TerminatedNCT03152526Updated Jul 9, 2020Results posted

CD3/CD19 Depleted or CD3 Depleted/CD56 Selected Haploidentical Donor Natural Killer (NK) Cell Based Therapy for AML Patients Not in CR

An interventional study of Interventions in Acute Myelogenous Leukemia, sponsored by Miltenyi Biotec B.V. & Co. KG. Terminated at 3 sites in United States. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2020-07-09.

Sponsored by Miltenyi Biotec B.V. & Co. KG · Not applicable, Interventional, and Treatment

Why this study was terminated

Slow enrollment

Phase: Not applicable
Study type: Interventional
Enrollment: 1
Allocation: Not applicable
Ages: 18 Years to 75 Years
Sex: All

Study summary

This is a phase II trial designed to test the safety and efficacy (complete response [CR]) of related donor HLA-haploidentical NK-cell based therapy for the treatment of acute myelogenous leukemia (AML).

Patients with newly diagnosed AML who failed to achieve a complete remission (CR) after one or two standard induction attempts receive after a preparative regimen of cyclophosphamide and fludarabine a single infusion of CD3-/CD19- NK cells or CD3-/CD56+ NK cells followed by a short course of Interleukin-2 (IL-2) to facilitate NK cell survival and expansion.

Read the detailed description

This trial uses a Simon's two-stage design to estimate the complete remission rate at day +42 post NK cell infusion. The trial includes an initial randomized sub- study of 24 patients during stage 1 to choose which of the enriched NK cell products (CD3-/CD19- versus CD3-/CD56+) should be used to complete the trial based on successful in vivo NK cell expansion. This parameter is defined as 40% donor DNA and 40% of lymphocytes are NK cells at day 7 post infusion OR 20% donor DNA and 20% of lymphocytes are NK cells at day 14 post infusion. Twelve patients will be randomized to each product.

Enrollment Plan:

Stage 1: Enroll 24 patients with 1:1 randomization for NK cell processing (CD3-/CD19- versus CD3-/CD56+) Stage 2: Enroll an additional 17 patients using the optimal NK cell product identified during stage 1.

If neither product achieves success at the end of stage 1, the study will stop and the platform redesigned

Conditions studied

Acute Myelogenous Leukemia

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In context

Leukemia

5,441 studies on the registry are indexed under Leukemia; 636 are open to participants now.

This study's enrollment of 1 is below the median of 38 across 4,247 interventional studies indexed under Leukemia.

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Lead sponsor

Miltenyi Biotec B.V. & Co. KG is the lead sponsor of 3 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 18 Years to 75 Years
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

Newly diagnosed with acute myelogenous leukemia (except acute promyelocytic leukemia) and has failed one or two prior standard induction attempts. Failure is defined as:
≥ 30% bone marrow blasts with at least 20% cellularity at mid-cycle bone marrow biopsy or residual AML on subsequent\~ day 28 bone marrow biopsy by morphology, flow, PCR or FISH
Patients enrolling after only 1 failed induction attempt must meet at least one of the following additional eligibility criteria of high risk: ≥ 60 years of age adverse cytogenetics or molecular characteristics
AML that progressed out of myelodysplastic syndrome (MDS) is eligible if the patient did not receive treatment directed at the MDS
HLA-haploidentical related donor (aged 12 to 70 years)
≥ 18, but \< 75 years of age
Karnofsky performance status ≥ 60%
Adequate organ function within 14 days of study registration (30 days for pulmonary and cardiac) as defined in section 4.5
Ability to be off prednisone and other immunosuppressive drugs for at least 3 days prior to the NK cell infusion (excluding preparative regimen pre-meds)
No prior hematopoietic transplant
Not pregnant or lactating
Sexually active females of childbearing potential and males with partners of child bearing potential must agree to use birth control

Exclusion criteria

Exclusion Criteria:

Pregnant or lactating as the treatments used in this study includes drugs that are FDA Pregnancy Category D.
Acute leukemias of ambiguous lineage
AML that transformed from previously treated myelodysplastic syndromes
Prior hematopoietic transplant
New or progressive pulmonary infiltrates on screening chest x-ray or chest CT scan that has not been cleared 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 - chronic asymptomatic viral hepatitis is allowed
Known hypersensitivity to one or more of the study agents used

Study design

Phase: Not applicable
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrollment: 1 participant (actual)

Study arms

Other
Single-arm trial
Multi-center, open-label, single-arm, phase I/II clinical trial
Device: Interventions

Interventions

DeviceInterventions
CliniMACS® CD3 and CD19 Reagent System
Also known as: Devise

What researchers measure

Primary outcomes

The Primary Endpoint of the Study is Complete Remission (±3 Days)
Both the number of patients with leukemia in complete remission and the number of patients with leukemia not in complete remission will be reported. Leukemia remission status will be assessed according to the Revised Recommendations of The International Working Group (J Clin Oncol 21:4642-4649, 2003).
Time frame: On Day+42 (+/- 3 days) after NK cell infusion

Secondary outcomes

The Secondary Endpoint is the Expansion and Persistence of NK Cells to be Used for the Remainder of the Study.
Successful expansion and persistence of NK cells is defined as ≥ 100 donor derived NK cells per µl blood.
Time frame: Day+7 to Day+42 after NK cell infusion

Results

Posted May 7, 2019

Limitations and caveats

As a result of poor subject accrual (n=1), statistically relevant efficacy data cannot be defined according to the protocol.

Participant flow

Participant flow — Overall Study
Milestone	CD3-/CD19- NK Cells Followed by IL-2	CD3-/CD56+ NK Cells Followed by IL-2
Started	0	1
Completed	0	0
Not completed	0	1
Withdrew: Withdrawal by subject	0	1

Outcome measures

PrimaryThe Primary Endpoint of the Study is Complete Remission (±3 Days)

Both the number of patients with leukemia in complete remission and the number of patients with leukemia not in complete remission will be reported. Leukemia remission status will be assessed according to the Revised Recommendations of The International Working Group (J Clin Oncol 21:4642-4649, 2003).

Time frame:: On Day+42 (+/- 3 days) after NK cell infusion

No measurements were reported for this outcome.

SecondaryThe Secondary Endpoint is the Expansion and Persistence of NK Cells to be Used for the Remainder of the Study.

Successful expansion and persistence of NK cells is defined as ≥ 100 donor derived NK cells per µl blood.

Time frame:: Day+7 to Day+42 after NK cell infusion

No measurements were reported for this outcome.

Adverse events

Collected over 3 months. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
CD3-/CD19- NK Cells Followed by IL-2	—	—	—
CD3-/CD56+ NK Cells Followed by IL-2	1/1 (100%)	1/1 (100%)	1/1 (100%)

Most frequent serious events

Most frequent serious events
Event	CD3-/CD19- NK Cells Followed by IL-2	CD3-/CD56+ NK Cells Followed by IL-2
Serious adverse eventRespiratory, thoracic and mediastinal disorders	—	1/1
Serious adverse eventNeoplasms benign, malignant and unspecified (incl cysts and polyps)	—	1/1

Most frequent other events

Most frequent other events
Event	CD3-/CD19- NK Cells Followed by IL-2	CD3-/CD56+ NK Cells Followed by IL-2
Febrile neutropeniaBlood and lymphatic system disorders	—	1/1
Chills, edema limbs, fever, injection site reactionGeneral disorders	—	1/1
Dyspnea, pneumonitisRespiratory, thoracic and mediastinal disorders	—	1/1
Hypertension, hypotensionVascular disorders	—	1/1

Baseline characteristics

No participants were enrolled on the CD3-/CD19- NK cells arm. The study was closed due to poor enrollment.

Age, Customized
Age, Customized(years)	CD3-/CD19- NK Cells Followed by IL-2	CD3-/CD56+ NK Cells Followed by IL-2	Total
Participant age (years)	—	52	52

Sex: Female, Male
Sex: Female, Male(Participants)	CD3-/CD19- NK Cells Followed by IL-2	CD3-/CD56+ NK Cells Followed by IL-2	Total
Female	—	1	1
Male	—	0	0

Race and Ethnicity Not Collected
Race and Ethnicity Not Collected(Participants)	CD3-/CD19- NK Cells Followed by IL-2	CD3-/CD56+ NK Cells Followed by IL-2	Total
Count of participants	—	—	0

Study locations

3 sites

Universtiy of Chicago
Chicago, Illinois 60637, United States
University of Minnesota
Minneapolis, Minnesota 55455, United States
Ohio State University
Columbus, Ohio 43210, United States

References and documents

Study documents

Protocol and statistical analysis plan · Mar 9, 2017

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Jul 9, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT03152526
Lead sponsor: Miltenyi Biotec B.V. & Co. KG
Collaborators: Masonic Cancer Center, University of Minnesota, University of Chicago, Ohio State University
Responsible party: Sponsor
First posted: May 15, 2017
Start date: Oct 18, 2017
Primary completion: Mar 21, 2018
Completion: Mar 21, 2018
Results posted: May 7, 2019
Last update: Jul 9, 2020

Oversight

Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: Yes

View the source record on ClinicalTrials.gov ↗

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This study is terminated, as verified in Feb 2020. You cannot join it, but the record below documents what was studied.

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