Chronic Myeloid Leukaemia: Treatment-Free Remission Registry

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

ANZCTR

Registry ID

ACTRN12625000236493

Acronym

CML-TFR Registry

Enrollment

Registered

2025-04-01

Start date

2025-11-26

Completion date

2028-04-14

Last updated

2026-06-01

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

None listed

Brief summary

The Chronic Myeloid Leukaemia: Treatment-Free Remission Registry (CML-TFR) is an observational registry for participants with CML in chronic phase who are in molecular remission on Tyrosine Kinase Inhibitor (TKI) treatment and are planning a trial of TKI drug cessation with the objective of Treatment-Free Remission (TFR). Who is it for? Participants may be eligible for this study if they are an adult who has been diagnosed with chronic myeloid leukaemia, have completed a minimum of 3 years TKI therapy, and are intending to stop TKI treatment based on their treating clinician’s advice. Study Details All participants in this study will enter this registry before stopping their TKI treatment. No intervention will be provided to participants as part of this study, participants will stop their TKI treatment as prescribed by their treating clinician. For this study, participants will undergo an informed consent process. Following this the screening process will begin where a series of data including blood test results relating to the participant’s CML diagnosis are collected from participants medical records. A research blood sample will be collected on the day before stopping their current TKI treatment. Results from CML blood tests requested by the treating clinician will be collected from participants medical records every 3 months for up to 5 years unless a participant experiences relapse or requires re-treatment. If a participant requires retreatment with TKI, participants will be followed for a further 1 year from commencing re-treatment. This registry will provide valuable descriptive data on TFR in Australia and will enable the validation of biomarkers to identify patients with a higher chance of achieving long-term TFR, as well as those with a lower chance of TFR in whom investigational approaches might improve the TFR outcome.

Interventions

Participants with a diagnosis of Chronic Myeloid Leukaemia (CML) in chronic phase who have received Tyrosine Kinase Inhibitor (TKI) treatment and are planning a trial of TKI drug cessation with the objective of Treatment-Free Remission (TFR). Screening Phase: Participants will enter the Registry before they cease TKI treatment. Retrospective data collected includes; demographics, CML diagnosis details, blood results at diagnosis, genetics results at diagnosis (if available), details of previous TFR attempts, CML treatment history and molecular response results data during the first 12 months of treatment after diagnosis. Baseline Timepoint (2-4 weeks post screening): Research blood sample collected and sent to SAHMRI Central Laboratory for processing, then participants will cease TKI the following day. Retrospective data collected includes; molecular response data in the lead-up to TKI cessation, other therapies and percentage lymphocytes at time of TKI cessation, previously failed TKIs and kinase domain mutations (if detected). Monitoring Phase: Retrospective CML blood results as requested by the treating clinician will be recorded in the Registry at a frequency of every 3 months for up to 1 year, unless relapse occurs. Results to be reviewed and actioned by treating clinician as standard of care. Follow-up Phase: Restrospective data collected will include, survival status, TFR status including current CML disease phase will be recorded in the Registry at a frequency of every 3 months for up to 4 years, unless relapse occurs. There will be a total of 5 years of the 'Monitoring' and 'Follow-up' phases combined, unless relapse/re-treatment occurs. Relapse/Retreatment: If retreatment with TKI occurs after a relapse of CML, participants will be followed for a further 1 year from commencing re-treatment. Including re-treatment details blood results as requested by the treating clinician indicating response to re-treatment. Retrospective data collected will include; TKI commenced, any treatment switches in the first 12 months after retreatment and molecular response results after retreatment.

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

1. Adults (>18 years of age) who can provide written informed consent. 2. Diagnosis of chronic myeloid leukaemia, expressing BCR::ABL1 quantifiable by real-time PCR or digital PCR. 3. Completed a minimum of 3 years TKI therapy. a. One or more previous TFR attempts are not an exclusion. Participants who have previously had a TFR attempt and re-started TKI treatment will still be eligible for the Registry if they have received at least 12 months of subsequent TKI treatment and meet all other inclusion criteria. 4. Currently, and for the preceding 12 months, maintaining MR4.0 response and receiving BCR::ABL1 TKI treatment over that period. 5. Intending to stop TKI treatment and not planning to resume CML treatment unless loss of MMR (i.e. BCR::ABL1 exceeds 0.1%). 6. Willing and able to provide blood samples for correlative sciences evaluation.

Exclusion criteria

1. Diagnosis of Philadelphia positive (Ph+) (or BCR::ABL1+) acute lymphoblastic leukaemia. 2. Current or prior blast crisis CML. Participants with prior accelerated phase (AP) may be enrolled at the discretion of the Investigator. 3. Prior allogeneic stem cell transplant for CML. 4. Life expectancy <2 years. 5. Unable or unwilling to comply with follow-up procedures.

Outcome results