New drug combo aims to stop blood cancer relapse after transplant
NCT ID NCT07183878
First seen Jun 25, 2026 · Last updated Sep 03, 2026 · Updated 3 times
Summary
This study tests whether adding the targeted drug venetoclax to standard chemotherapy before a stem cell transplant can help prevent cancer from returning in people with high-risk acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). About 138 participants aged 12 to 60 will be randomly assigned to receive either the enhanced or standard chemo regimen. The main goal is to see if the new combo improves how long patients stay cancer-free after transplant.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Venetoclax (a targeted cancer drug) added to standard chemotherapy (busulfan and cyclophosphamide)
- What this could lead to
- If it works, this could lower the chance of cancer returning after a stem cell transplant for people with high-risk AML or MDS.
- What could go wrong
- This is a mid-stage trial with 138 participants, so results are not yet proven. Adding venetoclax may increase side effects like infections or 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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Not a phased trial
Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.
- Participants
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About 138 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Aug 2025
- Expected to finish
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Aug 2028
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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12 to 60 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 acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) according to 2022 WHO classification * Age between 12 and 60 years * High-risk MDS as defined by at least one of the following: * IPSS intermediate-2/high risk or IPSS-R intermediate/high/very high risk * TP53 mutation * RAS pathway mutation (e.g., NRAS, KRAS, PTPN11, CBL, NF1, RIT1, FLT3, KIT) * Therapy-related MDS * High-risk AML as defined by at least one of the following: * TP53, RUNX1, or ASXL1 mutation * t(6;9)(p23;q34.1)/DEK-NUP214 * KMT2A rearrangement * BCR-ABL1 fusion * inv(3)(q21.3q26.2) or t(3;3)(q21.3;q26.2) * -5/del(5q), -7, -17/abn(17p) * Complex or monosomal karyotype * FLT3-ITD high with wild-type NPM1 * Initial WBC ≥ 10×10\^9/L * Secondary AML with history of MDS/MPN or therapy-related AML * AML with specific mutations (SRSF2, SF3B1, U2AF1, ZRSR2, ASXL1, EZH2, BCOR, STAG2) * MRD positive before transplantation * For AML: must have achieved CR or CRi prior to transplantation; for MDS: bone marrow blasts \< 20% * Availability of a matched related or unrelated donor (10/10 or 9/10 HLA match) * ECOG performance status 0-2 * Creatinine clearance ≥ 60 mL/min (Cockcroft-Gault) * AST/ALT ≤ 3 × ULN and total bilirubin ≤ 2 × ULN * LVEF ≥ 50% by echocardiogram * Life expectancy \> 8 weeks * Willingness to use effective contraception methods during and for a specified period after the study * Signed informed consent Exclusion Criteria: * Uncontrolled cardiovascular disease or New York Heart Association class III/IV heart failure * Other severe comorbid conditions that may interfere with study participation * Known HIV infection or uncontrolled active hepatitis B or C * Pregnant or breastfeeding women * More than one prior hematopoietic stem cell transplantation * Inability to understand the study protocol or provide informed consent * History of grade ≥ 3 non-hematologic adverse reaction to prior venetoclax therapy * Receipt of chemotherapy (except hydroxyurea/dexamethasone) or radiotherapy within 14 days before study treatment * Ongoing use of BCR-ABL1, IDH, or FLT3 inhibitors without proper washout (≥ 7 days)
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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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The First Affiliated Hospital of Zhejiang University School of Medicine
RECRUITINGHangzhou, Zhejiang, 310006, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can adding venetoclax make donor stem cell transplants safer for High-Risk blood cancers?
- Can an HDAC inhibitor wipe out residual leukemia cells?
- Can an experimental pill block a cancer-driving enzyme in hard-to-treat leukemia?
- Two-Drug combo targets leukemia that outsmarted its first treatment
- Tweaking donor cells may shield older transplant patients from a dangerous complication
- Can a drug and donor cells stop leukemia from returning after transplant?