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Chronic Myeloid LeukemiaNCT04048564

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

An observational study of Chronic Myeloid Leukemia, run by Incyte BioSciences France at 40 sites in France.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Chronic Myeloid Leukemia
How many people
120 participants
Where
40 sitesFrance
Study dates
23 Feb 2018to 3 Jul 2023
Phase
Observational
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Study population: 70 public and private French investigative centers of hematology will be recruited.

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

40 sites in 1 country.

40 sites in 1 country

France40 sites

  • CHU Amiens-Picardie- Site SUD

    Amiens, 80054

  • CHU D'Angers

    Angers, 49100

  • CH Annecy

    Annecy, 74370

  • Centre Hospitalier Argenteuil

    Argenteuil, 95107

  • Centre Hospitalier D'Avignon

    Avignon, 84000

  • CH Beziers

    Béziers, 34500

  • Institut Bergonie

    Bordeaux, 33076

  • Cabinet D'hematologie De La Clinique Du Parc

    Castelnau-le-Lez, 34170

  • CH William Morey

    Chalon-sur-Saône, 71321

  • CH Chambery

    Chambéry, 73000

  • CHU Estaing Clermont Ferrand

    Clermont-Ferrand, 63003

  • CHU Dijon, François Mitterrand

    Dijon, 21000

  • Centre Hospitalier De Dunkerque

    Dunkirk, 59240

  • Centre Hospitalier Departemental Vendee

    La Roche-sur-Yon, 85000

  • CH De Versailles (Andre Mignot)

    Le Chesnay, 78157

  • Hopital Bicetre

    Le Kremlin-Bicêtre, 94270

  • CH De Libourne

    Libourne, 33505

  • CHRU De Lille - Hôpital Huriez

    Lille, 59037

  • CH Limoges

    Limoges, 87042

  • Leon Berard, Lyon

    Lyon, 69373

  • Centre Hospitalier De Meaux

    Meaux, 77100

  • Hopitaux Prives Metz Centre De Belle-Isle

    Metz, 57000

  • CH De Metz (Hopital De Mercy - CHR Metz Thionville)

    Metz, 57530

  • CHU Montpellier

    Montpellier, 34090

  • Hopital Salpetriere

    Paris, 75013

  • La Pitié Salpêtrière - Paris

    Paris, 75013

  • Hopital Necker

    Paris, 75015

  • CH St Jean

    Perpignan, 66000

  • CHU De Poitiers

    Poitiers, 86021

  • Hôpital Rene Dubos

    Pontoise, 95300

  • CH De Cornouaille

    Quimper, 29107

  • CHU De Rennes

    Rennes, 35033

  • Hopital Victor Provo

    Roubaix, 59056

  • CHU SUD Reunion GHSR

    Saint-Pierre, Reunion, 97410

  • La Clinique Sainte-Anne

    Strasbourg, 67000

  • CHRU Strasbourg

    Strasbourg, 67200

  • Institut Universitaire Du Cancer Toulouse - Oncopo

    Toulouse, 31059

  • CH Troyes

    Troyes, 10000

  • CHRU De Nancy - Hôpitaux De Brabois

    Vandœuvre-lès-Nancy, 54511

  • CHU Sud, St Pierre - La Réunion

    Vandœuvre-lès-Nancy, 54511

Other trials in France

About this study

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

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.

Keywords
Chronic myeloid leukemia, Iclusig, ponatinib
Medical subject headings
Leukemia, Myelogenous, Chronic, BCR-ABL Positive

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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).

    Measured: from 24-60 months

  2. 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.

    Measured: from 24-60 months

Secondary outcomes (14)

  1. 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.

    Measured: from 24-60 months

  2. 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.

    Measured: from 24-60 months

  3. 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.

    Measured: from 24-60 months

  4. Duration of response

    Duration of CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.

    Measured: from 24-60 months

  5. 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.

    Measured: from 24-60 months

  6. Dose reduction (after response) in each cohort

    Includes level of response at the time of dose reduction and maintenance of response after dose reduction.

    Measured: from 24-60 months

  7. Time to response

    Time to CML response criteria as defined by the European LeukemiaNet Recommendations for Management of Chronic Myeloid Leukemia.

    Measured: from 24-60 months

  8. 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.

    Measured: from 24-60 months

  9. Progression-free survival (PFS)

    Survival without any progression to AP or BP according to ELN criteria.

    Measured: from 24-60 months

  10. Overall survival (OS)

    Overall survival defined according to ELN criteria.

    Measured: from 24-60 months

  11. 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.

    Measured: from 24-60 months

  12. 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.

    Measured: from 24-60 months

  13. Dose reductions (prior to response) in each dose cohort

    Reduction in the dose of Iclusig.

    Measured: from 24-60 months

  14. Dose interruptions in each dose cohort

    Interruption of Iclusig treatment.

    Measured: from 24-60 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted7 Aug 2019
  2. Study start23 Feb 2018
  3. Primary completion31 Dec 2022
  4. Study completion3 Jul 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Other
Enrolment
120 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04048564
Sponsor's protocol ID
2017-A01355-48
Status last verified
Sep 2023
First submitted
6 Aug 2019
Last updated
28 Sep 2023
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Huguet F, Guerci-Bresler A, Roth-Guepin G, Cayssials E, Slama B, Santagostino A, Penot A, Quittet P, Cony-Makhoul P, Saad A, Bastie JN, Hacini M, Coiteux V, Uzunov M, Roy L, Le Clech L, Berger M, Agneray AM, Messas E, Etienne G, Turhan A, Nicolini FE, Rousselot P. Clinical outcomes in patients in any phase of CML treated with ponatinib in France-Data from the TOPASE observational study. Br J Haematol. 2024 Dec;205(6):2295-2304. doi: 10.1111/bjh.19819. Epub 2024 Nov 6.

    PubMed 39506529

Questions about this trial

What is this clinical trial studying?

An observational study of Chronic Myeloid Leukemia, run by Incyte BioSciences France at 40 sites in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 28 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 40 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome 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 (time frame: from 24-60 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04048564, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Sep 2023.

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