New chemo cocktail aims to boost stem cell transplant success in blood cancers
NCT ID NCT02250937
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 2 trial tests whether adding venetoclax to a specific chemotherapy regimen before a donor stem cell transplant can improve outcomes for people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The study enrolls 116 adults aged 18-70 who have a matched donor. The goal is to see if this approach helps destroy cancer cells more effectively and leads to longer survival without the disease returning.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- chemotherapy (venetoclax, busulfan, cladribine, fludarabine) followed by donor stem cell transplant
- What this could lead to
- If successful, this approach could improve long-term survival and reduce relapse for people with AML or MDS undergoing a donor stem cell transplant.
- What could go wrong
- This is a mid-stage trial with only 116 participants, so results may not apply to everyone. The intense chemo regimen carries risks like infection, organ damage, and graft-versus-host disease.
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
-
About 116 people
The number the study aims to enrol. It can still change while the study runs.
- Started
-
Oct 2014
- Expected to finish
-
Oct 2027
An estimate. End dates often move.
- Lead sponsor
-
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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2 to 70 years
- Sex
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Anyone
- Healthy volunteers
-
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: * Patients with biopsy-proven acute myeloid leukemia or myelodysplastic syndrome with persistent disease or in remission * Human leukocyte antigen (HLA)-identical sibling or 8/8 matched unrelated donor transplant * Patients age 18 to 70 years old; eligibility for pediatric patients will be determined in conjunction with an MD Anderson Cancer Center (MDACC) pediatrician; patients age 2-17 years may be enrolled after at least 10 adults (ages 18-70 years old) have been assessed for safety at day 30 * Direct bilirubin \< 1 mg/dl * Alanine aminotransferase (ALT) \< 3 times upper limit of normal * Creatinine clearance \> 50 ml/min (calculated creatinine clearance is permitted) * Forced expiratory volume in 1 second (FEV1) \>= 50% of expected corrected for hemoglobin and/or volume * Forced vital capacity (FVC) \>= 50% of expected corrected for hemoglobin and/or volume * Diffusing capacity of the lungs for carbon monoxide (DLCO) \>= 50% of expected corrected for hemoglobin and/or volume * Children unable to perform pulmonary function tests (e.g., less than 7 years old) pulse oximetry of \>= 92% on room air * Left ventricular ejection fraction (LVEF) \>= 40% * Patient, legally authorized representative (LAR), or parent able to sign informed consent; able to give assent for patients age 7-17 * Negative beta human chorionic gonadotropin (HCG) test in a woman with child bearing potential, defined as not post-menopausal for 12 months or no previous surgical sterilization; women of child bearing potential must be willing to use an effective contraceptive measure while on study * Performance score of \>= 70 by Karnofsky/Lansky or performance status (PS) 0 or 1 (Eastern Cooperative Oncology Group \[ECOG\] =\< 1) Exclusion Criteria: * Prior allogeneic or autologous transplantation * Uncontrolled infections * Human immunodeficiency virus (HIV) seropositivity * Hematopoietic cell transplantation (HCT) co-morbidity index score \> 3; the principal investigator is the final arbiter of eligibility for comorbidity score \> 3 * Patients with prior coronary artery disease
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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
-
M D Anderson Cancer Center
Houston, Texas, 77030, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a Platelet-Boosting drug help control a rare bone marrow disorder?
- 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?