Haploidentical Stem Cell Transplant With or Without NK Cell Infusion in AML and MDS (Bigeminy)

Institute of Hospitalization and Scientific Care (IRCCS)

Status and phase: Terminated; Phase 2

Conditions: Myelodysplasia; Acute Myeloid Leukemia

Treatments: Biological: Haplo SCT; Biological: Haplo SCT with NK cells

Study type: Interventional

Funder types: Other

Identifiers

NCT04166929

2588 (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 6 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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6 participants in 2 patient groups

Haplo-stem cell transplant followed by NK infusion

Experimental group

Description:

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

Treatment:

Biological: Haplo SCT with NK cells

Haplo-stem cell transplant

Active Comparator group

Description:

Patients in this arm receive standard haplo stem cell transplant (from bone marrow or apheresis) transplant without subsequent NK cell infusion.

Treatment:

Biological: Haplo SCT