Acute Myeloid Leukemia Clinical Trial

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

Summary

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

View Full Description

Full Description

High risk acute myeloid leukemia (AML) 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 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.

View Eligibility Criteria

Eligibility Criteria

Inclusion Criteria:

Must be ≥18 and ≤70 years of age.
Must have confirmed diagnosis of AML in first or second complete remission (CR1 or CR2) or have bone marrow blasts ≤10% without circulating blasts.
AML sample from the patient must have evidence of CD33 expression (>0%)
AML must have intermediate or high-risk disease-related genetics and the presence of minimal residual disease (MRD). Subjects in CR2 or with persistent morphologic blasts; may have favorable disease-related genetics.
Candidate for HLA-matched allogeneic HCT using a myeloablative conditioning regimen.
Must have a related or unrelated stem cell donor that is a 10/10 match for HLA-A, -B, -C, -DRB1 and -DQB1.

Must have adequate performance status and organ function as defined below:

Performance Status: Karnofsky score of ≥70.
Cardiac: left ventricular ejection fraction (LVEF) ≥50%
Pulmonary: diffusing capacity of lung for carbon monoxide (DLCO), forced vital capacity (FVC), and forced expiratory volume in one second (FEV1) ≥66%.
Renal: estimated glomerular filtration rate (GFR) >60 mL/min
Hepatic: total bilirubin <1.5 × ULN, or if ≥1.5 × ULN direct bilirubin
Exclusion Criteria:

Prior autologous or allogeneic stem cell transplantation.
Presence of the following disease-related genetics: t(15; 17)(q22; q21), or t(9; 22)(q34; q11), or other evidence of acute promyelocytic leukemia or chronic myeloid leukemia.
Prior treatment with Mylotarg™ (gemtuzumab ozogamicin).
Active central nervous system (CNS) leukemia or history of other active malignancy(ies).
Patients diagnosed with Gilbert's syndrome.
Uncontrolled bacterial, viral, or fungal infections; or known human immunodeficiency virus (HIV), Hepatitis B, or Hepatitis C infection.

Study is for people with:

Acute Myeloid Leukemia

Phase:

Phase 1

Estimated Enrollment:

18

Study ID:

NCT04849910

Recruitment Status:

Recruiting

Sponsor:

Vor Biopharma

Check Your Eligibility

Let’s see if you might be eligible for this study.

What is your age and gender ?

Submit

There are 9 Locations for this study

See Locations Near You

University of California San Diego Moores Cancer Center
La Jolla California, 92037, United States More Info
Olivia Nolan
Contact
858-246-5794
[email protected]
Divya Koura, MD
Principal Investigator
Miami Cancer Institute
Miami Florida, 33176, United States More Info
Guenther Koehne, MD, PhD
Contact
786-527-8427
[email protected]
Guenther Koehne, MD, PhD
Principal Investigator
National Institutes of Health, Clinical Center
Bethesda Maryland, 20892, United States More Info
Monica Epstein
Contact
240-281-4207
[email protected]
Nirali Shah, MD, MHSc
Principal Investigator
Washington University School of Medicine Siteman Cancer Center
Saint Louis Missouri, 63110, United States More Info
Brandon Christen
Contact
314-454-5096
[email protected]
John DiPersio, MD
Principal Investigator
John Theurer Cancer Center at Hackensack University Medical Center
Hackensack New Jersey, 07601, United States More Info
Hyung Suh, MD
Contact
551-996-5863
[email protected]
Hyung Suh, MD
Principal Investigator
Memorial Sloan Kettering Cancer Center
New York New York, 10065, United States More Info
Christina Cho, MD
Contact
646-608-3785
[email protected]
Christina Cho, MD
Principal Investigator
University Hospitals Seidman Cancer Center
Cleveland Ohio, 44106, United States More Info
Cancer Information Service Line
Contact
800-641-2422
[email protected]
Brenda Cooper, MD
Principal Investigator
Fred Hutchinson Cancer Research Center
Seattle Washington, 98109, United States More Info
Nicole Stinnett
Contact
206-667-5226
[email protected]
Roland Walter, MD, PhD, MS
Principal Investigator
Hôpital Maisonneuve-Rosemont
Montréal Quebec, H1T2M, Canada More Info
Lea Bernard, MD
Contact
514-252-3404
[email protected]
Lea Bernard, MD
Principal Investigator

How clear is this clinincal trial information?

Study is for people with:

Acute Myeloid Leukemia

Phase:

Phase 1

Estimated Enrollment:

18

Study ID:

NCT04849910

Recruitment Status:

Recruiting

Sponsor:


Vor Biopharma

How clear is this clinincal trial information?

×

Introducing, the Journey Bar

Use this bar to access information about the steps in your cancer journey.