, MD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Antonello Di Paolo, MD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Francesca Guerrini, BiolD', 'role': 'SUB_INVESTIGATOR'}], 'facility': 'Santa Chiara University Hospital', 'geoPoint': {'lat': 43.70853, 'lon': 10.4036}}, {'zip': '00144', 'city': 'Roma', 'status': 'RECRUITING', 'country': 'Italy', 'contacts': [{'name': 'Elisabetta Abruzzese, MD', 'role': 'CONTACT', 'email': 'elisabetta.abruzzese@uniroma2.it', 'phone': '+39 06 51001'}], 'facility': 'Ospedale S. Eugenio ASL 2 Roma', 'geoPoint': {'lat': 44.99364, 'lon': 11.10642}}], 'centralContacts': [{'name': 'Sara Galimberti, PhD', 'role': 'CONTACT', 'email': 'sara.galimberti@unipi.it', 'phone': '+39 050 993111', 'phoneExt': '3488'}, {'name': 'Claudia Baratè, MD', 'role': 'CONTACT', 'email': 'claudia.barate@gmail.com', 'phone': '+39 050 992111'}], 'overallOfficials': [{'name': 'Sara Galimberti, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Pisa - Dept. Clinical and Experimental Medicine'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Pisa', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor of Pharmacology', 'investigatorFullName': 'Antonello Di Paolo, M.D., Ph.D.', 'investigatorAffiliation': 'University of Pisa'}}}}