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CAR T-cell Therapy

Genetically Modified T-cell Therapy for Leukemia

Phase 1

Waitlist Available

Led By Lihua E. Budde

Research Sponsored by City of Hope Medical Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

If a research participant has undergone prior allogeneic stem cell transplant, he/she must be off all immunosuppressants for graft versus host disease (GVHD) for at least 2 weeks before undergoing leukapheresis

If a research participant has undergone prior allogeneic stem cell transplant, he/she must be off all immunosuppressants for GVHD for at least 7 days before beginning lymphodepletion

Must not have

However, all participants must be able to reduce steroid requirement to no more than physiological replacement doses prior to start of lymphodepletion

Active autoimmune disease requiring systemic immunosuppressive therapy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 15 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing the side effects and best dosage of genetically modified T-cells, which are a type of immune cell, in patients with leukemia or a certain type of cancer of the blood and bone marrow.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Possible side effects include reactions related to immune system activation such as fever, fatigue, inflammation of organs; complications from low blood counts due to chemotherapy; allergic reactions; and potential worsening of underlying autoimmune diseases.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I stopped taking immunosuppressants for GVHD 2 weeks before leukapheresis.

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

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I am able to care for myself but may not be able to do active work.

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I have undergone a treatment to reduce my white blood cells.

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My kidney function is within the required range.

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I have a matched donor or stem cell source for my transplant.

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I don't need extra oxygen and my oxygen levels are 90% or higher without assistance.

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I have stopped all leukemia treatments except for lymphodepleting regimens 7 days before CAR T cell therapy.

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My liver is working well, with bilirubin levels at or below 3.0 mg/dl.

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My AML cancer returned after a period of no signs of the disease.

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I have BPDCN, have been treated before, and my disease is still present or has come back.

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My kidney function has not worsened significantly since my initial health check.

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I am a child weighing more than 50 kg.

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I am able to care for myself but may not be able to do active work.

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I have suitable veins for blood draws or a special catheter if needed.

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I do not have any uncontrolled active infections.

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I can understand and am willing to sign the consent form.

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My heart condition is stable without the need for intensive support.

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My cancer is not fully gone and shows signs of remaining disease.

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My kidney function, measured by creatinine clearance, is within the normal range.

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My AML did not respond to the first 2 chemotherapy cycles.

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My donor does not have hepatitis B or C.

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Side effects from my previous treatments are mild or back to normal, except for those that won’t get better.

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My stem cell donor for the transplant is the same person who donated before.

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I have at least 1% CAR T cells in my blood.

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My T cells are ready for CAR T cell therapy.

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I do not have significant new brain or nerve problems.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

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I am on medication to suppress my immune system due to an autoimmune disease.

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I understand the study's purpose, procedures, and the risks/benefits of participating.

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I rely on corticosteroids for my health condition.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 15 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 15 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 15 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Disease response (CR or CRi)

Dose-limiting toxicity (DLT) defined as any grade 3 or higher toxicity as assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0

Incidence of adverse events as assessed by NCI CTCAE version 4.0

Secondary study objectives

CAR123-specific antibody level

Duration of response

Engraftment of transferred CD123+ CAR T cells

+2 more

Other study objectives

Number of CD123+ leukemic cells in peripheral blood and bone marrow

Number of CD123+ normal cells in peripheral blood and bone marrow

Blood Cells

+2 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment (lymphodepletion, T-cell immunotherapy)Experimental Treatment5 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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

cyclophosphamide

1994

Completed Phase 3

~8140

Fludarabine Phosphate

1997

Completed Phase 3

~2390