This phase I trial tests the safety, side effects, and best dose of imetelstat in combination with fludarabine and cytarabine in treating patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) or juvenile myelomonocytic leukemia (JMML) that has not responded to previous treatment (refractory) or that has come back after a period of improvement (recurrent). Imetelstat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Chemotherapy drugs, such as fludarabine and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving imetelstat in combination with fludarabine and cytarabine may work better in treating patients with refractory or recurrent AML, MDS, and JMML.
PRIMARY OBJECTIVE: I. To estimate the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of imetelstat administered in combination with fludarabine and cytarabine to children with second or greater relapse of acute myeloid leukemia or first or greater relapse of myelodysplastic syndrome (MDS) or juvenile myelomonocytic leukemia (JMML). SECONDARY OBJECTIVES: I. To define and describe the toxicities of imetelstat administered on this schedule in combination with fludarabine and cytarabine in patients with refractory and/or relapsed AML, MDS, or JMML. II. To characterize the pharmacokinetics of imetelstat in combination with fludarabine and cytarabine in patients with refractory and/or relapsed AML, MDS, or JMML. III. To describe the antileukemic activity of imetelstat (CR \[complete remission\]/PR \[partial response\]/CRi \[complete remission with incomplete blood count recovery\] and rates of minimal residual disease \[MRD\] negative response after up to two cycles of therapy) in combination with fludarabine and cytarabine within the limits of a phase 1 study. IV. To estimate the overall survival (OS) of children with second or greater relapse of AML or first or greater relapse of MDS or JMML treated with imetelstat administered in combination with fludarabine and cytarabine. EXPLORATORY OBJECTIVES; I. To conduct pharmacodynamics studies of imetelstat in combination with fludarabine and cytarabine in patients with refractory and/or relapsed AML, MDS, or JMML. II. To analyze telomerase activity in peripheral blood mononuclear cells. III. To evaluate baseline and change of cytogenetic abnormalities after treatment with imetelstat in combination with fludarabine and cytarabine. IV. To evaluate baseline mutational status and change of mutational status after treatment with imetelstat in combination with fludarabine and cytarabine. OUTLINE: This is a dose-escalation study of imetelstat. Patients receive imetelstat intravenously (IV) over 2 hours on days 1 and 8, fludarabine IV over 1 hour on days 2-6, and cytarabine IV over 1-3 hours on days 2-6 of each cycle. Patients also receive cytarabine intrathecally (IT) alone or with methotrexate IT, and hydrocortisone IT at the provider's discretion. Patients may also receive leucovorin calcium IV or orally (PO) 24 and 30 hours after each IT triples dose. Treatment repeats every 28 days for up to 2 cycles in the absence of disease progression or unacceptable toxicity. Patients undergo echocardiography (ECHO), bone marrow biopsy and/or aspiration, blood sample collection, and lumbar puncture for cerebrospinal fluid (CSF) sample collection during screening and on the trial. After completion of study treatment, patients are followed up for 5 years.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
36
Undergo blood and CSF sample collection
Undergo bone marrow biopsy and aspiration
Undergo bone marrow biopsy and aspiration
Given IV and IT
Undergo ECHO
Given IV
Given IT
Given IV
Given IV or PO
Undergo lumbar puncture
Given IT
Children's Hospital of Alabama
Birmingham, Alabama, United States
RECRUITINGChildren's Hospital of Orange County
Orange, California, United States
RECRUITINGUCSF Medical Center-Mission Bay
San Francisco, California, United States
RECRUITINGChildren's Hospital Colorado
Aurora, Colorado, United States
RECRUITINGChildren's National Medical Center
Washington D.C., District of Columbia, United States
RECRUITINGChildren's Healthcare of Atlanta - Arthur M Blank Hospital
Atlanta, Georgia, United States
RECRUITINGLurie Children's Hospital-Chicago
Chicago, Illinois, United States
RECRUITINGRiley Hospital for Children
Indianapolis, Indiana, United States
RECRUITINGDana-Farber Cancer Institute
Boston, Massachusetts, United States
RECRUITINGC S Mott Children's Hospital
Ann Arbor, Michigan, United States
RECRUITING...and 9 more locations
Dose limiting toxicities of imetelstat administered in combination with fludarabine and cytarabine
Frequency percent (%) of patients with acute myeloid leukemia (AML) in second or greater relapse or refractory to relapse therapy who experience a cycle 1 dose limiting toxicity to imetelstat administered in combination with fludarabine and cytarabine stratified by dose level.
Time frame: During cycle 1 of therapy (each cycle is 28 days)
Incidence of adverse events of imetelstat administered in combination with fludarabine and cytarabine
Frequency (%) of patients with acute myeloid leukemia in second or greater relapse or refractory to relapse therapy who experience adverse events at least possibly attributable to imetelstat administered in combination with fludarabine and cytarabine stratified by dose level graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0.
Time frame: Up to 2 years from study entry
Area under the drug concentration curve of imetelstat administered in combination with fludarabine and cytarabine
Median (minimum \[min\], maximum \[max\]) of the area under the drug concentration curve of imetelstat administered in combination with fludarabine and cytarabine assessed during cycle 1 at 0, 0.5, 1, 4-5, 6-8 and 24-hours post administration.
Time frame: Up to 24 hours
Maximum serum concentration of imetelstat administered in combination with fludarabine and cytarabine
Median (min, max) of the maximum serum concentration of imetelstat administered in combination with fludarabine and cytarabine assessed during cycle 1 at 0, 0.5, 1, 4-5, 6-8 and 24-hours post administration.
Time frame: Up to 24 hours
Clearance of imetelstat administered in combination with fludarabine and cytarabine
Median (min, max) of the clearance of imetelstat administered in combination with fludarabine and cytarabine assessed during cycle 1 at 0, 0.5, 1, 4-5, 6-8 and 24-hours post administration.
Time frame: Up to 24 hours
Half-life of imetelstat administered in combination with fludarabine and cytarabine
Median (min, max) of the half-life of imetelstat administered in combination with fludarabine and cytarabine assessed during cycle 1 at 0, 0.5, 1, 4-5, 6-8 and 24-hours post administration.
Time frame: Up to 24 hours
PK parameters: Volume of distribution
Median (min, max) volume of distribution of imetelstat administered in combination with fludarabine and cytarabine assessed during cycle 1 at 0, 0.5, 1, 4-5, 6-8, and 24 hours post administration.
Time frame: Up to 24 hours
Antileukemic activity of imetelstat administered in combination with fludarabine and cytarabine
Frequency (%) of patients with best overall response of complete remission, partial response, or complete remission with incomplete blood count recovery after up to two cycles of therapy with imetelstat administered in combination with fludarabine and cytarabine.
Time frame: Up to 56 days
Overall survival (OS) of imetelstat administered in combination with fludarabine and cytarabine
Kaplan-Meier survival curves will estimate the median (95% CI) overall time-to-death due to any cause and stratified by dose level.
Time frame: Up to 5 years from study entry
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.