Status:
TERMINATED
Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment With Mylotarg, for Patients With CD33+ AML or MDS
Lead Sponsor:
Vor Biopharma
Conditions:
Leukemia, Myeloid, Acute
Myelodysplastic Syndromes
Eligibility:
All Genders
18-70 years
Phase:
PHASE1
PHASE2
Brief Summary
This is a Phase 1/2a, multicenter, open-label, first-in-human (FIH) study of VOR33 in participants with AML or MDS who are undergoing human leukocyte antigen (HLA)-matched allogeneic hematopoietic cel...
Detailed Description
High risk acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) frequently relapses despite hematopoietic stem cell transplant (HCT). Post-HCT targeted therapy to reduce relapse is limited ...
Eligibility Criteria
Inclusion
- Must be ≥18 and ≤70 years of age.
- Patients with AML must have one of the following groups of features that are known to be a risk factor for leukemia relapse:
- BM in morphological remission (\<5% blasts) with adverse-risk disease related genetics at presentation (according to European Leukemia-Net guidelines \[ELN, Döhner 2017\]), or
- Intermediate risk genetics in morphologic remission (\<5% blasts) with other recognized high risk criteria such as MRD+ following therapy, or
- BM with evidence of persistent leukemia 5-10% blasts post induction/salvage therapy. Patients with BM Blast count \>10% may participate with Sponsor Medical Monitor approval. (Note: these patients may have disease-related genetics of any risk criteria at presentation), or
- Any patient in second or greater remission.
- Patients with MDS must have all of the following:
- Previous or current IPSS-R score of High or Very High risk; AND
- Previous or current MDS-IB1 or MDS-IB2 per the 2022 WHO criteria (Khoury 2022)
- AML sample from the patient must have evidence of CD33 expression (\>0%)
- Candidate for HLA-matched allogeneic HCT using a myeloablative conditioning regimen.
- Must have a related or unrelated stem cell donor that is a 8/8 match for HLA-A, -B, -C, and -DRB1.
- Must have adequate performance status and organ function as defined below:
- Performance Status: Karnofsky score of ≥70.
- Cardiac: left ventricular ejection fraction (LVEF) ≥50%
- Pulmonary: diffusing capacity of lung for carbon monoxide (DLCO), forced vital capacity (FVC), and forced expiratory volume in one second (FEV1) ≥66%.
- Renal: estimated glomerular filtration rate (GFR) \>60 mL/min
- Hepatic: total bilirubin \<1.5 × ULN, or if ≥1.5 × ULN direct bilirubin \<ULN and ALT/AST \<1.5 × ULN (per institutional criteria).
Exclusion
- Prior autologous or allogeneic stem cell transplantation.
- Presence of the following disease-related genetics: t(15; 17)(q22; q21), or t(9; 22)(q34; q11), or other evidence of acute promyelocytic leukemia or chronic myeloid leukemia.
- Prior treatment with Mylotarg™ (gemtuzumab ozogamicin) in the past 3.5 months.
- Active central nervous system (CNS) leukemia.
- Patients diagnosed with Gilbert's syndrome.
- Uncontrolled bacterial, viral, or fungal infections; or known human immunodeficiency virus (HIV), Hepatitis B, or Hepatitis C infection.
Key Trial Info
Start Date :
December 16 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 28 2025
Estimated Enrollment :
67 Patients enrolled
Trial Details
Trial ID
NCT04849910
Start Date
December 16 2021
End Date
May 28 2025
Last Update
July 29 2025
Active Locations (15)
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1
University of California San Diego Moores Cancer Center
La Jolla, California, United States, 92037
2
Stanford Cancer Institute
Stanford, California, United States, 94305
3
Miami Cancer Institute
Miami, Florida, United States, 33176
4
Winship Cancer Institute Emory University
Atlanta, Georgia, United States, 30322