- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03572764
CPX-351 (Vyxeos™) for Transplant Eligible, Higher Risk Patients With Myelodysplastic Syndrome
April 6, 2024 updated by: Washington University School of Medicine
A Pilot Study of CPX-351 (Vyxeos™) for Transplant Eligible, Higher Risk Patients With Myelodysplastic Syndrome
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.
Study Overview
Status
Active, not recruiting
Conditions
Study Type
Interventional
Enrollment (Actual)
20
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
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Florida
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Tampa, Florida, United States, 33612
- Moffitt Cancer Center
-
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Missouri
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Saint Louis, Missouri, United States, 63110
- Washington University School of Medicine
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Washington
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Seattle, Washington, United States, 98109
- Fred Hutchinson Cancer Research Center
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 70 years (Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: CPX-351
|
-CPX-351 will be provided by Jazz Pharmaceuticals
Other Names:
-And/or buccal swab
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
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
Time Frame: Through 56 days after the last dose
|
Through 56 days after the last dose
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Overall response rate in MDS patients treated with CPX-351
Time Frame: 56 days after the last dose
|
|
56 days after the last dose
|
Best overall response in MDS patients treated with CPX-351
Time Frame: 56 days after the last dose
|
-Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
|
56 days after the last dose
|
Remission duration in MDS patients treated with CPX-351
Time Frame: Through 5 years
|
|
Through 5 years
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Relapse-free survival in MDS patients treated with CPX-351
Time Frame: Through 5 years
|
-Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
|
Through 5 years
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Progression-free survival in MDS patients treated with CPX-351
Time Frame: Through 5 years
|
|
Through 5 years
|
Overall survival in MDS patients treated with CPX-351
Time Frame: Through 5 years
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-Defined as the date of first dose of study drug to the date of death from any cause.
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Through 5 years
|
Complete remission + marrow complete remission rates in patients treated with CPX-351
Time Frame: 56 days after the last dose
|
-Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
|
56 days after the last dose
|
Post-induction mortality in MDS patients treated with CPX-351
Time Frame: Day 30
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-Rate of death
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Day 30
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Post-induction mortality in MDS patients treated with CPX-351
Time Frame: Day 60
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-Rate of death
|
Day 60
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Safety and feasibility of CPX-351 consolidation therapy in MDS patients as measured by the proportion of patients who experience an adverse event by patient, type of event, and grade of event
Time Frame: Through 56 days after the last dose
|
Through 56 days after the last dose
|
|
Proportion of MDS patients treated with CPX-351 proceeding to allogeneic hematopoietic cell transplant
Time Frame: Through 56 days after the last dose
|
Through 56 days after the last dose
|
|
Overall survival in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
Time Frame: Day 100
|
-Defined as the date of first dose of study drug to the date of death from any cause.
|
Day 100
|
Overall survival in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
Time Frame: 1 year
|
-Defined as the date of first dose of study drug to the date of death from any cause.
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1 year
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Non-relapse mortality in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
Time Frame: Day 100
|
Day 100
|
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Non-relapse mortality in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
Time Frame: 1 year
|
1 year
|
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Event-free survival in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
Time Frame: Day 100
|
|
Day 100
|
Event-free survival in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
Time Frame: 1 year
|
|
1 year
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Collaborators
Investigators
- Principal Investigator: Meagan Jacoby, M.D., Ph.D., Washington University School of Medicine
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
December 14, 2018
Primary Completion (Actual)
November 27, 2021
Study Completion (Estimated)
March 25, 2027
Study Registration Dates
First Submitted
June 18, 2018
First Submitted That Met QC Criteria
June 18, 2018
First Posted (Actual)
June 28, 2018
Study Record Updates
Last Update Posted (Actual)
April 9, 2024
Last Update Submitted That Met QC Criteria
April 6, 2024
Last Verified
April 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Neoplasms
- Disease
- Bone Marrow Diseases
- Hematologic Diseases
- Precancerous Conditions
- Syndrome
- Myelodysplastic Syndromes
- Preleukemia
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Anti-Infective Agents
- Antiviral Agents
- Enzyme Inhibitors
- Antimetabolites, Antineoplastic
- Antimetabolites
- Antineoplastic Agents
- Immunosuppressive Agents
- Immunologic Factors
- Topoisomerase II Inhibitors
- Topoisomerase Inhibitors
- Antibiotics, Antineoplastic
- Cytarabine
- Daunorubicin
Other Study ID Numbers
- 201807148
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
product manufactured in and exported from the U.S.
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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-
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AbbVieCelgene; Genentech, Inc.CompletedMyelodysplastic Syndromes (MDS)United States, Australia, Germany
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-
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Clinical Trials on CPX-351
-
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-
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Jazz PharmaceuticalsIqvia Pty LtdCompleted
-
Groupe Francophone des MyelodysplasiesCompleted
-
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-
Yale UniversityWithdrawn
-
Jazz PharmaceuticalsCompletedAcute Myeloid Leukemia | Myelodysplastic Syndromes | Acute Lymphoblastic Leukemia | Acute Lymphocytic Leukemia | Hematologic MalignancyUnited States, Canada
-
Jazz PharmaceuticalsCompletedAcute Myeloid LeukemiaUnited States
-
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