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

Leukemia, Myeloid, Acute Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05309733
• Other study ID #: VBP201
• Status: Recruiting
• Start date: April 15, 2022
• Est. completion date: January 2040

Study information

• Verified date: January 2024
• Source: Vor Biopharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

VOR33 long-term follow-up (LTFU) study

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 36
• Est. completion date: January 2040
• Est. primary completion date: January 2040
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Leukemia
• Leukemia, Myeloid
• Leukemia, Myeloid, Acute

Intervention

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

Locations

Country	Name	City	State
Canada	Hôpital Maisonneuve-Rosemont	Montréal	Quebec
United States	National Institutes of Health, Clinical Center	Bethesda	Maryland
United States	University Hospitals Seidman Cancer Center	Cleveland	Ohio
United States	John Theurer Cancer Center at Hackensack University Medical Center	Hackensack	New Jersey
United States	University of California San Diego Moores Cancer Center	La Jolla	California
United States	Miami Cancer Institute	Miami	Florida
United States	Columbia University Medical Center - Herbert Irving Comprehensive Cancer Center	New York	New York
United States	Memorial Sloan Kettering Cancer Center	New York	New York
United States	Washington University School of Medicine Siteman Cancer Center	Saint Louis	Missouri
United States	Fred Hutchinson Cancer Research Center	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Vor Biopharma

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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