Clinical trial NCT04048564: Observational Study on CML Patients in Any Phase of the Disease Treated With Ponatinib (Iclusig®)
Official title: Observational Study on CML Patients in Any Phase of the Disease Treated With Ponatinib (Iclusig®): A Multicentre, Ambispective Cohort Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Chronic Myeloid Leukemia
Keywords: Chronic myeloid leukemia
Iclusig
ponatinib
Lead sponsor: Incyte Corporation
Sponsor class: INDUSTRY
First posted: 2019-08-07
Enrollment: 120
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 70 public and private French investigative centers of hematology will be recruited.

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.


Eligibility 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.

Primary outcomes:
measure: 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
timeFrame: from 24-60 months
measure: 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
timeFrame: from 24-60 months
Secondary outcomes:
measure: Proportion of participants in CP-CML phase who achieved complete hematologic response
timeFrame: from 24-60 months
measure: Proportion of participants in AP and BP phases who achieved major (complete + partial) cytogenetic response
timeFrame: from 24-60 months
measure: Proportion of participants who achieved major molecular response and/or depth molecular response: (MR4 or MR4.5 or MR5)
timeFrame: from 24-60 months
measure: Duration of response
timeFrame: from 24-60 months
measure: Time to progression to AP-CML or BP-CML (for those participants not in AP-CML or BP-CML)
timeFrame: from 24-60 months
measure: Dose reduction (after response) in each cohort
timeFrame: from 24-60 months
measure: Time to response
timeFrame: from 24-60 months
measure: Rate of progression to accelerated phase (AP-) or blast phase (BP-) CML
timeFrame: from 24-60 months
measure: Progression-free survival (PFS)
timeFrame: from 24-60 months
measure: Overall survival (OS)
timeFrame: from 24-60 months
measure: Rate of adverse events
timeFrame: from 24-60 months
measure: Rate of discontinuation due to adverse events in each dose cohort
timeFrame: from 24-60 months
measure: Dose reductions (prior to response) in each dose cohort
timeFrame: from 24-60 months
measure: Dose interruptions in each dose cohort
timeFrame: from 24-60 months
Other outcomes:
Locations:
city: Saint-Pierre
country: France
facility: CHU SUD Reunion GHSR
state: Reunion
city: Amiens Cedex
country: France
facility: CHU Amiens-Picardie- Site SUD
city: Angers
country: France
facility: CHU D'Angers
city: Annecy
country: France
facility: CH Annecy
city: Argenteuil Cedex
country: France
facility: Centre Hospitalier Argenteuil
city: Avignon
country: France
facility: Centre Hospitalier D'Avignon
city: Bordeaux
country: France
facility: Institut Bergonie
city: Béziers
country: France
facility: CH Beziers
city: Castelnau-Le-Lez
country: France
facility: Cabinet D'hematologie De La Clinique Du Parc
city: Chalon-sur-Saône
country: France
facility: CH William Morey
city: Chambéry
country: France
facility: CH Chambery
city: Clermont-Ferrand Cedex
country: France
facility: CHU Estaing Clermont Ferrand
city: Dijon
country: France
facility: CHU Dijon, François Mitterrand
city: Dunkerque
country: France
facility: Centre Hospitalier De Dunkerque
city: La Roche-sur-Yon
country: France
facility: Centre Hospitalier Departemental Vendee
city: Le Chesnay
country: France
facility: CH De Versailles (Andre Mignot)
city: Le Kremlin-Bicêtre
country: France
facility: Hopital Bicetre
city: Libourne
country: France
facility: CH De Libourne
city: Lille Cedex
country: France
facility: CHRU De Lille - Hôpital Huriez
city: Limoges Cedex
country: France
facility: CH Limoges
city: Lyon
country: France
facility: Leon Berard, Lyon
city: Meaux
country: France
facility: Centre Hospitalier De Meaux
city: Metz
country: France
facility: Hopitaux Prives Metz Centre De Belle-Isle
city: Metz
country: France
facility: CH De Metz (Hopital De Mercy - CHR Metz Thionville)
city: Montpellier
country: France
facility: CHU Montpellier
city: Paris
country: France
facility: Hopital Salpetriere
city: Paris
country: France
facility: La Pitié Salpêtrière - Paris
city: Paris
country: France
facility: Hopital Necker
city: Perpignan
country: France
facility: CH St Jean
city: Poitiers
country: France
facility: CHU De Poitiers
city: Pontoise
country: France
facility: Hôpital Rene Dubos
city: Quimper
country: France
facility: CH De Cornouaille
city: Rennes
country: France
facility: CHU De Rennes
city: Roubaix
country: France
facility: Hopital Victor Provo
city: Strasbourg
country: France
facility: La Clinique Sainte-Anne
city: Strasbourg
country: France
facility: CHRU Strasbourg
city: Toulouse
country: France
facility: Institut Universitaire Du Cancer Toulouse - Oncopo
city: Troyes
country: France
facility: CH Troyes
city: Vandoeuvre Lès Nancy
country: France
facility: CHRU De Nancy - Hôpitaux De Brabois
city: Vandoeuvre Lès Nancy
country: France
facility: CHU Sud, St Pierre - La Réunion
Study officials:
affiliation: Paris Sud University Hospitals-Bicetre
name: Ali G. Turnan, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: