Ziftomenib in Combination With Chemotherapy for Children With Relapsed/Refractory Acute Leukemia

Conditions

• Relapsed/Refractory KMT2A-r Acute Leukemia
• Relapsed/Refractory NPM1-m Acute Leukemia
• Relapsed/Refractory NUP98-r Acute Leukemia

Eligibility Criteria

Sex

ALL

Age

0 Years - 21 Years

Healthy Volunteers

Not accepted

Interventions

• Ziftomenib — DRUG
  Oral capsule
• Cytarabine — DRUG
  Intravenous (IV) infusion
• Fludarabine — DRUG
  IV infusion

Study Details

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).

Key Dates

Start date

Mar 18, 2025

Status verified

May 2025

Primary completion

Jan 31, 2027

Completion

Jan 31, 2027

Study Design

Enrollment

20 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Ziftomenib
  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.

Primary Outcome Measure

Number of Participants Who Experience a Dose-limiting Toxicity (DLT) [ Time Frame: Day 1 to Day 49 ]

Central Contacts

• Dr. Branko Cuglievan
  (713) 563-1499
  [email protected]
• Dr. Sarah Tasian, MD
  (215) 590-2299
  [email protected]

Locations (11)

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