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  • A Master Protocol for Biomarker-Based Treatment of AML (The Beat AML Trial) Rochester, Minn., Jacksonville, Fla. 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.
  • A Phase I, Multicenter, Open-Label, Dose-Escalation and Expansion, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-120 in Subjects with Advanced Hematologic Malignancies with an IDH1 Mutation, with a Substudy in Subjects Relapsed or Refractory Myelodysplastic Syndrome with a Substudy in Subjects with Advanced Hematologic Malignancies with Organ Impairment Rochester, Minn.

    The purpose of this Phase I, multicenter study is to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-120 in advanced hematologic malignancies that harbor an IDH1 mutation. The first portion of the study is a dose escalation phase where cohorts of patients will receive ascending oral doses of AG-120 to determine maximum tolerated dose (MTD) and/or the recommended Phase II dose. The second portion of the study is a dose expansion phase where three cohorts of patients will receive AG-120 to further evaluate the safety, tolerability, and clinical activity of the recommended Phase II dose. Study will not be terminated until subjects have had opportunity to be treated with AG-120 for at least 3 years after the first dose of the last subject enrolled and followed for survival for at least 12 months.

  • A Randomized, Double-blind, Placebo-controlled Phase III Multi-center Study of Azacitidine With or Without MBG453 for the Treatment of Patients With Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS) as Per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2) (STIMULUS-MDS2) Jacksonville, Fla.

    The purpose of this study is to evaluate the safety and effectiveness of MBG453, or placebo added to azacitidine, in adult subjects with intermediate, high or very high risk myelodysplastic syndrome (MDS) as per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2) who have an indication for treatment with azacitidine in first-line setting and are not eligible for intensive chemotherapy or hematopoietic stem cell transplantation (HSCT) according to medical judgment by the investigator.

  • A Randomized, Open-label, Controlled, Phase 2 Study of Pevonedistat, Venetoclax, and Azacitidine Versus Venetoclax Plus Azacitidine in Adults With Newly Diagnosed Acute Myeloid Leukemia Who Are Unfit for Intensive Chemotherapy Jacksonville, Fla. The purpose of this study is to determine whether the combination of pevonedistat + venetoclax + azacitidine improves event-free survival (EFS) compared with venetoclax + azacitidine in patients with newly diagnosed acute myeloid leukemia (AML) who are unfit for intensive chemotherapy.
  • A Simplified Patient Care Strategy to Decrease Early Deaths in Acute Promyelocytic Leukemia (APL) Jacksonville, Fla., Rochester, Minn.

    This clinical trial studies how well simplified patient care strategy works in decreasing early death in patients with acute promyelocytic leukemia. Implementing simplified acute promyelocytic leukemia guidelines along with support from acute promyelocytic leukemia experts may decrease deaths and improve survival.

  • ASTX727-03: A Randomized, Open-Label, Phase 1-2 Study of ASTX727 Low Dose (ASTX727 LD) Extended Schedule in Subjects With Lower Risk (IPSS Low or Intermediate-1) Myelodysplastic Syndromes (MDS) (MDS) Jacksonville, Fla.

    The purpose of this study of various ASTX727 LD doses and schedules to assess safety, pharmacodynamics, pharmacokinetics, and hematologic response in subjects with International Prognostic Scoring System (IPSS) risk category of low-risk or Intermediate-1 Myelodysplastic Syndromes (MDS). This study will be conducted in two phases. In phase 1 subjects will be randomized into 3 cohorts in a 28-day cycles. Phase 2, 80 new subjects will be randomized in a 1:1 ratio into 2 doses/schedules.

  • KO-MEN-001, A Phase 1/2A First in Human Study of the Menin-MLL(KMT2A) Inhibitor KO 539 in Patients With Relapsed or Refractory Acute Myeloid Leukemia Jacksonville, Fla., Rochester, Minn.

    The purpose of this study is to determine the maximum tolerated dose (MTD) of KO-539, a menin-MLL(KMT2A) inhibitor, in patients with refractory or relapsed  acute myeloid leukemia (AML) who have failed or are ineligible for any approved standard of care therapies, including hematopoietic stem cell transplantation (HSCT).

  • SLSG18-301: A Randomized, Open-Label Study of the Efficacy and Safety of Galinpepimut-S (GPS) Maintenance Monotherapy Compared to Investigator's Choice of Best Available Therapy in Subjects with Acute Myeloid Leukemia Who Have Achieved Complete Remission After Second-Line Salvage Therapy Jacksonville, Fla.

    This is an open-label, multicenter, randomized, parallel groups study of GPS vs. best

    available treatment (BAT) in patients with AML in second complete remission (CR2)

    or in second complete remission with incomplete platelet recovery (CRp2). All

    patients will have their historical bone marrow samples stained for WT1 via IHC by

    central pathology review. The primary goal of the study will be to demonstrate an

    advantage for GPS in overall survival in these patient populations. The study will

    enroll approximately 116 patients and will be conducted at up to 50 investigational

    sites. Patients will be randomized 1:1 to GPS or BAT stratified by whether they are

    in CR2 or CRp2.

    Patients on the BAT arm may be treated with 1. observation (whereby palliative

    management with hydroxyurea is allowed), 2. a hypomethylating agent (decitabine or

    azacitidine), and/or 3. Venetoclax and/or 4. low-dose ara-C. Patients whose

    remission in CR2 that can be maintained with other agents (e.g. FLT-3 or IDH

    inhibitors) will not be eligible. However, there are no restrictions on prior use of

    any agents in the CR1 setting. Patients can not receive GPS as an adjunct therapy to

    any other agents.

    Patients on the GPS arm will receive 70 μg of sargramostim (GM- CSF) on Day -2

    and Day 1 before each injection of GPS. The first two administrations of GM-CSF

    will take place at the same anatomical site as the planned administration of GPS within

    each treatment cycle. GPS will be administered as an immunization induction every

    2 weeks for 6 administrations (Weeks 0 – 10); this will be followed by a 4-week

    period of no treatment. Treatment will then resume for 6 administrations as an initial

    booster phase every 4 weeks (Weeks 14 – 34) which will again be followed by a

    period of no treatment lasting 6 weeks. GPS will be resumed after this period as a

    second booster phase and will be administered every 6 weeks for 3 administrations

    (Weeks 40 – 52). Following each administration of GM-CSF or GPS, patients will

    remain in the clinic for approximately 30 minutes for observation. An End of

    Treatment visit will be conducted 30 days following the last dose of GPS. Patients

    will then enter the long-term follow-up portion of the trial where they will be followed

    for recurrence of leukemia and overall survival.

    To ensure a comparable level of observation, patients randomized to the BAT arm

    will be seen every 4 weeks through Week 52. The Schedules of Procedures for both

    the GPS and the BAT arms can be found in Section 6 of the protocol.

    All patients will undergo bone marrow aspirates and biopsies at screening, Week 12

    and end of treatment. Bone marrow examinations will then be repeated as clinically

    indicated. Patients will be assessed for safety at each clinic encounter. The primary

    endpoint will be overall survival.

  • STML-401-0314 - Tagraxofusp (SL-401) in Patients with Chronic Myelomonocytic Leukemia (CMML) or Myelofibrosis (MF) Jacksonville, Fla.

    This is a non-randomized open label multi-center study. Patients with high-risk myeloproliferative neoplasms (systemic mastocytosis [SM], advanced symptomatic hypereosinoophic disorder [PED], myelofibrosis [MF], and chronic myelomonocytic leukemia [CMML]) will be treated with SL-401, which will be administered as a brief intravenous infusion for 3 consecutive days initially every 21 days for 4 cycles; every 28 days for cycles 5-7; then every 42 days. Stage 1 will consist of a period in which several doses of SL-401 are evaluated. The Stage 2 portion will enroll up to 18 patients with each of the 4 myeloproliferative malignancies: SM, PED, MF, and CMML. In entirety, the Stage 2 portion will consist of up to 72 patients who will be treated at a maximum tolerated dose or maximum tested dose in which multiple dose-limiting toxicities are not observed (identified in Stage 1).

  • The National Myelodysplastic Syndromes Natural History Study (MDS) Scottsdale/Phoenix, Ariz., Eau Claire, Wis.

    Multi-center study enrolling patients suspected or newly diagnosed with myelodysplastic syndromes (MDS), myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN) overlap disorder, or idiopathic cytopenia of undetermined significance (ICUS). Participants will be followed long term. Clinical data, blood, and tissue samples will be collected to establish a biorepository to facilitate the study of the natural history of MDS.

Closed for Enrollment