Study of Adoptive Cellular Therapy Using Autologous T Cells Transduced With Lentivirus to Express a CD33 Specific Chimeric Antigen Receptor in Patients With Relapsed or Refractory CD33-Positive Acute Myeloid Leukemia

NCT ID: NCT03126864

Last Updated: 2019-11-04

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1

Total Enrollment

11 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-08-04

Study Completion Date

2019-10-10

Brief Summary

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Any time the words "you," "your," "I," or "me" appear, it is meant to apply to the potential participant.

The goal of this clinical research study is to learn about the safety and tolerability of 3 different doses of CD33-CAR-T cells (referred to throughout the consent as "T-cells") in patients who have CD33-positive acute myeloid leukemia (AML) that is relapsed (has come back) or refractory (has not responded to treatment).

CD33-CAR-T is made by genetically modifying (changing) your T-cells (a type of white blood cell). T-cells are genetically changed to help target leukemia cells.

This is an investigational study. CD33-CAR-T is not FDA approved or commercially available. It is currently being used for research purposes only. The study doctor can explain how the study drug is designed to work.

Up to 39 participants will be enrolled in this study. All will take part at MD Anderson.

Detailed Description

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Conditions

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Hematopoietic/Lymphoid Cancer Acute Myeloid Leukemia

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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CD33-CAR-T cells - Adult Group

After enrollment, steady state leukapheresis performed to collect apheresis material.

Fludarabine administered by vein on Days -5 to -3.

Cyclophosphamide administered by vein on Day -3.

CD33-CAR-T cell infusion administered by vein on Day 0. First group of participants receive the lowest dose level. Each new group will receive a higher dose than the one before it, if no intolerable side effects were seen. This will continue until the highest tolerable dose of T-cells is found.

Group Type EXPERIMENTAL

Leukapheresis

Intervention Type PROCEDURE

After enrollment, steady state leukapheresis performed to collect apheresis material. The goal is to achieve a target total nucleated cell (TNC) yield of at least 5 x 10\^9 (expected range 5 x 10\^8 - 5 x 10\^10), for up to two days.

Fludarabine

Intervention Type DRUG

25 mg/m2 administered by vein on Days -5 to -3.

Cyclophosphamide

Intervention Type DRUG

900 mg/m2 administered by vein on Day -3.

CD33-CAR-T Cell Infusion

Intervention Type BIOLOGICAL

CD33-CAR-T cell infusion administered by vein on Day 0. First group of participants receive the lowest dose level. Each new group will receive a higher dose than the one before it, if no intolerable side effects were seen. This will continue until the highest tolerable dose of T-cells is found. Starting dose level is \> 1.5 x 105/kg but ≤ 4.5 x 105/kg.

CD33-CAR-T cells - Pediatric Group

After enrollment, steady state leukapheresis performed to collect apheresis material.

Fludarabine administered by vein on Days -5 to -3.

Cyclophosphamide administered by vein on Day -3.

CD33-CAR-T cell infusion administered by vein on Day 0. First group of participants receive the lowest dose level. Each new group will receive a higher dose than the one before it, if no intolerable side effects were seen. This will continue until the highest tolerable dose of T-cells is found.

Group Type EXPERIMENTAL

Leukapheresis

Intervention Type PROCEDURE

After enrollment, steady state leukapheresis performed to collect apheresis material. The goal is to achieve a target total nucleated cell (TNC) yield of at least 5 x 10\^9 (expected range 5 x 10\^8 - 5 x 10\^10), for up to two days.

Fludarabine

Intervention Type DRUG

25 mg/m2 administered by vein on Days -5 to -3.

Cyclophosphamide

Intervention Type DRUG

900 mg/m2 administered by vein on Day -3.

CD33-CAR-T Cell Infusion

Intervention Type BIOLOGICAL

CD33-CAR-T cell infusion administered by vein on Day 0. First group of participants receive the lowest dose level. Each new group will receive a higher dose than the one before it, if no intolerable side effects were seen. This will continue until the highest tolerable dose of T-cells is found. Starting dose level is \> 1.5 x 105/kg but ≤ 4.5 x 105/kg.

Interventions

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Leukapheresis

After enrollment, steady state leukapheresis performed to collect apheresis material. The goal is to achieve a target total nucleated cell (TNC) yield of at least 5 x 10\^9 (expected range 5 x 10\^8 - 5 x 10\^10), for up to two days.

Intervention Type PROCEDURE

Fludarabine

25 mg/m2 administered by vein on Days -5 to -3.

Intervention Type DRUG

Cyclophosphamide

900 mg/m2 administered by vein on Day -3.

Intervention Type DRUG

CD33-CAR-T Cell Infusion

CD33-CAR-T cell infusion administered by vein on Day 0. First group of participants receive the lowest dose level. Each new group will receive a higher dose than the one before it, if no intolerable side effects were seen. This will continue until the highest tolerable dose of T-cells is found. Starting dose level is \> 1.5 x 105/kg but ≤ 4.5 x 105/kg.

Intervention Type BIOLOGICAL

Other Intervention Names

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Fludarabine Phosphate Fludara Cytoxan Neosar

Eligibility Criteria

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Inclusion Criteria

1. Age 1-80 years of age. The pediatric cohort is defined as age younger than 18 years.
2. Patients with active (blood or bone marrow blasts \>5%) relapsed or refractory CD33+ acute myeloid leukemia (AML) de novo, or secondary. a. Relapsed AML is defined as patients that had a first complete remission (CR) before developing recurrent disease. b. Refractory AML defined as patients that have not achieved a CR after 2 cycles of induction chemotherapy.
3. Patients must have bone marrow and peripheral blood studies available for confirmation of diagnosis of AML; CD33 positivity must be confirmed by either flow cytometry or immunohistochemistry; cytogenetics, flow cytometry, and molecular studies (such as FMS-like tyrosine kinase-3 \[Flt-3\] status) will be obtained as per standard practice.
4. ECOG performance status score \</= 2.
5. Pretreatment calculated or measured creatinine clearance (absolute value) of \>= 60 mL/minute.
6. Serum bilirubin =\< 3.0 mg/dL.
7. Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) =\< 5 times the institutional upper limits of normal.
8. Ejection fraction measured by echocardiogram (ECHO) or multi gated acquisition scan (MUGA) \>50%.
9. Subject does not require supplemental oxygen or mechanical ventilation, and oxygen saturation by pulse oximetry is 94% or higher on room air.
10. Negative serum pregnancy test.
11. Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control or abstinence) prior to study entry and for six months following duration of study participation; should a woman become pregnant or suspect that she is pregnant while participating on the trial, she should inform her treating physician immediately.
12. Patients who have undergone allo-SCT are eligible if they are at least 3 months post SCT, have relapsed AML, are not on treatment or prophylaxis for GVHD, and have no active GVHD.
13. All patients or legally responsible parent or guardian must have the ability to understand and willingness to sign a written informed consent

Exclusion Criteria

1. Diagnosis of acute promyelocytic leukemia (APL M3): t(15;17)(q22;q12); (promyelocytic leukemia \[PML\]/retinoic acid receptor \[RAR\] alpha \[a\]) and variants excluded.
2. Patients with extramedullary disease as their sole site of relapsed AML.
3. Acceptable allogeneic stem cell donor with imminent plans to proceed with allo-SCT.
4. Known central nervous system (CNS) leukemic involvement that is refractory to intrathecal chemotherapy and/or cranio-spinal radiation; patients with a history of CNS disease that have been effectively treated to complete remission (\< 5 white blood cell \[WBC\]/mm\^3 and no blasts in cerebrospinal fluid \[CSF\]) will be eligible.
5. Ongoing or active or uncontrolled 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.
6. Human immunodeficiency virus (HIV) seropositivity, or active hepatitis B or C infection based on testing performed within 4 weeks of enrollment.
7. Currently enrolled in another investigational therapy protocol for AML.
8. Participants with presence of other active malignancy within 2 years of study entry; participants with history of prior malignancy treated with curative intent and achieved CR within 2 years are eligible.
9. Pregnant and lactating women are excluded from this study
10. Failure of research participant or legally responsible parent or guardian to understand the basic elements of the protocol and/or the risks/benefits of participating in this phase I study.
11. History of allergic reactions attributed to compounds of similar chemical or biological composition to cetuximab (anti-EGFR).
12. History of allergic reactions to products containing mouse and bovine protein antibodies.
13. Receiving corticosteroids at \>20 mg (age \>17) or \>0.5mg/kg (age \<18) daily prednisone dose or equivalent.
14. Active autoimmune disease requiring systemic immunosuppressive therapy.
15. Patient, who in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.
Minimum Eligible Age

1 Year

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Intrexon Corporation

INDUSTRY

Sponsor Role collaborator

M.D. Anderson Cancer Center

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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William G. Wierda, MD, PHD, BS

Role: PRINCIPAL_INVESTIGATOR

M.D. Anderson Cancer Center

Locations

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University of Texas MD Anderson Cancer Center

Houston, Texas, United States

Site Status

Countries

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United States

Related Links

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http://www.mdanderson.org

University of Texas MD Anderson Cancer Center Website

Other Identifiers

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NCI-2018-01167

Identifier Type: REGISTRY

Identifier Source: secondary_id

2016-0341

Identifier Type: -

Identifier Source: org_study_id

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