Study of Biomarker-Based Treatment of Acute Myeloid Leukemia

• Status: Recruiting
• Trial end date: 2023-12-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Beat AML, LLC
• Treatments: Azacitidine; AZD5991; Cytarabine; Daunorubicin; Decitabine; Pevonedistat; Venetoclax

Criteria

Inclusion Criteria:

• 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 or B is allowed by the sub-study where age 18 and older is allowed.

• Subjects or their legal representative must be able to understand and provide written informed consent

• Cohort Inclusion Criteria - Group A: Subjects must have previously untreated acute myeloid leukemia (AML) according to the WHO classification with no prior treatment other than hydroxyurea. Prior therapy for myelodysplastic syndrome (MDS), myeloproliferative syndromes (MPD), or aplastic anemia is permitted but not with hypomethylating agents.

• Cohort Inclusion Criteria - Group B: Subjects must have relapsed or refractory AML according to the WHO classification. For study purposes, refractory AML is defined as failure to ever achieve CR or recurrence of AML within 6 months of achieving CR; relapsed AML is defined as all others with disease after prior remission.

Exclusion Criteria:

• Isolated myeloid sarcoma (meaning, patients must have blood or marrow involvement with AML to enter the study)

• Acute promyelocytic leukemia

• Symptomatic central nervous system (CNS) involvement by AML

• 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, 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