Study of Biomarker-Based Treatment of Acute Myeloid Leukemia

Recruiting now · Phase 1/Phase 2

Conditions studied: Previously Untreated Relapsed Refractory Acute Myeloid Leukemia

In brief

This screening and multi-sub-study Phase 1b/2 trial will establish a method for genomic screening followed by assigning and accruing simultaneously to a multi-study "Master Protocol (BAML-16-001-M1)." The specific subtype of acute myeloid leukemia will determine which sub-study, within this protocol, a participant will be assigned to evaluate investigational therapies or combinations with the ultimate goal of advancing new targeted therapies for approval. The study also includes a marker negative sub-study which will include all screened patients not eligible for any of the biomarker-driven sub-studies. Patients with myeloid malignancies \[e.g. myelodysplastic syndrome (MDS) or other diseases\], will be allowed to enroll to Master protocol if there is an available sub-study.

Key facts

Study ID

NCT03013998

Run by

Beat AML, LLC

People needed

3000

Starts

2016-11-01

Expected to finish

2028-12-01

Last updated by the study team

2025-12-17

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Adults, age 60 years or older at the time of diagnosis unless in a specific known cytogenetic and genomic group for which treatment in Group A, B, or C is allowed by the sub-study where age 18 and older is allowed. In such case, waiting for Foundation Medicine test results would not be required to proceed with sub-study treatment. Patients < 60 years old who are screened but do not fall within the cytogenetic and genomic open sub-studies would still be followed on the M1 Master Protocol and not considered screen fails.
• Patients must be able to understand and provide written informed consent
• Cohort Inclusion Criteria - Group A: Patients must have previously untreated acute myeloid leukemia (AML) according to the WHO classification with no prior treatment other than hydroxyurea. Patients with myeloid malignancies [e.g. myelodysplastic syndrome (MDS) or other disease], will be allowed to enroll to this group. For previously untreated subjects with ≥ 20% blasts in bone marrow or blood only: Prior therapy for myelodysplastic syndrome (MDS), myeloproliferative syndromes (MPD), or aplastic anemia is permitted. For select group, patients who cannot wait or choose not to wait for results of genomic testing as specified in this protocol, will be allowed to enroll to select sub-studies that allow enrollment and treatment of all patients regardless of their genomic mutations or cytogenetics. For this group, patients will proceed to enroll to that select sub-study without waiting for results of genomic testing and genomic samples will be collected to be analyzed retrospectively after patients' enrollment.
• Cohort Inclusion Criteria - Group B: Patients must have relapsed or refractory AML according to the WHO classification. For study purposes, refractory AML is defined as failure to ever achieve a complete response (CR) or recurrence of AML within 6 months of achieving CR; relapsed AML is defined as all others with disease after prior remission. For select genomic aberrations specified in the sub-studies, patients ≥ 18 years of age may be allowed to enroll in this portion of the study. Patients with relapsed or refractory myeloid malignancies (e.g., MDS or other diseases) will be allowed to enroll to this group.
• Cohort Inclusion Criteria - Group C: For select sites which are not part of the Beat AML core sites. These sites will only participate in select sub-studies. Patients in this group will enroll under the Beat AML M1 Master protocol with the intent to enroll into these select sub-studies and following screening on Beat M1 Master protocol, they will come off M1 Master protocol.

You may not qualify if…

• Acute promyelocytic leukemia
• Clinically active central nervous system (CNS) involvement by AML. A patient may be considered eligible if CNS leukemia is showing response to treatment at study entry and should continue to receive intrathecal therapy as clinical indicated. Patients who require or are undergoing craniospinal irradiation of disease control would not be eligible for participation.
• Signs of leukostasis requiring urgent therapy
• Disseminated intravascular coagulopathy with active bleeding or signs of thrombosis
• Patients with psychological, familial, social, or geographic factors that otherwise preclude them from giving informed consent, following the protocol (including failure to collect genomics samples for screening), or potentially hamper compliance with study treatment and follow-up
• Any other significant medical condition, including psychiatric illness or laboratory abnormality, that would preclude the patient participating in the trial or would confound the interpretation of the results of the trial.
• BAML-16-001-S17 - Inclusion Criteria:
• Patients, age 60 years or older at the time of diagnosis with untreated AML according to the International Consensus Classification (ICC) 2022 guidelines, that have NPM1 mutated or MLL rearranged disease and who are not candidates for or do not wish to pursue intensive induction chemotherapy.
• Patients must be able to understand and provide written informed consent.
• Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2.
• Aspartate aminotransferase (AST) < 5 x upper limit of normal (ULN), alanine aminotransferase (ALT) < 5 x ULN, and total bilirubin < 2 x ULN (except for patients with known or suspected Gilbert's syndrome and with direct bilirubin within normal range) for the local laboratory.
• Adequate renal function as defined by calculated creatinine clearance ≥ 60 mL/min for the local laboratory.
• Females must be non-child bearing, postmenopausal, surgically sterile or meet certain criteria if of childbearing potential. Males must adhere to criteria if with females of child bearing potentia
• Patients must have previously untreated AML with no prior treatment other than hydroxyurea. No chemotherapy for AML outside of hydroxyurea for treatment of leukostasis or ATRA for initially suspected APL (that is ruled out) is allowed as well as one dose of intrathecal chemotherapy for suspected CNS involvement (that is ruled out) is allowed. Prior therapy for myelodysplastic syndrome is (MDS) allowed except for hypomethylating agents.
• If the patient has co-morbid illness or malignancy, life expectancy attributed to this must be greater than 2 years.
• BAML-16-001-S17 - Exclusion Criteria:
• Isolated myeloid sarcoma (meaning, patients must have blood or marrow involvement with AML to enter the study).
• Acute promyelocytic leukemia (FAB M3).
• Favorable risk cytogenetics [Core Binding Factor (CBF) AML].
• Active central nervous system (CNS) involvement by AML.
• Signs of leukostasis requiring urgent therapy.
• Patients with WBC ≥ 25,000/μl. (Patients must have WBC < 25,000/μl to begin therapy and Hydroxyurea may be used to obtain this level).
• Patients willing and able to receive intensive induction chemotherapy.
• Disseminated intravascular coagulopathy with active bleeding or signs of thrombosis.
• Patients with psychological, familial, social, or geographic factors that otherwise preclude them from giving informed consent, following the protocol, or potentially hamper compliance with study treatment and follow-up.

Where it is running

• UNC Hospitals, University of North Carolina at Chapel Hill — Chapel Hill, North Carolina, United States (enrolling)
• University of Cincinnati Medical Center — Cincinnati, Ohio, United States (enrolling)
• Ohio State University — Columbus, Ohio, United States (enrolling)
• Oregon Health & Science University — Portland, Oregon, United States (enrolling)
• UCLA Ronald Reagan Medical Center — Los Angeles, California, United States (enrolling)
• University of Texas Southwestern — Dallas, Texas, United States (enrolling)
• Emory University — Atlanta, Georgia, United States (enrolling)
• University of Chicago — Chicago, Illinois, United States (enrolling)
• University of Kansas Clinical Research Center — Fairway, Kansas, United States (enrolling)
• University of Maryland Medical Center — Baltimore, Maryland, United States (enrolling)
• University of California, San Francisco — San Francisco, California, United States (enrolling)
• Memorial Sloan Kettering Cancer Center — New York, New York, United States (enrolling)
• Huntsman Cancer Institute, University of Utah — Salt Lake City, Utah, United States
• University of Colorado — Denver, Colorado, United States
• University of Florida Health Shands Cancer Hospital — Gainesville, Florida, United States
• Mayo Clinic Florida — Jacksonville, Florida, United States
• Mayo Clinic Minnesota — Rochester, Minnesota, United States
• UPMC Hillman Cancer Center — Pittsburgh, Pennsylvania, United States
• Mayo Clinic Arizona — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

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