MG4101 for Refractory or Relapsed AML
Condition: Acute Myeloid Leukemia
Study type: Interventional
Phase: Phase 2

Summary: 
      This study is a single center, single arm, open-labeled phase 2 clinical study. The aim of
      this study is to investigate the efficacy and safety of allogeneic natural killer cell
      (MG4101). After lymphodepletion with fludarabine and cyclophosphamide, the patient will
      receive MG4101. Each cycle consists of 28 days, and a total of 2 cycles of MG4101 will be
      administered with IL-2 to activate the study drug. The efficacy of MG4101 will be evaluated
      after 8 weeks from the first day of treatment. We will evaluate the safety of the drug
      measuring the vital sign, laboratory tests, and adverse events.
    

Detailed description: 
      Acute myeloid leukemia is a hematologic malignancy of myeloid lines leukocyte. In Korea,
      acute leukemia accounts for 87% of all leukemia and the incidence of acute myeloid leukemia
      is twice as high as acute lymphoblastic leukemia. The general treatment strategy for AML has
      not changed over the past 30 years. In adult AML, about 70 to 80% of the patients achieve
      complete remission after the intensive induction chemotherapy, but disease recurrence is
      relatively common. After the recurrence, the patients with good physical condition receive
      intensive salvage chemotherapy followed by allogeneic hematopoietic stem cell
      transplantation. But even with the intensive treatment, the long-term survival rate is only
      about 25%.

      MG4101 is the natural killer (NK) cell product that is activated in vitro after obtaining
      through leukapheresis from a healthy donor. The allogeneic NK cell is well known to have
      anti-leukemic effect in allogeneic stem cell transplantation. As it is widely reported that
      the lymphodepletion is essential in adoptive cell transfer therapy, MG4101 will be
      administered after the conditioning with cyclophosphamide and fludarabine. And after the
      infusion of MG4101, IL-2 will be infused together to activate the study drug.

      In the dose-finding phase 1 study of MG4101 (NCT01212341), the maximal tolerated dose was
      estimated to exceed 3x10^7 cells/kg. The patients will receive 2.0x10^9 to 5.0x10^6 cells in
      each cycle, based on the weight.

      The protocol is as follows:

      Cyclophosphamide and fludarabine will be administered at the dose of 250 mg/m2/day and 20
      mg/m2/day, respectively, for 3 days from the start of the treatment. On the 4th, 11th and
      18th day, the study drug, MG4101, will be administered intravenously, followed by 3 days of
      IL-2. The response will be assessed on the 28th, 56th and 112th day. The adverse event will
      be observed for 56 days after the initiation of the treatment.
    

Intervention name(s): MG4101
Intervention type(s): Drug
Intervention description(s): Administration of study drug (MG4101) for the patients with refractory or relapsed acute myeloid leukemia (Single arm)
Other intervention name(s): Allogeneic natural killer cell
Intervention model: Single Group Assignment
Intervention model description: Single center, single arm, open-labeled phase 2 trial
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Allogeneic natural killer cell, MG4101