Clinical Trials

The purpose of this study is to explore the safety, tolerability and preliminary biological and clinical activity of CC-96191 as a single-agent in the setting of Relapsed or refractory acute myeloid leukemia (R/R AML). The dose escalation (Part A) of the study will explore escalating intravenous doses of CC-96191 to estimate the MTD and/or RP2D of CC-96191 as monotherapy. The expansion (Part B), will further evaluate the safety and effectiveness of CC-96191 administered at or below the MTD in one or more expansion cohorts in order to determine the RP2D.

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).

The primary objective of the United States Food and Drug Administration (FDA) for this study is to demonstrate non-inferiority in subjects who received an allogeneic BMT for subjects randomized to Rezafungin for Injection compared to subjects randomized to the standard antimicrobial regimen (SAR) for fungal-free survival at Day 90 (±7 days).

The primary objective of the European Medicines Agency (EMA) for this study is to demonstrate superiority in subjects who received an allogeneic BMT randomized to Rezafungin for Injection compared to subjects randomized to the SAR for fungal-free survival at Day 90 (±7 days).

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.

The purpose of this study is to determine the safety and preliminary efficacy of voruciclib as monotherapy in subjects with relapsed or refractory B-cell malignancies or AML, and in combination with venetoclax in subjects with relapsed or refractory AML after treatment with standard therapy.

This study will include a Phase 1 dose-finding portion (Cohorts A and B) and a four-arm expansion portion. The primary objectives of the study are to determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of H3B-8800 administered orally in participants with Myelodysplastic Syndromes (MDS), Acute Myeloid Leukemia (AML), or Chronic Myelomonocytic Leukemia (CMML) and to assess the safety and tolerability of H3B-8800 as a single agent administered orally once daily on a 5 days on/9 days off repeated dosing schedule in 28-day cycles in participants with MDS, AML, or CMML.

This phase I trial studies the best dose and side effects of recombinant vesicular stomatitis virus carrying the human NIS and IFN beta genes (VSV-hIFNbeta-sodium iodide symporter [NIS]) in treating patients with multiple myeloma, acute myeloid leukemia, or T-cell lymphoma that has come back or does not respond to treatment. A virus, called VSV-hIFNbeta-NIS, which has been changed in a certain way, may be able to kill cancer cells without damaging normal cells.

The purpose of this study is to compare standard chemotherapy to therapy with CPX-351 and/or gilteritinib for patients with newly diagnosed acute myeloid leukemia with or without FLT3 mutations. 

The purpose of this study is to determine how well cytarabine and idarubicin or daunorubicin with or without pembrolizumab work in treating patients with newly-diagnosed acute myeloid leukemia. Drugs used in chemotherapy, 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.

This is a phase 1, dose-escalation study (using 3 + 3 dose-limiting toxicity (DLT) criteria) evaluating the safety and tolerability of XmAb18968, as well as establishing a recommended phase II dose (RP2D) in subjects with T cell acute lymphoblastic leukemia (T-ALL) and T cell lymphoblastic (lymphoma) T-LBL (Group A) and acute myeloid leukemia (AML) (Group B).