RecruitingPhase 1 / Phase 2

Pilot Study of Memory-like Natural Killer (ML NK) Cells After TCRαβ T Cell Depleted Haploidentical Transplant in AML

A Phase I/II Pilot Study of Memory-like NK Cells to Consolidate TCRαβ T Cell Depleted Haploidentical Transplant in High-risk AML

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests whether special immune cells called memory-like natural killer (ML NK) cells can be made and safely given after a stem cell transplant with certain T cells removed. It is for people age 40 or younger with high-risk acute myeloid leukemia (AML), a blood cancer, who have a partly matched family donor.

This trial represents a single institution phase I/II pilot study with the primary objective of establishing the safety and feasibility of generating and infusing ML NK cells after TCRαβ haplo-HCT.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be age 40 or younger and meet the study’s score for daily activity and self-care.
  • Group 1: Have high-risk AML with less than 5% leukemia cells in the bone marrow. You must also meet rules about high-risk features, past remission, or AML linked to earlier cancer treatment or a bone marrow disorder. One prior stem cell transplant is allowed.
  • Group 2: Have AML that has not gone into remission after treatment, or that has returned after at least two stem cell transplants.
  • Group 2: Usually have less than 25% leukemia cells in the bone marrow. Special rules apply if the marrow has very few cells or test results differ greatly.
  • Meet the study’s liver, kidney, heart, and lung function requirements.
  • Have a partly matched family donor who meets donor safety rules and agrees to two blood cell collections. Donors are usually adults; donors age 12 or older may qualify in special cases.
  • Agree to use birth control or avoid sex that could cause pregnancy before and during the study and for 24 months after transplant.
  • You or your guardian must understand the study and sign a consent form.

Who can't join

  • Active graft-versus-host disease, when donated cells attack your body. If you had it before, you must be off immune-suppressing drugs for at least three months before study treatment.
  • Active cancer other than a blood cancer, or active leukemia in the brain, spinal cord, or outside the bone marrow. Past disease that is now gone may be allowed.
  • Taking another experimental treatment at transplant, or unable to stop drugs that may block ML NK cell activity.
  • Past allergic reactions to treatments similar to those used in this study.
  • High levels of antibodies against the donor’s tissue markers, which may attack donated cells.
  • Another major illness that makes transplant unsafe, or an uncontrolled illness such as an infection or certain serious heart problems.
  • Fanconi anemia or Down syndrome.
  • Pregnant or breastfeeding. Those who can become pregnant must have a negative pregnancy test within 14 days before transplant preparation starts.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Patient Inclusion Criteria - Cohort 1: 1. High risk acute myeloid leukemia (AML) in either: 1. Complete remission (CR) defined by \< 5% marrow blasts by morphology in the context of hematological recovery (ANC ≥ 0.5× 10\^9/L, platelet count ≥ 50 × 10\^9/L). 2. Morphological leukemia free state (MLFS) defined by the absence of hematological recovery and \< 5% marrow blasts by morphology 2. Patients must further meet one of the below for inclusion into the study: 1. De novo AML in CR1 with any of the following high-risk features: * MRD ≥ 1% after first induction course * MRD ≥ 0.1% after second induction course * RPN1-MECOM * RUNX1-MECOM * NPM1-MLF1 * DEK-NUP214 * KAT6A-CREBBP (if ≥ 90 days at diagnosis) * FUS-ERG * KMT2A-AFF1 * KMT2A-AFDN * KMT2A-ABI1 * KMT2A-MLLT1 * 11p15 rearrangement (NUP98 - any partner gene) * 12p13.2 rearrangement (ETV6 - any partner gene) * Deletion 12p to include 12p13.2 (loss of ETV6) * Monosomy 5/Del(5q) to include 5q31 (loss of EGR1) * Monosomy 7 * 10p12.3 rearrangement (MLLT10 - any partner gene) * FLT3/ITD with allelic ratio \> 0.1%, without bZIP CEBPA or NPM1 * RAM phenotype as evidenced by flow cytometry * Other high-risk features not explicitly stated here, after discussion/approval with protocol PI. 2. De novo AML in ≥ CR2 3. Therapy-related AML in CR1 4. AML evolving from myelodysplastic syndrome (MDS) 3. One prior hematopoietic cell transplant is allowed, provided remission criteria as defined above are met. Patient Inclusion Criteria - Cohort 2: 1. High risk acute myeloid leukemia (AML) defined by either of the following: 1. Treatment refractory disease: AML that is not in complete remission despite prior standard or salvage therapies. 2. Multiply relapsed disease: AML that has relapsed after 2 or more hematopoietic cell transplantations. 2. BM disease burden criteria: 1. Bone marrow blasts must be \<25% by morphologic assessment on aspirate smear, based on a manual differential count of at least 200 nucleated cells. 2. If an absolute discrepancy of ≥ 20 percentage points exists between morphologic blast evaluation and ancillary studies - including flow cytometry, cytogenetics, and molecular analyses, eligibility determination is at the investigator's discretion. 3. Hypocellular / Aplastic Marrow Exception: If bone marrow cellularity on core biopsy is less than 25%, the blast percentage threshold and the 200-cell minimum differential count requirement are waived. Patient Inclusion Criteria - Both Cohorts: 1. Less than or equal to 40 years of age. 2. Lansky (\<16 years) or Karnofsky (≥16 years) performance status of \>60%. 3. Adequate organ function as defined below: 1. Total bilirubin ≤ 3 x IULN for age 2. AST(SGOT)/ALT(SGPT) ≤ 5 x IULN for age 3. GFR ≥ 60 mL/min/1.73m2 as estimated by (1) updated Schwartz formula for ages 1-17 years or Cockcroft-Gault formula for ages ≥ 18 years, (2) 24-hour creatinine clearance, or (3) renal scintigraphy. If GFR is abnormal for age based on updated Schwartz or Cockcroft-Gault formula, accurate measurement should be obtained by either 24-hour creatinine clearance or renal scintigraphy. 4. Renal function may also be estimated by serum creatinine based on age/gender. A serum creatinine \< 2 x IULN for age/gender is required for inclusion on this protocol. 4. Adequate cardiac function, defined by left ventricular ejection fraction (LVEF) at rest ≥50% or shortening fraction (SF) ≥27% (via echocardiogram or MUGA). 5. Adequate pulmonary function, defined by: 1. FEV1, FVC, and DLCO ≥50% of predicted. 2. O2 saturation ≥ 92% on room air by pulse oximetry and no supplemental O2 at rest for children \< 8 years of age or those unable to perform pulmonary function testing (PFT). For children unable to perform PFT, a high-resolution CT chest should be obtained. 6. The effects of these treatments on the developing human fetus are unknown. For this reason, women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control, abstinence) prior to study entry, for the duration of study participation, and for 24 months following transplant. Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately. 7. Ability to understand and willingness to sign an IRB approved written informed consent document, or patient has a guardian who has the ability to understand and willingness to sign an IRB approved written informed consent document. 8. Available familial haploidentical donor. The HCT donor must be available and willing to undergo 2 leukapheresis procedures: (I) one mobilized collection for the HPC graft and (II) one non-mobilized leukapheresis collection for the manufacturing of ML NK cells. 9. Donor and recipient must be identical at a minimum of one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, HLA-DRB1, and HLA- DQB1. A minimum of 5/10 match is required and will be considered sufficient evidence that the donor and recipient share one HLA haplotype. Patient Exclusion Criteria - Both Cohorts 1. Active GvHD. If patient had prior GvHD, patient must be off immunosuppression for at least 3 months prior to starting study treatment. 2. Active non-hematologic malignancy. History of other malignancy is acceptable as long as therapy has been completed and there is no current evidence of disease. 3. Currently receiving any other investigational agents at the time of transplant. 4. Active CNS or extramedullary disease. History of CNS or extramedullary disease currently in remission is acceptable. 5. A history of allergic reactions attributed to compounds of similar chemical or biologic composition to agents used in the study. 6. Inability to discontinue medications that are likely to interfere with ML NK cell activity, i.e., glucocorticoids and other immunosuppressants. 7. Presence of significant anti-donor HLA antibodies per institutional standards. Anti-donor HLA - Antibody Testing is defined as a positive crossmatch test of any titer (by complement dependent cytotoxicity or flow cytometric testing) or the mean fluorescence intensity (MFI) of any anti-donor HLA antibody by solid phase immunoassay \> 3000. 8. Presence of a second major disorder deemed a contraindication for HCT. 9. Patients with Fanconi Anemia or Down Syndrome. 10. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection (bacterial, viral with clinical instability, or fungal), symptomatic congestive heart failure, or unstable cardiac arrhythmia. 11. Pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 14 days of the start of conditioning. Donor Eligibility Criteria - Both Cohorts 1. The preferred donor should be an adult aged 18 years or older. However, in circumstances where no suitable adult donor is available, consideration may be given to a minor donor aged 12 years or older. This exception only applies when all identified, otherwise eligible adult donors meet one or more of the following criteria: * A medical condition that poses unacceptable risk, including autoimmune disease, infection, hematologic disorder, malignancy or a pathogenic germline mutation. * Comorbidities that preclude safe administration of granulocyte colony-stimulating factor (G-CSF), placement of a pheresis catheter and/or stem cell collection. * Served as donor in prior haploidentical HCT. * Significant psychosocial or logistical barriers. 2. Donor must be HLA haploidentical (≥ 5/10 and ≤ 9/10 allele match at the -A, -B, -C, DRB1 and DQ loci) by high resolution typing and related to the patient. 3. Donor must meet the selection criteria as defined by the Foundation for the Accreditation of Hematopoietic Cell Therapy (FACT). 4. Donor must be available and willing to undergo one mobilized and one non-mobilized leukapheresis procedure. 5. Donor may not be pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 7 days prior to initiation of recipient's conditioning regimen, within 7 days of donor stem cell mobilization regimen and prior to second non-mobilized leukapheresis.. 6. Donor must be able to understand and willing to sign an IRB-approved written informed consent document.

Study sites (1)

MO

  • Washington University School of Medicine · St Louis, MO 63110Thomas M Pfeiffer, M.D. · Study contact · 314-273-2070 · pthomas@wustl.eduThomas M Pfeiffer, M.D. · Principal investigatorAmanda Cashen, M.D. · Sub-investigatorTodd Fehniger, M.D., Ph.D. · Sub-investigatorMore trials in St Louis