Experimental chemo combo aims to boost transplant success in MDS
NCT ID NCT03572764
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase study tests CPX-351 (Vyxeos), a two-drug chemotherapy, in 20 adults aged 18-70 with higher-risk myelodysplastic syndrome (MDS) who are eligible for a stem cell transplant. The main goal is to see if the drug is safe and tolerable, with secondary goals of measuring remission rates and how many patients can proceed to transplant. Researchers will also collect blood, bone marrow, and skin samples to understand how the treatment works.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- CPX-351 (Vyxeos), a combination of two chemotherapy drugs (daunorubicin and cytarabine)
- What this could lead to
- If this works, it could help more high-risk MDS patients achieve remission and proceed to a stem cell transplant, potentially improving long-term outcomes.
- What could go wrong
- This is a very early (Phase 1) pilot study with only 20 participants, so results may not apply broadly. Chemotherapy carries risks like infection and organ damage.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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20 people
The number who actually took part.
- Started
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Dec 2018
- Expected to finish
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Mar 2027
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 70 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109, United States
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Moffitt Cancer Center
Tampa, Florida, 33612, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
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