Leukemia, Myeloid, Acute
Conditions
Keywords
AML, Acute Myeloid Leukemia, CD33, allogeneic, HCT, hematopoietic stem cell transplant, leukemia
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.
Interventions
GENETICVOR33
VOR33 is an allogeneic CRISPR/Cas9 genome-edited hematopoietic stem and progenitor cell therapy product that lacks the CD33 myeloid protein.
Sponsors
Vor Biopharma
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
Canada, United States
Outcome results
None listed