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

Stopped early · Phase 1/Phase 2

Conditions studied: Leukemia, Myeloid, Acute, Myelodysplastic Syndromes

In brief

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 cell transplant (HCT).

Key facts

Study ID

NCT04849910

Run by

Vor Biopharma

People needed

67

Starts

2021-12-16

Expected to finish

2025-05-28

Last updated by the study team

2025-07-29

Who can join

Age: 18 and older, up to 70. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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).

You may not qualify if…

• 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.

Where it is running

• University of California San Diego Moores Cancer Center — La Jolla, California, United States
• Stanford Cancer Institute — Stanford, California, United States
• Miami Cancer Institute — Miami, Florida, United States
• Winship Cancer Institute Emory University — Atlanta, Georgia, United States
• The University of Kansas Cancer Center — Fairway, Kansas, United States
• National Institutes of Health, Clinical Center — Bethesda, Maryland, United States
• Masonic Cancer Center, University of Minnesota — Minneapolis, Minnesota, United States
• Washington University School of Medicine Siteman Cancer Center — St Louis, Missouri, United States
• John Theurer Cancer Center at Hackensack University Medical Center — Hackensack, New Jersey, United States
• Columbia University Medical Center - Herbert Irving Comprehensive Cancer Center — New York, New York, United States
• Memorial Sloan Kettering Cancer Center — New York, New York, United States
• University Hospitals Seidman Cancer Center — Cleveland, Ohio, United States
• University of Utah Huntsman Cancer Institute — Salt Lake City, Utah, United States
• Fred Hutchinson Cancer Research Center — Seattle, Washington, United States
• Hôpital Maisonneuve-Rosemont — Montreal, Quebec, Canada

Full record on ClinicalTrials.gov

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