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

Active:	Yes
Cancer Type:	Cancer-Related Syndrome Leukemia Myelodysplastic Syndromes (MDS)	NCT ID:	NCT04849910
Trial Phases:	Phase I Phase II	Protocol IDs:	VBP101 (primary) NCI-2022-00073
Eligibility:	18 - 70 Years, Male and Female	Study Type:	Treatment
Study Sponsor:	Vor Biopharma
NCI Full Details:	http://clinicaltrials.gov/show/NCT04849910

Summary

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).

Objectives

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.

Treatment Sites in Georgia

Winship Cancer Institute of Emory University
1365 Clifton Road NE
Building C
Atlanta, GA 30322
404-778-5180
winshipcancer.emory.edu

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