Stopping TKI to Assess Treatment-Free Remission in Pediatric Chronic Myeloid Leukemia

What is the Purpose of this Study?

This study focuses on children, adolescents and young adults who have been diagnosed with chronic myeloid leukemia (CML) and whose level of disease is very low (molecular remission). To stay in molecular remission, treatment for CML requires patients to take a type of medication called a tyrosine kinase inhibitor (TKI) every day. In this study, researchers aim to determine whether children, adolescents, and young adults can stop taking their TKI medication and remain in molecular remission, in addition to assessing whether patients who restart TKI will re-achieve molecular remission. The study will also evaluate how stopping TKI medications affects social, emotional and behavioral functioning (neurocognitive function).

Eligibility

Patient must have been diagnosed with CML-CP at < 18 years of age.
Patient must have histologic verification of CML-CP at original diagnosis
Patient must be in molecular remission (MR) with a BCR-ABL1 level of =< 0.01% BCR-ABL1 as measured using the International Scale (IS) by RQ-PCR for >= 2 consecutive years at the time of enrollment

Inclusion Criteria:

Patient must have been diagnosed with CML-CP at < 18 years of age.
Patient must have histologic verification of CML-CP at original diagnosis
Patient must be in molecular remission (MR) with a BCR-ABL1 level of =< 0.01% BCR-ABL1 as measured using the International Scale (IS) by RQ-PCR for >= 2 consecutive years at the time of enrollment
Please note: The lab evaluating disease status and molecular response for this study must be College of American Pathology (CAP) and/or Clinical Laboratory Improvement Amendments (CLIA) certified (United States [US] only), sites in other countries must be certified by their accredited authorities. All labs must use the International Scale guidelines with a sensitivity of detection assay =< 0.01% BCR-ABL1 and be able to report results in =< 2 weeks
Patient must have received any TKI for a minimum of 3 consecutive years at time of enrollment
Patient agrees to discontinue TKI therapy
REGULATORY REQUIREMENTS
All patients and/or their parents or legal guardians must sign a written informed consent
All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
ELIGIBILITY FOR PATIENT-REPORTED OUTCOMES (PROs):
Age >= 8 years at the time of enrollment
Ability to understand English or Spanish
Cognitive ability to complete instruments according to the primary team
ELIGIBILITY FOR AAML18P1 NEUROCOGNITIVE STUDY:
Patient must be 5 years or older at the time of enrollment
English-, French- or Spanish-speaking
No known history of neurodevelopmental disorder prior to diagnosis of CML (e.g., Down syndrome, Fragile X, William syndrome, mental retardation)
No significant visual or motor impairment that would prevent computer use or recognition of visual test stimuli

Exclusion Criteria:

Known T3151 mutation
Additional clonal chromosomal abnormalities in Philadelphia chromosome (Ph) positive (+) cells at any time prior to enrollment that include "major route" abnormalities (second Ph, trisomy 8, isochromosome 17q, trisomy 19), complex karyotype or abnormalities of 3q26.2
History of accelerated phase or blast crisis CML
Female patients who are pregnant
Lactating females are not eligible unless they have agreed not to breastfeed their infants
Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained

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Where can I participate?

Cedars-Sinai Cancer at SOCC : Amy Oppenheim

More about this Clinical Trial

What is the full name of this clinical trial?

AAML18P1: Stopping Tyrosine Kinase Inhibitors to Assess Treatment-Free Remission in Pediatric Chronic Myeloid Leukemia - Chronic Phase ^

Study Details

Disease Type/Condition

Myeloid and Monocytic Leukemia

Principal Investigator

Majlessipour, Fataneh

Co-Investigators

Behrooz Hakimian, Leo Mascarenhas, Nicole Baca

Age Group

Both

Phase

IRB Number

STUDY00002830

ClinicalTrials.gov ID

NCT03817398

Key Eligibility

ClinicalTrials.gov

How do I learn more about this study?

Name

Amy Oppenheim

Phone

+1 310-423-3713

amy.oppenheim@cshs.org