Myelodysplastic SyndromesPhase 1NCT03572764
CPX-351 (Vyxeos™) for Transplant Eligible, Higher Risk Patients With Myelodysplastic Syndrome
A Phase 1 study testing CPX-351, Research skin biopsy and Research blood draw in people with Myelodysplastic Syndromes, run by Washington University School of Medicine at 3 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- CPX-351, Research skin biopsy and Research blood draw
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 3 sitesUnited States
- Study dates
- 14 Dec 2018to 25 Mar 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Show all 12Show fewer
- 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).
Who cannot join
- 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.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109
Washington University School of Medicine
St Louis, Missouri, 63110
Moffitt Cancer Center
Tampa, Florida, 33612
About this study
Official title: 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.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- CPX-351, Research skin biopsy, Research blood draw, Research bone marrow aspirate
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CPX-351Drug
Also called Vyxeos™, Daunorubicin and cytarabine
-CPX-351 will be provided by Jazz Pharmaceuticals
Research skin biopsyProcedure or surgery
-And/or buccal swab
- Pre-treatment
- Post-induction (no earlier than Day 28 and no later than Day 56 from last induction)
Research blood drawProcedure or surgery
- 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 aspirateProcedure or surgery
- 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)
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: CPX-351; Procedure: Research skin biopsy; Procedure: Research blood draw; Procedure: Research bone marrow aspirate |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Through 56 days after the last dose
Secondary outcomes (17)
Overall response rate in MDS patients treated with CPX-351
- Overall response rate = complete remission + marrow complete remission + partial response + hematologic improvement
- Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
Measured: 56 days after the last dose
Best overall response in MDS patients treated with CPX-351
-Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
Measured: 56 days after the last dose
Remission duration in MDS patients treated with CPX-351
- Defined as the interval from the date complete remission is documented to the date of recurrence. This is determined only for patients achieving a complete remission.
- Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
Measured: Through 5 years
Relapse-free survival in MDS patients treated with CPX-351
-Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
Measured: Through 5 years
Progression-free survival in MDS patients treated with CPX-351
- Defined as the interval from the date of first dose of study drug to disease progression or death from MDS.
- Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
Measured: Through 5 years
Overall survival in MDS patients treated with CPX-351
-Defined as the date of first dose of study drug to the date of death from any cause.
Measured: Through 5 years
Complete remission + marrow complete remission rates in patients treated with CPX-351
-Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
Measured: 56 days after the last dose
Post-induction mortality in MDS patients treated with CPX-351
-Rate of death
Measured: Day 30
Post-induction mortality in MDS patients treated with CPX-351
-Rate of death
Measured: Day 60
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
Measured: Through 56 days after the last dose
Proportion of MDS patients treated with CPX-351 proceeding to allogeneic hematopoietic cell transplant
Measured: Through 56 days after the last dose
Overall survival in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
-Defined as the date of first dose of study drug to the date of death from any cause.
Measured: Day 100
Overall survival in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
-Defined as the date of first dose of study drug to the date of death from any cause.
Measured: 1 year
Non-relapse mortality in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
Measured: Day 100
Non-relapse mortality in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
Measured: 1 year
Event-free survival in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
- Defined as the interval from the date of first dose of study drug to date of treatment failure, recurrence, or death due to any cause.
- Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
Measured: Day 100
Event-free survival in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
- Defined as the interval from the date of first dose of study drug to date of treatment failure, recurrence, or death due to any cause.
- Patients will be assessed for response according to modified International Working Group (IWG) criteria for MDS
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2018
- Study start14 Dec 2018
- Primary completion27 Nov 2021
- Study completion25 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by Washington University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Jazz Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Meagan Jacoby, M.D., Ph.D., Principal investigator, Washington University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03572764
- Sponsor's protocol ID
- 201807148
- Status last verified
- Apr 2026
- First submitted
- 18 Jun 2018
- Last updated
- 14 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of CPX-351 (Vyxeos™) for Transplant Eligible, Higher Risk Patients With Myelodysplastic Syndrome
- ClinicalTrials.gov ID
- NCT03572764
- Acronym / sponsor ID
- 201807148
- Conditions
- Myelodysplastic Syndromes
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Collaborators
- Jazz Pharmaceuticals
- Study start
- 14 Dec 2018 (actual)
- Primary completion
- 27 Nov 2021 (actual)
- Study completion
- 25 Mar 2027 (estimated)
- First posted
- 28 Jun 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CPX-351 | Experimental | Drug: CPX-351, Procedure: Research skin biopsy, Procedure: Research blood draw, Procedure: Research bone marrow aspirate* 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 |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- 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)
- Total bilirubin ≤ 2.0 x IULN
- AST(SGOT)/ALT(SGPT) ≤ 3.0 x IULN
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CPX-351Also: Vyxeos™, Daunorubicin and cytarabine-CPX-351 will be provided by Jazz Pharmaceuticals | Drug | CPX-351 |
| 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) | Procedure or surgery | CPX-351 |
| 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) | Procedure or surgery | CPX-351 |
| 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) | Procedure or surgery | CPX-351 |
Primary outcome
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
17 secondary outcomes
Overall response rate in MDS patients treated with CPX-351
Time frame: 56 days after the last dose
Best overall response in MDS patients treated with CPX-351
Time frame: 56 days after the last dose
Remission duration in MDS patients treated with CPX-351
Time frame: Through 5 years
Relapse-free survival in MDS patients treated with CPX-351
Time frame: Through 5 years
Progression-free survival in MDS patients treated with CPX-351
Time frame: Through 5 years
Overall survival in MDS patients treated with CPX-351
Time frame: Through 5 years
Complete remission + marrow complete remission rates in patients treated with CPX-351
Time frame: 56 days after the last dose
Post-induction mortality in MDS patients treated with CPX-351
Time frame: Day 30
Post-induction mortality in MDS patients treated with CPX-351
Time frame: Day 60
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
Proportion of MDS patients treated with CPX-351 proceeding to allogeneic hematopoietic cell transplant
Time frame: 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
Overall survival in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
Time frame: 1 year
Non-relapse mortality in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
Time frame: Day 100
Non-relapse mortality in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
Time frame: 1 year
Event-free survival in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
Time frame: Day 100
Event-free survival in MDS patients treated with CPX-351 in patients undergoing allogeneic hematopoietic cell transplant
Time frame: 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Apr 2026.
- Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of MedicineLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Meagan Jacoby, M.D., Ph.D. principal investigator
Washington University School of Medicine
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CPX-351, Research skin biopsy and Research blood draw in people with Myelodysplastic Syndromes, run by Washington University School of Medicine at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 14 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Apr 2026.