Italian Treatment Free Remission Registry

  • STATUS
    Recruiting
  • End date
    Feb 8, 2030
  • participants needed
    800
  • sponsor
    University of Turin, Italy
Updated on 26 November 2021
remission
interferon
chronic phase chronic myeloid leukemia
Accepts healthy volunteers

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.

Description

This is an observational, retrospective and prospective study of CP-CML patients who discontinue TKIs in Italy. The study will not imply additional costs for the participating centers. Data already present in the clinical charts will be collected.

The patients, answering to inclusion/exclusion criteria will be enrolled in one of the follow cohorts

  • Cohort 1 retrospective/prospective study: 293 patients enrolled and reported in Fava et al 2019 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 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 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.

Patients participating in the study will not be subjected to any procedure that falls outside the clinical practice; in the same way, clinical variables that will be collected for the study are those that are commonly collected by physician in daily clinical practice.

Any decision about drug administration or suspension is made by the physician based on his clinical judgment in the context of clinical practice, independently from decision to include the patient in this study.

Registration of enrolled patients will be done on-line on a key restricted accessible web-database.

Primary objective and endpoint To assess the TFR rate at 1 year from discontinuation of TKIs. Secondary objectives and endpoints

  • To further describe the observed population of patients in terms of:
  • treatment-free duration; o survival in MR4.5;
  • survival in MR3;
  • event-free survival;
  • progression-free survival;
  • deaths after treatment discontinuation;
  • rate of achievement of MMR and DMR after treatment re-initiation
  • To identify clinical and biological prognostic factors affecting the persistence of off-therapy remission.
  • To identify optimal timing and modalities of molecular monitoring of BCR-ABL1 transcript
  • Evaluation of the safe minimum monitoring during and after treatment discontinuation
  • Evaluation of the maximum MRD positivity level acceptable to safely stay off-treatment
  • Evaluation of short-term adverse events (withdrawal syndrome: fever, arthralgia, myalgia, bone pain, weight loss) after treatment discontinuation
  • Evaluation of long-term adverse events (i.e. cardiovascular adverse events, second neoplasia) after treatment discontinuation
  • Validation of the Italian version of the "Phase 2" of a questionnaire developed by an expert panel of eight CML patients to capture the experiences of people along all phases of the TFR
  • Evaluation of patient-reported outcomes describing quality of life of patients off therapy and after resumption of TKIs

Details
Condition Chronic myeloid leukemia, Treatment Free Remission, Chronic Myeloid Leukemia, chronic myelogenous leukemia
Clinical Study IdentifierNCT04769947
SponsorUniversity of Turin, Italy
Last Modified on26 November 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

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

Exclusion Criteria

Patients who were diagnosed with accelerated or blastic phase CML will be excluded
Clear my responses

How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar

Primary Contact

site

0/250

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider

Loading...

Browse trials for

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer

user name

Added by • 

 • 

Private

Reply by • Private
Loading...

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.
Loading...

No annotations made yet

Add a private note
  • abc Select a piece of text from the left.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.
Add a private note