Gene test could guide leukemia treatment after chemo
NCT ID NCT02756962
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study looks at whether checking for leftover leukemia-related gene mutations after initial chemotherapy can help predict relapse and survival in adults with acute myeloid leukemia (AML). Researchers will follow 107 patients aged 18-60 who are in remission after chemo. Those whose mutations have cleared will receive standard consolidation chemo, while those with persistent mutations may get a stem cell transplant. The goal is to see if this genetic approach improves survival compared to historical averages.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Cytarabine (chemotherapy) and allogeneic stem cell transplant
- What this could lead to
- If successful, this could help doctors better predict which AML patients need more aggressive treatment after initial chemotherapy, potentially improving survival rates.
- What could go wrong
- This is a Phase 2 study with only 107 participants, so results may not apply to all patients. The approach relies on genetic testing that may not capture all mutations, and the benefit of changing treatment based on results is not yet proven.
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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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107 people
The number who actually took part.
- Started
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Jul 2016
- Expected to finish
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Jul 2029
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 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: * Age 18-60 years. * Considered to be suitable intensive (cytotoxic) induction candidates. * Has previously untreated, de novo, non-M3 AML with intermediate-risk disease (Intermediate-I or Intermediate-II) as defined by ELN criteria OR normal cytogenetics with mutated NPM1 without FLT3-ITD. Monoallelic CEBPA mutations are not considered favorable risk and are therefore eligible. * Has undergone cytotoxic induction therapy * In a morphologic complete remission with incomplete blood count recovery, or morphologic complete remission post-induction after no more than 2 induction cycles as defined by revised IWG criteria * Patients at Washington University must be enrolled in HRPO# 201011766 ("Tissue Acquisition for Analysis of Genetic Progression Factors in Hematologic Diseases").This is not a requirement for secondary sites. However, secondary sites must provide informed consent forms that document that permission for whole genome, whole exome, and/or genome wide sequencing, and data sharing among institutions, was obtained. Because we will be also be sequencing non-diseased (normal) tissue, the informed consent forms must explicitly ask if patients wish to be informed, (or in the case of their death, their next-of-kin) if a deleterious mutation is identified in their non-diseased tissue, as this may be heritable. * 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 for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately. * Able to understand and willing to sign an IRB approved written informed consent document. * Willing to comply with the treatment assignment: * Intent to proceed with HiDAC consolidation for LAM VAF \<2.5% * Intent to proceed with either HiDAC consolidation or allogeneic stem cell transplantation, at the discretion of the treating physician, for LAM ≥2.5% Exclusion Criteria: * Diagnosis acute promyelocytic leukemia (APL) with t(15;17)(q22;q12); PML-RARA. * Therapy-related AML (defined as occurrence of AML due to prior exposure to chemotherapy or radiation for malignancy). * Secondary AML (defined as development of AML in patients with an antecedent hematological malignancy). * Has a medical or psychosocial conditions that would prevent study compliance. * Known seropositivity for or 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 vaccine are eligible. * History of allergic reaction to compounds of similar chemical or biologic composition to cytarabine. * Pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 3 days of signing consent.
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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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University of Florida
Gainesville, Florida, 32608, United States
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University of Rochester
Rochester, New York, 14642, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
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