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

NCT04621851 | Completed

• 1 other identifier: TFR-PRO
• Study Type: observational
• Target: 260
• Locations: 4 countries
• Sites: 26

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.

Conditions

Chronic Myeloid Leukemia

Outcome Measures

Primary Outcomes (1)

• The quantification of the risk of progression

  To quantify the risk of progression to accelerated phase (AP) or blast phase (BP), expressed as time adjusted rate (TAR), after TKI discontinuation in CML patients who undergo a first or subsequent TKI discontinuation attempt

  36 Month

Secondary Outcomes (5)

• To compare the time adjusted rate (TAR) of progression from Chronic phase-Chronic Myeloid Leukemia to Accelerated phase (AP) or Blastic phase (BP) by using the percentage of blasts, promyelocytes, basophils or platelet in blood or bone marrow

  36 Month

• Progression free survival (PFS) after TKI discontinuation.

  36 Month

• Rate of molecular relapse (loss of MR3 or MMR)

  36 Month

• Relapse free survival (RFS) after TKI discontinuation.

  36 Month

• Percentage of relapsed patients who obtain a new deep molecular response (DMR) within 6-12 months of treatment resumption among all patients who restart TKI treatment because of a molecular relapse after TKI discontinuation.

  36 Month

Study Arms (3)

Retrospective cohort

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

Prospective cohort

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

Retrospective/Prospective cohort

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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

The target population will include all CML patients presently eligible for treatment discontinuation independently of whether they discontinued or not. For the time they did not discontinue they will contribute to the reference cohort, while after TD they will contribute data to the discontinuation cohort. In case of treatment resumption, the patient will still contribute to the discontinuation cohort for 36 months after treatment resumption, while he/she will contribute to the reference cohort thereafter, assuming a new TD did not occur.

You may qualify if:

• Signed and dated IRB/IEC-approved informed consent for the prospective cohort patients.
• Age \>= 18 years.
• Male or female patients with CML diagnosed in chronic phase (CP).
• At least 4 years of TKI treatment.
• At least 18 months of DMR.

You may not qualify if:

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

Sponsors & Collaborators

• University of Milano Bicocca: lead

Study Sites (26)

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

Montreal, Quebec, H3T 1E2, Canada

Location

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

Berlin, 13353, Germany

Location

University of Mannheim, Mannheim, Germania

Mannheim, Germany

Location

ASST-Monza

Monza, Italy/MB, 20900, Italy

Location

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico UOC di Ematologia

Milan, Italy/Milano, 20162, Italy

Location

Universita di Tor Vergata Ospedale S. Eugenio

Rome, Italy/Rome, 00142, Italy

Location

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

Bologna, Italy

Location

CTMO Ematologia Ospedale "Businco"

Cagliari, Italy

Location

Università di Catania Cattedra di Ematologia Ospedale "Ferrarotto"

Catania, Italy

Location

SOC Ematologia Az. Ospedaliera Pugliese Ciaccio (AOPC)

Catanzaro, Italy

Location

Ematologia Ospedale Cuneo

Cuneo, Italy

Location

UO Ematologia O spedale Milano S. Raffaele

Miano, Italy

Location

Azienda Ospedaliera di Rilievo Nazionale "A. Cardarelli"

Napoli, Italy

Location

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

Napoli, Italy

Location

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

Palermo, Italy

Location

Unità operativa Ematologia e CTMO Az Ospedaliera Universitaria

Parma, Italy

Location

Fondazione IRCCS Policlinico San Matteo

Pavia, Italy

Location

Università di Pisa Azienda Ospedaliera Pisana Divisione di Ematologia

Pisa, Italy

Location

Azienda Unità Sanitaria Locale IRCCS

Reggio Emilia, Italy

Location

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

Torino, Italy

Location

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

Torino, Italy

Location

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

Varese, Italy

Location

U.O. di Ematologia Ospedale dell'Angelo Mestre

Venezia, Italy

Location

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

Verona, Italy

Location

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

Vicenza, Italy

Location

University Hospital Clínic de Barcelona

Barcelona, Spain

Location

MeSH Terms

Conditions

Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Elisabetta Abruzzese, MD

  Ospedale S. Eugenio Roma

  PRINCIPAL INVESTIGATOR

• Vincenzo Accurso, MD

  A.U. Policlinico "Paolo Giaccone" Palermo

  PRINCIPAL INVESTIGATOR

• Mario Annunziata, MD

  Azienda Ospedaliera di Rilievo Nazionale "A. Cardarelli" Napoli

  PRINCIPAL INVESTIGATOR

• Francesco Passamonti, MD

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

  PRINCIPAL INVESTIGATOR

• Massimo Bonifacio, MD

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

  PRINCIPAL INVESTIGATOR

• Giovanni Caocci, MD

  CTMO - Ospedale "Businco" Cagliari

  PRINCIPAL INVESTIGATOR

• Francesca Lunghi, MD

  Ospedale Milano S. Raffaele Milano

  PRINCIPAL INVESTIGATOR

• Chiara Elena, MD

  Fondazione IRCCS Policlinico San Matteo di Pavia

  PRINCIPAL INVESTIGATOR

• Monica Crugnola, MD

  Az Ospedaliera Universitaria Parma

  PRINCIPAL INVESTIGATOR

• Sara Galimberti, MD

  Azienda Ospedaliera Pisana Pisa

  PRINCIPAL INVESTIGATOR

• Alessandra Iurlo, MD

  Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano Milano

  PRINCIPAL INVESTIGATOR

• Luciano Levato, MD

  Az. Ospedaliera Pugliese - Ciaccio (AOPC) Catanzaro

  PRINCIPAL INVESTIGATOR

• Maria Cristina Miggiano, MD

  Azienda ULSS 8 "Berica" Ospedale San Bortolo Vicenza

  PRINCIPAL INVESTIGATOR

• Patrizia Pregno, MD

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

  PRINCIPAL INVESTIGATOR

• Davide Rapezzi, MD

  Ospedale Cuneo

  PRINCIPAL INVESTIGATOR

• Rosaria Sancetta, MD

  Ospedale dell'Angelo Mestre Venezia

  PRINCIPAL INVESTIGATOR

• Fabio Stagno, MD

  P.O. Gaspare Rodolico, Catania

  PRINCIPAL INVESTIGATOR

• Luigia Luciano, MD

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

  PRINCIPAL INVESTIGATOR

• Carmen Fava, MD

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

  PRINCIPAL INVESTIGATOR

• Philipp leCoutre, MD

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

  PRINCIPAL INVESTIGATOR

• Susanne Saussele, MD

  University of Mannheim

  PRINCIPAL INVESTIGATOR

• Sarit Assouline, MD

  Jewish General Hospital

  PRINCIPAL INVESTIGATOR

• Alberto Álvarez-Larrán, MD

  University Hospital Clínic de Barcelona

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Target Duration: 36 Months
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 14, 2020
• First Posted: November 9, 2020
• Study Start: September 30, 2020
• Primary Completion: September 30, 2021
• Study Completion: August 14, 2023
• Last Updated: July 29, 2025

Record last verified: 2023-12

Locations

DE (2); CA (1); ES (1); IT (22)