Study on Stopping Tyrosine Kinase Inhibitors for Treatment-Free Remission in Chronic Phase Chronic Myeloid Leukemia Patients
Sponsor: Children's Oncology Group
ClinicalTrials ID:NCT03817398
This clinical trial is exploring whether patients with chronic myeloid leukemia in the chronic phase can maintain remission without taking tyrosine kinase inhibitors (TKIs) after achieving a very low level of disease.
Patient Parameters
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Program Overview
This study is investigating if patients with chronic myeloid leukemia (CML) in the chronic phase can stop taking tyrosine kinase inhibitors (TKIs) and still keep their disease in remission. Remission means the disease is at a very low level, known as molecular remission. TKIs are medications that help maintain this low level. The study aims to find out if stopping TKIs after being in remission for a certain time can help patients continue or regain remission without the medication.
Description
The main goals of this study are to see if children, adolescents, and young adults with chronic phase chronic myeloid leukemia (CML-CP) can stay in remission for two years after stopping tyrosine kinase inhibitors (TKIs), and to find out how well they return to remission if they need to restart TKIs. Researchers will also look at factors that might affect staying in remission and restarting treatment, such as patient characteristics and previous treatment details.
The study will also explore changes in height, long-term health outcomes like hormone and bone health, and patient-reported health status after stopping TKIs. It will examine any withdrawal symptoms from stopping TKIs and changes in cognitive function using tests and questionnaires.
Participants will stop taking TKIs within 10 days of joining the study. They will have regular blood tests to monitor their remission status every 4 weeks in the first year, every 6 weeks in the second year, and every 12 weeks in the third year. If they lose remission, they can restart TKIs and will be monitored similarly. After the study treatment, participants will have annual follow-ups.
Eligibility Criteria
Inclusion Criteria
- Diagnosed with chronic phase chronic myeloid leukemia (CML-CP) before age 18.
- Confirmed diagnosis of CML-CP at the start.
- In molecular remission with a BCR-ABL1 level of 0.01% or less for at least 2 years.
- Received any TKI for at least 3 consecutive years.
- Agree to stop TKI therapy.
- Must sign informed consent.
- Meet all regulatory requirements for human studies.
- For patient-reported outcomes: Age 8 or older, able to understand English or Spanish, and able to complete questionnaires.
- For neurocognitive study: Age 5 or older, English, French, or Spanish-speaking, no history of neurodevelopmental disorders, and no significant visual or motor impairments.
Exclusion Criteria
- Known T3151 mutation.
- Additional chromosomal abnormalities in Philadelphia chromosome-positive cells.
- History of accelerated phase or blast crisis CML.
- Pregnant or breastfeeding females.
- Females of childbearing potential without a negative pregnancy test.
Locations
| FACILITY | ZIP | CITY | STATE |
|---|---|---|---|
| Children's Hospital of Alabama | 35233 | Birmingham | Alabama |
| USA Health Strada Patient Care Center | 36604 | Mobile | Alabama |
| Banner Children's at Desert | 85202 | Mesa | Arizona |
| Banner University Medical Center - Tucson | 85719 | Tucson | Arizona |
| Arkansas Children's Hospital | 72202-3591 | Little Rock | Arkansas |
| Kaiser Permanente Downey Medical Center | 90242 | Downey | California |
| Miller Children's and Women's Hospital Long Beach | 90806 | Long Beach | California |
| Children's Hospital Los Angeles | 90027 | Los Angeles | California |
| Cedars Sinai Medical Center | 90048 | Los Angeles | California |
| Mattel Children's Hospital UCLA | 90095 | Los Angeles | California |
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