Acute Myeloid LeukemiaPhase 2NCT04214249
BLAST MRD AML-1: BLockade of PD-1 Added to Standard Therapy to Target Measurable Residual Disease in Acute Myeloid Leukemia 1- A Randomized Phase 2 Study of Anti-PD-1 Pembrolizumab in Combination With Intensive Chemotherapy as Frontline Therapy in Patients With Acute Myeloid Leukemia
A Phase 2 study testing Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy in people with Acute Myeloid Leukemia and Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, run by National Cancer Institute (NCI) at 10 sites in United States.
Trial summary
This phase II trial studies how well cytarabine and idarubicin or daunorubicin with or without pembrolizumab work in treating patients with newly-diagnosed acute myeloid leukemia. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Acute Myeloid Leukemia Post Cytotoxic Therapy
- What is being tested?
- Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 49 participants
- Where
- 10 sitesUnited States
- Study dates
- 17 Feb 2021to 30 Jan 2027
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Newly diagnosed and pathologically-confirmed AML, confirmed by a bone marrow aspirate and/or biopsy and/or peripheral blood with >= 20% myeloid blasts. Bone marrow biopsy, or aspirate or peripheral blood that were obtained up to 3 weeks before signing consent are allowed for purposes of confirming AML diagnosis for eligibility purposes. Secondary AML (that is arising from prior myelodysplastic syndrome [MDS] as well as therapy-related [t]-AML) are also allowed. Clarifications: AML arising from myeloproliferative neoplasms (MPN), MPN/MDS overlap (including chronic myelomonocytic leukemia [CMML]) or another myeloid malignancy are NOT allowed. Note 1: Patients must have evidence of bone marrow involvement on aspirate or biopsy. Patients with only extramedullary disease and no bone marrow involvement will be excluded. Note 2: Every effort should be made to get an aspirate for central flow assessment at screening and all subsequent required time points, but in cases where an aspirate cannot be collected-including dry taps-the patient will not be excluded and assessments will be performed on peripheral blood (PB) which should be collected at every time that bone marrow (BM) is collected. Note 3: Some patients with AML require initiation of therapy quickly after diagnosis, and full metaphase karyotype results in some centers can take 2-3 weeks to result. To avoid this issue being an impediment to accrual to study or to cause delays in initiation of therapy in patients who need fast initiation of therapy, we allow use of karyotype and/or fluorescence in situ hybridization (FISH) results (as well as FLT3 results) on samples from blood or marrow that were obtained up to 3 weeks before signing consent for purposes of eligibility and stratification. In any case, results from FISH or karyotype should show if core-binding factor (CBF) abnormalities are present by time of randomization as the presence of CBF abnormalities is a required stratification factor
- Age >= 18 and =< 75 years
- Because no dosing or adverse event (AE) data are currently available on the use of pembrolizumab (MK-3475) in patients < 18 years of age, children are excluded from this study, but will be eligible for future pediatric trials
- Eastern Cooperative Oncology Group (ECOG) performance status =< -2
- The patient has to be eligible to receive intensive "7+3" induction chemotherapy as judged by the treating physician
- Prior use of hypomethylating agents (HMA), lenalidomide, erythropoiesis-stimulating agents (ESAs), and growth factors is allowed if used to treat prior MDS. AML must be previously untreated except as outlined below (hydroxyurea, or tretinoin [ATRA], or leukapheresis). Note: One dose of prophylactic intrathecal therapy is allowed during or before screening if a lumbar puncture is performed to rule out central nervous system (CNS) involvement
Show all 23Show fewer
- Hydroxyurea/leukapheresis allowed for control of hyperleukocytosis but hydroxyurea must be discontinued day prior to start of chemotherapy
- Creatinine =< 1.5 x upper limit of normal (ULN) OR measured or calculated creatinine clearance (CrCl) >= 60 mL/min for patient with creatinine levels > 1.5 x institutional ULN (within 3 days prior to the first day of 7+3)
- Creatinine clearance (CrCl) should be calculated per institutional standard
- Glomerular filtration rate (GFR) can also be used in place of creatinine or CrCl
- Total bilirubin =< 1.5 x ULN or direct bilirubin =< ULN for patients with total bilirubin levels > 1.5 x ULN (within 3 days prior to the first day of 7+3)
- Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT]) and alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 3 x ULN OR =< 5 x ULN for patients with liver metastases (within 3 days prior to the first day of 7+3)
- International normalized ratio (INR) or prothrombin time (PT) =< 1.5 x ULN unless patient is receiving anticoagulant therapy as long as PT or partial thromboplastin time (PTT) is within therapeutic range of intended use of anticoagulants (within 3 days prior to the first day of 7+3)
- Activated partial thromboplastin time (aPTT) =< 1.5 x ULN unless patient is receiving anticoagulant therapy as long as PT or PTT is within therapeutic range of intended use of anticoagulants (within 3 days prior to the first day of 7+3)
- Patients with a known history of being human immunodeficiency virus (HIV) positive may participate IF they meet the following eligibility requirements:
- They must be stable on their anti-retroviral regimen, and they must be healthy from an HIV perspective
- Patients must have an undetectable HIV viral load
- Patients with a known history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load. For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated
- Patients who have undergone major surgery must have recovered adequately from the toxicity and/or complications from the intervention prior to starting therapy
- Patients with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association functional classification. To be eligible for this trial, patients should be class 2B or better
- Female patients of childbearing potential must have a negative urine or serum pregnancy test within 72 hours prior to receiving the first dose of 7+3 treatment. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required. A female of childbearing potential is any woman, regardless of sexual orientation or whether they have undergone tubal ligation, who meets the following criteria: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 12 consecutive months (i.e., has had menses at any time in the preceding 12 consecutive months). Female patients of childbearing potential must be willing to use an adequate method of contraception for the course of the study through 120 days after the last dose of study medication. Male patients with female partners of childbearing potential must agree to use an adequate method of contraception, starting with the first dose of study therapy through 120 days after the last dose of study therapy
- NOTE: Abstinence is acceptable if this is the usual lifestyle and preferred contraception for the patient
- Ability to understand and the willingness to sign a written informed consent document. Participants with impaired decision-making capacity (IDMC) who have a legally-authorized representative (LAR) and/or family member available will also be eligible
Who cannot join
- Patients with a known additional malignancy that is progressing or requires active treatment. Exceptions include basal cell carcinoma of the skin or squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer. Prior treatment with the following are not allowed:
- Patients who have received anthracyclines for treatment of a prior, unrelated, curatively-treated malignancy which would limit their ability to receive 7 + 3 chemotherapy treatment on study
- Anti-PD-1, anti-PD-L1, or anti-PD-L2, for a prior, unrelated, curatively-treated malignancy, within last 3 months of enrollment in the study
- Anti-cancer monoclonal antibody (mAb) within 4 weeks, for a prior, unrelated, curatively-treated malignancy, prior to study registration or have not recovered (recovery defined as baseline or =< grade 1) from AEs due to agents administered more than 4 weeks earlier
- Experimental treatment within 4 weeks prior to study registration
- Patients who have had chemotherapy (except hydroxyurea and all trans retinoic acid [ATRA] which are allowed but have to be stopped the day before induction therapy starts), targeted small molecule therapy (aside from imatinib, dasatinib, or nilotinib), or curative-intent radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C), for a prior curatively treated malignancy, prior to entering the study
Show all 35Show fewer
- Patients who have received prior anthracyclines not to exceed 150 mg/m^2 of daunorubicin or equivalent for treatment of a prior, unrelated, curatively-treated malignancy which would limit their ability to receive 7 + 3 chemotherapy treatment on study
- Patients with a cardiac ejection fraction less than 50% as determined by echocardiogram or radionuclide ventriculogram scan (MUGA) scan
- Other active primary malignancy (other than non-melanomatous skin cancer or carcinoma in situ of the cervix) requiring treatment or limiting expected survival to =< 2 years
- NOTE: If there is a history of prior malignancy, they must not be receiving other specific treatment (other than hormonal therapy for their cancer)
- Patients who have FLT3-mutated AML
- FLT3-ITD or TKD mutations are defined as a mutation with a ratio of mutant to wild-type allele >= 0.05 or variant allele fraction of >= 5% by polymerase chain reaction (PCR) or next generation sequencing from either bone marrow or peripheral blood
- Note 1: FLT3, karyotype, or FISH results from bone marrow or peripheral blood that were performed up to 3 weeks before initiation of trial therapy are acceptable for eligibility determination or therapy stratification as long as they are performed in a Clinical Laboratory Improvement Act (CLIA) certified laboratory. Note 2: Patients are stratified based on age (younger than 65 versus [vs] 65 and older), presence of core-binding abnormalities by FISH or karyotype (yes/no), and by having t- AML or AML arising from prior/antecedent MDS (yes/no)
- Patients who have not recovered from AEs due to prior anti-cancer therapy (i.e., have not returned to baseline or have residual toxicities > grade 1) with the exception of =< grade 2 neuropathy and alopecia
- NOTE: Participants must have recovered from all radiation-related toxicities, not require corticosteroids, and not have had radiation pneumonitis. A 1-week washout is permitted for palliative radiation (=< 2 weeks of radiotherapy) to non-CNS disease
- Patients currently participating and receiving study therapy or have participated in a study of an investigational agent and received study therapy or used an investigational device within 4 weeks of the first dose of treatment are ineligible
- History of hypersensitivity to pembrolizumab (MK-3475) or any of its excipients, or other agents used in this study
- Current use of corticosteroids
- EXCEPTION: Low doses of steroids (< 10 mg of prednisone or equivalent dose of other steroid) used for treatment of non-hematologic medical condition (e.g., chronic adrenal insufficiency) is permitted
- Patients who underwent prior allogenic transplant
- Patient with a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the subject's participation for the full duration of the trial, or is not in the best interest of the subject to participate, in the opinion of the treating investigator
- Patient with known active CNS disease and/or carcinomatous meningitis before study enrollment. Assessment of the cerebral spinal fluid (CSF) is not required to enroll in the study unless there is clinical suspicion for CNS involvement. However, if CSF assessment is performed for any reason, there should be no evidence of active leukemia in the CSF as per investigator judgement. Up to one dose of prophylactic intrathecal chemotherapy is allowed prior to study enrollment. Subjects with previously treated brain metastases may participate provided they are stable (without evidence of progression by imaging for at least four weeks prior to the first dose of protocol treatment and any neurologic symptoms have returned to baseline), have no evidence of new or enlarging brain metastases, and are not using steroids for at least 7 days prior to protocol treatment. This exception does not include carcinomatous meningitis which is excluded regardless of clinical stability
- Patients with active autoimmune disease except for patients with hypothyroidism and vitiligo that has required systemic treatment in the past 2 years (i.e., with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment
- Patients with a known history of non-infectious pneumonitis that required the use of steroids or current pneumonitis
- Patients with active, uncontrolled infection as deemed by the treating investigator
- Patients with a known history of active TB (Bacillus tuberculosis)
- Patients with uncontrolled intercurrent illness
- Patients with psychiatric illness/social situations that would limit compliance with study requirements
- Pregnant women are excluded from this study because pembrolizumab (MK-3475) is humanized antibody with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with pembrolizumab (MK-3475), breastfeeding should be discontinued if the mother is treated with pembrolizumab (MK-3475). These potential risks may also apply to other agents used in this study
- Patient who have received a live vaccine within 30 days of planned start of study therapy
- NOTE: Seasonal influenza vaccines for injection are generally inactivated flu vaccines and are allowed; however intranasal influenza vaccines (e.g., Flu-Mist [registered trademark]) are live attenuated vaccines, and are not allowed
- Active hemolytic anemia requiring immunosuppressive therapy or other pharmacologic treatment. Patients who have a positive Coombs test but no evidence of hemolysis are NOT excluded from participation
- Patients with clinically significant disseminated intravascular coagulation (DIC), which cannot be managed with supportive care including transfusions, as assessed by treating physician, will be excluded from study
- Patients with no bone marrow involvement will be excluded (i.e., those with only extramedullary disease)
- Patients with acute promyelocytic leukemia will be excluded
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
University of Alabama at Birmingham Cancer Center
Birmingham, Alabama, 35233
Northwestern University
Chicago, Illinois, 60611
Wake Forest University at Clemmons
Clemmons, North Carolina, 27012
Mayo Clinic in Florida
Jacksonville, Florida, 32224-9980
Dartmouth Hitchcock Medical Center/Dartmouth Cancer Center
Lebanon, New Hampshire, 03756
Yale University
New Haven, Connecticut, 06520
UC Irvine Health/Chao Family Comprehensive Cancer Center
Orange, California, 92868
VCU Massey Comprehensive Cancer Center
Richmond, Virginia, 23298
Wake Forest Baptist Health - Wilkes Medical Center
Wilkesboro, North Carolina, 28659
Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157
About this study
Official title: BLockade of PD-1 Added to Standard Therapy to Target Measurable Residual Disease in Acute Myeloid Leukemia 1 (BLAST MRD AML-1): A Randomized Phase 2 Study of the Anti-PD-1 Antibody Pembrolizumab in Combination With Conventional Intensive Chemotherapy as Frontline Therapy in Patients With Acute Myeloid Leukemia
This phase II trial studies how well cytarabine and idarubicin or daunorubicin with or without pembrolizumab work in treating patients with newly-diagnosed acute myeloid leukemia. Chemotherapy drugs, such as cytarabine, idarubicin, and daunorubicin, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving induction chemotherapy with pembrolizumab may work better than induction chemotherapy alone in treating patients with acute myeloid leukemia.
Read the detailed description
PRIMARY OBJECTIVE:
- To assess the percentage of patients with minimal residual disease (MRD) negative complete remission (CR) (MRD-CR), or with minimal residual disease (MRD) negative complete remission with incomplete recovery (CRi) (MRD-CRi) as measured by flow cytometry at the end of first cycle of consolidation therapy with chemotherapy + pembrolizumab (MK-3475) and compare between the two study arms.
SECONDARY OBJECTIVES:
- Assess the rate of complete remission (CR)/complete remission with incomplete count recovery (CRi) as defined per European Leukemia Net 2017 response criteria at time of count recovery after induction therapy with chemotherapy + pembrolizumab (MK-3475) (Dohner et al., 2017).
II. Rates of complete remission with partial recovery count (CRh) and hematologic improvement (HI) to red blood cells and platelets.
III. Assess the rates of MRD negativity at day 14, MRD-negative CR at end of induction therapy and MRD negative CR after last consolidation cycle.
IV. Assess event free survival (EFS), measured from randomization to failure to achieve CR/CRi, relapse or death from any cause, and relapse free survival (RFS), calculated as the time from first documentation of CR/CRi to either disease relapse or death from any cause.
- Assess the duration of response (DOR, defined as the time from first CR/CRi to the date of the first documented relapse or death, whichever occurs first) and overall survival (OS), defined as time from randomization to death from any cause.
VI. Assess safety endpoints including proportion of patients who develop severe toxicity as defined in the protocol.
EXPLORATORY OBJECTIVES:
- MRD assessment by duplex sequencing (DS) and comparing DS and multiparameter flow cytometry for MRD detection as an exploratory biomarker.
II. Assessment of immune-checkpoint expression and dynamic change of immune cell subsets in response to the combination of checkpoint-inhibition and backbone combination in acute myeloid leukemia (AML).
III. High-throughput sequencing of the T-cell receptor (TCR) Vb CDR3 regions on flow cytometrically sorted t-cell subsets to assess the effect of immunotherapy on the diversity of the t-cell repertoire and assess for correlation to clinical outcomes.
IV. Investigation of protein signatures and ribonucleic acid (RNA) signatures associated with response and efficacy using O-link cytokine panel and RNA-sequencing (seq), respectively.
- Determination of mutational load by whole exome sequencing to assess for correlation with clinical outcomes, immune infiltrating profile, and T cell repertoire diversity and clonality.
VI. Correlate gut microbiome at baseline and changes in the microbiome with clinical response, both in standard chemotherapy and immunotherapy/chemotherapy therapy settings.
VII. MRD assessment using duplex sequencing strategy for circulating cell-free tumor deoxyribonucleic acid (DNA) and correlation with long-term outcomes.
OUTLINE: Patients are randomized to 1 of 2 arms.
INDUCTION PHASE:
ARM I: Patients receive cytarabine via continuous intravenous (IV) infusion on days 1-7 and idarubicin hydrochloride IV over 15-30 minutes or daunorubicin hydrochloride IV over 15-30 minutes on days 1-3 of a 28-35 day cycle. Patients who have evidence of residual leukemia receive cytarabine via continuous IV infusion over days 1-5 and idarubicin hydrochloride IV over 15-30 minutes or daunorubicin hydrochloride IV over 15-30 minutes on days 1-2 for an additional cycle in the absence of disease progression or unacceptable toxicity. Beginning day 8, patients receive pembrolizumab IV over 25-40 minutes. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients who achieve a CR or a CRi may undergo hematopoietic stem cell transplantation (HSCT) per physician discretion or continue to consolidation therapy.
ARM II: Patients receive cytarabine via continuous IV infusion on days 1-7 and idarubicin hydrochloride IV over 15-30 minutes or daunorubicin hydrochloride IV over 15-30 minutes on days 1-3 of a 28-35 day cycle. Patients who have evidence of residual leukemia receive cytarabine via continuous IV infusion over days 1-5 and idarubicin hydrochloride IV over 15-30 minutes or daunorubicin hydrochloride IV over 15-30 minutes on days 1-2 for an additional cycle in the absence of disease progression or unacceptable toxicity. Patients who achieve a CR or a CRi may undergo HSCT per physician discretion or continue to consolidation therapy.
CONSOLIDATION THERAPY:
ARM I: Within 4 weeks of remission status documentation, patients receive high-dose cytarabine (HiDAC) IV over 1-3 hours every 10-12 hours on days 1, 3, and 5 for a total of 6 doses and pembrolizumab IV over 25-40 minutes. Cycles with HiDAC repeat every 28-42 days and cycles with pembrolizumab repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients who remain in CR or CRi receive up to 3 additional cycles of HiDAC and pembrolizumab in the absence of disease progression or unacceptable toxicity and continue to maintenance therapy.
ARM II: Within 4 weeks of remission status documentation, patients receive HiDAC IV over 1-3 hours every 12 hours on days 1, 3, and 5 for a total of 6 doses in the absence of disease progression or unacceptable toxicity. Patients who remain in CR or CRi receive up to 3 additional cycles of HiDAC in the absence of disease progression or unacceptable toxicity.
MAINTENANCE THERAPY:
ARM I: Patients receive pembrolizumab IV over 25-40 minutes. Cycles repeat every 3 weeks for up to 2 years in the absence of disease progression or unacceptable toxicity.
All patients also undergo a skin punch biopsy during screening and undergo bone marrow biopsy and aspiration, collection of blood samples, and echocardiography (ECHO) or multigated acquisition scan (MUGA) during screening and on study. Patients may optionally undergo computed tomography (CT) scan during screening.
After completion of study treatment, patients are followed up at 30 days and then every 6 months for up to 5 years. Patients who undergo HSCT are also followed up at 100 days post-transplant.
- Conditions
- Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Acute Myeloid Leukemia Post Cytotoxic Therapy, Secondary Acute Myeloid Leukemia
- Being tested
- Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Computed Tomography, Cytarabine
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biospecimen CollectionProcedure or surgery
Also called Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection
Undergo collection of blood
Bone Marrow AspirationProcedure or surgery
Undergo bone marrow aspiration
Bone Marrow BiopsyProcedure or surgery
Also called Biopsy of Bone Marrow, Biopsy, Bone Marrow
Undergo bone marrow biopsy
Computed TomographyProcedure or surgery
Also called CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography
Undergo CT
CytarabineDrug
Also called .beta.-Cytosine arabinoside, 1-.beta.-D-Arabinofuranosyl-4-amino-2(1H)pyrimidinone, 1-.beta.-D-Arabinofuranosylcytosine, 1-Beta-D-arabinofuranosyl-4-amino-2(1H)pyrimidinone
Given via continuous IV infusion
Daunorubicin HydrochlorideDrug
Also called Cerubidin, Cerubidine, Cloridrato de Daunorubicina, Daunoblastin
Given IV
Echocardiography TestProcedure or surgery
Also called EC, Echocardiography
Undergo ECHO
Hematopoietic Cell TransplantationProcedure or surgery
Also called HCT, Hematopoietic Stem Cell Infusion, HEMATOPOIETIC STEM CELL TRANSPLANT, Hematopoietic Stem Cell Transplantation
Undergo HSCT
Idarubicin HydrochlorideDrug
Also called Idamycin, Idamycin PFS, Idarubicin HCl, IMI-30
Given IV
Multigated Acquisition ScanProcedure or surgery
Also called Blood Pool Scan, Equilibrium Radionuclide Angiography, Gated Blood Pool Imaging, Gated Heart Pool Scan
Undergo MUGA
PembrolizumabBiological
Also called BCD-201, GME 751, GME751, Keytruda
Given IV
Punch BiopsyProcedure or surgery
Also called BIOPSY, PUNCH, Punch Biopsy of Skin
Undergo a skin punch biopsy
| Study group | Role | What they receive |
|---|---|---|
| Arm I (cytarabine, idarubicin,daunorubicin,pembrolizumab,HSCT) | Experimental | See Detailed Description. Receives: Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Biopsy; Procedure: Computed Tomography; Drug: Cytarabine; Drug: Daunorubicin Hydrochloride; Procedure: Echocardiography Test; Procedure: Hematopoietic Cell Transplantation; Drug: Idarubicin Hydrochloride; Procedure: Multigated Acquisition Scan; Biological: Pembrolizumab; Procedure: Punch Biopsy |
| Arm II (cytarabine, idarubicin, daunorubicin, HSCT) | Active comparator | See Detailed Description. Receives: Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Biopsy; Procedure: Computed Tomography; Drug: Cytarabine; Drug: Daunorubicin Hydrochloride; Procedure: Echocardiography Test; Procedure: Hematopoietic Cell Transplantation; Drug: Idarubicin Hydrochloride; Procedure: Multigated Acquisition Scan; Procedure: Punch Biopsy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of Minimal Residual Disease (MRD) Negative - Complete Response (CR)/Complete Remission With Incomplete Recovery (CRi)
MRD will be assessed by multicolor flow cytometry at a central laboratory as an integral biomarker.
Measured: 78 days (mean)
Rate of MRD-negative CR
Measured: 33 days (mean)
Secondary outcomes (7)
Rate of CR/CRi
Will be defined per European LeukemiaNet 2017.
Measured: 33 days (mean) post induction
MRD Negativity
Measured: At day 14
Percentage of Patients With MRD-CR Using a MRD Cutoff of 0.01%
Measured: 33 days (mean)
Event-free Survival
Measured: From randomization to failure to achieve CR/CRi, relapse or death from any cause, 157 days (mean)
Relapse-free Survival (RFS)
Median RFS will be estimated with Kaplan-Meier curves with 95% confidence interval calculated based on the Brookmeyer-Crowley method
Measured: From first documentation of CR/CRi to either disease relapse or death from any cause, 33 days (mean)
Duration of Response (DOR)
Median DOR will be estimated with Kaplan-Meier curves with 95% confidence interval calculated based on the Brookmeyer-Crowley method.
Measured: From first CR to the date of the first documented relapse or death, whichever occurs first, 244 days (mean)
Incidence of Adverse Events
Will be assessed per Common Terminology Criteria for Adverse Events version 5.0. Presented are a count of those that experienced at least one adverse event.
Measured: Up to 35 days from start of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jan 2020
- Study start17 Feb 2021
- Primary completion27 Nov 2024
- Study completion30 Jan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 49 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Amer M Zeidan, Principal investigator, Yale University Cancer Center LAO
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04214249
- Sponsor's protocol ID
- NCI-2019-08626
- Other IDs
- NCI-2019-08626, 2000028858, 10300, 10300, UM1CA186689
- Status last verified
- Jun 2026
- First submitted
- 30 Dec 2019
- Last updated
- 12 Jun 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page.
Study at a glance
From the registry record, last updated by the sponsor on 12 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- BLockade of PD-1 Added to Standard Therapy to Target Measurable Residual Disease in Acute Myeloid Leukemia 1 (BLAST MRD AML-1): A Randomized Phase 2 Study of the Anti-PD-1 Antibody Pembrolizumab in Combination With Conventional Intensive Chemotherapy as Frontline Therapy in Patients With Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT04214249
- Acronym / sponsor ID
- NCI-2019-08626
- Conditions
- Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Acute Myeloid Leukemia Post Cytotoxic Therapy, Secondary Acute Myeloid Leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 49 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 17 Feb 2021 (actual)
- Primary completion
- 27 Nov 2024 (actual)
- Study completion
- 30 Jan 2027 (estimated)
- First posted
- 2 Jan 2020 (actual)
- Results
- Posted 4 Mar 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase II trial studies how well cytarabine and idarubicin or daunorubicin with or without pembrolizumab work in treating patients with newly-diagnosed acute myeloid leukemia. Chemotherapy drugs, such as cytarabine, idarubicin, and daunorubicin, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving induction chemotherapy with pembrolizumab may work better than induction chemotherapy alone in treating patients with acute myeloid leukemia.
Detailed description
PRIMARY OBJECTIVE:
- To assess the percentage of patients with minimal residual disease (MRD) negative complete remission (CR) (MRD-CR), or with minimal residual disease (MRD) negative complete remission with incomplete recovery (CRi) (MRD-CRi) as measured by flow cytometry at the end of first cycle of consolidation therapy with chemotherapy + pembrolizumab (MK-3475) and compare between the two study arms.
SECONDARY OBJECTIVES:
- Assess the rate of complete remission (CR)/complete remission with incomplete count recovery (CRi) as defined per European Leukemia Net 2017 response criteria at time of count recovery after induction therapy with chemotherapy + pembrolizumab (MK-3475) (Dohner et al., 2017).
II. Rates of complete remission with partial recovery count (CRh) and hematologic improvement (HI) to red blood cells and platelets.
III. Assess the rates of MRD negativity at day 14, MRD-negative CR at end of induction therapy and MRD negative CR after last consolidation cycle.
IV. Assess event free survival (EFS), measured from randomization to failure to achieve CR/CRi, relapse or death from any cause, and relapse free survival (RFS), calculated as the time from first documentation of CR/CRi to either disease relapse or death from any cause.
- Assess the duration of response (DOR, defined as the time from first CR/CRi to the date of the first documented relapse or death, whichever occurs first) and overall survival (OS), defined as time from randomization to death from any cause.
VI. Assess safety endpoints including proportion of patients who develop severe toxicity as defined in the protocol.
EXPLORATORY OBJECTIVES:
- MRD assessment by duplex sequencing (DS) and comparing DS and multiparameter flow cytometry for MRD detection as an exploratory biomarker.
II. Assessment of immune-checkpoint expression and dynamic change of immune cell subsets in response to the combination of checkpoint-inhibition and backbone combination in acute myeloid leukemia (AML).
III. High-throughput sequencing of the T-cell receptor (TCR) Vb CDR3 regions on flow cytometrically sorted t-cell subsets to assess the effect of immunotherapy on the diversity of the t-cell repertoire and assess for correlation to clinical outcomes.
IV. Investigation of protein signatures and ribonucleic acid (RNA) signatures associated with response and efficacy using O-link cytokine panel and RNA-sequencing (seq), respectively.
- Determination of mutational load by whole exome sequencing to assess for correlation with clinical outcomes, immune infiltrating profile, and T cell repertoire diversity and clonality.
VI. Correlate gut microbiome at baseline and changes in the microbiome with clinical response, both in standard chemotherapy and immunotherapy/chemotherapy therapy settings.
VII. MRD assessment using duplex sequencing strategy for circulating cell-free tumor deoxyribonucleic acid (DNA) and correlation with long-term outcomes.
OUTLINE: Patients are randomized to 1 of 2 arms.
INDUCTION PHASE:
ARM I: Patients receive cytarabine via continuous intravenous (IV) infusion on days 1-7 and idarubicin hydrochloride IV over 15-30 minutes or daunorubicin hydrochloride IV over 15-30 minutes on days 1-3 of a 28-35 day cycle. Patients who have evidence of residual leukemia receive cytarabine via continuous IV infusion over days 1-5 and idarubicin hydrochloride IV over 15-30 minutes or daunorubicin hydrochloride IV over 15-30 minutes on days 1-2 for an additional cycle in the absence of disease progression or unacceptable toxicity. Beginning day 8, patients receive pembrolizumab IV over 25-40 minutes. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients who achieve a CR or a CRi may undergo hematopoietic stem cell transplantation (HSCT) per physician discretion or continue to consolidation therapy.
ARM II: Patients receive cytarabine via continuous IV infusion on days 1-7 and idarubicin hydrochloride IV over 15-30 minutes or daunorubicin hydrochloride IV over 15-30 minutes on days 1-3 of a 28-35 day cycle. Patients who have evidence of residual leukemia receive cytarabine via continuous IV infusion over days 1-5 and idarubicin hydrochloride IV over 15-30 minutes or daunorubicin hydrochloride IV over 15-30 minutes on days 1-2 for an additional cycle in the absence of disease progression or unacceptable toxicity. Patients who achieve a CR or a CRi may undergo HSCT per physician discretion or continue to consolidation therapy.
CONSOLIDATION THERAPY:
ARM I: Within 4 weeks of remission status documentation, patients receive high-dose cytarabine (HiDAC) IV over 1-3 hours every 10-12 hours on days 1, 3, and 5 for a total of 6 doses and pembrolizumab IV over 25-40 minutes. Cycles with HiDAC repeat every 28-42 days and cycles with pembrolizumab repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients who remain in CR or CRi receive up to 3 additional cycles of HiDAC and pembrolizumab in the absence of disease progression or unacceptable toxicity and continue to maintenance therapy.
ARM II: Within 4 weeks of remission status documentation, patients receive HiDAC IV over 1-3 hours every 12 hours on days 1, 3, and 5 for a total of 6 doses in the absence of disease progression or unacceptable toxicity. Patients who remain in CR or CRi receive up to 3 additional cycles of HiDAC in the absence of disease progression or unacceptable toxicity.
MAINTENANCE THERAPY:
ARM I: Patients receive pembrolizumab IV over 25-40 minutes. Cycles repeat every 3 weeks for up to 2 years in the absence of disease progression or unacceptable toxicity.
All patients also undergo a skin punch biopsy during screening and undergo bone marrow biopsy and aspiration, collection of blood samples, and echocardiography (ECHO) or multigated acquisition scan (MUGA) during screening and on study. Patients may optionally undergo computed tomography (CT) scan during screening.
After completion of study treatment, patients are followed up at 30 days and then every 6 months for up to 5 years. Patients who undergo HSCT are also followed up at 100 days post-transplant.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm I (cytarabine, idarubicin,daunorubicin,pembrolizumab,HSCT) | Experimental | Procedure: Biospecimen Collection, Procedure: Bone Marrow Aspiration, Procedure: Bone Marrow Biopsy, Procedure: Computed Tomography, Drug: Cytarabine, Drug: Daunorubicin Hydrochloride, Procedure: Echocardiography Test, Procedure: Hematopoietic Cell Transplantation, Drug: Idarubicin Hydrochloride, Procedure: Multigated Acquisition Scan, Biological: Pembrolizumab, Procedure: Punch BiopsySee Detailed Description. |
| Arm II (cytarabine, idarubicin, daunorubicin, HSCT) | Active comparator | Procedure: Biospecimen Collection, Procedure: Bone Marrow Aspiration, Procedure: Bone Marrow Biopsy, Procedure: Computed Tomography, Drug: Cytarabine, Drug: Daunorubicin Hydrochloride, Procedure: Echocardiography Test, Procedure: Hematopoietic Cell Transplantation, Drug: Idarubicin Hydrochloride, Procedure: Multigated Acquisition Scan, Procedure: Punch BiopsySee Detailed Description. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (23 listed)
- Newly diagnosed and pathologically-confirmed AML, confirmed by a bone marrow aspirate and/or biopsy and/or peripheral blood with >= 20% myeloid blasts. Bone marrow biopsy, or aspirate or peripheral blood that were obtained up to 3 weeks before signing consent are allowed for purposes of confirming AML diagnosis for eligibility purposes. Secondary AML (that is arising from prior myelodysplastic syndrome [MDS] as well as therapy-related [t]-AML) are also allowed. Clarifications: AML arising from myeloproliferative neoplasms (MPN), MPN/MDS overlap (including chronic myelomonocytic leukemia [CMML]) or another myeloid malignancy are NOT allowed. Note 1: Patients must have evidence of bone marrow involvement on aspirate or biopsy. Patients with only extramedullary disease and no bone marrow involvement will be excluded. Note 2: Every effort should be made to get an aspirate for central flow assessment at screening and all subsequent required time points, but in cases where an aspirate cannot be collected-including dry taps-the patient will not be excluded and assessments will be performed on peripheral blood (PB) which should be collected at every time that bone marrow (BM) is collected. Note 3: Some patients with AML require initiation of therapy quickly after diagnosis, and full metaphase karyotype results in some centers can take 2-3 weeks to result. To avoid this issue being an impediment to accrual to study or to cause delays in initiation of therapy in patients who need fast initiation of therapy, we allow use of karyotype and/or fluorescence in situ hybridization (FISH) results (as well as FLT3 results) on samples from blood or marrow that were obtained up to 3 weeks before signing consent for purposes of eligibility and stratification. In any case, results from FISH or karyotype should show if core-binding factor (CBF) abnormalities are present by time of randomization as the presence of CBF abnormalities is a required stratification factor
- Age >= 18 and =< 75 years
- Eastern Cooperative Oncology Group (ECOG) performance status =< -2
- The patient has to be eligible to receive intensive "7+3" induction chemotherapy as judged by the treating physician
- Prior use of hypomethylating agents (HMA), lenalidomide, erythropoiesis-stimulating agents (ESAs), and growth factors is allowed if used to treat prior MDS. AML must be previously untreated except as outlined below (hydroxyurea, or tretinoin [ATRA], or leukapheresis). Note: One dose of prophylactic intrathecal therapy is allowed during or before screening if a lumbar puncture is performed to rule out central nervous system (CNS) involvement
Key exclusion criteria (35 listed)
- Patients with a known additional malignancy that is progressing or requires active treatment. Exceptions include basal cell carcinoma of the skin or squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer. Prior treatment with the following are not allowed:
- Patients who have had chemotherapy (except hydroxyurea and all trans retinoic acid [ATRA] which are allowed but have to be stopped the day before induction therapy starts), targeted small molecule therapy (aside from imatinib, dasatinib, or nilotinib), or curative-intent radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C), for a prior curatively treated malignancy, prior to entering the study
- Patients who have received prior anthracyclines not to exceed 150 mg/m^2 of daunorubicin or equivalent for treatment of a prior, unrelated, curatively-treated malignancy which would limit their ability to receive 7 + 3 chemotherapy treatment on study
- Patients with a cardiac ejection fraction less than 50% as determined by echocardiogram or radionuclide ventriculogram scan (MUGA) scan
- Other active primary malignancy (other than non-melanomatous skin cancer or carcinoma in situ of the cervix) requiring treatment or limiting expected survival to =< 2 years
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Biospecimen CollectionAlso: Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen CollectionUndergo collection of blood | Procedure or surgery | Arm I (cytarabine, idarubicin,daunorubicin,pembrolizumab,HSCT), Arm II (cytarabine, idarubicin, daunorubicin, HSCT) |
| Bone Marrow AspirationUndergo bone marrow aspiration | Procedure or surgery | Arm I (cytarabine, idarubicin,daunorubicin,pembrolizumab,HSCT), Arm II (cytarabine, idarubicin, daunorubicin, HSCT) |
| Bone Marrow BiopsyAlso: Biopsy of Bone Marrow, Biopsy, Bone MarrowUndergo bone marrow biopsy | Procedure or surgery | Arm I (cytarabine, idarubicin,daunorubicin,pembrolizumab,HSCT), Arm II (cytarabine, idarubicin, daunorubicin, HSCT) |
| Computed TomographyAlso: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial TomographyUndergo CT | Procedure or surgery | Arm I (cytarabine, idarubicin,daunorubicin,pembrolizumab,HSCT), Arm II (cytarabine, idarubicin, daunorubicin, HSCT) |
| CytarabineAlso: .beta.-Cytosine arabinoside, 1-.beta.-D-Arabinofuranosyl-4-amino-2(1H)pyrimidinone, 1-.beta.-D-Arabinofuranosylcytosine, 1-Beta-D-arabinofuranosyl-4-amino-2(1H)pyrimidinoneGiven via continuous IV infusion | Drug | Arm I (cytarabine, idarubicin,daunorubicin,pembrolizumab,HSCT), Arm II (cytarabine, idarubicin, daunorubicin, HSCT) |
| Daunorubicin HydrochlorideAlso: Cerubidin, Cerubidine, Cloridrato de Daunorubicina, DaunoblastinGiven IV | Drug | Arm I (cytarabine, idarubicin,daunorubicin,pembrolizumab,HSCT), Arm II (cytarabine, idarubicin, daunorubicin, HSCT) |
| Echocardiography TestAlso: EC, EchocardiographyUndergo ECHO | Procedure or surgery | Arm I (cytarabine, idarubicin,daunorubicin,pembrolizumab,HSCT), Arm II (cytarabine, idarubicin, daunorubicin, HSCT) |
| Hematopoietic Cell TransplantationAlso: HCT, Hematopoietic Stem Cell Infusion, HEMATOPOIETIC STEM CELL TRANSPLANT, Hematopoietic Stem Cell TransplantationUndergo HSCT | Procedure or surgery | Arm I (cytarabine, idarubicin,daunorubicin,pembrolizumab,HSCT), Arm II (cytarabine, idarubicin, daunorubicin, HSCT) |
| Idarubicin HydrochlorideAlso: Idamycin, Idamycin PFS, Idarubicin HCl, IMI-30Given IV | Drug | Arm I (cytarabine, idarubicin,daunorubicin,pembrolizumab,HSCT), Arm II (cytarabine, idarubicin, daunorubicin, HSCT) |
| Multigated Acquisition ScanAlso: Blood Pool Scan, Equilibrium Radionuclide Angiography, Gated Blood Pool Imaging, Gated Heart Pool ScanUndergo MUGA | Procedure or surgery | Arm I (cytarabine, idarubicin,daunorubicin,pembrolizumab,HSCT), Arm II (cytarabine, idarubicin, daunorubicin, HSCT) |
| PembrolizumabAlso: BCD-201, GME 751, GME751, KeytrudaGiven IV | Biological | Arm I (cytarabine, idarubicin,daunorubicin,pembrolizumab,HSCT) |
| Punch BiopsyAlso: BIOPSY, PUNCH, Punch Biopsy of SkinUndergo a skin punch biopsy | Procedure or surgery | Arm I (cytarabine, idarubicin,daunorubicin,pembrolizumab,HSCT), Arm II (cytarabine, idarubicin, daunorubicin, HSCT) |
Primary outcome
Rate of Minimal Residual Disease (MRD) Negative - Complete Response (CR)/Complete Remission With Incomplete Recovery (CRi)
Time frame: 78 days (mean)
MRD will be assessed by multicolor flow cytometry at a central laboratory as an integral biomarker.
Rate of MRD-negative CR
Time frame: 33 days (mean)
7 secondary outcomes
Rate of CR/CRi
Time frame: 33 days (mean) post induction
MRD Negativity
Time frame: At day 14
Percentage of Patients With MRD-CR Using a MRD Cutoff of 0.01%
Time frame: 33 days (mean)
Event-free Survival
Time frame: From randomization to failure to achieve CR/CRi, relapse or death from any cause, 157 days (mean)
Relapse-free Survival (RFS)
Time frame: From first documentation of CR/CRi to either disease relapse or death from any cause, 33 days (mean)
Duration of Response (DOR)
Time frame: From first CR to the date of the first documented relapse or death, whichever occurs first, 244 days (mean)
Incidence of Adverse Events
Time frame: Up to 35 days from start of treatment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jun 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page.
Related trials for Acute Myeloid Leukemia
RecruitingPhase 1
A Study of CHM-029 in Participants With NPM1 Mutated, KMT2A or NUP98 Rearranged AML
AML (Acute Myeloid Leukemia)
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
RecruitingPhase 1/2
Venetoclax and CLAG-M for the Treatment of Acute Myeloid Leukemia and High-Grade Myeloid Neoplasms
Acute Biphenotypic Leukemia, Acute Myeloid Leukemia
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Amer M Zeidan principal investigator
Yale University Cancer Center LAO
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy in people with Acute Myeloid Leukemia and Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, run by National Cancer Institute (NCI) at 10 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 23 inclusion and 35 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 12 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 4 Mar 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate of Minimal Residual Disease (MRD) Negative - Complete Response (CR)/Complete Remission With Incomplete Recovery (CRi) (time frame: 78 days (mean)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Jun 2026.