NCT04166929

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.

Trial Health

57
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Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
6

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jan 2020

Typical duration for phase_2

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 14, 2019

Completed
4 days until next milestone

First Posted

Study publicly available on registry

November 18, 2019

Completed
2 months until next milestone

Study Start

First participant enrolled

January 15, 2020

Completed
3.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 28, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 28, 2023

Completed
Last Updated

October 4, 2023

Status Verified

May 1, 2021

Enrollment Period

3.7 years

First QC Date

November 14, 2019

Last Update Submit

October 2, 2023

Conditions

Acute Myeloid LeukemiaMyelodysplasia

Outcome Measures

Primary Outcomes (1)

  • Leukemia relapse

    Cumulative incidence of leukemia relapse.

    1 year after bone marrow transplant

Study Arms (2)

Haplo-stem cell transplant followed by NK infusion

EXPERIMENTAL

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

Biological: Haplo SCT with NK cells

Haplo-stem cell transplant

ACTIVE COMPARATOR

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

Biological: Haplo SCT

Interventions

Haplo SCT with NK cellsBIOLOGICAL

Patients in this arm receive haplo-SCT followed by one boost of NK cells one/two weeks after the transplant. Lymphocyte are collected by apheresis from the donor one-two weeks after BM harvest. CD3 negative/CD56 positive cells are selected by immunomagnetic columns (other lymphocytes are stored to be used as DLI)

Also known as: haplo SCT followed by NK cell infusion
Haplo-stem cell transplant followed by NK infusion
Haplo SCTBIOLOGICAL

Patients in this arm receive a standard haplo-SCT. All administerd therapies are the same as in other arm, except for the NK cell boost..

Also known as: Standard haplo SCT without subsequent NK cell boost.
Haplo-stem cell transplant

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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) .

You may not qualify if:

  • Positive serologic markers for human immunodeficiency virus (HIV)
  • Acute hepatitis B virus (HBV) or acute hepatitis C virus (HCV) infection

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Fondazione Policlinico Universitario A. Gemelli IRCCS

Roma, RM, 00168, Italy

Location

MeSH Terms

Conditions

Leukemia, Myeloid, AcuteAnemia, Refractory, with Excess of Blasts

Condition Hierarchy (Ancestors)

Leukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesAnemia, RefractoryAnemiaMyelodysplastic SyndromesBone Marrow Diseases

Study Officials

  • Andrea Bacigalupo, Prof

    Fondazione Policlinico Gemelli IRCCS

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 14, 2019

First Posted

November 18, 2019

Study Start

January 15, 2020

Primary Completion

September 28, 2023

Study Completion

September 28, 2023

Last Updated

October 4, 2023

Record last verified: 2021-05

Locations

IT(1)