Conditions: Relapsed Acute Lymphoblastic Leukemia Relapsed Acute Myelogenous Leukemia
Keywords: Relapsed Minimal Residual Disease MRD Acute Lymphoblastic Leukemia Acute Myelogenous Leukemia ALL AML GNKG168 Pediatric Childhood
Allocation: NON_RANDOMIZED | Intervention model: SINGLE_GROUP
Enrollment: 4
Healthy volunteers: no

Brief summary:
This is a phase I trial of an investigational drug called GNKG168 in patients with relapsed and refractory acute lymphoblastic leukemia (ALL) and acute myelogenous leukemia (AML) who are in morphologic remission but are positive for Minimum Residual Disease (MRD).

Detailed description:
GNKG168 is a Toll-like receptor (TLR) agonist. TLR agonists are a novel approach to stimulate an effective anti-tumor immune response as they are able to stimulate both innate and adaptive immune responses. There will be two strata i.e patients who have received hematopoietic stem cell transplant (HSCT) and patients who have never undergone HSCT. GNKG168 will be administered as a 60 min iv infusion. One 14-day cycle consists of 5-day treatment followed by 9 day-rest. Patients will receive 2 cycles before evaluation. The primary objective is to determine the maximum tolerated dose of GNKG168 in relapsed ALL and AML patients. The completion of this study and the correlative studies will provide a solid foundation for subsequent phase 2 study and the development of clinical applications of CpG-ODN-based immune therapy as a treatment of childhood leukemia. If safety and efficacy of GNKG168 is proven, it will be intriguing to explore its role to maintain CR in a randomized manner.

Arms:
arm 1: Patients who have a history of hematopoetic stem cell transplantation (HSCT). GNKG168 will be administered as a 60 minute intravenous infusion daily for 5 consecutive days. It may be repeated every 14 days. Patients should receive minimum 2 courses and up to 6 courses may be administered. Dose will be assigned at study entry. arm 2: Patients who have never undergone HSCT. GNKG168 will be administered as a 60 minute intravenous infusion daily for 5 consecutive days. It may be repeated every 14 days. Patients should receive minimum 2 courses and up to 6 courses may be administered. Dose will be assigned at study entry.

Interventions: intervention 1: GNKG168
Intervention descriptions: intervention 1: GNKG168 will be given intravenously over 1 hour on days 1 through 5 followed by 9 days of rest. Dose will be assigned at study entry.

Dose Level 0: 0.15 mg/kg Dose Level 1: 0.25 mg/kg Dose Level 2: 0.75 mg/kg Dose Level 3: 1.5 mg/kg

The starting dose level for the trial will be 0.25 mg/kg. If that dose level proves to be intolerable, the dose will be reduced to 0.15 mg/kg (dose level 0). If the 0.15 mg/kg dose level is intolerable due to DLTs, TACL, the Principal Investigator, and Medical Monitor will then decide the best course of action for subsequent administration of GNKG168.

Protocol document: