Observational Study on CML Patients in Any Phase of the Disease Treated With Ponatinib (Iclusig®)
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
|
Study Design
Outcome Measures
Primary Outcome Measures
- For participants in chronic myeloid leukemia in chronic phase (CP-CML): Proportion of participants who achieve a complete or partial cytogenetic response after the initiation of study treatment [from 24-60 months]
Chronic myeloid leukemia (CML) response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia (Baccarani et al. Blood 2013).
- 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 [from 24-60 months]
CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.
Secondary Outcome Measures
- Proportion of participants in CP-CML phase who achieved complete hematologic response [from 24-60 months]
CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.
- Proportion of participants in AP and BP phases who achieved major (complete + partial) cytogenetic response [from 24-60 months]
CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.
- Proportion of participants who achieved major molecular response and/or depth molecular response: (MR4 or MR4.5 or MR5) [from 24-60 months]
CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.
- Duration of response [from 24-60 months]
Duration of CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.
- Time to progression to AP-CML or BP-CML (for those participants not in AP-CML or BP-CML) [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.
- Dose reduction (after response) in each cohort [from 24-60 months]
Includes level of response at the time of dose reduction and maintenance of response after dose reduction.
- Time to response [from 24-60 months]
Time to CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.
- Rate of progression to accelerated phase (AP-) or blast phase (BP-) CML [from 24-60 months]
Rate of progression to AP- or BP-CML as defined in European LeukemiaNet (ELN) criteria.
- Progression-free survival (PFS) [from 24-60 months]
Survival without any progression to AP or BP according to ELN criteria.
- Overall survival (OS) [from 24-60 months]
Overall survival defined according to ELN criteria.
- Rate of adverse events [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.
- Rate of discontinuation due to adverse events in each dose cohort [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.
- Dose reductions (prior to response) in each dose cohort [from 24-60 months]
Reduction in the dose of Iclusig.
- Dose interruptions in each dose cohort [from 24-60 months]
Interruption of Iclusig treatment.
Eligibility Criteria
Criteria
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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | CHU SUD Reunion GHSR | Saint-Pierre | Reunion | France | 97410 |
| 2 | CHU Amiens-Picardie- Site SUD | Amiens Cedex | France | 80054 | |
| 3 | CHU D'Angers | Angers | France | 49100 | |
| 4 | CH Annecy | Annecy | France | 74370 | |
| 5 | Centre Hospitalier Argenteuil | Argenteuil Cedex | France | 95107 | |
| 6 | Centre Hospitalier D'Avignon | Avignon | France | 84000 | |
| 7 | Institut Bergonie | Bordeaux | France | 33076 | |
| 8 | CH Beziers | Béziers | France | 34500 | |
| 9 | Cabinet D'hematologie De La Clinique Du Parc | Castelnau-Le-Lez | France | 34170 | |
| 10 | CH William Morey | Chalon-sur-Saône | France | 71321 | |
| 11 | CH Chambery | Chambéry | France | 73000 | |
| 12 | CHU Estaing Clermont Ferrand | Clermont-Ferrand Cedex | France | 63003 | |
| 13 | CHU Dijon, François Mitterrand | Dijon | France | 21000 | |
| 14 | Centre Hospitalier De Dunkerque | Dunkerque | France | 59240 | |
| 15 | Centre Hospitalier Departemental Vendee | La Roche-sur-Yon | France | 85000 | |
| 16 | CH De Versailles (Andre Mignot) | Le Chesnay | France | 78157 | |
| 17 | Hopital Bicetre | Le Kremlin-Bicêtre | France | 94270 | |
| 18 | CH De Libourne | Libourne | France | 33505 | |
| 19 | CHRU De Lille - Hôpital Huriez | Lille Cedex | France | 59037 | |
| 20 | CH Limoges | Limoges Cedex | France | 87042 | |
| 21 | Leon Berard, Lyon | Lyon | France | 69373 | |
| 22 | Centre Hospitalier De Meaux | Meaux | France | 77100 | |
| 23 | Hopitaux Prives Metz Centre De Belle-Isle | Metz | France | 57000 | |
| 24 | CH De Metz (Hopital De Mercy - CHR Metz Thionville) | Metz | France | 57530 | |
| 25 | CHU Montpellier | Montpellier | France | 34090 | |
| 26 | Hopital Salpetriere | Paris | France | 75013 | |
| 27 | Pitie Salpetriere | Paris | France | 75013 | |
| 28 | Hopital Necker | Paris | France | 75015 | |
| 29 | CH St Jean | Perpignan | France | 66000 | |
| 30 | CHU De Poitiers | Poitiers | France | 86021 | |
| 31 | Hôpital Rene Dubos | Pontoise | France | 95300 | |
| 32 | CH De Cornouaille | Quimper | France | 29107 | |
| 33 | CHU De Rennes | Rennes | France | 35033 | |
| 34 | Hopital Victor Provo | Roubaix | France | 59056 | |
| 35 | La Clinique Sainte-Anne | Strasbourg | France | 67000 | |
| 36 | CHRU Strasbourg | Strasbourg | France | 67200 | |
| 37 | Institut Universitaire Du Cancer Toulouse - Oncopo | Toulouse | France | 31059 | |
| 38 | CH Troyes | Troyes | France | 10000 | |
| 39 | CHRU De Nancy - Hôpitaux De Brabois | Vandoeuvre Lès Nancy | France | 54511 | |
| 40 | CHU SUD Reunion GHSR | Vandoeuvre Lès Nancy | France | 54511 |
Sponsors and Collaborators
- Incyte BioSciences France
Investigators
- Principal Investigator: Ali G. Turnan, MD, PhD, Paris Sud University Hospitals-Bicetre
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 2017-A01355-48