New drug cocktail aims to beat back tough leukemia
NCT ID NCT03629171
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase II trial is testing whether combining two drugs—CPX-351 (a chemotherapy) and venetoclax (a targeted therapy)—can help people with acute myeloid leukemia (AML) that has come back or not responded to prior treatment. About 52 adults will take part at MD Anderson Cancer Center. The goal is to see if this combo can achieve remission and how safe it is.
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 (a liposomal combination of two chemotherapy drugs, daunorubicin and cytarabine) and venetoclax (a targeted drug)
- What this could lead to
- If successful, this combination could offer a new treatment option for people with AML that has come back or not responded to other therapies.
- What could go wrong
- This is a small, early-phase (Phase II) trial, so results may not apply broadly. Side effects from chemotherapy and venetoclax can be serious, including infection and bleeding risks.
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 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 52 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Oct 2018
- Expected to finish
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Dec 2026
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 years and older
- 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: * For the lead in phase: Patients \>= 18 years of age with a diagnosis of relapsed and/or refractory AML will be eligible. Patients who have had prior treatment with venetoclax will be allowed to participate in the lead in phase and cohort A * For the dose expansion cohort A (relapsed/refractory \[R/R\] AML): Patients \>= 18 years of age with a diagnosis of relapsed and/or refractory AML will be eligible * For the dose expansion cohort B (de novo AML): Patients \>= 18 years to 69 years of age; patients in this cohort must have received no prior therapy for AML * Prior therapy with hydroxyurea, hematopoietic growth factors, or tretinoin (ATRA) (for emergency use for stabilization) is allowed with no washout. A cumulative dose of ara-C of up to 3 g for emergency stabilization in patients with rapidly proliferating disease is also allowed provided it was administered \> 48 hrs prior to enrollment * Bilirubin =\< 2 mg/dL * Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) =\< 3 x upper limit of normal (ULN) or \< 5 x ULN if related to leukemic involvement * Creatinine =\< 1.5 x ULN * Known cardiac ejection fraction of \> or = 45% within the past 3 months * Eastern Cooperative Oncology Group (ECOG) performance status of =\< 2 * A negative urine or serum pregnancy test is required within 1 week for all women of childbearing potential prior to enrolling on this trial. A woman of childbearing potential is defined as a woman who has not been naturally postmenopausal for at least 12 consecutive months, or who had no previous surgical sterilization * Patient must have the ability to understand the requirements of the study and signed informed consent. A signed informed consent by the patient or his legally authorized representative is required prior to their enrollment on the protocol Exclusion Criteria: * Pregnant women are excluded from this study because the agents used in this study have the potential for teratogenic or abortifacient effects. Because there is a potential risk for adverse events in nursing infants secondary to treatment of the mother with the chemotherapy agents, breastfeeding should also be avoided * Uncontrolled intercurrent illness including, but not limited to active uncontrolled infection, symptomatic congestive heart failure (New York Heart Association \[NYHA\] class III or IV), unstable angina pectoris, clinically significant cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements * Patient with documented hypersensitivity to any of the components of the chemotherapy program * Patients with acute promyelocytic leukemia (M3) or core-binding factor AML * Patients with active central nervous system (CNS) leukemia are excluded since the antileukemia activity of the treatment components against CNS leukemia are not known * Men and women of childbearing potential who do not practice contraception. Women of childbearing potential and men must agree to use contraception prior to study entry and for the duration of study participation * Prior treatment with CPX-351 or venetoclax. Patients with prior treatment with venetoclax will be allowed in patients with relapsed/refractory (R/R) disease including those in the lead-in phase as well as those in cohort A
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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M D Anderson Cancer Center
RECRUITINGHouston, Texas, 77030, United States
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