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

Sponsor

Vor Biopharma (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05309733

Collaborator

(none)

Enrollment

Locations

199.5

Anticipated Duration (Months)

4.5

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

VOR33 long-term follow-up (LTFU) study

Condition or Disease	Intervention/Treatment	Phase
Leukemia, Myeloid, Acute	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 Design

Study Type:

Observational

Anticipated Enrollment :

36 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

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

Actual Study Start Date :

Apr 15, 2022

Anticipated Primary Completion Date :

Nov 30, 2038

Anticipated Study Completion Date :

Nov 30, 2038

Arms and Interventions

Arm	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.

Arm

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.

Outcome Measures

Primary Outcome Measures

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). [Years 1-15]
Incidence of VOR33-related AEs/SAEs or deaths.

Secondary Outcome Measures

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]
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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

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

Has received any part of or all of a VOR33 infusion

Exclusion Criteria:

There are no exclusion criteria.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of California San Diego Moores Cancer Center	La Jolla	California	United States	92037
2	Miami Cancer Institute	Miami	Florida	United States	33176
3	Washington University School of Medicine Siteman Cancer Center	Saint Louis	Missouri	United States	63110
4	John Theurer Cancer Center at Hackensack University Medical Center	Hackensack	New Jersey	United States	07601
5	Memorial Sloan Kettering Cancer Center	New York	New York	United States	10065
6	University Hospitals Seidman Cancer Center	Cleveland	Ohio	United States	44106
7	Fred Hutchinson Cancer Research Center	Seattle	Washington	United States	98109
8	Hôpital Maisonneuve-Rosemont	Montréal	Quebec	Canada	H1T 2M4