Expanded Natural Killer Cells Following Haploidentical HSCT for AML/MDS

NCT03300492 | Recruiting Phase 1

• 1 other identifier: Haplo-NK-DLI for AML/MDS
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

Myelodysplastic Syndromes; Acute Myeloid Leukemia

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of adverse events including GvHD and infections.

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

  1 year following haplo HSCT

Secondary Outcomes (5)

• Progression-free survival (PFS)

  1 year following haplo HSCT

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

  1 year following haplo HSCT

• Incidence of graft rejection

  1 year following haplo HSCT

• Number of NK cells given per kg body weight

  30 days following haplo-HSCT

• Number of NK-DLI infusions applied

  30 days following haplo-HSCT

Study Arms (1)

NK-DLI

EXPERIMENTAL

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

Other: NK-DLI

Interventions

NK-DLI OTHER

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.

NK-DLI

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• University Hospital, Basel, Switzerland: lead

Study Sites (1)

University Hospital Basel

Basel, 4031, Switzerland

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases

Study Officials

• Jakob Passweg, Prof. MD

  University Hospital Basel, Basel Switzerland

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Matyas Ecsedi, MD-PhD

CONTACT

+41612652525; matyas.ecsedi@usb.ch

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Prospective, single center, open-label, single arm study

Study Record Dates

• First Submitted: July 30, 2017
• First Posted: October 3, 2017
• Study Start: November 12, 2018
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: December 4, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

CH (1)