110 Participants Needed

Stopping Tyrosine Kinase Inhibitors for Chronic Myeloid Leukemia

Recruiting at 182 trial locations
Age: < 65
Sex: Any
Trial Phase: Phase 2
Sponsor: Children's Oncology Group
Must be taking: Tyrosine kinase inhibitors
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

What is the purpose of this trial?

This phase II trial studies how stopping tyrosine kinase inhibitors will affect treatment-free remission in patients with chronic myeloid leukemia in chronic phase. When the level of disease is very low, it's called molecular remission. TKIs are a type of medication that help keep this level low. However, after being in molecular remission for a specific amount of time, it may not be necessary to take tyrosine kinase inhibitors. It is not yet known whether stopping tyrosine kinase inhibitors will help patients with chronic myeloid leukemia in chronic phase continue or re-achieve molecular remission.

Will I have to stop taking my current medications?

Yes, you will need to stop taking tyrosine kinase inhibitors (TKIs) if you join this trial, as the study is focused on observing the effects of stopping these medications.

What data supports the effectiveness of stopping the drug Tyrosine Kinase Inhibitors for Chronic Myeloid Leukemia?

Research shows that many patients with chronic myeloid leukemia can safely stop taking tyrosine kinase inhibitors if they have achieved a deep and sustained response to the treatment. Studies indicate that a significant number of patients maintain remission without the drug, suggesting that stopping the drug can be effective for some individuals.12345

Is it safe to stop taking tyrosine kinase inhibitors for chronic myeloid leukemia?

Stopping tyrosine kinase inhibitors for chronic myeloid leukemia is generally considered safe for patients who have achieved a deep and sustained response to the treatment. Clinical trials and studies have shown that many patients can successfully stop the medication without their condition worsening, although some may experience side effects like fatigue and diarrhea while on the medication.13456

How is the drug Tyrosine Kinase Inhibitor unique in treating chronic myeloid leukemia?

Tyrosine Kinase Inhibitors are unique because they allow some patients with chronic myeloid leukemia to stop treatment after achieving a deep and sustained molecular response, potentially leading to long-term remission without ongoing medication. This approach differs from traditional treatments that require continuous therapy to manage the disease.12457

Research Team

SC

Sonali Chaudhury

Principal Investigator

Children's Oncology Group

Eligibility Criteria

This trial is for patients diagnosed with chronic myeloid leukemia in chronic phase (CML-CP) who were under 18 at diagnosis, have been in deep molecular remission for at least 2 years, and have taken tyrosine kinase inhibitors (TKIs) for a minimum of 3 years. Participants must be over the age of 8, able to understand English or Spanish, and not pregnant or breastfeeding. Those with certain genetic mutations or additional chromosomal abnormalities are excluded.

Inclusion Criteria

All patients and/or their parents or legal guardians must sign a written informed consent
No significant visual or motor impairment that would prevent computer use or recognition of visual test stimuli
All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
See 11 more

Exclusion Criteria

Lactating females are not eligible unless they have agreed not to breastfeed their infants
I am currently pregnant.
I am not pregnant or have confirmed it with a negative pregnancy test.
See 3 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Discontinuation

Participants stop taking TKI medication within 10 days after enrollment

10 days
1 visit (in-person)

Monitoring

Participants undergo peripheral blood collection to monitor loss of MMR every 4 weeks in year 1, every 6 weeks in year 2, and every 12 weeks in year 3

36 months
Regular visits every 4-12 weeks

Re-initiation of Treatment

Participants who lose their molecular remission may restart TKI medication and are monitored every 4 weeks in year 1, every 6 weeks in year 2, and every 12 weeks in year 3

Up to 36 months
Regular visits every 4-12 weeks

Follow-up

Participants are followed up annually after completion of study treatment

Annually

Treatment Details

Interventions

  • Drug Withdrawn
  • Tyrosine Kinase Inhibitor
Trial Overview The study is examining if stopping TKI medication affects the ability to maintain or re-enter molecular remission in CML-CP patients. It involves questionnaire administration, biospecimen collection to monitor disease levels, and quality-of-life assessments after drug withdrawal.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: Basic Science (stop taking TKI, biospecimen collection)Experimental Treatment5 Interventions
Patients stop taking TKI medication within 10 days after enrollment. Patients undergo peripheral blood collection to monitor loss of MMR every 4 weeks in year 1, every 6 weeks in year 2, and every 12 weeks in year 3. Patients who lose their molecular remission may restart TKI medication and are monitored every 4 weeks in year 1, every 6 weeks in year 2, and every 12 weeks in year 3.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Children's Oncology Group

Lead Sponsor

Trials
467
Recruited
241,000+

National Cancer Institute (NCI)

Collaborator

Trials
14,080
Recruited
41,180,000+

Findings from Research

In a study of 293 Italian patients with chronic myeloid leukemia (CML) who discontinued tyrosine kinase inhibitors (TKI) after achieving deep molecular response, 62% maintained treatment-free remission (TFR) after a median follow-up of 34 months, indicating that stopping TKI can be safe and effective.
The study found no disease progression during the follow-up period, supporting the feasibility of TKI discontinuation in clinical practice, with the majority of patients stopping treatment due to shared decision-making with their physician.
Observational study of chronic myeloid leukemia Italian patients who discontinued tyrosine kinase inhibitors in clinical practice.Fava, C., Rege-Cambrin, G., Dogliotti, I., et al.[2020]
In the LAST study involving 172 patients with chronic myeloid leukemia (CML), discontinuation of tyrosine kinase inhibitors (TKIs) was found to be safe, with 60.8% of patients achieving treatment-free remission (TFR) after stopping their medication.
Patients who discontinued TKIs reported significant improvements in quality of life, including reductions in fatigue (80.4%), depression (34.8%), and diarrhea (87.5%), highlighting the potential benefits of stopping TKIs beyond just cancer management.
Assessment of Outcomes After Stopping Tyrosine Kinase Inhibitors Among Patients With Chronic Myeloid Leukemia: A Nonrandomized Clinical Trial.Atallah, E., Schiffer, CA., Radich, JP., et al.[2022]

References

Handling challenging questions in the management of chronic myeloid leukemia: when is it safe to stop tyrosine kinase inhibitors? [2022]
Immunosurveillance markers may predict patients who can discontinue imatinib therapy without relapse. [2021]
Treatment outcome in a population-based, 'real-world' cohort of patients with chronic myeloid leukemia. [2018]
Observational study of chronic myeloid leukemia Italian patients who discontinued tyrosine kinase inhibitors in clinical practice. [2020]
Handling challenging questions in the management of chronic myeloid leukemia: when is it safe to stop tyrosine kinase inhibitors? [2023]
Assessment of Outcomes After Stopping Tyrosine Kinase Inhibitors Among Patients With Chronic Myeloid Leukemia: A Nonrandomized Clinical Trial. [2022]
Discontinuation of tyrosine kinase inhibitor therapy in chronic myeloid leukaemia (EURO-SKI): a prespecified interim analysis of a prospective, multicentre, non-randomised, trial. [2019]