Overview

Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment With Mylotarg, for Patients With CD33+ AML

Status:
Recruiting
Trial end date:
2025-09-01
Target enrollment:
0
Participant gender:
All
Summary
This is a Phase 1/2a, multicenter, open-label, first-in-human (FIH) study of VOR33 in participants with AML who are undergoing human leukocyte antigen (HLA)-matched allogeneic hematopoietic cell transplant (HCT).
Phase:
Phase 1/Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Vor Biopharma
Treatments:
Gemtuzumab
Criteria
Inclusion Criteria:

1. Must be ≥18 and ≤70 years of age.

2. Must have confirmed diagnosis of AML in first or second complete remission (CR1 or
CR2) or have bone marrow blasts ≤10% without circulating blasts.

3. AML sample from the patient must have evidence of CD33 expression (>0%)

4. AML must have intermediate or high-risk disease-related genetics and the presence of
minimal residual disease (MRD). Subjects in CR2 or with persistent morphologic blasts;
may have favorable disease-related genetics.

5. Candidate for HLA-matched allogeneic HCT using a myeloablative conditioning regimen.

6. Must have a related or unrelated stem cell donor that is a 10/10 match for HLA-A, -B,
-C, -DRB1 and -DQB1.

7. Must have adequate performance status and organ function as defined below:

1. Performance Status: Karnofsky score of ≥70.

2. Cardiac: left ventricular ejection fraction (LVEF) ≥50%

3. Pulmonary: diffusing capacity of lung for carbon monoxide (DLCO), forced vital
capacity (FVC), and forced expiratory volume in one second (FEV1) ≥66%.

4. Renal: estimated glomerular filtration rate (GFR) >60 mL/min

5. Hepatic: total bilirubin <1.5 × ULN, or if ≥1.5 × ULN direct bilirubin ALT/AST <1.5 × ULN (per institutional criteria).

Exclusion Criteria:

1. Prior autologous or allogeneic stem cell transplantation.

2. 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.

3. Prior treatment with Mylotarg™ (gemtuzumab ozogamicin).

4. Active central nervous system (CNS) leukemia or history of other active
malignancy(ies).

5. Patients diagnosed with Gilbert's syndrome.

6. Uncontrolled bacterial, viral, or fungal infections; or known human immunodeficiency
virus (HIV), Hepatitis B, or Hepatitis C infection.