CAR T CELL Therapy for Pediatric, Adolescent and Young Adult Patients With CD19-Positive Leukemia

NCT ID: NCT06847269

Last Updated: 2025-10-27

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 25 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-05-21
• Study Completion Date: 2031-04-30

Brief Summary

CAR19PK is a research study evaluating the use of lymphodepleting chemotherapy and chimeric antigen receptor (CAR) T cell therapy, a type of cellular therapy, for the treatment of refractory and/or relapsed leukemia. For this type of therapy, peripheral (circulating) immune cells are collected and then modified so that they can recognize an antigen, which is a particle present on the surface of a cancer cell. The CD19-CAR T cell 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:

• Evaluate different doses of fludarabine prior CAR T cell infusion
• How your body processes fludarabine and cyclophosphamide,
• How long the CAR T cells last in the body,
• Whether or not treatment with this therapy is effective in treating people with refractory or relapsed leukemia, and
• The side effects of this therapy.

Detailed Description

CAR19PK is a Phase II study evaluating lymphodepleting chemotherapy (age-based fludarabine dosing and cyclophosphamide), followed by infusion of CD19-CAR T cells, in pediatric and young adult patients ≤ 21 years old with relapsed/refractory CD19-positive leukemia. Treatment will include a single course of lymphodepleting chemotherapy followed by CAR T cell infusion. Lymphodepletion will include fludarabine (dosing based on age) and cyclophosphamide. The CAR T cell infusion will include a single infusion of 3x10^6 CD19-CAR T cells/kg patient weight.

This protocol contains a two-part consent process: 1) to proceed with autologous apheresis and 2) to proceed with treatment with lymphodepleting chemotherapy and infusion of the CD19-CAR T cell product.

Conditions

Acute Lymphoblastic Leukemia; Refractory Acute Lymphoblastic Leukemia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

CAR19PK Therapy

This study contains two phases.

Collection and Manufacturing Phase:

Patient blood cells will be collected, and possibly frozen, via a process called apheresis. These cells will then be changed to improve their ability to recognize and kill cancer cells.

Treatment Phase:

Patients that meet eligibility for treatment will receive lymphodepleting chemotherapy with fludarabine and cyclophosphamide, followed by an infusion of CD19-CAR T cells that were made in the Collection and Manufacturing Phase.

Group Type: EXPERIMENTAL

Fludarabine

Intervention Type: DRUG

Given IV

Cyclophosphamide

Intervention Type: DRUG

Given IV

Mesna

Intervention Type: DRUG

Given IV

CD19-CAR T cell Infusion

Intervention Type: BIOLOGICAL

Patients will receive the CD19-CAR T cells by vein, through either an IV or a central line.

Interventions

Fludarabine

Given IV

Intervention Type: DRUG

Cyclophosphamide

Given IV

Intervention Type: DRUG

Mesna

Given IV

Intervention Type: DRUG

CD19-CAR T cell Infusion

Patients will receive the CD19-CAR T cells by vein, through either an IV or a central line.

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

• CD19+ leukemia** with any of the following:

• Refractory disease (primary or in relapse)
• 2nd or greater relapse
• Any relapse after allogeneic hematopoietic cell transplantation
• 1st relapse if patient requires an allogeneic HCT as part of standard of care relapse therapy, but is found to be ineligible and/or unsuitable for HCT

• must be confirmed to be CD19+ within 3 months prior to enrollment for treatment
• Age: ≤ 21 years of age
• Karnofsky or Lansky (age-dependent) performance score ≥ 50 (Appendix A)
• Estimated life expectancy of > 12 weeks. Patients with a history of prior allogeneic hematopoietic cell transplantation [HCT] must be clinically recovered from prior HCT therapy, have no evidence of active GVHD and have not received a donor lymphocyte infusion (DLI) within the 28 days prior to apheresis
• For females of child bearing age:

• Not lactating with intent to breastfeed
• Not pregnant with negative serum pregnancy test within 7 days prior to enrollment

• Age: ≤ 21 years of age
• Estimated life expectancy of > 8 weeks
• Detectable disease
• Prior to planned CAR T cell infusion, patients with a history of prior allogeneic HCT must:

• be at least 3 months from HCT
• have no evidence of active GVHD
• have not received a donor lymphocyte infusion (DLI) within the 28 days prior to planned infusion
• Adequate cardiac function defined as left ventricular ejection fraction > 40%, or shortening fraction ≥ 25%
• EKG without evidence of clinically significant arrhythmia
• Adequate renal function defined as creatinine clearance or radioisotope GFR ³ 50 ml/min/1.73m2 (GFR ³ 40 ml/min/1.73m2 if < 2 years of age)
• Adequate pulmonary function defined as forced vital capacity (FVC) ≥ 50% of predicted value; or pulse oximetry ≥ 92% on room air if patient is unable to perform pulmonary function testing
• Karnofsky or Lansky (age-dependent) performance score ≥ 50 (Appendix A)
• Total Bilirubin ≤ 3 times the upper limit of normal for age, except in subjects with Gilbert's syndrome
• Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≤ 5 times the upper limit of normal for age
• Has recovered from all NCI CTAE grade III-IV, non-hematologic acute toxicities from prior therapy
• For patients of child bearing age:

• Not lactating with intent to breastfeed
• Not pregnant with negative serum pregnancy test within 7 days prior to enrollment
• If sexually active, agreement to use birth control until 6 months after T cell infusion.

Exclusion Criteria

• Known primary immunodeficiency
• History of HIV infection
• Severe intercurrent bacterial, viral or fungal infection
• History of hypersensitivity reactions to murine protein-containing products
• Known contraindication to receiving protocol defined lymphodepleting chemotherapy regimen

Treatment

• Active CNS-3 disease
• Known primary immunodeficiency
• History of HIV infection
• Evidence of active, uncontrolled neurologic disease
• Severe, uncontrolled bacterial, viral or fungal infection
• History of hypersensitivity reactions to murine protein-containing products
• Known contraindication to receiving protocol defined lymphodepleting chemotherapy regimen

• Maximum Eligible Age: 21 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

St. Jude Children's Research Hospital

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Aimee Talleur, MD

Role: PRINCIPAL_INVESTIGATOR

St. Jude Children's Research Hospital

Locations

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Aimee Talleur, MD

Role: CONTACT

referralinfo@stjude.org

8662785833

Facility Contacts

Aimee Talleur, MD

Role: primary

referralinfo@stjude.org

866-278-5833

Related Links

http://www.stjude.org

St. Jude Children's Research Hospital

http://www.stjude.org/protocols

Clinical Trials Open at St. Jude

Other Identifiers

CAR19PK

Identifier Type: -

Identifier Source: org_study_id