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

Completed

• Conditions: Chronic Myeloid Leukemia

• Registration Number: NCT04048564
• Lead Sponsor: Incyte BioSciences France

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2018-02-23
• Study First Submitted: 2019-08-06
• Study First Submitted QC: 2019-08-06
• Study First Posted: 2019-08-07
• Primary Completion: 2022-12-31
• Study Completion: 2023-07-03
• Status Verified: 2023-09
• Last Update Submitted: 2023-09-26
• Last Update Posted: 2023-09-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 120

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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	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	from 24-60 months	CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.

Secondary Outcome Measures

Name	Time	Method
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.
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.
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.
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.
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.
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.
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.
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.
Overall survival (OS)	from 24-60 months	Overall survival defined according to ELN criteria.
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.

Trial Locations

Locations (40)

Hopital Bicetre; 🇫🇷 Le Kremlin-Bicêtre, France

CH De Libourne; 🇫🇷 Libourne, France

CHRU De Lille - Hôpital Huriez; 🇫🇷 Lille Cedex, France

CH Limoges; 🇫🇷 Limoges Cedex, France

CHU Amiens-Picardie- Site SUD; 🇫🇷 Amiens Cedex, France

CHU SUD Reunion GHSR; 🇫🇷 Saint-Pierre, Reunion, France

CHU D'Angers; 🇫🇷 Angers, France

CH Annecy; 🇫🇷 Annecy, France

Centre Hospitalier Argenteuil; 🇫🇷 Argenteuil Cedex, France

Institut Bergonie; 🇫🇷 Bordeaux, France

Cabinet D'hematologie De La Clinique Du Parc; 🇫🇷 Castelnau-Le-Lez, France

CH William Morey; 🇫🇷 Chalon-sur-Saône, France

CH Chambery; 🇫🇷 Chambéry, France

CHU Dijon, François Mitterrand; 🇫🇷 Dijon, France

Centre Hospitalier De Dunkerque; 🇫🇷 Dunkerque, France

Centre Hospitalier Departemental Vendee; 🇫🇷 La Roche-sur-Yon, France

CH De Versailles (Andre Mignot); 🇫🇷 Le Chesnay, France

Hopitaux Prives Metz Centre De Belle-Isle; 🇫🇷 Metz, France

CH De Metz (Hopital De Mercy - CHR Metz Thionville); 🇫🇷 Metz, France

CHU Montpellier; 🇫🇷 Montpellier, France

La Pitié Salpêtrière - Paris; 🇫🇷 Paris, France

Hopital Necker; 🇫🇷 Paris, France

CHU De Poitiers; 🇫🇷 Poitiers, France

CH De Cornouaille; 🇫🇷 Quimper, France

CHU De Rennes; 🇫🇷 Rennes, France

Hopital Victor Provo; 🇫🇷 Roubaix, France

La Clinique Sainte-Anne; 🇫🇷 Strasbourg, France

CHU Sud, St Pierre - La Réunion; 🇫🇷 Vandoeuvre Lès Nancy, France

CH Beziers; 🇫🇷 Béziers, France

Centre Hospitalier De Meaux; 🇫🇷 Meaux, France

Hopital Salpetriere; 🇫🇷 Paris, France

CH St Jean; 🇫🇷 Perpignan, France

Hôpital Rene Dubos; 🇫🇷 Pontoise, France

CHRU Strasbourg; 🇫🇷 Strasbourg, France

CH Troyes; 🇫🇷 Troyes, France

Institut Universitaire Du Cancer Toulouse - Oncopo; 🇫🇷 Toulouse, France

CHRU De Nancy - Hôpitaux De Brabois; 🇫🇷 Vandoeuvre Lès Nancy, France

Centre Hospitalier D'Avignon; 🇫🇷 Avignon, France

CHU Estaing Clermont Ferrand; 🇫🇷 Clermont-Ferrand Cedex, France

Leon Berard, Lyon; 🇫🇷 Lyon, France