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

Recruiting

• Conditions: Leukemia, Myeloid, Acute
• Interventions: Genetic: VOR33

• Registration Number: NCT05309733
• Lead Sponsor: Vor Biopharma

Brief Summary

VOR33 long-term follow-up (LTFU) study

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 Timeline

• Study First Submitted: 2022-03-24
• Study First Submitted QC: 2022-03-24
• Study First Posted: 2022-04-04
• Study Start: 2022-04-15
• Status Verified: 2024-01
• Last Update Submitted: 2024-01-24
• Last Update Posted: 2024-01-26
• Primary Completion: 2040-01
• Study Completion: 2040-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 36

Inclusion Criteria

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

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

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Exclusion Criteria

• There are no exclusion criteria.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Observational Cohort 1	VOR33	All patients who have received any part of or all of a VOR33 genome-edited hematopoietic stem and progenitor cell therapy product.

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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.

Trial Locations

Locations (10)

University Hospitals Seidman Cancer Center; 🇺🇸 Cleveland, Ohio, United States

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

Washington University School of Medicine Siteman Cancer Center; 🇺🇸 Saint Louis, Missouri, 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

Fred Hutchinson Cancer Research Center; 🇺🇸 Seattle, Washington, United States

John Theurer Cancer Center at Hackensack University Medical Center; 🇺🇸 Hackensack, New Jersey, United States

Hôpital Maisonneuve-Rosemont; 🇨🇦 Montréal, Quebec, Canada