Evaluation of CD19-Specific CAR Engineered Autologous T-Cells for Treatment of Relapsed/Refractory CD19+ Acute Lymphoblastic Leukemia
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|ClinicalTrials.gov Identifier: NCT03573700|
Recruitment Status : Recruiting
First Posted : June 29, 2018
Last Update Posted : November 12, 2019
SJCAR19 is a research study seeking to evaluate the use of chimeric antigen receptor (CAR) T cell therapy, a type of cellular therapy, for the treatment of pediatric, adolescent and young adult patients with relapsed or refractory CD19+ acute lymphoblastic leukemia (ALL). CAR therapy combines two of the body's basic disease fighters: antibodies and T Cells. For this type of therapy, peripheral (circulating) immune cells are collected and then undergo a manufacturing process to engineer them to more effectively kill cancer cells. The SJCAR19 product will be manufactured at the St. Jude Children's Research Hospital's Good Manufacturing Practice (GMP) facility.
The main purpose of this study is to determine:
- The largest dose of SJCAR19 that is safe to give,
- How long SJCAR19 cells last in the body,
- The side effects of SJCAR19, and
- Whether or not treatment with SJCAR19 is effective in treating people with refractory or relapsed ALL.
|Condition or disease||Intervention/treatment||Phase|
|Acute Lymphoblastic Leukemia, in Relapse Acute Lymphoblastic Leukemia, Refractory||Drug: Cyclophosphamide Drug: Fludarabine Drug: Mesna Device: CliniMACS Biological: CD19- specific CAR engineered autologous T-cells (SJCAR19 product)||Phase 1 Phase 2|
SJCAR19 is a Phase I/II clinical trial evaluating the use of SJCAR19 (CD19- specific CAR engineered autologous T-cells) in pediatric, adolescent and young adult patients with relapsed/ refractory CD19+ ALL. Treatment will include a single treatment course, with most patients receiving a lymphodepleting chemotherapy preparative regimen of fludarabine/ cyclophosphamide, followed by a single infusion of SJCAR19.
This protocol contains a 3-part consent process: 1) to proceed with autologous apheresis, 2) to proceed with manufacturing of the SJCAR19 product, and 3) to receive treatment with the SJCAR19 product (initially as Phase I, then proceeding to Phase II). The Phase I portion will evaluate the safety and maximum tolerated dose (MTD) of SJCAR19.
The Phase II portion will evaluate the efficacy, and provide further safety evaluation, of SJCAR19 in an expansion cohort at the MTD determined in the Phase I portion of the study. Additionally, for both the Phase I/II portions of the study there are correlative studies evaluating the biology of this treatment as well assessments into patient/caregiver experiences with undergoing this treatment.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||35 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||SJCAR19: A Phase I/II Study Evaluating SJCAR19 (CD19-Specific CAR Engineered Autologous T-Cells) in Pediatric and Young Adult Patients ≤ 21 Years of Age With Relapsed or Refractory CD19+ Acute Lymphoblastic Leukemia|
|Actual Study Start Date :||July 24, 2018|
|Estimated Primary Completion Date :||July 1, 2023|
|Estimated Study Completion Date :||July 1, 2024|
Experimental: SJCAR19 Therapy
Patients in both the Phase I and Phase II portion of the study will receive lymphodepleting chemotherapy (unless determined by PI that lymphodepletion is not necessary), followed by a single infusion of the patient-derived SJCAR19 cellular product. The most commonly used lymphodepleting chemotherapy regimen will consist of the agents: Fludarabine and Cyclophosphamide. They will also receive Mesna. Dosing of SJCAR19 on the Phase I study will follow a dose escalation schema, with dose changes based on dose-limiting toxicities. In the Phase II study, SJCAR19 dosing with follow the maximum tolerated dose, as determined in the Phase I portion.
Cells for infusion are prepared using the CliniMACS System.
Other Name: Cytoxan
Other Name: Fludara
Other Name: Mesnex
The mechanism of action of the CliniMACS Cell Selection System is based on magnetic-activated cell sorting (MACS). The CliniMACS device is a powerful tool for the isolation of many cell types from heterogeneous cell mixtures, (e.g. apheresis products). These can then be separated in a magnetic field using an immunomagnetic label specific for the cell type of interest.
Biological: CD19- specific CAR engineered autologous T-cells (SJCAR19 product)
- Maximum Tolerated Dose and Dose-limiting Toxicities [ Time Frame: 4 weeks post-SJCAR19 infusion ]The primary objectives for the Phase I study portion are to determine the maximum tolerated dose (MTD) and characterize the safety profile and dose-limiting toxicities (DLTs) of treatment with SJCAR19 in pediatric and young adult patient's ≤ 21 years of age, with relapsed or refractory CD19+ ALL.
- Complete Response Rate [ Time Frame: 4 weeks post-SJCAR19 infusion ]The primary objective for the Phase II study portion is to evaluate the complete response (CR) rates of SJCAR19 in pediatric and young adult patient's ≤ 21 years of age, with relapsed or refractory CD19+ ALL.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03573700
|Contact: Aimee C. Talleur, MDfirstname.lastname@example.org|
|United States, Tennessee|
|St. Jude Children's Research Hospital||Recruiting|
|Memphis, Tennessee, United States, 38105|
|Contact: Aimee C. Talleur, MD 866-278-5833 email@example.com|
|Principal Investigator: Aimee C. Talleur, MD|
|Principal Investigator:||Aimee C. Talleur, MD||St. Jude Children's Research Hospital|