AzTrials
AzTrials

Refractory Acute Myelogenous LeukemiaPhase 1NCT03081780

Open Label NK Cell Infusion (FATE-NK100) With Subq IL-2 in Adults With AML

A Phase 1 study testing FATE-NK100 in people with Refractory Acute Myelogenous Leukemia and Relapsed Acute Myelogenous Leukemia, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This is a Phase I open-label dose escalation study of a single infusion of FATE-NK100 and a short course of subcutaneous interleukin-2 (IL-2) administered after lymphodepleting chemotherapy (CY/FLU) in subjects with refractory or relapsed acute myelogenous leukemia (AML). Read more about this study

Who is it for?
Ages 18 to 70 years
What is being tested?
FATE-NK100
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
1 siteUnited States
Study dates
27 Apr 2017to 1 Dec 2020
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • ≥18 but ≤ 70 years of age
  • Diagnosis of acute myeloid leukemia (AML) and meets one of the following disease criteria:
  • Primary induction failure:
  • De Novo AML: no CR after 2, 3 or 4 induction attempts with high dose chemotherapy
  • Secondary AML (from MDS or treatment related): no CR after 1, 2 or 3 cycles of high dose chemotherapy
  • Relapsed:
Show all 33Show fewer
  • Not in CR after 1 or 2 cycles of standard re-induction therapy
  • Relapse diagnosed at the time of the 6 months post-HCT standard of care follow-up or later (i.e. based on bone marrow biopsy performed Day +170 or later) and without evidence of graft versus host disease (GVHD)
  • For patients > 60 years of age, the minimum of 1 cycle of standard chemotherapy is not required.
  • Available HLA-matched or better but not fully HLA-matched (2/4 or 3/4 antigens) related donor (aged 18 to 75 years) with donor/recipient match based on a minimum of intermediate resolution DNA based Class I typing of the A and B locus who is CMV seropositive.
  • Karnofsky Performance Status ≥ 60%
  • Adequate organ function within 14 days of study registration (28 days for pulmonary and cardiac) defined as:
  • Creatinine: Estimated glomerular filtration rate (eGFR) ≥ 50 mL/min/1.73m^2 per current institutional calculation formula
  • Hepatic: AST and ALT ≤ 3 x upper limit of institutional normal
  • Pulmonary Function: oxygen saturation ≥ 90% on room air; PFT's required only if symptomatic or prior known impairment - must have pulmonary function >50% corrected DLCO and FEV1
  • Cardiac Function: LVEF ≥ 40% by echocardiography or MUGA
  • No symptomatic active conduction system abnormalities
  • Able to be off prednisone or other immunosuppressive medications for at least 3 days prior to FATE-NK100 cell infusion (excluding preparative regimen premedications)
  • Sexually active females of child bearing potential and males with partners of child bearing potential must agree to use effective contraception during therapy and for 4 months after completion of therapy
  • Voluntary written consent prior to the performance of any research related procedures
  • Arm Specific Inclusion Criteria
  • High-Risk aGVHD (ARM 1):
  • Pediatric or adult (ages 0-76 years) HCT recipients with high-risk acute GVHD, as determined either by the refined MN acute GVHD risk score [28]: http://z.umn.edu/MNAcuteGVHDRiskScore OR high risk on the basis of blood biomarkers (Ann Arbor Score 3) [31]. Patients in this arm must start treatment within the first 7 days after onset of high-risk aGVHD.
  • or
  • Steroid- Dependent aGVHD (ARM 2A):
  • Pediatric or adult (ages 0-76 years) HCT recipient with grade II-IV steroid-dependent acute GVHD, defined as any one of the following: Flare of acute GVHD of at least grade II/IV severity within 8 weeks of tapering down or (off of) immunosuppression for acute GVHD, with the flare occurring on ≤0.5 mg/kg prednisone. This can include late-onset aGVHD and overlap syndrome.
  • or
  • Steroid-Refractory aGVHD (ARM 2B):
  • Pediatric or adult (ages 0-76 years) HCT recipient with grade II-IV steroid refractory acute GVHD, defined as any one of the following:
  • No response of acute GVHD after at least 4 days of systemic corticosteroids of at least 2 mg/kg prednisone or equivalent
  • Progression of acute GVHD within 3 days of systemic corticosteroids of at least 2 mg/kg prednisone or equivalent
  • Failure to improve to at least grade II acute GVHD after 14 days of systemic corticosteroids, with initial doses of at least 2 mg/kg prednisone or equivalent
  • Flare of acute GVHD of at least grade II/IV severity while on steroids at a dose >0.5/mg/kg/day. This can include late-onset aGVHD and overlap syndrome.

Who cannot join

  • Myocardial Infraction (MI) within the previous 6 months
  • Acute leukemias of ambiguous lineage
  • Pregnant or breastfeeding - The agents used in this study include those that fall under Pregnancy Category D - have known teratogenic potential. Women of child bearing potential must have a negative pregnancy test at screening
  • History of or known active CNS involvement with AML
  • Active autoimmune disease requiring systemic immunosuppressive therapy
  • History of severe asthma and currently on chronic systemic medications (mild asthma requiring inhaled steroids only is eligible)
Show all 9Show fewer
  • New or progressive pulmonary infiltrates on screening chest X-ray or chest CT scan unless cleared for study by Pulmonary. Infiltrates attributed to infection must be stable/improving (with associated clinical improvement) after 1 week of appropriate therapy (4 weeks for presumed or documented fungal infections).
  • Uncontrolled bacterial, fungal or viral infections including HIV-1/2 or active hepatitis C/B - chronic asymptomatic viral hepatitis is allowed
  • Received any investigational agent within the 14 days before the start of study treatment (1st dose of fludarabine)

Trial locations and contacts

1 site in 1 country.

United States1 site

  • University of Minnesota, Masonic Cancer Center

    Minneapolis, Minnesota, 55455

Other trials in United States

About this study

Official title: Open Label Dose Escalation Trial of an Adaptive Natural Killer (NK) Cell Infusion (FATE-NK100) With Subcutaneous IL-2 in Adults With Refractory or Relapsed Acute Myelogenous Leukemia (AML)

This is a Phase I open-label dose escalation study of a single infusion of FATE-NK100 and a short course of subcutaneous interleukin-2 (IL-2) administered after lymphodepleting chemotherapy (CY/FLU) in subjects with refractory or relapsed acute myelogenous leukemia (AML). FATE-NK100 is a natural killer (NK) cell product that is enriched for NK cells with an "adaptive", or human cytomegalovirus (CMV)-induced, phenotype. The NK cell product is comprised of peripheral blood (PB) leukocytes sourced from a related donor (HLA-haploidentical or better but not fully HLA-matched) that is seropositive for cytomegalovirus (CMV+), and enriched for adaptive NK cells by depletion of CD3+ (T-lymphocytes) and CD19+ (B-lymphocytes) cells followed by ex-vivo culture expansion.

Being tested
FATE-NK100
Keywords
AML
Medical subject headings
Leukemia, Myeloid, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • FATE-NK100Biological

    Preparative regimen:

    • Fludarabine 25 mg/m2 x 5 days start Day -6
    • Cyclophosphamide 60 mg/kg x 2 days on Day -5 and -4

    Apheresis cell collection (collected from the Donor Day - 8) will be enriched for FATE-NK100 per CMC. IL-2 at 6 million IU subcutaneously (SC) every other day (EOD) for 6 doses with Dose 1 on Day 0 (no sooner than 4 hours post NK cells) and last dose no later than Day +12.

Study groups in NCT03081780
Study groupRoleWhat they receive
FATE NK-100ExperimentalNot described
Receives: Biological: FATE-NK100

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Maximum tolerated dose (MTD)

    Maximum FATE-NK100 dose which could be given to 3 participants such that not more than 1 participant experienced a DLT.

    Measured: 3 months

Secondary outcomes (8)

  1. Clinical activity by CR/CRp leukemia clearance

    Incidence of CRp defined as leukemia clearance (≤5%marrow blasts and no circulating peripheral blasts)

    Measured: Day +42

  2. Clinical activity by CR/CRp neutrophil recovery

    Incidence of CRp defined as neutrophil recovery (ANC >500 cells/microliter) but with incomplete platelet recovery

    Measured: Day +42

  3. In vivo expansion of NK cells

    Incidence of in vivo expansion (≥ 100 donor derived NK cells per uL blood) of NK cells

    Measured: Day +14

  4. Treatment Related Mortality (TRM)

    Incidence of treatment related mortality (TRM)

    Measured: 6 months

  5. Minimal residual disease (MRD) by bone marrow morphology

    Incidence of minimal residual disease (MRD) clearance by bone marrow morphology after NK Cell infusion

    Measured: up to Day 28

  6. Minimal residual disease (MRD) by flow cytometry

    Incidence of minimal residual disease (MRD) clearance by flow cytometry after NK Cell infusion

    Measured: up to Day 28

  7. Leukemia free survival (LFS)

    Incidence of Leukemia free survival (LFS)

    Measured: 1 year

  8. Overall survival (OS)

    Incidence of overall survival (OS)

    Measured: 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Mar 2017
  2. Study start27 Apr 2017
  3. Primary completion1 Aug 2020
  4. Study completion1 Dec 2020

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03081780
Sponsor's protocol ID
2016LS153
Status last verified
Mar 2021
First submitted
6 Mar 2017
Last updated
12 Mar 2021
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing FATE-NK100 in people with Refractory Acute Myelogenous Leukemia and Relapsed Acute Myelogenous Leukemia, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 33 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 12 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Maximum tolerated dose (MTD) (time frame: 3 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03081780.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Mar 2021.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in