Phase 1b Study of the Safety and Pharmacokinetics of Pivekimab Sunirine in Pediatric Patients with Relapsed or Refractory CD123-Positive Acute Myeloid Leukemia

The primary objective is to evaluate the safety and pharmacokinetics of pivekimab sunirine in pediatric subjects diagnosed with relapsed or refractory acute myeloid leukemia expressing CD-123. The secondary objective includes:

• Estimation of anti-tumor activity following pivekimab sunirine monotherapy.

This clinical trial involves 80 pediatric subjects diagnosed with acute myeloid leukemia. The study population includes both male and female patients with relapsed or refractory disease. Eligible participants must exhibit CD123-positive myeloid leukemic blasts via flow cytometry. A minimum of 5% myeloid leukemic blasts must be present in the bone marrow at the time of relapse or refractory disease. Required performance status includes a Lansky score of at least 50 for those under 16 years of age, a Karnofsky score of at least 50 for those 16 years of age or older, or an ECOG score of 2 or less. The population may include patients with central nervous system disease, provided there are no clinical neurologic symptoms. Non-central nervous system extramedullary disease is also permitted.

This Phase 1b study is designed to evaluate the safety and pharmacokinetics of pivekimab sunirine in pediatric subjects diagnosed with relapsed or refractory acute myeloid leukemia. The research methodology focuses on determining treatment-emergent adverse events and measuring specific pharmacokinetic parameters, including Cmax, AUC, and Tmax for both the intact antibody-drug conjugate and its payload. The study involves a screening process to confirm histological diagnosis, CD123-positive myeloid leukemic blasts via flow cytometry, and specific performance status requirements. Following screening, participants receive the investigational product via intravenous infusion of a powder for infusion. Clinical assessments are conducted to monitor for complete remission and its various composite forms, alongside the duration of complete remission. Participant involvement continues through scheduled monitoring of safety and efficacy endpoints until the conclusion of the study protocols.

The experimental medication is pivekimab sunirine, which is supplied as a powder for infusion. This substance is administered via intravenous use to pediatric subjects with relapsed or refractory acute myeloid leukemia.

Efficacy assessment in this clinical trial focuses on evaluating clinical responses in pediatric subjects with relapsed or refractory acute myeloid leukemia. Secondary endpoints include the rate of complete remission, composite complete remission consisting of CR plus CRi or CRh, and the duration of complete remission or composite complete remission.