NCT05656248

Study of CPX-351 (VYXEOS) in Individuals < 22 Years With Secondary Myeloid Neoplasms

A Prospective,Single Site, Single-Arm Pilot Study of CPX-351 (VYXEOS) in Individuals < 22 Years With Secondary Myeloid Neoplasms

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 25 (actual)
• Sponsor: St. Jude Children's Research Hospital · Academic / Other
• Sex: All
• Age: 1 Year – 21 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to learn the effects of treatment with an investigational drug, CPX-351 in patients with secondary myeloid neoplasms (SMNs).

Detailed description

Primary Objective * Determine the composite complete remission (CR) and complete remission with incomplete peripheral blood recovery (CRi) rates, safety and tolerability in patients under 22 years of age with SMN treated with one or two courses of CPX-351 before HSCT. Secondary Objectives * Describe the toxicity profile of patients with SMN treated with one or two courses of CPX-351. * Describe the biologic correlates of response in patients with SMN after one or two courses of CPX-351. * Estimate the 3-year overall survival of patients who received one or two courses of CPX-351 followed by HSCT. Participants who meet the inclusion criteria and consent will receive up to 2 cycles of CPX-351 for remission induction, and then will proceed to allogeneic HSCT or other therapies as per institutional practice. If a patient attains remission and has negative MRD after the first course of CPX-351, and HSCT can occur within 3 to 4 weeks from the evaluation date of the first course, the patient can proceed to HSCT without receiving the second course of CPX-351.

Conditions

• Myeloid Neoplasm

Interventions

Type	Name	Description
DRUG	CPX-351	Given Intraveneously (IV)
DRUG	MHA	Given Intrathecal (IT)
PROCEDURE	Allogeneic Hematopoietic Stem Cell Transplantation	Undergo HSCT

Timeline

• Start date: 2023-01-17
• Primary completion: 2025-08-28
• Completion: 2028-08-01
• First posted: 2022-12-19
• Last updated: 2026-02-04

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study