An observational study in Leukemia, Myeloid, Acute, sponsored by Vor Biopharma. Terminated at 11 sites in 2 countries. Per ClinicalTrials.gov, last updated 2025-07-29.
Sponsored by Vor Biopharma · Observational
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.
3,667 studies on the registry are indexed under Leukemia, Myeloid, Acute; 816 are open to participants now.
This study's enrollment of 10 is below the median of 114 across 395 observational studies indexed under Leukemia, Myeloid, Acute.
Browse Leukemia, Myeloid, Acute studies →Vor Biopharma is the lead sponsor of 7 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
All patients who have received any part of or all of a VOR33 genome-edited hematopoietic stem and progenitor cell therapy product.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
A patient is included in this study if he/she:
Exclusion Criteria:
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.
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
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
Plan to share: No
No publications or documents are linked to this record.
Ran from Apr 2022 to primary completion in May 2025, 37 months.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in May 2025. You cannot join it, but the record below documents what was studied.
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