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

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

Eligibility Criteria

Sex

ALL

Age

1 Year - 18 Years

Healthy Volunteers

Not accepted

Interventions

• Biospecimen Collection — PROCEDURE
  Undergo blood and CSF sample collection
• Bone Marrow Aspiration — PROCEDURE
  Undergo bone marrow biopsy and aspiration
• Bone Marrow Biopsy — PROCEDURE
  Undergo bone marrow biopsy and aspiration
• Cytarabine — DRUG
  Given IV and IT
• Echocardiography Test — PROCEDURE
  Undergo ECHO
• Fludarabine — DRUG
  Given IV
• Hydrocortisone Sodium Succinate — DRUG
  Given IT
• Imetelstat — BIOLOGICAL
  Given IV
• Leucovorin Calcium — DRUG
  Given IV or PO
• Lumbar Puncture — PROCEDURE
  Undergo lumbar puncture
• Methotrexate — DRUG
  Given IT

Study Details

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.

Key Dates

Start date

Feb 4, 2025

Status verified

Apr 2026

Primary completion

Mar 31, 2028

Completion

Mar 31, 2028

Study Design

Enrollment

36 participants (estimated)

Allocation

NA

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Treatment (Imetelstat, fludarabine, cytarabine)
  Patients receive imetelstat 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 IT alone or with methotrexate IT, and hydrocortisone IT at the provider's discretion. Patients may also receive leucovorin calcium IV or 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 ECHO, bone marrow biopsy and/or aspiration, blood sample collection, and lumbar puncture for CSF sample collection during screening and on the trial.

Primary Outcome Measure

Dose limiting toxicities of imetelstat administered in combination with fludarabine and cytarabine [ Time Frame: During cycle 1 of therapy (each cycle is 28 days) ]

Locations (19)

Find similar trials in Birmingham, AL

Related Studies

• HA-1 T TCR T Cell Immunotherapy for the Treatment of Patients With Relapsed or Refractory Acute Leukemia After Donor Stem Cell Transplant
  PHASE1 · Recruiting · Fred Hutchinson Cancer Center · Seattle, Washington
• FH-FOLR1 Chimeric Antigen Receptor T Cell Therapy for Treating Pediatric Patients With Relapsed or Refractory Acute Myeloid Leukemia
  PHASE1 · Recruiting · Fred Hutchinson Cancer Center · Seattle, Washington
• Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling
  PHASE1 · Recruiting · Florida International University · Miami, Florida