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C123-CD33cCAR T cells transduced with a lentiviral vector to express two distinct units of anti-CD123 and CD33 CARs

CD123-CD33 cCAR T cells transduced with a lentiviral vector to express two distinct units of anti-CD123 and CD33 CARs.

CD123-CD33 cCAR T cells

Kevin Pinz

Fang Liu, MD, PhD

Hongyu Zhang, MD, PhD

Phase I, interventional, single arm, open label, treatment study to evaluate the safety and tolerability of CD123-CD33 cCAR in patients with relapsed and/or refractory, high risk hematologic malignancies.

AML bears heterogeneous cells that can consequently offset killing by single-CAR-based therapy, which results in disease relapse. Leukemic stem cells (LSCs) associated with CD123 expression comprise a rare population that also plays an important role in disease progression and relapse for myeloid malignancies. CD33 is widely expressed in AML, high risk myelodysplastic syndromes (MDS) and myeloproliferative neoplasms. Targeting both CD33 and CD123 surface antigens together may offer two distinct benefits. First, targeting both bulk disease and leukemic stem cells together allows for a more comprehensive ablation of the disease. Second, dual targeting of myeloid malignancies by both CD33 and CLL1 directed therapy overcomes the pitfalls of single-antigen therapy by preventing relapse due to antigen loss. While loss of a single antigen under antigen-specific selection pressure is possible, loss of two antigens simultaneously is much less likely.

CD123-CD33 cCAR is a compound Chimeric Antigen Receptor (cCAR) immunotherapy with two distinct functional CAR molecules expressed on a T-cell, directed against the surface proteins CLL1 and CD33. cCAR intends to target the mechanisms of single-CAR relapse, specifically antigen escape and leukemic stem cells.

CD123-CD33 cCAR in Patients With Relapsed and/or Refractory, High Risk Hematologic Malignancies

Phase I, Interventional, Single Arm, Open Label, Treatment Study to Evaluate The Safety and Tolerability of CD123-CD33 cCAR in Patients With Relapsed and/or Refractory, High Risk Hematologic Malignancies

Assessment of morphologic complete remission (CR), complete remission with incomplete recovery of counts (CR1), no residual disease as analyzed by flow cytometry analysis, and molecular remission by molecular studies

Peking University Shenzhen Hospital

Chengdu Military General Hospital

iCAR Bio Therapeutics Ltd.

iCell Gene Therapeutics

All patients who have received any part of or all of a VOR33 genome-edited hematopoietic stem and progenitor cell therapy product.

VOR33 is an allogeneic CRISPR/Cas9 genome-edited hematopoietic stem and progenitor cell therapy product that lacks the CD33 myeloid protein.

VOR33

Michelle Padilla

Divya Koura, MD

Guenther Koehne, MD, PhD

Monica Epstein

Nirali Shah, MD, MHSc

Cathy Wiggins

John DiPersio, MD

Hyung Suh, MD

Nurse Navigator

Markus Y Mapara, MD, PhD

Christina Cho, MD

Cancer Information Service Line

Brenda Cooper, MD

Roland Walter, MD, PhD, MS

Lea Bernard, MD

VBP201 study is the VOR33 long-term follow-up (LTFU) study focusing on assessing long term safety and efficacy of VOR33. This study may last up to 15 years (counted from the date of VOR33 infusion completion). All patients who have participated in a study involving the drug product VOR33 and have completed the applicable study or prematurely discontinued, but have at least received the VOR33 infusion, will be asked to participate in the LTFU study.

A Long-term Follow-up Study of Patients Who Received VOR33

Incidence of VOR33-related AEs/SAEs or deaths.

Percentage of CD33-negative hematopoiesis and change over time. Overall rates of RFS measured from the time of hematopoietic cell transplantation (HCT) to the date of disease relapse, and OS rates defined as the time from HCT to the date of death from any cause.

Vor Biopharma

The trial will be conducted in a manner of simon two-stage design with anti-CD33-CAR-transduced T cells, beginning in the first stage with the aim of over 30% reaction rate among 15 patients with myeloid malignancies. Only when the expected reaction rate is achieved the 30 patients left can be recruited.

The first 3 enrolled patients will receive autologous-derived CD33-targeted CAR-T cells on day 1, 2 and 3 with respective 10%, 30% and 60% of the total expected dosage after receiving lymphodepleting chemotherapy. If the 3 patients don't display severe toxicity,the next patients enrolled will get infused in 2 days with respective 40% and 60% total dosage.

Anti-CD33-CAR-transduced T cells

Cheng Qian, MD, PhD

Shiqi Li, MD

Cheng Qian, PhD

Zhi Yang, PhD

The overall purpose of this study is to explore the therapeutic effect of CD33-targeted chimeric antigen receptor T(CAR-T) cells in the treatment of Myeloid Malignancies.

Great progress has been made in the treatment of relapsed and refractory B cell malignancies with CD19-targeted CAR-T cells. However, for myeloid malignancies, which has higher morbidity, trials of CAR-T is few. CD33 is expressed on most myeloid leukemia cells so it is a ideal target for CAR-T. This trial is designed and conducted to test the safety and effectiveness of CD33-targeted CAR-T.

A Clinical Research of CD33-Targeted CAR-T in Myeloid Malignancies

Determine the toxicity profile of the CD33 targeted CAR T cells with Common Toxicity Criteria for Adverse Effects (CTCAE) version 4.0.

Southwest Hospital, China

Principal Investigator of Biotherapy Center

CLL1-CD33cCAR T cells transduced with a lentiviral vector to express two distinct units of anti-CLL1 and CD33 CARs

CLL1-CD33 cCAR T cells transduced with a lentiviral vector to express two distinct units of anti-CLL1 and CD33 CARs.

CLL1-CD33 cCAR T cells

Phase I, interventional, single arm, open label, treatment study to evaluate the safety and tolerability of CLL1-CD33 cCAR in patients with relapsed and/or refractory, high risk hematologic malignancies.

AML bears heterogeneous cells that can consequently offset killing by single-CAR-based therapy, which results in disease relapse. Leukemic stem cells (LSCs) associated with CLL1 expression comprise a rare population that also plays an important role in disease progression and relapse for myeloid malignancies. CD33 is widely expressed in AML, high risk myelodysplastic syndromes (MDS) and myeloproliferative neoplasms. Targeting both CD33 and CLL1 surface antigens together may offer two distinct benefits. First, targeting both bulk disease and leukemic stem cells together allows for a more comprehensive ablation of the disease. Second, dual targeting of myeloid malignancies by both CD33 and CLL1 directed therapy overcomes the pitfalls of single-antigen therapy by preventing relapse due to antigen loss. While loss of a single antigen under antigen-specific selection pressure is possible, loss of two antigens simultaneously is much less likely.

CLL1-CD33 cCAR is a compound Chimeric Antigen Receptor (cCAR) immunotherapy with two distinct functional CAR molecules expressed on a T-cell, directed against the surface proteins CLL1 and CD33. cCAR intends to target the mechanisms of single-CAR relapse, specifically antigen escape and leukemic stem cells.

CLL1-CD33 cCAR in Patients With Relapsed and/or Refractory, High Risk Hematologic Malignancies

Phase I, Interventional, Single Arm, Open Label, Treatment Study to Evaluate The Safety and Tolerability of CLL1-CD33 cCAR in Patients With Relapsed and/or Refractory, High Risk Hematologic Malignancies.

The General Hospital of Western Theater Command

Acute Myeloblastic Leukemia Without Maturation

Acute Myeloblastic Leukemia With Maturation

All Drugs and Chemicals

Immunomodulating Agents

Enrolled patients will receive CAR-NK cells immunotherapy with a novel specific chimeric antigen receptor targeting CD33 antigen by infusion.

The allogeneic NK cells (NK-92 cell line for clinical use) are engineered to contain anti-CD33 attached to TCRzeta, CD28 and 4-1BB signaling domains. These modified cells are called chimeric antigen receptor NK cells with specificity for CD33.

anti-CD33 CAR-NK cells

Lin Yang, Ph.D

Yangyi Bao, MD

Xiang Sun, MD

The purpose of this clinical trial is to study genetically engineered NK92 cell therapy in treating patients with CD33 positive acute myeloid leukemias that is relapsed (after stem cell transplantation or intensive chemotherapy) or refractory to further chemotherapy.

PRIMARY OBJECTIVES:

Determine the safety and feasibility of the chimeric antigen receptor NK92 cells transduced with the anti-CD33 vector (referred to as anti-CD33 CAR-NK cells).

SECONDARY OBJECTIVES:

I. For patients with detectable disease, measure anti-leukemia response due to anti-CD33 CAR-NK cell infusions.

II. For patients with stored or accessible leukemia blasts, determine leukemia cell killing by anti-CD33 CAR-NK in vitro.

III. Determine if cellular or humoral host immunity develops against the murine anti-CD33.

OUTLINE: Patients are assigned to 1 group according to order of enrollment.

Patients receive anti-CD33 CAR-NK (coupled with CD28, CD137 and CD3 zeta signalling domains) vector-transduced NK92 cell line on days 0,3, and 5 in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed intensively for 6 months, every 3 months for 2 years, and annually thereafter for 10 years.

CAR-pNK Cell Immunotherapy for Relapsed/Refractory CD33+ AML

Clinical Investigation of Chimeric CD(Cluster of Differentiation)33 Antigen Receptor-modified NK92 Cells in Relapsed and/or Refractory Acute Myeloid Leukemias

(1) The use of a controlled access approach, using a transparent and robust system to review requests and provide secure data access; (2) Seeking consent for sharing IPD from trial participants in all future clinical trials with adequate assurance that patient privacy and confidentiality can be maintained; and (3) Establishing an approach to resource the sharing of IPD which would include support from trial funders, sponsor organisations and users of IPD.

Defined as \>= Grade 3 signs/symptoms, laboratory toxicities, and clinical events that are possibly, likely, or definitely related to study treatment

Anti-leukemia responses to anti-CD33 CAR-NK cell infusions

The First People's Hospital of Hefei

Hefei Binhu Hospital

PersonGen BioTherapeutics (Suzhou) Co., Ltd.

Anemia, Refractory, with Excess of Blasts

Precancerous Conditions

Myelodysplastic Syndrome With Excess Blasts

Antineoplastic Agents

Gemtuzumab

Antineoplastic Agents, Immunological

Immunotoxins

Immunoconjugates

Immunologic Factors

VOR33 infusion followed by Mylotarg Dose Level 1

VOR33 infusion followed by Mylotarg Dose Level 2

VOR33 infusion followed by Mylotarg Dose Level 3

Allogeneic, human leukocyte antigen (HLA) matched, genome edited hematopoietic stem and progenitor cell (HSPC) therapy product lacking the CD33 myeloid protein

Mylotarg

Glen Raffel, MD, PhD

Olivia Nolan

Muhammad U Mushtaq, MD

Maggie Nash

Roni Tamari, MD

This is a Phase 1/2a, multicenter, open-label, first-in-human (FIH) study of VOR33 in participants with AML or MDS who are undergoing human leukocyte antigen (HLA)-matched allogeneic hematopoietic cell transplant (HCT).

High risk acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) frequently relapses despite hematopoietic stem cell transplant (HCT). Post-HCT targeted therapy to reduce relapse is limited by toxicity to the engrafted cells. VOR33, an allogeneic CRISPR/Cas9 genome-edited hematopoietic stem and progenitor cell (HSPC) therapy product, lacking the CD33 protein, is being investigated for participants with CD33+ AML or MDS at high risk for relapse after HCT to allow post-HCT targeting of residual CD33+ acute AML cells using Mylotarg™ without toxicity to engrafted VOR33 cells. Participants will undergo a myeloablative HCT with matched related or unrelated donor CD34+-selected hematopoietic stem and progenitor cells (HSPCs) engineered to remove CD33 expression (VOR33 product). Mylotarg™ will be given after engraftment for up to 4 cycles. The primary endpoint assessing safety of VOR33 will be the incidence of successful engraftment at 28 days. Part 1 of this study will evaluate the safety of escalating Mylotarg™ dose levels to determine the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D). Part 2 will expand the number of participants to evaluate the Mylotarg™ RP2D.

Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment With Mylotarg, for Patients With CD33+ AML or MDS

A First-In-Human, Open-Label, Multicenter Study of VOR33 in Patients With Acute Myeloid Leukemia Who Are at High-Risk for Leukemia Relapse Following Hematopoietic Cell Transplantation

Cumulative incidence of patients who achieve neutrophil engraftment (first day of 3 consecutive days of absolute neutrophil count (ANC) ≥500 cells/mm3) by Day 28.

Time to neutrophil engraftment after HCT from Day 0; calculated as the first day of 3 consecutive laboratory values obtained on separate days where the ANC is ≥500 cells/mm3.

Time to platelet recovery defined as first day of a sustained platelet count \>20,000/ μL with no platelet transfusion in the preceding seven days.

Incidence of primary and secondary graft failure measured by day 28 post HCT. Secondary graft failure is defined as initial neutrophil engraftment by Day 28 followed by subsequent decline.

Percent donor myeloid chimerism and CD33-negative myeloid cells in peripheral blood.

OS defined as the time from HCT to the date of death from any cause

Allogeneic Anti-CD33 Chimeric Antigen Receptor (CAR) T Cell therapy

VCAR33

Jennifer Whangbo, MD, PhD

Krisma Montalvo

Alyssa Kanegai

Lori Muffly, MD

John Magenau, MD

Marie Ventimiglia

Abhinav Deol, MD

Whitney Anderson

John DiPersio, MD, PhD

Andrew McConnell

Jacques Azzi, MD

Jonathan Lagdameo

Collind Boyington

Brian McClune, MD

This is a Phase 1/2, multicenter, open-label, first-in-human (FIH) study of donor-derived anti-CD33 Chimeric Antigen Receptor (CAR) T cell therapy (VCAR33) in patients with relapsed or refractory Acute Myeloid Leukemia (AML) after human leukocyte antigen (HLA)-matched allogeneic hematopoietic cell transplant (alloHCT).

CD33 is a preferential target for AML CAR T cell therapy due to its surface expression on the majority (\>80%) of AML blasts and due to the extensive prior clinical experience demonstrating safety and efficacy of targeting CD33 with Mylotarg (gemtuzumab ozogamicin). VCAR33 is being developed as a potential new treatment for patients with relapsed/refractory (R/R) AML after alloHCT. In this Phase 1/2 trial, the safety and efficacy of lentiviral-transduced CD33-directed CAR T cells (VCAR33) generated from the patient's prior allogeneic stem cell donor will be tested. It is hypothesized that CAR T cell production from healthy donors will not only eliminate delays in production due to lymphopenia but also reduce concerns for suboptimal T cell function from exposure to systemic immunosuppression or chemotherapeutic agents. Approximately 24 eligible patients with R/R AML after alloHCT will be enrolled in one of two separate arms based on disease burden (morphologic disease versus measurable residual disease (MRD ) positive). The maximum tolerated dose of VCAR33 will be determined using a 3+3 trial design within each arm. Dose escalation can only occur after a minimum of 3 patients have completed the dose-limiting toxicity (DLT) observation period.

Donor-Derived Anti-CD33 CAR T Cell Therapy (VCAR33) in Patients With Relapsed or Refractory AML After Allogeneic Hematopoietic Cell Transplant

Phase 1/2 Study of Donor-Derived Anti-CD33 Chimeric Antigen Receptor Expressing T Cells (VCAR33) in Patients With Relapsed or Refractory Acute Myeloid Leukemia After Allogeneic Hematopoietic Cell Transplantation

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
CD123-CD33 cCAR in Patients With Relapsed and/or Refractory, High Risk Hematologic Malignancies	NCT04156256	Hematologic Mal... Acute Myeloid L... Myelodysplastic... Myeloproliferat... Chronic Myeloid...	CD123-CD33 cCAR...	-	iCell Gene Therapeutics
A Long-term Follow-up Study of Patients Who Received VOR33	NCT05309733	Leukemia, Myelo...	VOR33	-	Vor Biopharma
A Long-term Follow-up Study of Patients Who Received VOR33	NCT05309733	Leukemia, Myelo...	VOR33	-	Vor Biopharma
A Clinical Research of CD33-Targeted CAR-T in Myeloid Malignancies	NCT02958397	Myeloid Maligna...	Anti-CD33-CAR-t...	14 Years - 75 Years	Southwest Hospital, China
CLL1-CD33 cCAR in Patients With Relapsed and/or Refractory, High Risk Hematologic Malignancies	NCT03795779	Hematologic Mal... Acute Myeloid L... Myelodysplastic... Myeloproliferat... Chronic Myeloid...	CLL1-CD33 cCAR ...	-	iCell Gene Therapeutics
CAR-pNK Cell Immunotherapy for Relapsed/Refractory CD33+ AML	NCT02944162	Acute Myelogeno... Acute Myeloid L... Acute Myeloid L... Acute Myeloid L... ANLL	anti-CD33 CAR-N...	3 Years - 80 Years	PersonGen BioTherapeutics (Suzhou) Co., Ltd.
Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment With Mylotarg, for Patients With CD33+ AML or MDS	NCT04849910	Leukemia, Myelo... Myelodysplastic...	VOR33 Mylotarg	18 Years - 70 Years	Vor Biopharma
CAR-pNK Cell Immunotherapy for Relapsed/Refractory CD33+ AML	NCT02944162	Acute Myelogeno... Acute Myeloid L... Acute Myeloid L... Acute Myeloid L... ANLL	anti-CD33 CAR-N...	3 Years - 80 Years	PersonGen BioTherapeutics (Suzhou) Co., Ltd.
Donor-Derived Anti-CD33 CAR T Cell Therapy (VCAR33) in Patients With Relapsed or Refractory AML After Allogeneic Hematopoietic Cell Transplant	NCT05984199	Leukemia, Myelo...	VCAR33	18 Years -	Vor Biopharma

Spots Global Cancer Trial Database for cd33

Every month we try and update this database with for cd33 cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for cd33

Every month we try and update this database with for cd33 cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for cd33 cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.