Observational Study on CML Patients in Any Phase of the Disease Treated With Ponatinib (Iclusig®): A Multicentre, Ambispective Cohort Study
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Incyte BioSciences France
- Enrollment
- 120
- Locations
- 40
- Primary Endpoint
- 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
Study Overview
Brief Summary
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 Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Other
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
Outcomes
Primary Outcomes
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).
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
- Proportion of participants in CP-CML phase who achieved complete hematologic response(from 24-60 months)
- Proportion of participants in AP and BP phases who achieved major (complete + partial) cytogenetic response(from 24-60 months)
- Duration of response(from 24-60 months)
- Proportion of participants who achieved major molecular response and/or depth molecular response: (MR4 or MR4.5 or MR5)(from 24-60 months)
- Dose reduction (after response) in each cohort(from 24-60 months)
- Time to response(from 24-60 months)
- Time to progression to AP-CML or BP-CML (for those participants not in AP-CML or BP-CML)(from 24-60 months)
- Rate of progression to accelerated phase (AP-) or blast phase (BP-) CML(from 24-60 months)
- Progression-free survival (PFS)(from 24-60 months)
- Rate of adverse events(from 24-60 months)
- Dose reductions (prior to response) in each dose cohort(from 24-60 months)
- Dose interruptions in each dose cohort(from 24-60 months)
- Overall survival (OS)(from 24-60 months)
- Rate of discontinuation due to adverse events in each dose cohort(from 24-60 months)