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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 15 years

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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 outcome measures

CAR123-specific antibody level

Duration of response

Engraftment of transferred CD123+ CAR T cells

+2 more

Other outcome measures

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

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

Fludarabine Phosphate

1997

Completed Phase 3

~2390

cyclophosphamide

1994

Completed Phase 3

~8140

0 / 27Eligibility criteria met

Find a Location

Who is running the clinical trial?

City of Hope Medical CenterLead Sponsor

565 Previous Clinical Trials

1,921,429 Total Patients Enrolled

1 Trials studying Interleukin-3 Receptor Subunit Alpha Positive

National Cancer Institute (NCI)NIH

13,657 Previous Clinical Trials

40,933,636 Total Patients Enrolled

1 Trials studying Interleukin-3 Receptor Subunit Alpha Positive

Mustang Bio, Inc.Industry Sponsor

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How successful have past experiments been utilizing Autologous CD123CAR-CD28-CD3zeta-EGFRt-expressing T Lymphocytes?

"Currently, there are 889 ongoing medical studies exploring Autologous CD123CAR-CD28-CD3zeta-EGFRt-expressing T Lymphocytes with 161 of them being in the final phase. These experiments primarily take place in Philadelphia, Pennsylvania but can be found across 28446 sites globally."

Answered by AI

Is recruitment for this experiment proceeding according to plan?

"This research endeavour has already reached its quota of participants and is no longer accepting applications. Initially posted on December 15th 2015, the project's last update was February 17th 2022. Patients who are interested in alternative trials may consider 4737 studies related to minimal residual disease or 889 medical investigations that involve Autologous CD123CAR-CD28-CD3zeta-EGFRt-expressing T Lymphocytes."

Answered by AI

How many individuals are enrolled in this trial to date?

"This trial has closed its recruitment period and is no longer searching for patients. It was first published on December 15th, 2015 with the last edit taking place February 17th 2022. There are currently 4737 trials looking to recruit participants suffering from minimal residual disease, along with 889 studies requiring Autologous CD123CAR-CD28-CD3zeta-EGFRt expressing T Lymphocytes volunteers."

Answered by AI

What medical conditions have been treated through the utilization of Autologous CD123CAR-CD28-CD3zeta-EGFRt-expressing T Lymphocytes?

"Autologous CD123CAR-CD28-CD3zeta-EGFRt-expressing T Lymphocytes has been proven to be an effective treatment for multiple sclerosis, mixed cell type lymphoma and acute myelocytic leukemia."

Answered by AI

Has the FDA granted permission for Autologous CD123CAR-CD28-CD3zeta-EGFRt-expressing T Lymphocytes to be used in therapeutic treatments?

"Our appraisal of Autologous CD123CAR-CD28-CD3zeta-EGFRt-expressing T Lymphocytes' safety is 1 due to its current Phase 1 status; meaning concrete evidence for both efficacy and safety has yet to be established."

Answered by AI

What potential benefits are the researchers hoping to gain from this trial?

"As outlined by Mustang Bio, Inc., the primary outcome of this trial will be monitored over a 28 day period and is focused on adverse events as indicated in NCI CTCAE version 4.0. Additionally, there are two secondary objectives: measuring CAR123-specific antibody levels with associated 95% Clopper and Pearson binomial confidence limits; engraftment of transferred CD123+ CAR T cells also accompanied by respective confidence intervals; and descriptive statistics for duration of response to treatment stratified across disease arms."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Genetically Modified T-cell Immunotherapy in Treating Patients With Relapsed/Refractory Acute Myeloid Leukemia and Persistent/Recurrent Blastic Plasmacytoid Dendritic Cell Neoplasm

2015. "Genetically Modified T-cell Immunotherapy in Treating Patients With Relapsed/Refractory Acute Myeloid Leukemia and Persistent/Recurrent Blastic Plasmacytoid Dendritic Cell Neoplasm". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02159495.

All > Acute Myeloid Leukemia > Aml