Stopping Tyrosine Kinase Inhibitors in Affecting Treatment-Free Remission in Patients with Chronic Phase Chronic Myeloid Leukemia

Active:	Yes
Cancer Type:	Leukemia	NCT ID:	NCT03817398
Trial Phases:	Phase II	Protocol IDs:	AAML18P1 (primary) AAML18P1 NCI-2018-03439
Eligibility:	0 - 25 Years, Male and Female	Study Type:	Treatment
Study Sponsor:	Children's Oncology Group
NCI Full Details:	http://clinicaltrials.gov/show/NCT03817398

Summary

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.

Objectives

PRIMARY OBJECTIVES:
I. To determine the 2-year treatment free remission (TFR) rate of children, adolescents, and young adults with chronic myeloid leukemia - chronic phase (CML-CP) following discontinuation tyrosine kinase inhibitor (TKI).
II. To estimate the re-induction rate and maintenance of molecular remission (BCR-ABL1 =< 0.1%) at 1 year after restarting TKI for children, adolescents, and young adults.

SECONDARY OBJECTIVE:
I. To describe clinical factors and laboratory correlates affecting the persistence of major molecular remission (MMR) and re-initiation of treatment after stopping TKI (e.g. patient demographics, duration and level of prior molecular remission, duration and type of TKI, clinical presentation at diagnosis and immune studies).

EXPLORATORY OBJECTIVES:
I. To describe change in height standard deviation score over time in patients who are able to discontinue their TKI.
II. To describe the long-term health outcomes including but not limited to gonadal function, endocrine function, and bone metabolism in patients who are able to discontinue TKI as well as those that need to restart TKIs.
III. To describe differences in patient-reported health status after stopping TKIs, including those who need to resume TKI after stopping.
IV. To describe the incidence and characteristics of TKI withdrawal syndrome in children.
V. To evaluate changes in neurocognitive outcomes of patients enrolled on this study using a patient-completed, performance-based, computerized measure of neuropsychological functioning and a parent-report/self-report questionnaire.

OUTLINE:
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.

After completion of study treatment, patients are followed up annually.

Treatment Sites in Georgia

Aflac Cancer and Blood Disorders Center of Children’s at Egleston
1405 Clifton Road NE
3rd Floor
Atlanta, GA 30322
404-785-0853
www.choa.org

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