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

VOR33 long-term follow-up (LTFU) study

Study Overview

• Status: Terminated
• Conditions: Leukemia, Myeloid, Acute
• Intervention / Treatment: Genetic: VOR33

Detailed Description

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.

• Study Type: Observational
• Enrollment (Actual): 10

Contacts and Locations

Study Locations

• Canada
  • Quebec
    • Montréal, Quebec, Canada, H1T2M4
      • Hôpital Maisonneuve-Rosemont
• United States
  • California
    • La Jolla, California, United States, 92037
      • University of California San Diego Moores Cancer Center
  • Florida
    • Miami, Florida, United States, 33176
      • Miami Cancer Institute
  • Maryland
    • Bethesda, Maryland, United States, 20892
      • National Institutes of Health, Clinical Center
  • Missouri
    • Saint Louis, Missouri, United States, 63110
      • Washington University School of Medicine Siteman Cancer Center
  • New Jersey
    • Hackensack, New Jersey, United States, 07601
      • John Theurer Cancer Center at Hackensack University Medical Center
  • New York
    • New York, New York, United States, 10065
      • Memorial Sloan Kettering Cancer Center
    • New York, New York, United States, 10029
      • ICAHN School of Medicine at Mount Sinai
    • New York, New York, United States, 10032
      • Columbia University Medical Center - Herbert Irving Comprehensive Cancer Center
  • Ohio
    • Cleveland, Ohio, United States, 44106
      • University Hospitals Seidman Cancer Center
  • Washington
    • Seattle, Washington, United States, 98109
      • Fred Hutchinson Cancer Research Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

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

Description

Inclusion Criteria:

• A patient is included in this study if he/she:

  1. Has received any part of or all of a VOR33 infusion

Exclusion Criteria:

• There are no exclusion criteria.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Observational Cohort 1; All patients who have received any part of or all of a VOR33 genome-edited hematopoietic stem and progenitor cell therapy product.	Genetic: VOR33; VOR33 is an allogeneic CRISPR/Cas9 genome-edited hematopoietic stem and progenitor cell therapy product that lacks the CD33 myeloid protein.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Long term safety of VOR33 in patients who participated in a VOR33 study in terms of study product-related Adverse Events/Serious Adverse Events (AEs/SAEs).	Incidence of VOR33-related AEs/SAEs or deaths.	Years 1-15

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Long term efficacy of VOR33 in patients who participated in a VOR33 study in terms of persistence of CD33-negative hematopoiesis, overall survival (OS), and monthly relapse free survival (RFS).	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.	Years 1-15

Collaborators and Investigators

• Sponsor: Vor Biopharma

Study record dates

Study Major Dates

• Study Start (Actual): April 15, 2022
• Primary Completion (Actual): May 28, 2025
• Study Completion (Actual): May 28, 2025

Study Registration Dates

• First Submitted: March 24, 2022
• First Submitted That Met QC Criteria: March 24, 2022
• First Posted (Actual): April 4, 2022

Study Record Updates

• Last Update Posted (Actual): July 29, 2025
• Last Update Submitted That Met QC Criteria: July 25, 2025
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Keywords: Acute Myeloid Leukemia; AML; HCT; leukemia; CD33; allogeneic; hematopoietic stem cell transplant
• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Hematologic Diseases; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute
• Other Study ID Numbers: VBP201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No