NCT05309733

Brief Summary

VOR33 long-term follow-up (LTFU) study

Trial Health

60
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Apr 2022

Typical duration for all trials

Geographic Reach
2 countries

11 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 24, 2022

Completed
11 days until next milestone

First Posted

Study publicly available on registry

April 4, 2022

Completed
11 days until next milestone

Study Start

First participant enrolled

April 15, 2022

Completed
3.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 28, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 28, 2025

Completed
Last Updated

July 29, 2025

Status Verified

May 1, 2025

Enrollment Period

3.1 years

First QC Date

March 24, 2022

Last Update Submit

July 25, 2025

Conditions

Keywords

AMLAcute Myeloid LeukemiaCD33allogeneicHCThematopoietic stem cell transplantleukemia

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (1)

  • 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).

    Years 1-15

Study Arms (1)

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

Interventions

VOR33GENETIC

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

Observational Cohort 1

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-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.

You may qualify if:

  • A patient is included in this study if he/she:
  • Has received any part of or all of a VOR33 infusion

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (11)

University of California San Diego Moores Cancer Center

La Jolla, California, 92037, United States

Location

Miami Cancer Institute

Miami, Florida, 33176, United States

Location

National Institutes of Health, Clinical Center

Bethesda, Maryland, 20892, United States

Location

Washington University School of Medicine Siteman Cancer Center

St Louis, Missouri, 63110, United States

Location

John Theurer Cancer Center at Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

Columbia University Medical Center - Herbert Irving Comprehensive Cancer Center

New York, New York, 10032, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

University Hospitals Seidman Cancer Center

Cleveland, Ohio, 44106, United States

Location

Fred Hutchinson Cancer Research Center

Seattle, Washington, 98109, United States

Location

Hôpital Maisonneuve-Rosemont

Montreal, Quebec, H1T2M4, Canada

Location

MeSH Terms

Conditions

Leukemia, Myeloid, AcuteLeukemia

Condition Hierarchy (Ancestors)

Leukemia, MyeloidNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic Diseases

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 24, 2022

First Posted

April 4, 2022

Study Start

April 15, 2022

Primary Completion

May 28, 2025

Study Completion

May 28, 2025

Last Updated

July 29, 2025

Record last verified: 2025-05

Data Sharing

IPD Sharing
Will not share

Locations