NCT04668885

CPX-351 as a Novel Approach for the Treatment of Older Patients With AML and MDS

Lower Doses of CPX-351 as a Novel Approach for the Treatment of Older Patients With Relapsed or Refractory Acute Myeloid Leukemia and Myelodysplastic Syndromes

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: Case Comprehensive Cancer Center · Academic / Other
• Sex: All
• Age: 60 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate how effective lower doses of CPX-351 are in older participants with relapsed/refractory acute myeloid leukemia (AML) who are not eligible to receive intensive chemotherapy and in participants with myelodysplastic syndromes (MDS) after Hypomethylating Agents (HMA) failure.

Detailed description

Currently, elderly patients with AML and high risk MDS, who are ineligible to receive induction chemotherapy and fail HMA +/- combination, have very poor outcomes and there is no FDA-approved therapy outside of some targeted therapies which can only be applied to a small patient population. CPX-351 is an investigational (experimental) drug that works by combining two anti-cancer drugs cytarabine and daunorubicin. CPX-351 is experimental because it is only FDA approved for the treatment of adults with two types of AML: newly diagnosed therapy-related AML or AML with myelodysplasia-related changes. This is an open label clinical trial of lower doses of CPX-351 in relapsed/primary refractory older AML and MDS patients ineligible to receive intensive chemotherapy. The first arm is for particpants with primary refractory/relapsed AML and the second arm is for higher risk MDS participants after HMA failure.

Conditions

• Refractory Acute Myeloid Leukemia
• Relapsed Acute Myelomonocytic Leukemia
• Myelodysplastic Syndromes

Interventions

Type	Name	Description
DRUG	CPX-351	Induction phase: CPX-351 15 mg/m\^2 on days 1 and 3 of each 28-day cycle for up to a total of 6 cycles in the absence of unacceptable toxicity Maintenance phase: CPX-351 7.5 mg/m\^2 on days 1 and 3 for two cycles alternating with 15 mg/m\^2 for one cycle. Participants may receive up to 12 cycles of maintenance phase in the absence of unacceptable toxicity.

Timeline

• Start date: 2021-01-14
• Primary completion: 2025-10-10
• Completion: 2026-10-10
• First posted: 2020-12-16
• Last updated: 2025-07-15

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study