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Lack of Funding
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VOR33 | Genetic | VOR33 is an allogeneic CRISPR/Cas9 genome-edited hematopoietic stem and progenitor cell therapy product that lacks the CD33 myeloid protein. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
A patient is included in this study if he/she:
Exclusion Criteria:
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All patients who have received any part of or all of a VOR33 genome-edited hematopoietic stem and progenitor cell therapy product.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California San Diego Moores Cancer Center | La Jolla | California | 92037 | United States | ||
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| ID | Term |
|---|---|
| D015470 | Leukemia, Myeloid, Acute |
| D007938 | Leukemia |
| ID | Term |
|---|---|
| D007951 | Leukemia, Myeloid |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
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| Years 1-15 |
| Miami Cancer Institute |
| Miami |
| Florida |
| 33176 |
| United States |
| National Institutes of Health, Clinical Center | Bethesda | Maryland | 20892 | United States |
| Washington University School of Medicine Siteman Cancer Center | St Louis | Missouri | 63110 | United States |
| John Theurer Cancer Center at Hackensack University Medical Center | Hackensack | New Jersey | 07601 | United States |
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | United States |
| Columbia University Medical Center - Herbert Irving Comprehensive Cancer Center | New York | New York | 10032 | United States |
| Memorial Sloan Kettering Cancer Center | New York | New York | 10065 | United States |
| University Hospitals Seidman Cancer Center | Cleveland | Ohio | 44106 | United States |
| Fred Hutchinson Cancer Research Center | Seattle | Washington | 98109 | United States |
| Hôpital Maisonneuve-Rosemont | Montreal | Quebec | H1T2M4 | Canada |
| D006425 |
| Hemic and Lymphatic Diseases |