CPX-351 (Vyxeos™) for Transplant Eligible, Higher Risk Patients With Myelodysplastic Syndrome

NCT ID: NCT03572764

Last Updated: 2025-04-09

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-12-14
• Study Completion Date: 2027-03-25

Brief Summary

This is a pilot and feasibility study of transplant eligible, higher risk myelodysplastic syndrome (MDS) patients to determine the safety and tolerability of a lower -dose and higher-dose CPX-351 regimen, with secondary objectives including complete remission (CR) rates and proportion of patients proceeding to transplant.

Conditions

Myelodysplastic Syndromes

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

CPX-351

• CPX-351 will be given according to the assigned dose level over a minimum of a 90-minutes via IV infusion on Days 1, 3, and 5 of the first induction
• If the treating physician elects to perform a day 14 bone marrow biopsy then, a second induction may be considered for patients in the absence of a chemoablated, hypocellular marrow on the Day 14 bone marrow assessment, if the patient has failed to achieve a marrow CR, and it is deemed safe to administer by the treating physician. The second induction uses a modified schedule in which CPX-351 will be given according to the assigned dose level on Days 1 and 3
• In the absence of disease progression or unacceptable toxicity, the patient may continue to consolidation at the discretion of the treating physician or the patient may proceed to alloHCT after induction at the discretion of the treating physician

Group Type: EXPERIMENTAL

CPX-351

Intervention Type: DRUG

-CPX-351 will be provided by Jazz Pharmaceuticals

Research skin biopsy

Intervention Type: PROCEDURE

-And/or buccal swab

• Pre-treatment
• Post-induction (no earlier than Day 28 and no later than Day 56 from last induction)

Research blood draw

Intervention Type: PROCEDURE

• Pre-treatment
• Post-induction (no earlier than Day 28 and no later than Day 56 from last induction)
• Post-consolidation 1 (if applicable)
• Post-consolidation 2 (if applicable)
• Post-transplant Day 30 (if applicable)
• Post-transplant Day 100 (if applicable)

Research bone marrow aspirate

Intervention Type: PROCEDURE

• Pre-treatment
• Post-induction (no earlier than Day 28 and no later than Day 56 from last induction)
• Post-consolidation 1 (if applicable)
• Post-consolidation 2 (if applicable)
• Post-transplant Day 30 (if applicable)
• Post-transplant Day 100 (if applicable)

Interventions

CPX-351

-CPX-351 will be provided by Jazz Pharmaceuticals

Intervention Type: DRUG

Research skin biopsy

-And/or buccal swab

• Pre-treatment
• Post-induction (no earlier than Day 28 and no later than Day 56 from last induction)

Intervention Type: PROCEDURE

Research blood draw

• Pre-treatment
• Post-induction (no earlier than Day 28 and no later than Day 56 from last induction)
• Post-consolidation 1 (if applicable)
• Post-consolidation 2 (if applicable)
• Post-transplant Day 30 (if applicable)
• Post-transplant Day 100 (if applicable)

Intervention Type: PROCEDURE

Research bone marrow aspirate

• Pre-treatment
• Post-induction (no earlier than Day 28 and no later than Day 56 from last induction)
• Post-consolidation 1 (if applicable)
• Post-consolidation 2 (if applicable)
• Post-transplant Day 30 (if applicable)
• Post-transplant Day 100 (if applicable)

Intervention Type: PROCEDURE

Other Intervention Names

Vyxeos™; Daunorubicin and cytarabine

Eligibility Criteria

Inclusion Criteria

• Diagnosis of myelodysplastic syndrome (MDS) with an IPSS-R score of Intermediate, High or Very High (see Appendix A) AND ≥ 5% myeloblasts in the bone marrow.
• Age 18-70 years.
• ECOG performance status ≤ 2 (see Appendix B)

Adequate renal and hepatic function as defined below:

*Total bilirubin ≤ 2.0 x IULN*

• AST(SGOT)/ALT(SGPT) ≤ 3.0 x IULN
• Serum creatinine ≤ 2.0 mg/dL
• Note: If, in the opinition of the treatment physician, the bilirubin is elevated secondary to hemolysis or Gilbert's disease, the patient may be eligible after discussion with the Washington University PI.

• Left ventricular cardiac ejection fraction ≥ 50% by echocardiography or MUGA.
• Deemed by the treating physician to be a suitable candidate for cytotoxic induction therapy and an alloHCT candidate at the time of enrollment.
• Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control, abstinence) prior to study entry and continuing until 30 days after the last study treatment.
• Ability to understand and willingness to sign an IRB approved written informed consent document (or that of legally authorized representative, if applicable).

Exclusion Criteria

• Prior treatment for MDS with disease-modifying therapy (conventional or investigational) (i.e. hypomethylator therapy, lenalidomide, or prior AML-like induction therapy intended for the therapy of MDS). Use of prior growth factor and ESA support is permitted.
• Currently receiving any other investigational agents.
• A history of allergic reactions attributed to compounds of similar chemical or biologic composition to CPX-351 or other agents used in the study.
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia.
• History of Wilson's disease or other copper-metabolism disorder.
• Pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 14 days of study entry.
• Known active viral infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV). Patients who are seropositive because of hepatitis B virus vaccine are eligible. Patients who are seropositive for HCV but have a negative viral load are also eligible provided that the patient has completed a course of therapy for HCV.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Jazz Pharmaceuticals

INDUSTRY

Sponsor Role: collaborator

Washington University School of Medicine

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Meagan Jacoby, M.D., Ph.D.

Role: PRINCIPAL_INVESTIGATOR

Washington University School of Medicine

Locations

Moffitt Cancer Center

Tampa, Florida, United States

Washington University School of Medicine

St Louis, Missouri, United States

Fred Hutchinson Cancer Research Center

Seattle, Washington, United States

Countries

United States

Related Links

http://www.siteman.wustl.edu

Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine

Other Identifiers

201807148

Identifier Type: -

Identifier Source: org_study_id