Neoplasms

Blood and Lymph Conditions

All Conditions

Rare Diseases

Leukemia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Neoplasms by Histologic Type

Hematologic Diseases

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Acute Graft Versus Host Disease

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

Symptoms and General Pathology

Syndrome

Preleukemia

Myelodysplastic Syndromes

Anemia, Refractory, with Excess of Blasts

Bone Marrow Diseases

Precancerous Conditions

Myelodysplastic Syndrome With Excess Blasts

Antineoplastic Agents

All Drugs and Chemicals

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

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
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
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

Spots Global Cancer Trial Database for vor33

Every month we try and update this database with for vor33 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 vor33

Every month we try and update this database with for vor33 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 vor33 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.