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

Vor Biopharma10 enrolled

Overview

VOR33 long-term follow-up (LTFU) study

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

Conditions

Leukemia, Myeloid, Acute

Interventions

VOR33GENETIC

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

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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.

Locations (11)

University of California San Diego Moores Cancer Center

La Jolla, California, United States

Miami Cancer Institute

Miami, Florida, United States

National Institutes of Health, Clinical Center

Bethesda, Maryland, United States

Outcomes

Primary Outcomes

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.

Time frame: Years 1-15

Secondary Outcomes

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.

Time frame: Years 1-15

Data from ClinicalTrials.gov

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