Ziftomenib + Chemotherapy for Leukemia

Recruiting at 14 trial locations

Overseen By Dr. Sarah Tasian, MD

Age: < 65

Sex: Any

Trial Phase: Phase 1

Sponsor: LLS PedAL Initiative, LLC

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

Compensation: Varies

Number of Visits: Unknown

20 Participants Needed

Trial Summary

What is the purpose of this trial?

The primary objective of the study is to determine the recommended phase 2 dose (RP2D) of ziftomenib in combination with chemotherapy (FLA) in children with relapsed or refractory KMT2A-r, NUP98-r, or NPM1-m acute leukemia based on safety and pharmacokinetics (PK).

Eligibility Criteria

This trial is for children with relapsed or refractory acute leukemia who have specific genetic alterations (KMT2A-r, NUP98-r, or NPM1-m). They must show a certain level of leukemic blasts in their bone marrow and not just isolated extramedullary disease. Participants should be aged 0-21 years, mostly under 18, and weigh at least 5 kg.

Inclusion Criteria

My tests show abnormal chromosomes in my cells.

My bone marrow sample shows more than 1% leukemia cells confirmed by tests.

I am 21 or younger and weigh at least 5 kg, with most participants being under 18.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Cycle 1

Participants receive escalating doses of ziftomenib in combination with FLA chemotherapy for 49 days

7 weeks

Daily visits for drug administration

Treatment Cycle 2

Participants continue with ziftomenib and FLA chemotherapy for 28 days if they respond to Cycle 1

4 weeks

Daily visits for drug administration

Extended Treatment

Participants may receive up to 10 additional cycles of ziftomenib monotherapy if there is a delay before HSCT

Up to 10 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 13 months

Treatment Details

Interventions

• Ziftomenib

Trial Overview The study aims to find the safest dose for Ziftomenib when combined with chemotherapy drugs Cytarabine and Fludarabine in children with certain types of acute leukemia. It will look at how well patients tolerate this combination and measure drug levels in the body.

Participant Groups

Group I: Ziftomenib Experimental Treatment 3 Interventions

During Cycle 1 (49 days), participants will receive escalating doses of ziftomenib once daily (QD) on Days 8 to 49. Participants will also receive FLA chemotherapy consisting of fludarabine at a dose of 30 mg/m\^2 (1 mg/kg/dose in infants \<1 year of age or ≤10 kg) and cytarabine at a dose of 2000 mg/m\^2 (67 mg/kg/dose in infants \<1 year of age or ≤10 kg) QD on Day 1 to Day 5. Participants with \<5% blasts will continue ziftomenib monotherapy until Day 49. Participants with a response and \>5% blasts will continue to Cycle 2. During Cycle 2 (28 days), participants will receive escalating doses of ziftomenib QD on Day 1 to Day 28 in combination with FLA chemotherapy on Day 1 to Day 5. Participants who respond to treatment, but experience a delay prior to hematopoietic stem cell transplantation (HSCT), may receive up to 10 additional cycles (28 days) of ziftomenib monotherapy. Participants may also receive intrathecal therapy prophylaxis, if needed, during all cycles.

Who Is Running the Clinical Trial?

LLS PedAL Initiative, LLC

Lead Sponsor

Trials

4

Recruited

1,100+

Kura Oncology

Collaborator

Trials

2

Recruited

110+