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

Chronic Myeloid Leukemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04048564
• Other study ID #: 2017-A01355-48
• Status: Completed
• Start date: February 23, 2018
• Est. completion date: July 3, 2023

Study information

• Verified date: September 2023
• Source: Incyte Corporation
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 120
• Est. completion date: July 3, 2023
• Est. primary completion date: December 31, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Chronic Myeloid Leukemia
• Leukemia, Myelogenous, Chronic, BCR-ABL Positive
• Leukemia, Myeloid

Locations

Country	Name	City	State
France	CHU Amiens-Picardie- Site SUD	Amiens Cedex
France	CHU D'Angers	Angers
France	CH Annecy	Annecy
France	Centre Hospitalier Argenteuil	Argenteuil Cedex
France	Centre Hospitalier D'Avignon	Avignon
France	CH Beziers	Béziers
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 Estaing Clermont Ferrand	Clermont-Ferrand Cedex
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	Hopital Bicetre	Le Kremlin-Bicêtre
France	CH De Libourne	Libourne
France	CHRU De Lille - Hôpital Huriez	Lille Cedex
France	CH Limoges	Limoges Cedex
France	Leon Berard, Lyon	Lyon
France	Centre Hospitalier De Meaux	Meaux
France	CH De Metz (Hopital De Mercy - CHR Metz Thionville)	Metz
France	Hopitaux Prives Metz Centre De Belle-Isle	Metz
France	CHU Montpellier	Montpellier
France	Hopital Necker	Paris
France	Hopital Salpetriere	Paris
France	La Pitié Salpêtrière - Paris	Paris
France	CH St Jean	Perpignan
France	CHU De Poitiers	Poitiers
France	Hôpital Rene Dubos	Pontoise
France	CH De Cornouaille	Quimper
France	CHU De Rennes	Rennes
France	Hopital Victor Provo	Roubaix
France	CHU SUD Reunion GHSR	Saint-Pierre	Reunion
France	CHRU Strasbourg	Strasbourg
France	La Clinique Sainte-Anne	Strasbourg
France	Institut Universitaire Du Cancer Toulouse - Oncopo	Toulouse
France	CH Troyes	Troyes
France	CHRU De Nancy - Hôpitaux De Brabois	Vandoeuvre Lès Nancy
France	CHU Sud, St Pierre - La Réunion	Vandoeuvre Lès Nancy

Sponsors (1)

Lead Sponsor	Collaborator
Incyte BioSciences France

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	Progression-free survival (PFS)	Survival without any progression to AP or BP according to ELN criteria.	from 24-60 months
Secondary	Overall survival (OS)	Overall survival defined according to ELN criteria.	from 24-60 months
Secondary	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
Secondary	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
Secondary	Dose reductions (prior to response) in each dose cohort	Reduction in the dose of Iclusig.	from 24-60 months
Secondary	Dose interruptions in each dose cohort	Interruption of Iclusig treatment.	from 24-60 months