Phase Ib Study of CD33 FPBMC in Patients With MRD+ AML or MDS
Phase Ib Study of Anti-CD3 x Anti-CD33 Bispecific Antibody (CD33Bi) Armed Fresh Peripheral Blood Mononuclear Cells (CD33 FPBMC) in Patients With Measurable Residual Disease (MRD)+ Acute Myeloid Leukemia or Myelodysplastic Syndrome
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
- Procedures mentioned: Infusions
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
- "Participants receive 4 weekly doses of CD33 FPBMC by intravenous infusion followed by 4-6 weeks of standard treatment with a hypomethylating agent (type of treatment such as decitabine or azacitidine) and possibly a drug called venetoclax."
- 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 the safety of CD33 FPBMC, blood cells armed with an antibody, and how well it may work. It is for adults with AML, a blood cancer, or MDS and certain related bone marrow diseases who still have disease after treatment. Treatment includes 4 weekly doses through a vein, followed by 4–6 weeks of standard drug treatment, and may repeat up to 4 times.
The purpose of this study is to understand the safety and estimate the efficacy of combining anti-CD3 x anti-CD33 bispecific antibody (CD33Bi) armed fresh peripheral blood mononuclear cells (CD33Bi FPBMC) for patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) where they still have detectable disease ("MRD+") after some treatment. Participants receive 4 weekly doses of CD33 FPBMC by intravenous infusion followed by 4-6 weeks of standard treatment with a hypomethylating agent (type of treatment such as decitabine or azacitidine) and possibly a drug called venetoclax. This is considered 1 cycle of study treatment and may be repeated up to 4 times during the study.
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 18 or older and able to give written consent.
- Have AML after strong starting chemotherapy or at least 2 cycles of lower-intensity treatment. MDS or related overlap diseases must have returned or not responded after the required prior treatment.
- Have remaining AML shown by blood, bone marrow, or gene tests. For MDS or overlap diseases, have signs of worsening disease, such as more immature blood cells, lower blood counts, or greater need for transfusions.
- Have remaining immature blood cells that carry CD33, a marker on cells. An earlier positive CD33 test may count in certain cases.
- If you have certain gene changes that drugs can target, have received that drug or made a decision not to receive it.
- Have a heart pumping strength of at least 45%. Be able to care for yourself and be out of bed or a chair for at least half the day.
- Agree to effective birth control during treatment and for 90 days afterward if required. Men must also avoid sperm donation during this time.
- For the dose-expansion part, have AML or MDS that returned or did not respond. Newly diagnosed disease and MDS overlap diseases are not allowed in that part.
Who can't join
- Being pregnant or breastfeeding, or having had treatment that targets CD33 before.
- Being actively considered for a stem cell transplant, unless the study and transplant doctors agree that joining first is in your best interest.
- Having donor cells attack your body after a past transplant and needing body-wide immune-suppressing drugs in the past 4 weeks, except low-dose prednisone or an equal dose of another drug.
- Having liver or kidney test results outside the study limits, very low lymphocytes (a type of white blood cell), or a serious infection not controlled by treatment.
- Having HIV with detectable virus, a positive hepatitis B surface antigen test, or known or suspected active hepatitis C. Some positive hepatitis antibody tests are allowed if no virus is detected.
- Having another cancer that could affect the study's safety or results.
- Having taken other leukemia drugs or chemotherapy too recently. The waiting period is 7 days or the time for 5 drug half-lives, whichever is shorter. Hypomethylating drugs and venetoclax are allowed; hydroxyurea may continue through the first cycle.
- Having an allergy to hypomethylating drugs, such as azacitidine or decitabine, or having blasts (immature blood cells) at 25% or higher.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: 1\. Adults: ≥18 years of age 2. Diagnosis of either: 1. Newly diagnosed or relapsed/refractory AML who have received either intensive induction chemotherapy or at least 2 cycles of a non-intensive options such as hypomethylating agent and venetoclax or other targeted agent 2. Relapsed/ refractory (R/R) MDS who have received at least 2 prior cycles of hypomethylating agent and venetoclax or single agent hypomethylating agent for at least 4 cycles 3. Relapsed/refractory (R/R) MDS/MPN overlap syndromes like CMML who have received at least 2 prior cycles of azacitidine and venetoclax or single agent hypomethylating agent for at least 4 cycles 3. For patients with targetable mutations (IDH1, IDH2, KMT2A, or FLT3): Receipt of and/or decision not to receive associated inhibitor. 4\. Patients with R/R MDS or R/R MDS/MPN overlap syndromes must have at least one of the following: 1. Bone marrow blasts ≥ 5% 2. Appearance of previously absent leukemic blasts in peripheral blood 3. Absolute neutrophil count \<1 x 109/L and 50% below best unsupported on-study value 4. Platelet count \<100 x 109/L and 50% below best unsupported on-study value 5. Hemoglobin \<11g/dL, and ≥2 g/dL reduction from best unsupported on-study value 6. Increase of the volume of transfused red blood cells by more than 30% in an 8-week period 7. Increase of the number of transfused platelet units by more than 30% in an 8-week period In the case of criteria 4-8 above, no reasonable alternative explanation such as drug toxicity should be identified. 5\. Patients with AML must have persistent or recurrent MRD positivity defined by presence of blasts ≥5% AND/OR disease detected by multiparametric flow cytometry (MFC) at a level of ≥0.1%, AND/OR persistent genomic mutations other than those found most with CHIP AND/OR persistent cytogenetic abnormalities related to underlying myeloid neoplasm 6\. Residual blasts must be positive for CD33 expression at any level. Note: Patients whose most recent disease-positive evaluation by flow cytometry showed CD33 expression but whose current assessment for MRD is only positive for genomics or cytogenetics may be included. 7\. Left Ventricular Ejection Fraction (LVEF) ≥ 45% at rest (MUGA or echocardiogram) 8\. Performance status ≤ 2 (ECOG Scale) 9\. Females of childbearing potential and males must agree to use an effective method for contraception for the duration of the treatment with study drug plus 90 days (duration of sperm turnover). Males must also abstain from sperm donations during study treatment and for at least 90 days after the last dose of study drug. 10\. Ability to provide informed consent and provision of written informed consent In order to be eligible to participate in the dose expansion portion of this study, an individual must meet all criteria that apply to the R/R MDS or R/R AML population (Newly diagnosed patients and patients with MDS/MPN overlap syndromes are not eligible for the expansion phase of the study). In order to be eligible to participate in the dose expansion portion of this study, an individual must meet all criteria that apply to the R/R MDS or R/R AML population (Newly diagnosed patients and patients with MDS/MPN overlap syndromes are not eligible for the expansion phase of the study). Exclusion Criteria: 1. Pregnancy or lactation 2. Prior treatment with anti-CD33 therapy 3. Patients who are being actively considered for stem cell transplant, unless participation in the study prior to the planned stem cell transplant is considered to be in the best interest of the patient in the opinion of the treating investigator in consultation with the transplant team. This does not exclude patients that may be eligible for stem cell transplant at some future (undetermined) date. 4. Past hematopoietic stem cell transplant (HSCT) with graft vs host disease requiring systemic immunosuppression other than low dose prednisone (10 mg) (or the equivalent dose of another immunosuppressant) within the 4 weeks before registration 5. Clinically significant organ dysfunction, defined as any of the following: * AST or ALT \>3x the upper limit of normal (ULN) * Total bilirubin \>1.5x the ULN, unless due to ongoing hemolysis or Gilbert's syndrome, in which case \> 3.0 mg/dL * Absolute lymphocyte count (ALC) \< 300 lymphocytes/microliter * Creatinine clearance \<30 mL/min * Active serious infection not controlled by oral or intravenous antibiotics (e.g. persistent fever or lack of clinical improvement despite antimicrobial treatment). 6. Known human immunodeficiency virus (HIV) with detectable viral load. 7. Known hepatitis B surface antigen seropositive or known or suspected active hepatitis C infection a. Note: Patients who have isolated positive hepatitis B core antibody (ie, in the setting of negative hepatitis B surface antigen and negative hepatitis B surface antibody) must have an undetectable hepatitis B viral load. Patients who have positive hepatitis C antibody may be included if they have an undetectable hepatitis C viral load. 8. Patients with a prior or concurrent malignancy whose natural history or treatment is anticipated to interfere with the safety or efficacy assessment of the investigational regimen (according to the treating investigator). 9. Treatment with any antileukemic agents or chemotherapy (other than hypomethylating agents or venetoclax) agents in the last 7 days or 5 half-lives (whichever is sooner) before study entry. Note: treatment with hydroxyurea may continue through the first cycle of study treatment. 10. Known allergy to hypomethylating agents 11. Blasts ≥ 25%
Study sites (1)
VA
- University of Virginia · Charlottesville, VA 22908Ashley Donihee · Study contact · 434-243-6377 · zwz6jm@uvahealth.orgAvani Hopkins · Study contact · 434-243-8108 · AJH7JQE@uvahealth.orgDaniel Reed, MD · Principal investigatorMore trials in Charlottesville