← Back to Search

CAR T-cell Therapy

CAR T-Cell Therapy for Acute Lymphoblastic Leukemia

Phase 1 & 2

Waitlist Available

Led By Aimee C. Talleur, MD

Research Sponsored by St. Jude Children's Research Hospital

Eligibility Criteria Checklist

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

Must have

Age ≤ 21 years old

CD19+ ALL with any of the following: Primary refractory disease despite at least 2 cycles of an intensive chemotherapy regimen designed to induce remission, Refractory disease despite salvage therapy, 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 for any of the following reasons: Patients that do not have an available allogeneic donor (defined as at least a 7/8 HLA-matched related/unrelated donor, 5/6 HLA-matched umbilical cord donor, or 3/6 HLA-matched haploidentical donor), Patients with refractory leukemia, for which allogeneic transplant is known to be less effective in the B-ALL population, and Patients who are unable to receive myeloablative total body irradiation (TBI), which is included in standard transplant regimens for patients with B - ALL., Detectable disease, Age: ≤ 21 years of age, Estimated life expectancy of > 8 weeks, Prior to planned SJCAR19 infusion, patients with a history of prior allogeneic HCT must be at least 3 months from HCT, have no evidence of active GVHD and 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 ≥50 ml/min/1.73m2 (GFR ≥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, 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, Hemoglobin > 8 g/dl (can be transfused), Platelet count > 20,000/μL (can be transfused), Has recovered from all NCI CTAE grade III-IV, non-hematologic acute toxicities from prior therapy, 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, If sexually active, agreement to use birth control until 6 months after T-cell infusion. Male partners should use a condom, Available SJCAR19 product with ≥ 15% expression of the CD19-CAR, and killing of CD19+ targets ≥ 20% in an in vitro cytotoxicity assay, Agreement to participate in long-term follow-up on protocol NCT00695279

Must not have

CNS-3 disease with or without neurologic changes

Receiving systemic steroids therapy exceeding the equivalent of 0.5 mg/ kg/day of methylprednisolone, in the 7 days prior to CAR T-cell infusion

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 4 weeks post-sjcar19 infusion

Awards & highlights

No Placebo-Only Group

Summary

This trial is for patients with relapsed or refractory CD19+ acute lymphoblastic leukemia (ALL) who are between the ages of 0 and 30. The trial will test the safety and effectiveness of a new treatment called chimeric antigen receptor (CAR) T cell therapy. In this therapy, patient's own immune cells are collected and then manufactured to be more effective at killing cancer cells. The product will be made at the St. Jude Children's Research Hospital's Good Manufacturing Practice (GMP) facility. The trial will test different doses of the therapy to find the largest safe dose, as well as how long the therapy lasts in the

Who is the study for?

This trial is for young people (≤21 years old) with a specific type of leukemia called CD19+ ALL that hasn't responded to other treatments or has come back. They should have a life expectancy over 8 weeks, be able to perform certain physical activities, and not be pregnant or breastfeeding. They must agree to use birth control and can't join if they have severe infections, HIV, CNS-3 disease with neurological changes, are on high-dose steroids or immunosuppressants.

What is being tested?

The study tests CAR T-cell therapy using engineered immune cells (SJCAR19 product) in children and young adults with relapsed/refractory leukemia. It aims to find the highest safe dose, how long these cells last in the body, their side effects, and effectiveness against this type of leukemia.

What are the potential side effects?

Possible side effects include reactions related to immune system activation such as fever and fatigue; organ inflammation; allergic reactions due to mouse protein content in the treatment; blood cell count changes; infection risk increase; and potential neurologic symptoms.

Eligibility Criteria

Inclusion Criteria

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

Select...

I am 21 years old or younger.

Select...

You have a type of leukemia called CD19+ ALL and meet any of the following conditions: your disease has not responded to the usual chemotherapy treatments, you have relapsed multiple times, you have failed to respond to other treatments or you are unable to undergo a stem cell transplant. You are under 21 years old and have a life expectancy of at least 8 weeks. You also need to meet certain health criteria such as having adequate heart, kidney, and lung function, and normal blood count levels. Women who may become pregnant must have a negative pregnancy test and agree to use birth control during and after treatment. Your cancer cells must have certain characteristics for you to be eligible for this study. You must also agree to long-term follow-up.

Exclusion Criteria

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

Select...

My cancer has spread to my brain, with or without symptoms.

Select...

I haven't taken high doses of steroids in the week before my CAR T-cell treatment.

Select...

I have not had chemotherapy injected into my spine within the last week.

Select...

I do not have any severe, uncontrolled infections.

Select...

I do not have a severe infection right now.

Select...

My brain or spinal cord disease has caused changes in my nervous system.

Select...

I have an active neurological condition that is not under control.

Select...

I have a history of HIV infection.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 4 weeks post-sjcar19 infusion

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~4 weeks post-sjcar19 infusion

This trial's timeline: 3 weeks for screening, Varies for treatment, and 4 weeks post-sjcar19 infusion for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Complete Response Rate

Maximum Tolerated Dose and Dose-limiting Toxicities

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: SJCAR19 TherapyExperimental Treatment5 Interventions

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Cyclophosphamide

2010

Completed Phase 4

~2310

Fludarabine

2012

Completed Phase 4

~1860