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NCT Number: NCT04048564

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

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.

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Key information

Conditions

Chronic Myeloid Leukemia Bone Marrow Diseases Chronic Disease Disease Attributes Hematologic Diseases Hemic and Lymphatic Diseases Leukemia Leukemia, Myelogenous, Chronic, BCR-ABL Positive Leukemia, Myeloid 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

CHU SUD Reunion GHSR, Saint-Pierre, Reunion, France

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Who can participate

Healthy volunteers accepted: No

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

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.

Treatment and study plan

Primary outcomes

  1. 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

    Time frame: from 24-60 months

    Chronic myeloid leukemia (CML) response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia (Hochhaus et al Leukemia 2020).

  2. 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

    Time frame: from 24-60 months

    CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.

Secondary outcomes

  1. Proportion of participants in CP-CML phase who achieved complete hematologic response

    Time frame: from 24-60 months

    CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.

  2. Proportion of participants in AP and BP phases who achieved major (complete + partial) cytogenetic response

    Time frame: from 24-60 months

    CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.

  3. Proportion of participants who achieved major molecular response and/or depth molecular response: (MR4 or MR4.5 or MR5)

    Time frame: from 24-60 months

    CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.

  4. Duration of response

    Time frame: from 24-60 months

    Duration of CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.

  5. Time to progression to AP-CML or BP-CML (for those participants not in AP-CML or BP-CML)

    Time frame: from 24-60 months

    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.

  6. Dose reduction (after response) in each cohort

    Time frame: from 24-60 months

    Includes level of response at the time of dose reduction and maintenance of response after dose reduction.

  7. Time to response

    Time frame: from 24-60 months

    Time to CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.

  8. Rate of progression to accelerated phase (AP-) or blast phase (BP-) CML

    Time frame: from 24-60 months

    Rate of progression to AP- or BP-CML as defined in European LeukemiaNet (ELN) criteria.

  9. Progression-free survival (PFS)

    Time frame: from 24-60 months

    Survival without any progression to AP or BP according to ELN criteria.

  10. Overall survival (OS)

    Time frame: from 24-60 months

    Overall survival defined according to ELN criteria.

  11. Rate of adverse events

    Time frame: from 24-60 months

    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.

  12. Rate of discontinuation due to adverse events in each dose cohort

    Time frame: from 24-60 months

    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.

  13. Dose reductions (prior to response) in each dose cohort

    Time frame: from 24-60 months

    Reduction in the dose of Iclusig.

  14. Dose interruptions in each dose cohort

    Time frame: from 24-60 months

    Interruption of Iclusig treatment.

Sponsors and collaborators

Lead sponsor

Incyte BioSciences France

Industry

Registry information

Official study title

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

Important dates

Study start
2018
Primary completion
2022
Study completion
2023
First posted
Aug 7, 2019
Registry last updated
Sep 28, 2023

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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