Phase I/II Clinical Trial of Universal Donor CD33 CAR Natural Killer Cells for AML
A Phase I/II Clinical Trial Evaluating the Safety and Efficacy of Universal Donor CD33 CAR Natural Killer Cells for Treatment of Relapsed/Refractory Acute Myeloid Leukemia
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 1 Year to 39 Years
- 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
- Time periods mentioned: 1 year, 15 years
- Procedures mentioned: Infusions, Blood draws, Physical exams, Biopsies, Spinal tap (lumbar puncture)
Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.
Show the study's own words
- "Patients will undergo screening, chemotherapy (Fludarabine and Cytarabine, in combination with Venetoclax) followed by the infusion of the CD33 CAR NK cells."
- "Patients will be hospitalized for the chemotherapy and CD33 CAR NK cell infusion for close monitoring and will remain in the hospital until blood counts recover."
- "If patients are discharged from the hospital before day 35, they will be followed in clinic weekly for blood work and a physical exam."
- "A bone marrow biopsy will be performed around day 28-35 to see if the patient's leukemia is in remission."
- "Lumbar puncture or imaging may also be done if the study doctor thinks it is necessary."
- "Patients will continue to be followed for research studies and clinical outcomes (leukemia relapse, survival) for 1 year."
- 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 immune cells from a healthy donor that are changed to attack acute myeloid leukemia (AML), a blood cancer. It is for people ages 1 to under 40 whose AML has come back or has not responded to treatment. The study will find a safe dose and test how well it works after chemotherapy.
This phase 1/2 study is testing a new treatment for acute myeloid leukemia (AML) that has come back or has not responded to other treatments. The treatment uses specially modified immune cells (called CD33 CAR-NK cells) from a healthy, unrelated donor to attack the cancer. The first part of the study (Phase I) will focus on finding the safest and most effective dose. The second part (Phase II) will test how well the treatment works at that dose. Patients will undergo screening, chemotherapy (Fludarabine and Cytarabine, in combination with Venetoclax) followed by the infusion of the CD33 CAR NK cells. Some patients may receive 2 doses of CD33 CAR NK cells infused 1 week apart. The investigator will let participants know if they will receive 1 or 2 doses. Patients will be hospitalized for the chemotherapy and CD33 CAR NK cell infusion for close monitoring and will remain in the hospital until blood counts recover. If patients are discharged from the hospital before day 35, they will be followed in clinic weekly for blood work and a physical exam. A bone marrow biopsy will be performed around day 28-35 to see if the patient's leukemia is in remission. Lumbar puncture or imaging may also be done if the study doctor thinks it is necessary. Patients will continue to be followed for research studies and clinical outcomes (leukemia relapse, survival) for 1 year. After 1 year, patients will have completed their study participation, but can be monitored for up to 15 years for potential long term side effects of the cell therapy. Some patients may undergo a bone marrow transplant after the study treatment. Patients who proceed to bone marrow transplant will have one blood sample drawn about a month after the transplant and then will have completed study participation.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Have AML with a marker called CD33 that has come back or has not cleared after 2 rounds of starting or repeat chemotherapy. Small amounts of cancer left after treatment may qualify.
- Be age 1 to under 40. The first 3 patients and the first patient at each dose level must be at least 16.
- Cancer only outside the bone marrow may qualify. Cancer in the brain or spinal cord is allowed only in Phase II.
- Meet study limits for kidney, liver, heart, and lung function. Seizures may be allowed if well controlled.
- If able to become pregnant, have a negative blood pregnancy test within 14 days before joining. Sexually active patients who could cause a pregnancy must use approved birth control for 6 months after the last chemotherapy or cell dose.
- Have a stem cell donor identified. Be able and willing to have a donor stem cell transplant if treatment stops the bone marrow from making blood cells.
- You or your legal guardian must understand the study and sign a consent form.
- Agree to join a separate study to track long-term effects of cell and gene therapy.
Who can't join
- Recent treatment: most AML drugs within 14 days before study treatment; CD33-targeting antibodies within 42 days before joining; brain or spinal cord radiation within 28 days; donor immune cells or other cell therapy within 30 days; or a donor stem cell transplant within 90 days. Hydroxyurea and certain drugs given into spinal fluid are exceptions.
- Use of medicines that lower immune activity within 14 days before joining, except allowed steroid doses. Hydrocortisone for low adrenal hormones and other steroids up to the study's dose limit may be allowed.
- Active graft-versus-host disease, when donor cells attack your body and medicines are needed to control symptoms.
- An uncontrolled illness or a social situation that makes study visits or care hard to follow, or poses an unacceptable risk.
- Breastfeeding or a past transplant of a solid organ, such as a kidney or liver.
- A score below 50 on the study's scale of ability to do daily activities.
- An infection that has not cleared or greatly improved with treatment. A virus found in the blood without symptoms does not by itself rule you out.
- An uncontrolled irregular heartbeat or uncontrolled heart disease that causes symptoms.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: 1. Patients with relapsed or primary refractory CD33+ AML, including: * Patients with relapsed AML (patients in second or subsequent relapse, or any relapse after HSCT, are eligible). * Refractory AML defined as failure to achieve a complete response after 2 cycles of induction or reinduction chemotherapy, including persistent MRD positivity. * Patients with isolated CNS or extramedullary disease are eligible. Patients with CNS disease are excluded from the phase I dose escalation portion but are eligible for the phase II portion of the study. 2. 1-39.99 years of age (note: the first three subjects treated AND the first subject on each dose level must be ≥ 16 years of age) 3. Negative serum test to rule out pregnancy within 14 days prior to enrollment in females of childbearing potential o Sexually active males and females of childbearing potential must agree to use a form of contraception considered effective and medically acceptable by the Investigator for 6 months after the last dose of chemotherapy and/or NK cell infusion 4. Organ function requirements: * Renal function: Creatinine ≤ 2 times the institutional upper limit of normal for age OR creatinine clearance \> 60 ml/min/1.73m2 (measured by 24 hour- urine specimen or radioisotope GFR) * Liver function: Total bilirubin ≤ 2 mg/dl (unless Gilbert's syndrome), AST and ALT ≤ 5 times the upper limit of normal (unless related to leukemic involvement). Upper limit of normal should be determined by the institutional defined normal laboratory range. * Cardiac function: left ventricular ejection fraction ≥ 40% or shortening fraction ≥20%. May be eligible after cardiology clearance if qualitatively normal function or repeat measures are normal. * CNS: Patients with seizure disorder may be eligible if seizures are well controlled * Pulmonary function: baseline oxygen saturation \>92% on room air at rest 5. Due to the risk of hematopoietic toxicity from CD33 targeting, enrolled subjects must have an allogeneic HCT donor identified and be eligible and willing to undergo a subsequent HSCT in the event of aplasia. 6. All patients or their legal guardian must be able to understand and willing to sign a written informed consent document. 7. All patients must consent to enroll in a separate long term follow up study for cell and gene therapy Exclusion Criteria: 1. Prior therapies: * AML directed therapies in the 14 days prior to beginning treatment on this protocol (except for hydroxyurea) Note: There is no waiting period required for patients having received intrathecal cytarabine, methotrexate and/or hydrocortisone * Gemtuzumab or other CD33-targeted antibody within 42 days of enrollment * CNS radiation within 28 days of enrollment * DLI or adoptive cell therapy within 30 days of enrollment * Allogeneic SCT within 90 days of enrollment * Patients with CNS disease are excluded from the phase I portion of the study but are eligible for the phase II expansion phase. * Patients on immunosuppressive therapy * Patients must be off of systemic immunosuppressive therapy for at least 14 days prior to enrollment with no evidence of recurrent GVHD * Patients on hydrocortisone for treatment of adrenal insufficiency are permitted on study * Patients on corticosteroids ≤ 0.5mg/kg/day (prednisone equivalent) for any other indication are permitted on study 2. Uncontrolled, symptomatic, intercurrent illness or social situations that would limit compliance with study requirements or in the opinion of the site PI would pose an unacceptable risk to the subject 3. Patients who are breastfeeding 4. Patients with prior solid organ transplantation 5. Performance status: Karnofsky or Lansky Performance Scale (PS) \< 50 6. Uncontrolled infection, defined as an infection which has not resolved or does not show evidence of significant resolution after initiating appropriate therapy o Asymptomatic viremia such as CMV, HPV, BK virus, HCV, etc. is NOT considered as an exclusion criterion 7. Uncontrolled arrhythmias or uncontrolled symptomatic cardiac disease 8. Active acute or chronic GVHD of any grade at the time of enrollment. "Active GVHD" is defined as a patient who requires immunosuppressive therapy for control of their GVHD symptoms.
Study sites (1)
OH
- Nationwide Children's Hospital · Columbus, OH 43205Clelie Peck · Study contact · 614-722-5634 · Clelie.Peck@nationwidechildrens.orgMore trials in Columbus