Study on Stem Cell Transplant Without CD33 Protein and Post-Transplant Treatment with Mylotarg for Patients with CD33+ AML or MDS

Sponsor: Vor Biopharma

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT04849910

Provider View

This clinical trial is testing a new approach to stem cell transplants for patients with certain types of leukemia (AML) or myelodysplastic syndromes (MDS). The study aims to see if removing a protein called CD33 from donor stem cells can make post-transplant treatment with Mylotarg safer and more effective.

Patient Parameters

Program Overview

This study is exploring a new type of stem cell transplant for patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) who are at high risk of the disease returning after a transplant. The trial uses donor stem cells that have been genetically modified to remove a protein called CD33. This allows doctors to use a drug called Mylotarg after the transplant to target any remaining cancer cells without harming the new stem cells. The study will test the safety of this approach and determine the best dose of Mylotarg to use.

Description

This clinical trial is for patients with high-risk acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) who are undergoing a stem cell transplant. The trial uses a new type of donor stem cell that has been genetically modified to remove a protein called CD33. This modification allows doctors to use a drug called Mylotarg after the transplant to target any remaining cancer cells without harming the new stem cells.

Participants will receive a stem cell transplant from a donor whose cells have been engineered to lack the CD33 protein. After the transplant, they will receive Mylotarg for up to four cycles to help eliminate any remaining cancer cells. The study will monitor the safety of this approach by checking if the new stem cells successfully grow in the patient's body within 28 days.

The trial is divided into two parts. The first part will test different doses of Mylotarg to find the highest dose that can be given safely. The second part will involve more participants to further evaluate the best dose of Mylotarg.

Eligibility Criteria

Inclusion Criteria

  • Aged between 18 and 70 years.
  • For AML patients, must have certain risk factors for the disease returning, such as:
    • In remission with specific genetic risks.
    • Intermediate genetic risk with other high-risk factors.
    • Persistent leukemia after initial treatment.
    • In second or later remission.
  • For MDS patients, must have:
    • High or very high risk according to specific scoring systems.
    • Specific types of MDS as per recent criteria.
  • AML sample must show CD33 protein.
  • Eligible for a matched donor stem cell transplant with a specific conditioning regimen.
  • Must have a donor who is a full match for certain genetic markers.
  • Must have good overall health and organ function, including:
    • Performance score of 70 or higher.
    • Heart function with an ejection fraction of 50% or more.
    • Lung function with specific capacity levels.
    • Kidney function with a filtration rate over 60 mL/min.
    • Liver function within specified limits.

Exclusion Criteria

  • Previous stem cell transplant.
  • Certain genetic markers related to other types of leukemia.
  • Recent treatment with Mylotarg within the last 3.5 months.
  • Active leukemia in the central nervous system.
  • Diagnosis of Gilbert's syndrome.
  • Uncontrolled infections or known infections with HIV, Hepatitis B, or Hepatitis C.

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