Clinical trial NCT03300492: 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
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Acute Myeloid Leukemia
Myelodysplastic Syndromes
Keywords: Natural Killer cells
NK cells
Immunotherapy
haploidentical hematopoietic stem cell transplantation
Lead sponsor: University Hospital, Basel, Switzerland
Sponsor class: OTHER
First posted: 2017-10-03
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Prospective, single center, open-label, single arm study
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Incidence and severity of adverse events including GvHD and infections.
timeFrame: 1 year following haplo HSCT
Secondary outcomes:
measure: Progression-free survival (PFS)
timeFrame: 1 year following haplo HSCT
measure: Incidence of AML/MDS-EB complete morphological and molecular remission (CR) at day + 30, + 90, +180 and 1 year post allo-HSCT
timeFrame: 1 year following haplo HSCT
measure: Incidence of graft rejection
timeFrame: 1 year following haplo HSCT
measure: Number of NK cells given per kg body weight
timeFrame: 30 days following haplo-HSCT
measure: Number of NK-DLI infusions applied
timeFrame: 30 days following haplo-HSCT
Other outcomes:
Locations:
city: Basel
country: Switzerland
facility: University Hospital Basel
Study officials:
affiliation: University Hospital Basel, Basel Switzerland
name: Jakob Passweg, Prof. MD
role: PRINCIPAL_INVESTIGATOR
References: