RecruitingPhase 1NCT06247787

A Study to Find the Highest Dose of Imetelstat in Combination With Fludarabine and Cytarabine for Patients With AML, MDS or JMML That Has Come Back or Does Not Respond to Therapy

A Phase 1 Study of GRN163L (Imetelstat) in Combination With Fludarabine and Cytarabine for Patients With Acute Myeloid Leukemia That is in Second or Greater Relapse or That is Refractory to Relapse Therapy; Myelodysplastic Syndrome or Juvenile Myelomonocytic Leukemia in First or Greater Relapse or is Refractory to Relapse Therapy

Sponsor

Children's Oncology Group

Enrollment

36 participants

Start Date

Feb 4, 2025

Study Type

INTERVENTIONAL

Conditions

Recurrent Childhood Acute Myeloid Leukemia Recurrent Childhood Myelodysplastic Syndrome Recurrent Juvenile Myelomonocytic Leukemia Refractory Childhood Acute Myeloid Leukemia Refractory Childhood Myelodysplastic Syndrome Refractory Juvenile Myelomonocytic Leukemia

Summary

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.

Eligibility

Min Age: 1 YearMax Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This study looks for the safest and most effective dose of a drug called imetelstat, given together with two chemotherapy drugs (fludarabine and cytarabine), for children and teens with certain blood cancers — including AML (acute myeloid leukemia), MDS (a blood disorder), or JMML (a rare childhood leukemia) — that have come back or haven't responded to prior treatment. **You may be eligible if...** - You are between 1 and 18 years old - You have AML, therapy-related AML, MDS, or JMML that has relapsed or is not responding to treatment - Your leukemia has come back at least twice, or your MDS or JMML has relapsed at least once - You do not have isolated cancer in the brain or testicles **You may NOT be eligible if...** - You have isolated central nervous system or testicular relapse only - You have not tried prior treatment - You have significant organ problems preventing treatment Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

PROCEDUREBiospecimen Collection

Undergo blood and CSF sample collection

PROCEDUREBone Marrow Aspiration

Undergo bone marrow biopsy and aspiration

PROCEDUREBone Marrow Biopsy

Undergo bone marrow biopsy and aspiration

DRUGCytarabine

Given IV and IT

PROCEDUREEchocardiography Test

Undergo ECHO

DRUGFludarabine

Given IV

DRUGHydrocortisone Sodium Succinate

Given IT

BIOLOGICALImetelstat

Given IV

DRUGLeucovorin Calcium

Given IV or PO

PROCEDURELumbar Puncture

Undergo lumbar puncture

DRUGMethotrexate

Given IT

Locations(19)

Children's Hospital of Alabama

Birmingham, Alabama, United States

Children's Hospital of Orange County

Orange, California, United States

UCSF Medical Center-Mission Bay

San Francisco, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Children's National Medical Center

Washington D.C., District of Columbia, United States

Children's Healthcare of Atlanta - Arthur M Blank Hospital

Atlanta, Georgia, United States

Lurie Children's Hospital-Chicago

Chicago, Illinois, United States

Riley Hospital for Children

Indianapolis, Indiana, United States

Dana-Farber Cancer Institute

Boston, Massachusetts, United States

C S Mott Children's Hospital

Ann Arbor, Michigan, United States

University of Minnesota/Masonic Cancer Center

Minneapolis, Minnesota, United States

Washington University School of Medicine

St Louis, Missouri, United States

NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center

New York, New York, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Saint Jude Children's Research Hospital

Memphis, Tennessee, United States

Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center

Houston, Texas, United States

Seattle Children's Hospital

Seattle, Washington, United States

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

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NCT06247787

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