Trial Outcomes & Findings for CD19/CD22 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory CD19/CD22-expressing B Cell Malignancies (NCT NCT03448393)

CD19/CD22 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory CD19/CD22-expressing B Cell Malignancies

Safety analyses will consist of tabulations of grades of toxicity by type of toxicity assessed by the Common Terminology Criteria for Adverse Events (CTCAE) v5.0. Grade 1 is mild, Grade 2 is moderate, Grade 3 is severe, Grade 4 is life-threatening, and Grade 5 is death related to adverse event.

MTD is defined as the dose level immediately below the level at which the enrollment is stopped due to dose-limiting toxicity (DLT). A DLT is defined as an adverse event that is at least possibly related to the cluster of differentiation 19/cluster of differentiation 22 (CD19/CD22)-chimeric antigen receptor (CAR) T cells with onset within the first 28 days after cell infusion.

Number of participants that have the targeted dose number of CAR cells successfully manufactured (i.e., number of participants enrolled where the correct number of cells are produces at the dose level that are enrolled) as measured by total number of viable cluster of differentiation 19 (CD19)/cluster of differentiation 22 (CD22) transduced T cells.

Overall survival (OS) will be determined as the time from the date of chimeric antigen receptor (CAR) infusion until death.

PFS is assessed from the date of chimeric antigen receptor (CAR) infusion until the documentation of disease progression or death due to any cause, whichever occurs first. Disease progression was assessed by the Response Criteria Lymphoma and is defined as individual node/lesion must be abnormal with LDI\>1.5 cm and increase by ≥50% from product of perpendicular diameters (PPD) nadir and an increase in longest transverse diameter of a lesion (LDI) or shortest axis perpendicular to LDi (SDI) from nadir 0.5 cm for lesions ≤2 cm, 1.0 cm for lesions \> 2 cm; and the International Working Group and is defined as worse marrow classification with at least a 50% increase in the percentage of marrow blasts.

Clinical activity (response) in children (age ≥3 years to 17 years) and young adults (age 18 years to ≤ 39 years) was measured by the Response Criteria Lymphoma. Complete Response (CR) is complete metabolic and/or radiographic response. Partial Response (PR) is partial metabolic response or partial remission. Stable Disease (SD) is no metabolic response or 50% decrease from baseline in the sum of products of diameters (SPD) of up to 6 dominant measurable nodes and extra nodal sites. Progressive Disease (PD) is individual node/lesion must be abnormal with longest transverse diameter of a lesion (LDI) \>1.5 cm and increase by ≥50% from product of perpendicular diameters (PPD) nadir and an increase in LDI or shortest axis perpendicular to LDi (SDI) from nadir 0.5 cm for lesions ≤2 cm and/or 1.0 cm for lesions \> 2 cm; also assessed by the International Working Group and PD is defined as worse marrow classification with at least a 50% increase in the percentage of marrow blasts.

A DLT is defined as an adverse event that is at least possibly related to the cluster of differentiation 19/cluster of differentiation 22 (CD19/CD22)-chimeric antigen receptor (CAR) T cells with onset within the first 28 days after cell infusion.

Here is the number of participants with serious and/or non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.