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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05309733
Recruitment Status : Recruiting
First Posted : April 4, 2022
Last Update Posted : August 10, 2022
Sponsor:
Information provided by (Responsible Party):
Vor Biopharma

Brief Summary:
VOR33 long-term follow-up (LTFU) study

Condition or disease Intervention/treatment
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.

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Study Type : Observational
Estimated 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 : April 15, 2022
Estimated Primary Completion Date : November 30, 2038
Estimated Study Completion Date : November 30, 2038


Group/Cohort 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.




Primary Outcome Measures :
  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). [ Time Frame: Years 1-15 ]
    Incidence of VOR33-related AEs/SAEs or deaths.


Secondary Outcome Measures :
  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). [ Time Frame: 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.



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-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.
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05309733


Locations
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United States, California
University of California San Diego Moores Cancer Center Not yet recruiting
La Jolla, California, United States, 92037
Contact: Michelle Padilla    858-822-5364    mpadilla@health.ucsd.edu   
Principal Investigator: Divya Koura, MD         
United States, Florida
Miami Cancer Institute Recruiting
Miami, Florida, United States, 33176
Contact: Guenther Koehne, MD, PhD    786-527-8427    guentherk@baptisthealth.net   
Principal Investigator: Guenther Koehne, MD, PhD         
United States, Missouri
Washington University School of Medicine Siteman Cancer Center Not yet recruiting
Saint Louis, Missouri, United States, 63110
Contact: Cathy Wiggins    314-454-8491    cwiggins@wustl.edu   
Principal Investigator: John DiPersio, MD         
United States, New Jersey
John Theurer Cancer Center at Hackensack University Medical Center Recruiting
Hackensack, New Jersey, United States, 07601
Contact: Hyung Suh, MD    551-996-5863    Hyung.Suh@hmhn.org   
Principal Investigator: Hyung Suh, MD         
United States, New York
Memorial Sloan Kettering Cancer Center Recruiting
New York, New York, United States, 10065
Contact: Christina Cho, MD    646-608-3785    ABMTTrials@mskcc.org   
Principal Investigator: Christina Cho, MD         
United States, Ohio
University Hospitals Seidman Cancer Center Recruiting
Cleveland, Ohio, United States, 44106
Contact: Cancer Information Service Line    800-641-2422    Crystal.Santo@UHhospitals.org   
Principal Investigator: Brenda Cooper, MD         
United States, Washington
Fred Hutchinson Cancer Research Center Not yet recruiting
Seattle, Washington, United States, 98109
Contact: Roland Walter, MD, PhD, MS       rwalter@fredhutch.org   
Principal Investigator: Roland Walter, MD, PhD, MS         
Canada, Quebec
Hôpital Maisonneuve-Rosemont Not yet recruiting
Montréal, Quebec, Canada, H1T 2M4
Contact: Nadia Bambace, MD    514-252-3404    nadia.bambace@umontreal.ca   
Principal Investigator: Nadia Bamace, MD         
Sponsors and Collaborators
Vor Biopharma
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Responsible Party: Vor Biopharma
ClinicalTrials.gov Identifier: NCT05309733    
Other Study ID Numbers: VBP201
First Posted: April 4, 2022    Key Record Dates
Last Update Posted: August 10, 2022
Last Verified: August 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Vor Biopharma:
AML
Acute Myeloid Leukemia
CD33
allogeneic
HCT
hematopoietic stem cell transplant
leukemia
Additional relevant MeSH terms:
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Leukemia
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Neoplasms by Histologic Type
Neoplasms