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

Phase 2

Terminated

• Conditions: Acute Myeloid Leukemia; Myelodysplasia
• Interventions: Biological: Haplo SCT; Biological: Haplo SCT with NK cells

• Registration Number: NCT04166929
• Lead Sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-11-14
• Study First Submitted QC: 2019-11-15
• Study First Posted: 2019-11-18
• Study Start: 2020-01-15
• Status Verified: 2021-05
• Primary Completion: 2023-09-28
• Study Completion: 2023-09-28
• Last Update Submitted: 2023-10-02
• Last Update Posted: 2023-10-04

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 6

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Haplo-stem cell transplant	Haplo SCT	Patients in this arm receive standard haplo stem cell transplant (from bone marrow or apheresis) transplant without subsequent NK cell infusion.
Haplo-stem cell transplant followed by NK infusion	Haplo SCT with NK cells	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

Primary Outcome Measures

Name	Time	Method
Leukemia relapse	1 year after bone marrow transplant	Cumulative incidence of leukemia relapse.

Trial Locations

Locations (1)

Fondazione Policlinico Universitario A. Gemelli IRCCS; 🇮🇹 Roma, RM, Italy