Haploidentical Stem Cell Transplantation With or Without NK Cell Infusion in Refractory/Relapsed Acute Myeloid Leukemia and Myelodysplastic Syndrome:The BigEMINy Study
Trial Snapshot
- Phase
- Phase 2
- Status
- Terminated
- Enrollment
- 6
- Locations
- 1
- Primary Endpoint
- Leukemia relapse
Study Overview
Brief Summary
Relapse after an allogeneic hematopoietic stem cell transplantation (HSCT) is high in patients with advanced AML, in the 50% range. NK cells have been shown to possess significant anti-leukemic activity and may be used to reduce the incidence of relapse in patients with advanced AML.
Investigators hypothesize that the administration of a purified boost of NK cells on day +7 post HSCT, will reduce the incidence of relapse from the current 50% to 25%. In a phase III multicenter clinical study, 116 patients will be randomized to receive or not a boost of donor NK cells on day +7 post-HSCT. The first 10 patients in the experimental arm will be analyzed for toxicity. The stopping rule will be a transplant related mortality of more than 50% in the first 20 patients who received NK cells.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 75 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Diagnosis of AML or MDS
- •Patients not in remission at the time of transplant
- •Age 18-75
- •ECOG performance status <2
- •Availability of a family HLA haploidentical donor, eligible to donate both marrow cells and unstimulated lymphocytes (1 apheresis procedure at day +7) .
Exclusion Criteria
- •Positive serologic markers for human immunodeficiency virus (HIV)
- •Acute hepatitis B virus (HBV) or acute hepatitis C virus (HCV) infection
Arms & Interventions
Haplo-stem cell transplant followed by NK infusion
Haplo stem cell transplant (from bone marrow or apheresis) is followed by a haplo-NK cell infusion (target dose ≥1*106/kg b.w.) at day 7.
Unstimulated haplo-NK cells are collected through apheresis Mononuclear cells are then subjected to a preliminary negative selection of CD3+ cells and to a subsequent positive selection of CD56+ cells. CD3 negative/CD56 positive cells are infused
Intervention: Haplo SCT with NK cells (Biological)
Haplo-stem cell transplant
Patients in this arm receive standard haplo stem cell transplant (from bone marrow or apheresis) transplant without subsequent NK cell infusion.
Intervention: Haplo SCT (Biological)
Outcomes
Primary Outcomes
Leukemia relapse
Time Frame: 1 year after bone marrow transplant
Cumulative incidence of leukemia relapse.
Secondary Outcomes
No secondary outcomes reported