Chemotherapy for Pediatric Acute Myeloid Leukemia

Age: < 65

Sex: Any

Trial Phase: Phase 1

Sponsor: M.D. Anderson Cancer Center

Disqualifiers: CNS pathology, Active infections, Hepatitis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Trial Summary

What is the purpose of this trial?

This phase I trial studies the best dose and side effects of liposomal cytarabine, daunorubicin, and gemtuzumab ozogamicin in treating pediatric patients with acute myeloid leukemia that has returned after treatment (relapsed) or does not respond to treatment (refractory). Chemotherapy drugs, such as liposomal cytarabine and daunorubicin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Gemtuzumab ozogamicin is a monoclonal antibody, called gemtuzumab, linked to a toxic agent called ozogamicin. Gemtuzumab attaches to CD33 positive cancer cells in a targeted way and delivers ozogamicin to kill them. Giving liposomal cytarabine and daunorubicin and gemtuzumab ozogamicin may help to control the disease.

Research Team

Branko Cuglievan

Principal Investigator

M.D. Anderson Cancer Center

Eligibility Criteria

This trial is for pediatric patients up to 21 years old with relapsed or refractory acute myeloid leukemia (AML) that's CD33 positive. They should have a certain level of organ function, not be pregnant or breastfeeding, and agree to use effective contraception. Excluded are those with heart issues, high doses of prior chemotherapy, active infections, other cancers in the last few years except certain skin cancers or localized cancers.

Inclusion Criteria

Total serum bilirubin =< 2 x upper limit of normal (ULN); =< 3 x ULN for patients with known Gilbert's syndrome

I can do most activities but need help with some, regardless of my age.

I have acute myeloid leukemia with at least 5% cancer cells in my blood or bone marrow.

Exclusion Criteria

I am currently receiving treatment for GvHD.

My heart's pumping ability is below normal.

I have active brain or spinal cord disease.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Induction 1

Patients receive CPX-351 IV over 90 minutes on days 1, 3, and 5 and GO IV over 2 hours on day 1

4 weeks

Induction 2

Patients who do not attain a defined clinical response after Induction 1 receive CPX-351 IV on days 1 and 3 and GO IV over 2 hours on day 1

4 weeks

Consolidation

Beginning 4 weeks after last induction, patients receive CPX-351 IV over 90 minutes on days 1 and 3 and GO IV over 2 hours on day 1

4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Cytarabine
• Daunorubicin
• Gemtuzumab Ozogamicin

Trial Overview The trial tests liposomal cytarabine and daunorubicin alongside gemtuzumab ozogamicin for AML in children who've had treatment fail before. It aims to find the best dose and monitor side effects by seeing how these drugs stop cancer cells from growing.

Participant Groups

Group I: Treatment (CPX-351, GO) Experimental Treatment 2 Interventions

INDUCTION 1 (28 days): Patients receive CPX-351 IV over 90 minutes on days 1, 3, and 5 and GO IV over 2 hours on day 1 in the absence of disease progression or unacceptable toxicity. INDUCTION 2: Patients who do not attain a defined clinical response after cycle Induction 1 receive CPX-351 IV on days 1 and 3 and GO IV over 2 hours on day 1 in the absence of disease progression or unacceptable toxicity. CONSOLIDATION: Beginning 4 weeks after last induction, patients receive CPX-351 IV over 90 minutes on days 1 and 3 and GO IV over 2 hours on day 1 in the absence of disease progression or unacceptable toxicity.

Cytarabine is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Cytosar-U for:

• Acute myeloid leukemia
• Acute lymphocytic leukemia
• Chronic myeloid leukemia
• Meningeal leukemia

Approved in European Union as Depocyt for:

• Lymphomatous meningitis

Approved in Canada as Cytosar-U for:

• Acute myeloid leukemia
• Acute lymphocytic leukemia
• Chronic myeloid leukemia

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials

3,107

Recruited

1,813,000+