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

NCT03572764 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: 201807148
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 3

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

Outcome Measures

Primary Outcomes (1)

• Safety and tolerability of a CPX-351 regimen in a transplant eligible, higher risk MDS population as measured by the proportion of participants who experience an adverse event by patient, type of event, and grade of event

  Through 56 days after the last dose

Secondary Outcomes (17)

• Overall response rate in MDS patients treated with CPX-351

  56 days after the last dose

• Best overall response in MDS patients treated with CPX-351

  56 days after the last dose

• Remission duration in MDS patients treated with CPX-351

  Through 5 years

• Relapse-free survival in MDS patients treated with CPX-351

  Through 5 years

• Progression-free survival in MDS patients treated with CPX-351

  Through 5 years

Study Arms (1)

CPX-351

EXPERIMENTAL

* 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

Drug: CPX-351; Procedure: Research skin biopsy; Procedure: Research blood draw; Procedure: Research bone marrow aspirate

Interventions

CPX-351 DRUG

-CPX-351 will be provided by Jazz Pharmaceuticals

Also known as: Vyxeos™, Daunorubicin and cytarabine

CPX-351

Research skin biopsy PROCEDURE

-And/or buccal swab * Pre-treatment * Post-induction (no earlier than Day 28 and no later than Day 56 from last induction)

CPX-351

Research blood draw 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)

CPX-351

Research bone marrow aspirate 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)

CPX-351

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Washington University School of Medicine: lead
• Jazz Pharmaceuticals: collaborator

Study Sites (3)

Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Fred Hutchinson Cancer Research Center

Seattle, Washington, 98109, United States

Location

Related Links

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

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

CPX-351; Daunorubicin; Cytarabine

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Anthracyclines; Naphthacenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polycyclic Compounds; Aminoglycosides; Glycosides; Carbohydrates; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Arabinonucleosides; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides

Study Officials

• Meagan Jacoby, M.D., Ph.D.

  Washington University School of Medicine

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 18, 2018
• First Posted: June 28, 2018
• Study Start: December 14, 2018
• Primary Completion: November 27, 2021
• Study Completion (Estimated): March 25, 2027
• Last Updated: April 14, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)