Observational Study on CML Patients in Any Phase of the Disease Treated With Ponatinib (Iclusig®)

Observational Study on CML Patients in Any Phase of the Disease Treated With Ponatinib (Iclusig®): A Multicentre, Ambispective Cohort Study

This is a multicentre ambispective cohort study involving French patients who have started or are receiving for less than 6 months a treatment with ponatinib. This study aims at better qualifying the ponatinib benefit-risk balance in real life and in relation with CML patients' therapeutic history.

Study Overview

• Status: Completed
• Conditions: Chronic Myeloid Leukemia

• Study Type: Observational
• Enrollment (Actual): 120

Contacts and Locations

Study Locations

• France
  • Amiens Cedex, France, 80054
    • CHU Amiens-Picardie- Site SUD
  • Angers, France, 49100
    • CHU d'Angers
  • Annecy, France, 74370
    • CH Annecy
  • Argenteuil Cedex, France, 95107
    • Centre Hospitalier Argenteuil
  • Avignon, France, 84000
    • Centre hospitalier d'Avignon
  • Bordeaux, France, 33076
    • Institut Bergonié
  • Béziers, France, 34500
    • Ch Beziers
  • Castelnau-Le-Lez, France, 34170
    • Cabinet D'hematologie De La Clinique Du Parc
  • Chalon-sur-Saône, France, 71321
    • CH William Morey
  • Chambéry, France, 73000
    • CH Chambery
  • Clermont-Ferrand Cedex, France, 63003
    • CHU Estaing Clermont Ferrand
  • Dijon, France, 21000
    • CHU Dijon, François Mitterrand
  • Dunkerque, France, 59240
    • Centre Hospitalier de Dunkerque
  • La Roche-sur-Yon, France, 85000
    • Centre Hospitalier Departemental Vendee
  • Le Chesnay, France, 78157
    • CH De Versailles (Andre Mignot)
  • Le Kremlin-Bicêtre, France, 94270
    • Hôpital Bicêtre
  • Libourne, France, 33505
    • CH de Libourne
  • Lille Cedex, France, 59037
    • CHRU de Lille - Hopital Huriez
  • Limoges Cedex, France, 87042
    • CH Limoges
  • Lyon, France, 69373
    • Leon Berard, Lyon
  • Meaux, France, 77100
    • Centre Hospitalier de Meaux
  • Metz, France, 57000
    • Hopitaux Prives Metz Centre De Belle-Isle
  • Metz, France, 57530
    • CH De Metz (Hopital De Mercy - CHR Metz Thionville)
  • Montpellier, France, 34090
    • CHU Montpellier
  • Paris, France, 75013
    • Hopital Salpetriere
  • Paris, France, 75015
    • Hopital Necker
  • Paris, France, 75013
    • La Pitié Salpêtrière - Paris
  • Perpignan, France, 66000
    • CH St Jean
  • Poitiers, France, 86021
    • Chu de Poitiers
  • Pontoise, France, 95300
    • Hôpital René Dubos
  • Quimper, France, 29107
    • CH de Cornouaille
  • Rennes, France, 35033
    • CHU de Rennes
  • Roubaix, France, 59056
    • Hopital Victor Provo
  • Strasbourg, France, 67000
    • La Clinique Sainte-Anne
  • Strasbourg, France, 67200
    • CHRU Strasbourg
  • Toulouse, France, 31059
    • Institut Universitaire Du Cancer Toulouse - Oncopo
  • Troyes, France, 10000
    • Ch Troyes
  • Vandoeuvre Lès Nancy, France, 54511
    • CHRU de Nancy - Hôpitaux de Brabois
  • Vandoeuvre Lès Nancy, France, 54511
    • CHU Sud, St Pierre - La Réunion
  • Reunion
    • Saint-Pierre, Reunion, France, 97410
      • CHU SUD Reunion GHSR

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

70 public and private French investigative centers of hematology will be recruited.

Description

Inclusion Criteria:

• Presenting a CML in any phase.
• Having initiated for less than six months a treatment with ponatinib.
• The ability to understand the requirements of the study and to comply with the study data collection procedures.

Exclusion Criteria:

• Patients previously treated with investigational ponatinib (within a clinical trial).
• Patients receiving an investigational agent.
• Patients who are pregnant and/or breastfeeding.
• Patients with contraindications for Ponatinib according to Summary of Products Characteristics.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
For participants in chronic myeloid leukemia in accelerated phase (AP-CML) or chronic myeloid leukemia in blast phase (BP-CML): Proportion of participants who achieve a complete hematologic response	CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.	from 24-60 months
For participants in chronic myeloid leukemia in chronic (CP-CML) phase: Proportion of participants who achieve a major molecular response after the initiation of study treatment	Chronic myeloid leukemia (CML) response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia (Hochhaus et al Leukemia 2020).	from 24-60 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of participants in CP-CML phase who achieved complete hematologic response	CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.	from 24-60 months
Proportion of participants in AP and BP phases who achieved major (complete + partial) cytogenetic response	CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.	from 24-60 months
Proportion of participants who achieved major molecular response and/or depth molecular response: (MR4 or MR4.5 or MR5)	CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.	from 24-60 months
Duration of response	Duration of CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.	from 24-60 months
Time to progression to AP-CML or BP-CML (for those participants not in AP-CML or BP-CML)	Time to progression to AP-CML defined as follows: Blasts in blood or marrow 15%-29%, or blasts plus promyelocytes in blood or marrow > 30%, with blasts < 30%; basophils in blood ≥ 20%; persistent thrombocytopenia (< 100 × 10^9/L) unrelated to therapy; clonal chromosome abnormalities in Ph1 cells (CCA/Ph1), major route, on treatment. Time to progression to BP-CML defined as follows: Blasts in blood or marrow ≥ 30%; extramedullary blast proliferation, apart from spleen.	from 24-60 months
Dose reduction (after response) in each cohort	Includes level of response at the time of dose reduction and maintenance of response after dose reduction.	from 24-60 months
Time to response	Time to CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.	from 24-60 months
Rate of progression to accelerated phase (AP-) or blast phase (BP-) CML	Rate of progression to AP- or BP-CML as defined in European LeukemiaNet (ELN) criteria.	from 24-60 months
Progression-free survival (PFS)	Survival without any progression to AP or BP according to ELN criteria.	from 24-60 months
Overall survival (OS)	Overall survival defined according to ELN criteria.	from 24-60 months
Rate of adverse events	Adverse event is any untoward medical occurrence in a patient or clinical study subject administered a medicinal (investigational or non-investigational) product. An adverse event does not necessarily have a causal relationship with the treatment.	from 24-60 months
Rate of discontinuation due to adverse events in each dose cohort	Adverse event is any untoward medical occurrence in a patient or clinical study subject administered a medicinal (investigational or non-investigational) product. An adverse event does not necessarily have a causal relationship with the treatment.	from 24-60 months
Dose reductions (prior to response) in each dose cohort	Reduction in the dose of Iclusig.	from 24-60 months
Dose interruptions in each dose cohort	Interruption of Iclusig treatment.	from 24-60 months

Collaborators and Investigators

• Sponsor: Incyte BioSciences France
• Investigators: Principal Investigator: Ali G. Turnan, MD, PhD, Paris Sud University Hospitals-Bicetre

Study record dates

Study Major Dates

• Study Start (Actual): February 23, 2018
• Primary Completion (Actual): December 31, 2022
• Study Completion (Actual): July 3, 2023

Study Registration Dates

• First Submitted: August 6, 2019
• First Submitted That Met QC Criteria: August 6, 2019
• First Posted (Actual): August 7, 2019

Study Record Updates

• Last Update Posted (Actual): September 28, 2023
• Last Update Submitted That Met QC Criteria: September 26, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Keywords: Chronic myeloid leukemia; Iclusig; ponatinib
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms by Histologic Type; Neoplasms; Disease Attributes; Bone Marrow Diseases; Hematologic Diseases; Myeloproliferative Disorders; Leukemia; Chronic Disease; Leukemia, Myeloid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive
• Other Study ID Numbers: 2017-A01355-48

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No