Genetically Modified T-cell Therapy for Leukemia

Overseen by Lihua E. Budde

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: City of Hope Medical Center

No Placebo Group

Trial Summary

What is the purpose of this trial?

This phase I trial studies the side effects and the best dose of genetically modified T-cells after lymphodepleting chemotherapy in treating patients with acute myeloid leukemia or blastic plasmacytoid dendritic cell neoplasm that has returned after a period of improvement or has not responded to previous treatment. An immune cell is a type of blood cell that can recognize and kill abnormal cells in the body. The immune cell product will be made from patient or patient's donor (related or unrelated) blood cells. The immune cells are changed by inserting additional pieces of deoxyribonucleic acid (DNA) (genetic material) into the cell to make it recognize and kill cancer cells. Placing a modified gene into white blood cells may help the body build an immune response to kill cancer cells.

Research Team

Lihua E. Budde

Principal Investigator

City of Hope Medical Center

Eligibility Criteria

This trial is for patients with certain blood cancers that have come back or didn't respond to treatment. It's open to those who meet specific health criteria like normal liver and kidney function, a good performance status score (able to carry out daily activities), and no severe active infections. They must not be pregnant, agree to use birth control, and can't have other active cancers or HIV.

Inclusion Criteria

My last cancer treatment was at least 2 weeks ago.

I stopped taking immunosuppressants for GVHD 2 weeks before leukapheresis.

I had a stem cell transplant and have not taken any immunosuppressants for GVHD in the last 7 days.

Exclusion Criteria

Research participants with uncontrolled intercurrent illness including, but not limited to ongoing or active or poorly controlled infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, poorly controlled pulmonary disease or psychiatric illness/social situations that would limit compliance with study requirements; such social situations include but are not limited to lack of reliable means of transportation for follow up, inability to make time for required clinic visits due to work or family needs, or lack of reliable ways of communication with the study team in the event that the participant is seriously ill

Research participants who have tested human immunodeficiency virus (HIV) positive, or have active hepatitis B or C infection based on testing performed within 4 weeks of enrollment

I can lower my steroid use to only what's needed for basic body functions before starting treatment.

Treatment Details

Interventions

• Allogeneic CD123CAR-CD28-CD3zeta-EGFRt-expressing T-lymphocytes (CAR T-cell Therapy)
• Autologous CD123CAR-CD28-CD3zeta-EGFRt-expressing T Lymphocytes (CAR T-cell Therapy)
• Cyclophosphamide (Alkylating agents)
• Fludarabine Phosphate (Anti-metabolites)

Trial Overview The trial tests genetically modified T-cells after chemotherapy in patients with acute myeloid leukemia or blastic plasmacytoid dendritic cell neoplasm. The T-cells are engineered from the patient's or donor's blood cells to target and kill cancer cells.

Participant Groups

Group I: Treatment (lymphodepletion, T-cell immunotherapy) Experimental Treatment 5 Interventions

Patients undergo a lymphodepleting regimen 3-10 days prior to CD123+ CAR T cell infusion as determined by the principal investigator and the protocol team. Patients receive either cyclophosphamide IV on days -4 and/or -3; fludarabine phosphate and cyclophosphamide IV on days -5 to -3; fludarabine phosphate IV on days -5 to -3 and cyclophosphamide IV on days -4 and/or -3. Patients receive autologous or allogeneic CD123+ CAR Tcells IV over 15 minutes on day 0. Patients with evidence of disease at \> 28 days, continuing expression of the CD123 antigen, and not having experienced a DLT may receive a second infusion of CD123+ CAR T cells after 28 days.

Who Is Running the Clinical Trial?

City of Hope Medical Center

Lead Sponsor

Trials

614

Recruited

1,924,000+

Robert Stone

City of Hope Medical Center

Chief Executive Officer since 2014

Juris Doctorate from the University of Chicago, Bachelor's degree in Political Science from the University of Redlands

Sumanta (Monty) Pal

City of Hope Medical Center

Chief Medical Officer since 2023

MD

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

Dr. Douglas R. Lowy

National Cancer Institute (NCI)

Chief Executive Officer since 2023

MD from New York University School of Medicine

Dr. Monica Bertagnolli

National Cancer Institute (NCI)

Chief Medical Officer since 2022

MD from Harvard Medical School

Mustang Bio, Inc.

Industry Sponsor

Trials

1

Recruited

30+