Retro-prospective Observational Study on Risk of Progression in CP-CML Patients Eligible for TKI Discontinuation

NCT ID: NCT04621851

Last Updated: 2025-07-29

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 260 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2020-09-30
• Study Completion Date: 2023-08-14

Brief Summary

The purpose of this study is to investigate the safety profile of TKI discontinuation in clinical practice, with particular regard on the risk of progression after treatment discontinuation.

Detailed Description

This study will enroll approximately 3000 CP-CML patients that must have a history of at least 4 years of TKI treatment and at least 18 months of DMR. Events developing in patients after the end of discontinuation and TKI resumption will be considered as linked to the discontinuation if they will develop within 36 months from the end of discontinuation. This rule will apply also to subsequent TD attempts. In case of a second or subsequent discontinuation attempt after the failure of a previous one (for molecular relapse), patients must have re-achieved a DMR with TKI therapy resumption and must keep DMR for at least 18 months before another TD.

Collection of data will be retrospective and prospective, as each center will collect the data for 24 months. Patients who discontinued before the opening of this study will contribute to the retrospective cohort, while those who will discontinue after it will contribute to the prospective cohort. Patients who discontinued before the opening of this study but will continue their discontinuation after it, will contribute to both cohorts. For patients prospectively recruited, monitoring of disease status will be performed to assess the maintenance of the molecular remission during the study period.

Patients with an atypical BCR-ABL1 fusion gene, which does not allow the use of Q-RT-PCR, will be monitored by qualitative PCR and will be analyzed separately. For these patients, negativity of nested qualitative RT-PCR will be considered a surrogate of DMR of patients monitored by Q-RT-PCR, while loss of negativity of first-round qualitative PCR will be considered a surrogate of loss of MMR (i.e. molecular relapse). Accordingly, for patients monitored by qualitative PCR, TKI resumption after TD will be provided in case of a new positivity of first-round PCR.

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Conditions

Chronic Myeloid Leukemia

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: OTHER

Study Groups

Retrospective cohort

Patients who discontinued before the opening of this study will contribute to the retrospective cohort.

No interventions assigned to this group

Prospective cohort

Patients who will discontinue after it will contribute to the prospective cohort.

No interventions assigned to this group

Retrospective/Prospective cohort

Patients who discontinued before the opening of this study but will continue their discontinuation after it, will contribute to both cohorts.

No interventions assigned to this group

Eligibility Criteria

Inclusion Criteria

1. Signed and dated IRB/IEC-approved informed consent for the prospective cohort patients.

2. Age >= 18 years.

3. Male or female patients with CML diagnosed in chronic phase (CP).

4. At least 4 years of TKI treatment.

5. At least 18 months of DMR.

Exclusion Criteria

• Allogeneic hematopoietic stem cell transplantation.
• CML diagnosed in AP or BC

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Milano Bicocca

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Elisabetta Abruzzese, MD

Role: PRINCIPAL_INVESTIGATOR

Ospedale S. Eugenio Roma

Vincenzo Accurso, MD

Role: PRINCIPAL_INVESTIGATOR

A.U. Policlinico "Paolo Giaccone" Palermo

Mario Annunziata, MD

Role: PRINCIPAL_INVESTIGATOR

Azienda Ospedaliera di Rilievo Nazionale "A. Cardarelli" Napoli

Francesco Passamonti, MD

Role: PRINCIPAL_INVESTIGATOR

Ospedale di Circolo e Fondazione Macchi Varese, ASST dei Sette Laghi Varese

Massimo Bonifacio, MD

Role: PRINCIPAL_INVESTIGATOR

Istituti Ospitalieri di Verona- Policlinico G.B. Rossi Verona

Giovanni Caocci, MD

Role: PRINCIPAL_INVESTIGATOR

CTMO - Ospedale "Businco" Cagliari

Francesca Lunghi, MD

Role: PRINCIPAL_INVESTIGATOR

Ospedale Milano S. Raffaele Milano

Chiara Elena, MD

Role: PRINCIPAL_INVESTIGATOR

Fondazione IRCCS Policlinico San Matteo di Pavia

Monica Crugnola, MD

Role: PRINCIPAL_INVESTIGATOR

Az Ospedaliera Universitaria Parma

Sara Galimberti, MD

Role: PRINCIPAL_INVESTIGATOR

Azienda Ospedaliera Pisana Pisa

Alessandra Iurlo, MD

Role: PRINCIPAL_INVESTIGATOR

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano Milano

Luciano Levato, MD

Role: PRINCIPAL_INVESTIGATOR

Az. Ospedaliera Pugliese - Ciaccio (AOPC) Catanzaro

Maria Cristina Miggiano, MD

Role: PRINCIPAL_INVESTIGATOR

Azienda ULSS 8 "Berica" Ospedale San Bortolo Vicenza

Patrizia Pregno, MD

Role: PRINCIPAL_INVESTIGATOR

A.O. Città della Salute e della Scienza di Torino S. G.Battista Torino

Davide Rapezzi, MD

Role: PRINCIPAL_INVESTIGATOR

Ospedale Cuneo

Rosaria Sancetta, MD

Role: PRINCIPAL_INVESTIGATOR

Ospedale dell'Angelo Mestre Venezia

Fabio Stagno, MD

Role: PRINCIPAL_INVESTIGATOR

P.O. Gaspare Rodolico, Catania

Luigia Luciano, MD

Role: PRINCIPAL_INVESTIGATOR

Azienda Ospedaliera Universitaria-Università degli Studi di Napoli "Federico II"

Carmen Fava, MD

Role: PRINCIPAL_INVESTIGATOR

A.S.O. Ordine Mauriziano, P.O. Umberto I Torino

Philipp leCoutre, MD

Role: PRINCIPAL_INVESTIGATOR

Charité University of Berlin · Medical Department, Division of Oncology and Hematology

Susanne Saussele, MD

Role: PRINCIPAL_INVESTIGATOR

University of Mannheim

Sarit Assouline, MD

Role: PRINCIPAL_INVESTIGATOR

Jewish General Hospital

Alberto Álvarez-Larrán, MD

Role: PRINCIPAL_INVESTIGATOR

University Hospital Clínic de Barcelona

Locations

McGill University - Jewish General Hospital Division of Hematology and Department of Oncology

Montreal, Quebec, Canada

Charité University of Berlin - Clinic of Medicine - Hematology and Oncology

Berlin, , Germany

University of Mannheim, Mannheim, Germania

Mannheim, , Germany

ASST-Monza

Monza, Italy/MB, Italy

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico UOC di Ematologia

Milan, Italy/Milano, Italy

Universita di Tor Vergata Ospedale S. Eugenio

Rome, Italy/Rome, Italy

Istituto di Ematologia "Lorenzo e A. Seragnoli" Policlinico S. Orsola Malpighi,

Bologna, , Italy

CTMO Ematologia Ospedale "Businco"

Cagliari, , Italy

Università di Catania Cattedra di Ematologia Ospedale "Ferrarotto"

Catania, , Italy

SOC Ematologia Az. Ospedaliera Pugliese Ciaccio (AOPC)

Catanzaro, , Italy

Ematologia Ospedale Cuneo

Cuneo, , Italy

UO Ematologia O spedale Milano S. Raffaele

Miano, , Italy

Azienda Ospedaliera di Rilievo Nazionale "A. Cardarelli"

Napoli, , Italy

Azienda Ospedaliera Universitaria Università degli Studi di Napoli "Federico II" Facoltà di Medicina e Chirurgia

Napoli, , Italy

U.O. di Ematologia con trapianto A.U. Policlinico "Paolo Giaccone"

Palermo, , Italy

Unità operativa Ematologia e CTMO Az Ospedaliera Universitaria

Parma, , Italy

Fondazione IRCCS Policlinico San Matteo

Pavia, , Italy

Università di Pisa Azienda Ospedaliera Pisana Divisione di Ematologia

Pisa, , Italy

Azienda Unità Sanitaria Locale IRCCS

Reggio Emilia, , Italy

Dipartimento di Oncologia ed Ematologia S.C. Ematologia 2 A.O. Città della Salute e della Scienza di Torino S. G.Battista

Torino, , Italy

Struttura Complessa a Dir. Universitaria Ematologia e Terapie Cellulari A.S.O. Ordine Mauriziano, P.O. U mberto I

Torino, , Italy

S.C. Ematologia, Ospedale di Circolo e Fondazione Macchi Varese, ASST dei Sette Laghi,

Varese, , Italy

U.O. di Ematologia Ospedale dell'Angelo Mestre

Venezia, , Italy

Istituti Ospitalieri di Verona Div. di Ematologia Policlinico G.B. Rossi

Verona, , Italy

U.O. Complessa di Ematologia Azienda ULSS 8 "Berica" Ospedale San Bortolo

Vicenza, , Italy

University Hospital Clínic de Barcelona

Barcelona, , Spain

Countries

Canada; Germany; Italy; Spain

Provided Documents

Document Type: Study Protocol

View Document

Other Identifiers

TFR-PRO

Identifier Type: -

Identifier Source: org_study_id