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Expanded Natural Killer Cells Following Haploidentical HSCT for AML/MDS

Primary Purpose

Acute Myeloid Leukemia, Myelodysplastic Syndromes

Status

Recruiting

Phase

Phase 1

Locations

Switzerland

Study Type

Interventional

Intervention

NK-DLI

About this trial

This is an interventional treatment trial for Acute Myeloid Leukemia focused on measuring Natural Killer cells, NK cells, Immunotherapy, haploidentical hematopoietic stem cell transplantation

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Patient:

>18 years of age
No HLA-matched related or unrelated donor available
AML or MDS-EB with indication for a haplo-HSCT according to the guidelines of the University Hospital Basel Stem Cell Transplant Team
Judged by the transplant physicians to have adequate organ function and no contraindications to haplo-HSCT
Available related haploidentical donor
Written informed consent

Donor:

>18 years old, haploidentical parent, sibling or other relative
Donor suitable for cell donation and apheresis according to standard criteria
Written informed consent

Exclusion Criteria:

Patient:

APL diagnosis
Presence of relevant (mean fluorescence intensity >2000) donor-specific anti-HLA antibodies
Pregnancy
Necessity of immunosuppression apart from GvHD prophylaxis

Exclusion Criteria:

Donor:

• Pregnancy

Sites / Locations

University Hospital BaselRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

NK-DLI

Arm Description

Preemptive immunotherapy with ex vivo expanded NK cells on days +10, +15 and +20 with increasing NK cell doses following haplo-HSCT.

Outcomes

Primary Outcome Measures

Incidence and severity of adverse events including GvHD and infections.

As defined by the CTCAE version 4.03 and the NIH Scoring of GvHD.

Secondary Outcome Measures

Progression-free survival (PFS)

Incidence of AML/MDS-EB complete morphological and molecular remission (CR) at day + 30, + 90, +180 and 1 year post allo-HSCT

rejection.

Incidence of graft rejection

Number of NK cells given per kg body weight

Number of NK-DLI infusions applied

Full Information

NCT ID

NCT03300492

First Posted

July 30, 2017

Last Updated

November 28, 2022

Sponsor

University Hospital, Basel, Switzerland

1. Study Identification

Unique Protocol Identification Number

NCT03300492

Brief Title

Expanded Natural Killer Cells Following Haploidentical HSCT for AML/MDS

Official Title

A Phase I/II Single Center Study to Assess the Safety, Tolerability and Feasibility of Pre-emptive Immunotherapy With in Vitro Expanded Natural Killer Cells in Patients Treated With Haplo-HSCT for AML/MDS

Study Type

Interventional

2. Study Status

Record Verification Date

November 2022

Overall Recruitment Status

Recruiting

Study Start Date

November 12, 2018 (Actual)

Primary Completion Date

January 31, 2023 (Anticipated)

Study Completion Date

January 31, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

University Hospital, Basel, Switzerland

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

The study examines the application of expanded natural killer cells (NK cells) following haploidentical allogeneic hematopoietic stem cell transplantation (haplo-HSCT) for AML or MDS. Haplo-HSCT is a preferred treatment option for patients with AML or MDS without a HLA-matched donor. With administration of cyclophosphamide post-transplant , the safety of the procedure is similar to a HSCT from a HLA-identical donor. Relapse of AML/MDS represents a serious problem following haplo-HSCT. NK cells are immune cells able to destroy tumor cells. Their potency has been established particularly in the setting of a haplo-HSCT. In the current study, study participants undergoing haplo-HSCT will receive expanded NK cells from their respective stem-cell donors following haplo-HSCT. The primary goal of the study is to establish the safety and feasibility of this approach. In addition, the activity of the NK cells will be examined.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Acute Myeloid Leukemia, Myelodysplastic Syndromes

Keywords

Natural Killer cells, NK cells, Immunotherapy, haploidentical hematopoietic stem cell transplantation

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

Model Description

Prospective, single center, open-label, single arm study

Masking

None (Open Label)

Allocation

N/A

Enrollment

10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

NK-DLI

Arm Type

Experimental

Arm Description

Preemptive immunotherapy with ex vivo expanded NK cells on days +10, +15 and +20 with increasing NK cell doses following haplo-HSCT.

Intervention Type

Other

Intervention Name(s)

NK-DLI

Intervention Description

Application of three infusions of ex vivo expanded NK cells on days +10, +15 and +20 with increasing NK cell doses (1x107/kg, 1x108/kg and the remaining cells up to 1x109/kg) following haplo-HSCT. Maximal cumulative T-cell dose is fixed at <1x105/kg.

Primary Outcome Measure Information:

Title

Incidence and severity of adverse events including GvHD and infections.

Description

As defined by the CTCAE version 4.03 and the NIH Scoring of GvHD.

Time Frame

1 year following haplo HSCT

Secondary Outcome Measure Information:

Title

Progression-free survival (PFS)

Time Frame

1 year following haplo HSCT

Title

Incidence of AML/MDS-EB complete morphological and molecular remission (CR) at day + 30, + 90, +180 and 1 year post allo-HSCT

Description

rejection.

Time Frame

1 year following haplo HSCT

Title

Incidence of graft rejection

Time Frame

1 year following haplo HSCT

Title

Number of NK cells given per kg body weight

Time Frame

30 days following haplo-HSCT

Title

Number of NK-DLI infusions applied

Time Frame

30 days following haplo-HSCT

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Patient: >18 years of age No HLA-matched related or unrelated donor available AML or MDS-EB with indication for a haplo-HSCT according to the guidelines of the University Hospital Basel Stem Cell Transplant Team Judged by the transplant physicians to have adequate organ function and no contraindications to haplo-HSCT Available related haploidentical donor Written informed consent Donor: >18 years old, haploidentical parent, sibling or other relative Donor suitable for cell donation and apheresis according to standard criteria Written informed consent Exclusion Criteria: Patient: APL diagnosis Presence of relevant (mean fluorescence intensity >2000) donor-specific anti-HLA antibodies Pregnancy Necessity of immunosuppression apart from GvHD prophylaxis Exclusion Criteria: Donor: • Pregnancy

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Matyas Ecsedi, MD-PhD

Phone

+41612652525

matyas.ecsedi@usb.ch

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Jakob Passweg, Prof. MD

Organizational Affiliation

University Hospital Basel, Basel Switzerland

Official's Role

Principal Investigator

Facility Information:

Facility Name

University Hospital Basel

City

Basel

ZIP/Postal Code

4031

Country

Switzerland

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Matyas Ecsedi, MD-PhD

Phone

+41612652525

matyas.ecsedi@usb.ch

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

Expanded Natural Killer Cells Following Haploidentical HSCT for AML/MDS

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