Italian Treatment Free Remission Registry

NCT04769947 | Recruiting DMC

• 1 other identifier: ITALY-TFR
• Study Type: observational
• Target: 800
• Locations: 1 country
• Sites: 1

Brief Summary

The investigator propose an extension of the previous National, multicentric study, promoting an observational registry, both retrospective and prospective, in order to expand and further characterize the series of Italian patients with Philadelphia-positive, chronic phase CML (CP-CML) who discontinue TKIs in an off-protocol setting. As safety concerns may arise for patients receiving long-term treatments, and due to the growing number of patients discontinuing TKIs in clinical practice, the study aim to collect all available data regarding feasibility and freedom from progression of the cohort of patients.

Conditions

Treatment Free Remission; Chronic Myeloid Leukemia

Outcome Measures

Primary Outcomes (1)

• TFR rate at 1 year from discontinuation of TKIs

  To assess the TFR rate at 1 year from discontinuation of TKIs

  1 year

Secondary Outcomes (1)

• Secondary objectives

  10 years

Study Arms (3)

Cohort 1

Cohort 1 retrospective/prospective study: 293 patients enrolled and reported in Fava et al 2019 (1) with at least 1 year of follow up at the end of the study (February 2017) and 107 patients enrolled but not reported in Fava et al 2019 since their follow-up was shorter than 1 year. The data for these patients will be collected since the end of previous study.

Cohort 2

Cohort 2 retrospective/prospective study: patient not enrolled in the previous study (Fava et al 2019). The data for these patients will be collected since patient diagnosis to the end of the study.

Cohort 3

Cohort 3 prospective study: patients who have discontinued TKI therapy after the study approval in each center. These patients will also participate in the validation process of Phase 2 of a questionnaire developed by an expert panel of eight CML patients with the purpose of capturing the experiences of people along all phases of the TFR.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with CP-CML who discontinued treatment with TKIs in Italy will be selected.

You may qualify if:

• Patients with CP-CML, treated with TKI monotherapy or TKI in association with other drugs (such as interferon, BCR-ABL1 peptidic vaccine and others)
• Treatment with TKI discontinued for any reason
• Deep Molecular Response (DMR), defined as MR4 (BCR-ABL1 ratio ≤ 0.01% with at least 10,000 ABL1 copies), or MR4.5 (BCR-ABL1 ratio ≤ 0.0032% with at least 32,000 ABL1 copies), or MR5 (BCR-ABL1 ratio ≤ 0.001% with at least 100,000 ABL1 copies), confirmed at least three times before TKI discontinuation. In patients who discontinued TKIs before the establishment of molecular standardization, DMR will be defined as a level of BCR-ABL1 transcript undetectable by qPCR or by qualitative PCR, confirmed in at least two controls.
• Participant is willing and able to give informed consent for participation in the study Data regarding patients discontinuing in less than DMR (as defined above) will be collected and analysed separately.

You may not qualify if:

• Patients who were diagnosed with accelerated or blastic phase CML will be excluded

Sponsors & Collaborators

• University of Turin, Italy: lead

Study Sites (1)

Division of Hematology-AO Mauriziano Hospital, University of Turin

Turin, 10128, Italy

RECRUITING

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

Central Study Contacts

Carmen Fava, MD, PhD

CONTACT

0115085523; carmen.fava@unito.it

Giulia Piraccini, MA

CONTACT

0115085523; giulia.piraccini1@gmail.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 10, 2021
• First Posted: February 25, 2021
• Study Start: August 9, 2021
• Primary Completion: July 8, 2022
• Study Completion (Estimated): February 8, 2030
• Last Updated: November 18, 2021

Record last verified: 2021-05

Locations

IT (1)