Leukemia, Myeloid, AcuteNCT05309733
A Long-term Follow-up Study of Patients Who Received VOR33
An observational study of Leukemia, Myeloid, Acute, run by Vor Biopharma at 11 sites in 2 countries.
Trial summary
VOR33 long-term follow-up (LTFU) study Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Leukemia, Myeloid, Acute
- What is being tested?
- VOR33
- How many people
- 10 participants
- Where
- 11 sitesin 2 countries
- Study dates
- 15 Apr 2022to 28 May 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: All patients who have received any part of or all of a VOR33 genome-edited hematopoietic stem and progenitor cell therapy product.
Who may be able to join
- A patient is included in this study if he/she:
- Has received any part of or all of a VOR33 infusion
Who cannot join
- There are no exclusion criteria.
Trial locations and contacts
11 sites in 2 countries.
11 sites in 2 countries
United States10 sites
National Institutes of Health, Clinical Center
Bethesda, Maryland, 20892
University Hospitals Seidman Cancer Center
Cleveland, Ohio, 44106
John Theurer Cancer Center at Hackensack University Medical Center
Hackensack, New Jersey, 07601
University of California San Diego Moores Cancer Center
La Jolla, California, 92037
Miami Cancer Institute
Miami, Florida, 33176
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
Columbia University Medical Center - Herbert Irving Comprehensive Cancer Center
New York, New York, 10032
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109
Washington University School of Medicine Siteman Cancer Center
St Louis, Missouri, 63110
About this study
VOR33 long-term follow-up (LTFU) study
Read the 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.
- Conditions
- Leukemia, Myeloid, Acute
- Being tested
- VOR33
- Keywords
- AML, Acute Myeloid Leukemia, CD33, allogeneic, HCT, hematopoietic stem cell transplant, leukemia
- Medical subject headings
- Leukemia, Myeloid, Acute, Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VOR33Genetic
VOR33 is an allogeneic CRISPR/Cas9 genome-edited hematopoietic stem and progenitor cell therapy product that lacks the CD33 myeloid protein.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Genetic: VOR33 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Years 1-15
Secondary outcomes (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).
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.
Measured: Years 1-15
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Apr 2022
- Study start15 Apr 2022
- Primary completion28 May 2025
- Study completion28 May 2025
Why it stopped: Lack of Funding
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leukemia, Myeloid, AcuteStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vor Biopharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05309733
- Sponsor's protocol ID
- VBP201
- Status last verified
- May 2025
- First submitted
- 24 Mar 2022
- Last updated
- 29 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-term Follow-up Study of Patients Who Received VOR33
- ClinicalTrials.gov ID
- NCT05309733
- Acronym / sponsor ID
- VBP201
- Conditions
- Leukemia, Myeloid, Acute
- Keywords
- AML, Acute Myeloid Leukemia, CD33, allogeneic, HCT, hematopoietic stem cell transplant, leukemia
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Lack of Funding
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Vor BiopharmaIndustry (pharma, biotech, devices)
- Study start
- 15 Apr 2022 (actual)
- Primary completion
- 28 May 2025 (actual)
- Study completion
- 28 May 2025 (actual)
- First posted
- 4 Apr 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Observational Cohort 1 | – | Genetic: VOR33All patients who have received any part of or all of a VOR33 genome-edited hematopoietic stem and progenitor cell therapy product. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All patients who have received any part of or all of a VOR33 genome-edited hematopoietic stem and progenitor cell therapy product.
Key inclusion criteria (2 listed)
- A patient is included in this study if he/she:
Key exclusion criteria (1 listed)
- There are no exclusion criteria.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VOR33VOR33 is an allogeneic CRISPR/Cas9 genome-edited hematopoietic stem and progenitor cell therapy product that lacks the CD33 myeloid protein. | Genetic | Observational Cohort 1 |
Primary outcome
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.
1 secondary outcome
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
Results and safety
The study was stopped early (reason given: Lack of Funding). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jul 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
11 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Leukemia, Myeloid, Acute, run by Vor Biopharma at 11 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 29 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Lack of Funding). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jul 2025.