A Phase 1 interventional study of CD19/CD22 CAR T-Cells and Fludarabine in Lymphoma, Non-Hodgkin, Leukemia, Lymphocytic, B Cell and B-Cell Lymphoma, sponsored by National Cancer Institute (NCI). Completed at 1 site in United States. Open to participants aged 3 Years to 39 Years. Per ClinicalTrials.gov, last updated 2025-06-04.
Sponsored by National Cancer Institute (NCI) · Phase 1, Interventional, and Treatment
Among 27 studies in Lymphoma, B-Cell by National Cancer Institute (NCI) — 4 open to participants now, 11 under way, 1 of them phase 3. Lymphoma, B-Cell · National Cancer Institute (NCI).
Among the 27 studies in Lymphoma, B-Cell by National Cancer Institute (NCI), this one is the 6th largest by enrollment of the 26 interventional ones, is one of 2 that started in 2018, is one of 18 in phase 1, and was the 16th registered.
885 studies on the registry are indexed under Lymphoma, B-Cell; 286 are open to participants now.
This study's enrollment of 54 is above the median of 34 across 773 interventional studies indexed under Lymphoma, B-Cell.
Browse Lymphoma, B-Cell studies →4 of the 27 studies in Lymphoma, B-Cell by National Cancer Institute (NCI) are open now, in 2 countries. Each count lists the studies.
Of the 209 Lymphoma, B-Cell studies recruiting now, 129 (62%) set no upper age limit and 60 (29%) stop at 75 or younger; 37 take children under 18; 3 take healthy volunteers.
This study: 3 Years to 39 Years · all sexes · no healthy volunteers.
The full table on the hub →27 studies; the count open now beside each. This study's row is marked.
First in 1991; this study’s year is marked. 2 started in 2018.
Median enrollment 32 across the 26 interventional studies, 902 across the 1 observational. Of the 15 that completed or were terminated, 8 (53%) have results posted.
National Cancer Institute (NCI) is the lead sponsor of 3,556 studies on the registry; 336 are open to participants now.
Of its 411 completed or terminated interventional studies of FDA-regulated products, 373 (91%) have results posted.
4 of the studies in Lymphoma, B-Cell by National Cancer Institute (NCI) that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT04759586Phase 3National Cancer Institute (NCI)Nivolumab in Combination With Chemo-Immunotherapy for the Treatment of Newly Diagnosed Primary Mediastinal B-Cell Lymphoma | 2027-06-30 |
| NCT05627245National Cancer Institute (NCI)Testing the Safety of the Anti-cancer Drugs Tazemetostat and Belinostat in Patients With Lymphomas That Have Resisted Treatment | 2027-06-01 |
| NCT05272384National Cancer Institute (NCI)Testing the Combination of Nivolumab and ASTX727 for Relapsed or Refractory B-Cell Lymphoma | 2027-06-28 |
| NCT05442515National Cancer Institute (NCI)CD19/CD22 Bicistronic Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory B Cell Malignancies | 2027-07-01 |
Among the 27 studies in Lymphoma, B-Cell by National Cancer Institute (NCI), from their registry updates:
Background:
B-cell leukemias and lymphomas are cancers that are often difficult to treat. The primary objective of this study is to determine the ability to take a patient's own cells (T lymphocytes) and grow them in the laboratory with the cluster of differentiation 19 (CD19/cluster of differentiation 22-chimeric antigen receptor (CD22-CAR) gene through a process called 'lentiviral transduction (also considered gene therapy) and growing them to large numbers to use as a treatment for hematologic cancers in children and young adults.. Researchers want to see if giving modified CD19/CD22-CAR T cells to people with these cancers can attack cancer cells. In addition, the safety of giving these gene modified cells to humans will be tested at different cell doses. Additional objectives are to determine if this therapy can cause regression of B cell cancers and to measure if the gene modified cells survive in patients' blood.
Objective:
To study the safety and effects of giving CD19/CD22-CAR T cells to children and young adults with B-cell cancer.
Eligibility:
People ages 3-39 with certain cancers that have not been cured by standard therapy. Their cancer tissue must express the CD19 protein.
Design:
A sample of participants blood or bone marrow will be sent to National Institutes of Health (NIH) and tested for leukemia.
Participants will be screened with:
Medical history
Physical exam
Urine and blood tests (including for human immunodeficiency virus (HIV)
Heart and eye tests
Neurologic assessment and symptom checklist.
Scans, bone marrow biopsy, and/or spinal tap
Some participants will have lung tests.
Participants will repeat these tests throughout the study and follow-up.
Participants will have leukapheresis. Blood will be drawn from a plastic tube (intravenous (IV) or needle in one arm then go through a machine that removes lymphocytes. The remaining blood will be returned to the participant's other arm.
Participants will stay in the hospital about 2 weeks. There they will get:
Two chemotherapy drugs by IV
Their changed cells by IV
Standard drugs for side effects
Participants will have frequent follow-up visits for 1 year, then 5 visits for the next 4 years. Then they will answer questions and have blood tests every year for 15 years.
...
Background:
Objectives:
-Assess the safety of administering escalating doses of autologous CD19/CD22-CAR engineered T cells that meet established release specifications in children and young adults with CD19+CD22+ B cell ALL, isolated central nervous system (CNS) ALL, or lymphoma following a cyclophosphamide/fludarabine conditioning regimen.
Eligibility:
-Participants between >= 3 years and \<= 39 years of age, with CD19+/CD22+ B cell ALL, isolated CNS ALL, or lymphoma who have relapsed or have refractory disease after at least one standard chemotherapy regimen and one salvage regimen, with no alternative curative options who meet standard Phase I eligibility criteria.
Design:
885 studies on the registry are indexed under Lymphoma, B-Cell; 286 are open to participants now.
This study's enrollment of 54 is above the median of 34 across 773 interventional studies indexed under Lymphoma, B-Cell.
Browse Lymphoma, B-Cell studies →National Cancer Institute (NCI) is the lead sponsor of 3,556 studies on the registry; 336 are open to participants now.
Of its 411 completed or terminated interventional studies of FDA-regulated products, 373 (91%) have results posted.
Compared within Lymphoma, B-Cell, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Lymphoma, Non-Hodgkin. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Study subjects with cluster of differentiation 19 (CD19) + cluster of differentiation 22 (CD22+) expressing B cell who have or are treatment refractory may enroll as defined by the following inclusion and exclusion criteria.
INCLUSION CRITERIA:
Diagnosis
CD22/CD19 expression
--CD19 expression must be detected on greater than 15% of the malignant cells by immunohistochemistry or greater than 90% by flow cytometry. The choice of whether to use flow cytometry or immunohistochemistry will be determined by what is the most easily available tissue sample in each participant. In general, immunohistochemistry will be used for lymph node , flow cytometry will be used for peripheral blood and bone marrow samples. CD22+ B cell malignancy is required and CD22 expression levels will be documented when available, but a specific level of expression is not an eligibility requirement; it may be documented as positive or negative.
Age:
--Greater than or equal to 3 years of age (and at least 15 kg) and less than or equal to 39 years of age at time of enrollment (greater than or equal to 3 years to less than or equal to 39 years). NOTE: The first participant in each dose cohort must be greater than or equal to 18 years of age.
Clinical Performance
--Clinical performance status: Participants greater than or equal to 16 years of age: greater than or equal to 50%; Participants \< 16 years of age: Lansky scale greater than or equal to 50%. Subjects who are unable to walk because of paralysis, but who are upright in a wheelchair will be considered ambulatory for the purpose of calculating the performance score.
Participants must have adequate organ and marrow function as defined below:
greater than or equal to 60 mL/min/1.73 m\^2 for participants with creatinine levels above institutional normal.
:
--Individuals of child-bearing or child-fathering potential (IOCBP or IOCFP) must be willing to practice birth control from the time of enrollment on this study and for 12 months after receiving the preparative regimen for IOCBP and for 4 months after receiving the preparative regimen for IOCFP
EXCLUSION CRITERIA:
Subjects meeting any of the following criteria are not eligible for participation in the study:
Subjects will be excluded related to the following prior therapy criteria:
Systemic chemotherapy, anti-neoplastic agents, or antibody-based therapies =\<2 weeks (6 weeks for clofarabine or nitrosoureas) prior to apheresis with the following exception:
---No time restriction with prior intrathecal chemotherapy, therapy, hydroxyurea or ALL maintenance type chemotherapy (vincristine, 6-mercaptopurine, oral methotrexate, or a tyrosine kinase inhibitor for participants with Philadelphia chromosome (Ph)+ ALL) provided there is recovery from any acute toxic effects.
Radiation therapy =\<3 weeks prior to apheresis with the following exception:
---No time restriction with radiation therapy if the volume of bone marrow treated is less than 10% and the subject has measurable/evaluable disease outside the radiation window.
History of stem cell transplantation prior to apheresis that meet the following criteria:
History of prior CAR therapy or other adoptive cell therapies prior to apheresis that meet the following criteria:
(HIV)/ virus (HBV)/ virus (HCV) Infection:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GVHD (graft-versus-host disease)
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cluster of differentiation 19 (CD19)/cluster of differentiation 22 (CD22)-chimeric antigen receptor (CAR)-transduced T cells at escalating doses.
Biological: CD19/CD22 CAR T-Cells · Drug: Fludarabine · Drug: Cyclophosphamide · Procedure: Apheresis · Other: Anti-emetic · Drug: Diphenhydramine · Drug: Acetaminophen · Diagnostic Test: ECG · Diagnostic Test: ECHO · Diagnostic Test: MRI Brain · Procedure: Bone marrow biopsy · Diagnostic Test: Cardiac MRI
Cluster of differentiation 19 (CD19)/cluster of differentiation 22 (CD22)-chimeric antigen receptor (CAR)-transduced T cells at maximum tolerated dose (MTD) or highest dose administered.
Biological: CD19/CD22 CAR T-Cells · Drug: Fludarabine · Drug: Cyclophosphamide · Procedure: Apheresis · Other: Anti-emetic · Drug: Diphenhydramine · Drug: Acetaminophen · Diagnostic Test: ECG · Diagnostic Test: ECHO · Diagnostic Test: MRI Brain · Procedure: Bone marrow biopsy · Diagnostic Test: Cardiac MRI
Cluster of differentiation 19 (CD19)/cluster of differentiation 22 (CD22) chimeric antigen receptor (CAR) T-cells will be infused on Day 0 after lymphodepleting chemotherapy regimen.
Also known as: cluster of differentiation 19 chimeric antigen receptor T-cells, cluster of differentiation 22 chimeric antigen receptor T-cells
Fludarabine is administered as an intravenous (IV) infusion in an appropriate solution over 30 minutes. To prevent undue toxicity the dose will be based on body surface area (BSA) (25-30 mg/m\^2/dose) on Days -4, -3, -2 or Days -5, -4, -3, -2.
Also known as: Fludara
Cyclophosphamide will be diluted in an appropriate solution and infused over one hour. The dose will be based on the body surface area (BSA), at 900 mg/m\^2/dose after fludarabine infusion on Day -2 or 600 mg/m\^2/dose on Days -3 \& -2.
Also known as: Cytoxan
According to institutional standards.
Prophylaxis and treatment.
Pre-medication: 0.5-1 mg/kg/dose (maximum 50 mg/dose) by mouth or intravenous over 10-15 minutes.
Also known as: Benadryl, Banophen, Nytol
Pre-medication: 15 mg/kg/dose (maximum 650 mg/dose by mouth).
Also known as: Tylenol, ofirmev, FeverAll
Pre-cell infusion.
Also known as: Electrocardiogram
Pre-cell infusion.
Also known as: Echocardiogram
Pre-cell infusion.
Also known as: Magnetic resonance imaging
Pre-cell infusion.
Also known as: BM biopsy
Screening
Also known as: Cardiac magnetic resonance imaging
Grades of Toxicity by Type of Toxicity
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.
Time frame: From start of lymphodepleting chemotherapy regimen through 30-days after chimeric antigen receptor (CAR) infusion (approximately 5 weeks).
Maximum Tolerated Dose (MTD)
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.
Time frame: First 28 days after cell infusion
Number of Participants That Have Successful Manufacture of the Targeted Dose Number of Chimeric Antigen Receptor (CAR) Cells
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.
Time frame: CAR infusion (Day 0)
Overall Survival
Overall survival (OS) will be determined as the time from the date of chimeric antigen receptor (CAR) infusion until death.
Time frame: Number of months from CAR cell infusion until date of death or time of censor (max 66.6 months)
Progression-free Survival (PFS)
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.
Time frame: Number of months from CAR cell infusion until time of disease progression, death, or date of censor (up to 67 months)
Clinical Activity (Response) in Children and Young Adults With B-cell Acute Lymphoblastic Leukemia (B-ALL), Isolated Central Nervous System (CNS) ALL, or Lymphoma Who Previously Received Chimeric Antigen Receptor (CAR) Therapy and Those That Are CAR Naive
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.
Time frame: At Day 28 (+/- 4 days) after CAR cell infusion
Number of Participants With a 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.
Time frame: First 28 days
Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0).
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.
Time frame: Adverse Events were monitored/assessed from the first study intervention, Study Day -4, through day 30 after the study agent (s) was/were administered, an average of 5 weeks.
| Milestone | GROUP 1: Cohort 1, Dose Level 1, 3 x10^5 Transduced T Cells/kg (+Or-20%) | GROUP 2: Cohort 1, Dose Level 2, 1x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 3: Cohort 1, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 4: Cohort 1A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 5: Cohort 1B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 6: Cohort 2A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 7: Cohort 2B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20% | GROUP 8: Cohort 2C, Dose Level 3, 3 x 10^6 Transduced T-cells/kg (+Or- 20%) | Enrolled and Not Assigned to a Treatment Assignment Code; Not Treated |
|---|---|---|---|---|---|---|---|---|---|
| Started | 4 | 4 | 5 | 2 | 1 | 0 | 0 | 0 | 8 |
| Completed | 4 | 4 | 4 | 2 | 1 | 0 | 0 | 0 | 0 |
| Not completed | 0 | 0 | 1 | 0 | 0 | 0 | 0 | 0 | 8 |
| Withdrew: Not treated - participant did not receive cell infusion. | 0 | 0 | 1 | 0 | 0 | 0 | 0 | 0 | 8 |
| Milestone | GROUP 1: Cohort 1, Dose Level 1, 3 x10^5 Transduced T Cells/kg (+Or-20%) | GROUP 2: Cohort 1, Dose Level 2, 1x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 3: Cohort 1, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 4: Cohort 1A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 5: Cohort 1B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 6: Cohort 2A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 7: Cohort 2B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20% | GROUP 8: Cohort 2C, Dose Level 3, 3 x 10^6 Transduced T-cells/kg (+Or- 20%) | Enrolled and Not Assigned to a Treatment Assignment Code; Not Treated |
|---|---|---|---|---|---|---|---|---|---|
| Started | 0 | 0 | 0 | 0 | 0 | 21 | 1 | 1 | 7 |
| Completed | 0 | 0 | 0 | 0 | 0 | 21 | 1 | 1 | 0 |
| Not completed | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 7 |
| Withdrew: Not treated - screen failure | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 7 |
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.
| toxicities | GROUP 1: Cohort 1, Dose Level 1, 3 x10^5 Transduced T Cells/kg (+Or-20%) | GROUP 2: Cohort 1, Dose Level 2, 1x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 3: Cohort 1, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 4: Cohort 1A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 5: Cohort 1B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 6: Cohort 2A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 7: Cohort 2B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20% | GROUP 8: Cohort 2C, Dose Level 3, 3 x 10^6 Transduced T-cells/kg (+Or- 20%) |
|---|---|---|---|---|---|---|---|---|
| Grade 3 Anemia | 2 | 2 | 1 | 1 | 1 | 13 | 1 | 0 |
| Grade 3 Febrile neutropenia | 1 | 2 | 1 | 1 | 1 | 13 | 0 | 0 |
| Grade 3 Sinus tachycardia | 0 | 0 | 0 | 0 | 1 | 0 | 0 | 0 |
| Grade 3 Abdominal pain | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 0 |
| Grade 3 Diarrhea | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 0 |
| Grade 3 Gastric hemorrhage | 0 | 0 | 0 | 0 | 0 | 1 | 0 | 0 |
| Grade 3 Fever | 1 | 2 | 0 | 0 | 1 | 3 | 0 | 0 |
| Grade 3 Cytokine release syndrome | 0 | 1 | 0 | 1 | 0 | 2 | 0 | 0 |
| Grade 3 Immune system disorders, other (GVHD) | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 0 |
| Grade 3 Sepsis | 0 | 0 | 0 | 0 | 0 | 1 | 0 | 0 |
| Grade 3 Alanine aminotransferase | 1 | 0 | 0 | 0 | 1 | 1 | 0 | 0 |
| Grade 3 Aspartate aminotransferase | 0 | 0 | 0 | 0 | 1 | 1 | 1 | 0 |
| Grade 3 GGT increased | 0 | 0 | 0 | 0 | 0 | 1 | 0 | 0 |
| Grade 3 Lymphocyte count decreased | 2 | 2 | 4 | 2 | 1 | 13 | 1 | 1 |
| Grade 3 Neutrophil count decreased | 3 | 2 | 3 | 2 | 0 | 17 | 1 | 1 |
| Grade 3 Platelet count decreased | 4 | 1 | 0 | 2 | 0 | 8 | 1 | 0 |
| Grade 3 White blood cell decreased | 3 | 0 | 3 | 2 | 0 | 16 | 1 | 0 |
| Grade 3 Anorexia | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 0 |
| Grade 3 Hypertriglyceridemia | 2 | 1 | 0 | 0 | 0 | 3 | 0 | 0 |
| Grade 3 Hypocalcemia | 0 | 0 | 0 | 0 | 0 | 1 | 0 | 0 |
| Grade 3 Hypokalemia | 0 | 1 | 0 | 0 | 0 | 4 | 0 | 0 |
| Grade 3 Back pain | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 0 |
| Grade 3 Myalgia | 0 | 0 | 0 | 1 | 0 | 1 | 0 | 0 |
| Grade 3 Dysphasia | 0 | 0 | 0 | 0 | 1 | 0 | 0 | 0 |
| Grade 3 Encephalopathy | 0 | 0 | 0 | 0 | 1 | 0 | 0 | 0 |
| Grade 3 Headache | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 0 |
| Grade 3 Nervous system disorder, other (ICANS) | 0 | 0 | 0 | 0 | 0 | 2 | 0 | 0 |
| Grade 3 Nervous system disorder, other (Neurotoxicity) | 0 | 0 | 0 | 0 | 1 | 0 | 0 | 0 |
| Grade 3 Seizure | 0 | 0 | 0 | 0 | 0 | 2 | 0 | 0 |
| Grade 3 Hypoxia | 0 | 0 | 0 | 0 | 0 | 1 | 0 | 0 |
| Grade 3 Hypertension | 0 | 0 | 0 | 0 | 1 | 0 | 0 | 0 |
| Grade 3 Hypotension | 0 | 2 | 0 | 1 | 0 | 6 | 0 | 0 |
| Grade 4 Anemia | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 0 |
| Grade 4 Fibrinogen decreased | 0 | 0 | 0 | 0 | 0 | 1 | 0 | 0 |
| Grade 4 Lymphocyte count decreased | 3 | 3 | 4 | 2 | 1 | 17 | 1 | 1 |
| Grade 4 Neutrophil count decreased | 3 | 1 | 2 | 2 | 0 | 14 | 1 | 0 |
| Grade 4 Platelet count decreased | 2 | 1 | 0 | 0 | 1 | 6 | 1 | 0 |
| Grade 4 White blood cell decreased | 3 | 2 | 0 | 1 | 0 | 10 | 1 | 0 |
| Grade 4 Hypertriglyceridemia | 0 | 0 | 0 | 0 | 0 | 1 | 0 | 0 |
| Grade 4 Hypokalemia | 0 | 0 | 0 | 0 | 1 | 0 | 0 | 0 |
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.
| 10^6 CAR T cells/kg | All Participants |
|---|---|
| Maximum Tolerated Dose (MTD) | 3 |
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.
| Participants | GROUP 1: Cohort 1, Dose Level 1, 3 x10^5 Transduced T Cells/kg (+Or-20%) | GROUP 2: Cohort 1, Dose Level 2, 1x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 3: Cohort 1, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 4: Cohort 1A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 5: Cohort 1B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 6: Cohort 2A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 7: Cohort 2B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20% | GROUP 8: Cohort 2C, Dose Level 3, 3 x 10^6 Transduced T-cells/kg (+Or- 20%) |
|---|---|---|---|---|---|---|---|---|
| Number of Participants That Have Successful Manufacture of the Targeted Dose Number of Chimeric Antigen Receptor (CAR) Cells | 4 | 4 | 5 | 2 | 1 | 21 | 1 | 1 |
Overall survival (OS) will be determined as the time from the date of chimeric antigen receptor (CAR) infusion until death.
| Months | GROUP 1: Cohort 1, Dose Level 1, 3 x10^5 Transduced T Cells/kg (+Or-20%) | GROUP 2: Cohort 1, Dose Level 2, 1x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 3: Cohort 1, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 4: Cohort 1A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 5: Cohort 1B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 6: Cohort 2A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 7: Cohort 2B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20% | GROUP 8: Cohort 2C, Dose Level 3, 3 x 10^6 Transduced T-cells/kg (+Or- 20%) |
|---|---|---|---|---|---|---|---|---|
| Overall Survival | 12.2 (8.9 to 27.8) | 17.8 (10.9 to 68.5) | 45.4 (13 to 66.6) | 15.3 (10.1 to 20.5) | 60 (60 to NA) | 20.7 (3 to 50.9) | 9.2 (9.2 to NA) | 8.3 (8.3 to NA) |
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.
| Months | GROUP 1: Cohort 1, Dose Level 1, 3 x10^5 Transduced T Cells/kg (+Or-20%) | GROUP 2: Cohort 1, Dose Level 2, 1x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 3: Cohort 1, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 4: Cohort 1A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 5: Cohort 1B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 6: Cohort 2A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 7: Cohort 2B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20% | GROUP 8: Cohort 2C, Dose Level 3, 3 x 10^6 Transduced T-cells/kg (+Or- 20%) |
|---|---|---|---|---|---|---|---|---|
| Progression-free Survival (PFS) | 6.4 (0.9 to 11.9) | 2.6 (1.0 to 8.6) | 20.1 (0.9 to 66.6) | 5.3 (2.4 to 8.1) | 60 (60 to NA) | 14.2 (0.9 to 50.9) | 5.7 (5.7 to NA) | 8.3 (8.3 to NA) |
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.
| percentage of participants | GROUP 4:Cohort 1A, CAR Naïve or CAR Pretreated With Interval Hematopoietic Stem Cell Transplantation | GROUP 5: Cohort 1B, Previously Received Chimeric Antigen Receptor (CAR) Participants | GROUP 6:Cohort 2A, CAR Naïve or CAR Pretreated With Interval Hematopoietic Stem Cell Transplantation | GROUP 7: Cohort 2B, Previously Received Chimeric Antigen Receptor (CAR) | Group 8:Cohort 2C, CAR Naïve or CAR Pretreated With Previous Hematopoietic Stem Cell Transplantation |
|---|---|---|---|---|---|
| CR in children and young adults | 50 (2.6 to 97.4) | 100 (5.1 to 100) | 90.5 (71.1 to 98.3) | 100 (5.1 to 100) | 100 (5.1 to 100) |
| PR in children and young adults | 50 (2.6 to 97.4) | 0 (0 to 94.9) | 0 (0 to 15.5) | 0 (0 to 94.9) | 0 (0 to 94.9) |
| SD in children and young adults | 0 (0 to 82.2) | 0 (0 to 94.9) | 0 (0 to 15.5) | 0 (0 to 94.9) | 0 (0 to 94.9) |
| PD in children and young adults | 0 (0 to 82.2) | 0 (0 to 94.9) | 9.5 (1.7 to 28.9) | 0 (0 to 94.9) | 0 (0 to 94.9) |
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.
| Participants | GROUP 1: Cohort 1, Dose Level 1, 3 x10^5 Transduced T Cells/kg (+Or-20%) | GROUP 2: Cohort 1, Dose Level 2, 1x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 3: Cohort 1, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 4: Cohort 1A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 5: Cohort 1B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 6: Cohort 2A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 7: Cohort 2B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20% | GROUP 8: Cohort 2C, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or- 20%) |
|---|---|---|---|---|---|---|---|---|
| Number of Participants With a Dose-limiting Toxicity (DLT) | 0 | 0 | 0 | 0 | 1 | 2 | 0 | 0 |
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.
| Participants | GROUP 1: Cohort 1, Dose Level 1, 3 x10^5 Transduced T Cells/kg (+Or-20%) | GROUP 2: Cohort 1, Dose Level 2, 1x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 3: Cohort 1, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 4: Cohort 1A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 5: Cohort 1B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 6: Cohort 2A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 7: Cohort 2B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20% | Group 8:Cohort 2C, CAR Naïve or CAR Pretreated With Previous Hematopoietic Stem Cell Transplantation |
|---|---|---|---|---|---|---|---|---|
| Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). | 4 | 4 | 4 | 2 | 1 | 21 | 1 | 1 |
Collected over All-Cause Mortality was monitored/assessed an average of 35 days. Adverse Events were monitored/assessed from the first study intervention (Study Day -4, through day 30 after the study agent (s) was/were administered) in any cycle, an average of 5 weeks.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| GROUP 1: Cohort 1, Dose Level 1, 3 x10^5 Transduced T Cells/kg (+Or-20%) | 4/4 (100%) | 2/4 (50%) | 4/4 (100%) |
| GROUP 2: Cohort 1, Dose Level 2, 1x10^6 Transduced T Cells/kg (+Or-20%) | 3/4 (75%) | 2/4 (50%) | 4/4 (100%) |
| GROUP 3: Cohort 1, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | 2/4 (50%) | 0/4 (0%) | 4/4 (100%) |
| GROUP 4: Cohort 1A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | 2/2 (100%) | 2/2 (100%) | 2/2 (100%) |
| GROUP 5: Cohort 1B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | 0/1 (0%) | 1/1 (100%) | 1/1 (100%) |
| GROUP 6: Cohort 2A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | 8/21 (38.1%) | 7/21 (33.3%) | 21/21 (100%) |
| GROUP 7: Cohort 2B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20% | 1/1 (100%) | 0/1 (0%) | 1/1 (100%) |
| Group 8:Cohort 2C, CAR Naïve or CAR Pretreated With Previous Hematopoietic Stem Cell Transplantation | 0/1 (0%) | 1/1 (100%) | 1/1 (100%) |
| Event | GROUP 1: Cohort 1, Dose Level 1, 3 x10^5 Transduced T Cells/kg (+Or-20%) | GROUP 2: Cohort 1, Dose Level 2, 1x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 3: Cohort 1, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 4: Cohort 1A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 5: Cohort 1B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 6: Cohort 2A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 7: Cohort 2B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20% | Group 8:Cohort 2C, CAR Naïve or CAR Pretreated With Previous Hematopoietic Stem Cell Transplantation |
|---|---|---|---|---|---|---|---|---|
| Nervous system disorders - Other, (Neurotoxicity)Nervous system disorders | 0/4 | 0/4 | 0/4 | 0/2 | 1/1 | 0/21 | 0/1 | 0/1 |
| RhabdomyolysisMusculoskeletal and connective tissue disorders | 0/4 | 0/4 | 0/4 | 0/2 | 0/1 | 0/21 | 0/1 | 1/1 |
| Cytokine release syndromeImmune system disorders | 1/4 | 2/4 | 0/4 | 1/2 | 0/1 | 1/21 | 0/1 | 0/1 |
| Febrile neutropeniaBlood and lymphatic system disorders | 0/4 | 1/4 | 0/4 | 1/2 | 0/1 | 0/21 | 0/1 | 0/1 |
| HypotensionVascular disorders | 0/4 | 0/4 | 0/4 | 1/2 | 0/1 | 1/21 | 0/1 | 0/1 |
| Immune system disorders - Other, GVHDImmune system disorders | 0/4 | 0/4 | 0/4 | 1/2 | 0/1 | 0/21 | 0/1 | 0/1 |
| FeverGeneral disorders | 0/4 | 1/4 | 0/4 | 0/2 | 0/1 | 3/21 | 0/1 | 0/1 |
| Flu like symptomsGeneral disorders | 1/4 | 0/4 | 0/4 | 0/2 | 0/1 | 0/21 | 0/1 | 0/1 |
| HypertensionVascular disorders | 1/4 | 0/4 | 0/4 | 0/2 | 0/1 | 0/21 | 0/1 | 0/1 |
| Upper respiratory infectionInfections and infestations | 1/4 | 0/4 | 0/4 | 0/2 | 0/1 | 0/21 | 0/1 | 0/1 |
| Event | GROUP 1: Cohort 1, Dose Level 1, 3 x10^5 Transduced T Cells/kg (+Or-20%) | GROUP 2: Cohort 1, Dose Level 2, 1x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 3: Cohort 1, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 4: Cohort 1A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 5: Cohort 1B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 6: Cohort 2A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 7: Cohort 2B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20% | Group 8:Cohort 2C, CAR Naïve or CAR Pretreated With Previous Hematopoietic Stem Cell Transplantation |
|---|---|---|---|---|---|---|---|---|
| Abdominal painGastrointestinal disorders | 2/4 | 2/4 | 2/4 | 2/2 | 1/1 | 10/21 | 0/1 | 0/1 |
| Activated partial thromboplastin time prolongedInvestigations | 1/4 | 1/4 | 2/4 | 1/2 | 1/1 | 9/21 | 1/1 | 0/1 |
| AgitationPsychiatric disorders | 0/4 | 0/4 | 0/4 | 0/2 | 1/1 | 0/21 | 1/1 | 0/1 |
| Alanine aminotransferase increasedInvestigations | 2/4 | 3/4 | 2/4 | 2/2 | 1/1 | 9/21 | 0/1 | 1/1 |
| Alkaline phosphatase increasedInvestigations | 1/4 | 2/4 | 2/4 | 0/2 | 0/1 | 5/21 | 1/1 | 0/1 |
| AnemiaBlood and lymphatic system disorders | 4/4 | 4/4 | 4/4 | 2/2 | 1/1 | 19/21 | 1/1 | 1/1 |
| AnorexiaMetabolism and nutrition disorders | 3/4 | 2/4 | 3/4 | 2/2 | 0/1 | 13/21 | 1/1 | 0/1 |
| AnxietyPsychiatric disorders | 1/4 | 2/4 | 2/4 | 2/2 | 1/1 | 10/21 | 0/1 | 1/1 |
| Aspartate aminotransferase increasedInvestigations | 3/4 | 2/4 | 0/4 | 2/2 | 1/1 | 13/21 | 1/1 | 1/1 |
| Back painMusculoskeletal and connective tissue disorders | 0/4 | 1/4 | 3/4 | 1/2 | 1/1 | 13/21 | 0/1 | 0/1 |
Data collected for participants enrolled and not treated are reported here.
| Age, Categorical(Participants) | GROUP 1: Cohort 1, Dose Level 1, 3 x10^5 Transduced T Cells/kg (+Or-20%) | GROUP 2: Cohort 1, Dose Level 2, 1x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 3: Cohort 1, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 4: Cohort 1A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 5: Cohort 1B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 6: Cohort 2A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 7: Cohort 2B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20% | GROUP 8: Cohort 2C, Dose Level 3, 3 x 10^6 Transduced T-cells/kg (+Or- 20%) | Enrolled and Not Assigned to a Treatment Assignment Code; Not Treated | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| <=18 years | 1 | 0 | 2 | 0 | 1 | 8 | 1 | 0 | 5 | 18 |
| Between 18 and 65 years | 3 | 4 | 3 | 2 | 0 | 13 | 0 | 1 | 10 | 36 |
| >=65 years | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Age, Continuous(years) | GROUP 1: Cohort 1, Dose Level 1, 3 x10^5 Transduced T Cells/kg (+Or-20%) | GROUP 2: Cohort 1, Dose Level 2, 1x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 3: Cohort 1, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 4: Cohort 1A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 5: Cohort 1B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 6: Cohort 2A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 7: Cohort 2B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20% | GROUP 8: Cohort 2C, Dose Level 3, 3 x 10^6 Transduced T-cells/kg (+Or- 20%) | Enrolled and Not Assigned to a Treatment Assignment Code; Not Treated | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| Mean | 15.88 ± 6.06 | 24.75 ± 3.97 | 19.12 ± 8.73 | 25.75 ± 3.45 | 9.7 ± 0 | 21 ± 10.86 | 18.2 ± 0 | 22.6 ± 0 | 21.75 ± 10.91 | 20.69 ± 10.09 |
| Sex: Female, Male(Participants) | GROUP 1: Cohort 1, Dose Level 1, 3 x10^5 Transduced T Cells/kg (+Or-20%) | GROUP 2: Cohort 1, Dose Level 2, 1x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 3: Cohort 1, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 4: Cohort 1A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 5: Cohort 1B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 6: Cohort 2A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 7: Cohort 2B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20% | GROUP 8: Cohort 2C, Dose Level 3, 3 x 10^6 Transduced T-cells/kg (+Or- 20%) | Enrolled and Not Assigned to a Treatment Assignment Code; Not Treated | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| Female | 1 | 0 | 2 | 1 | 1 | 9 | 1 | 0 | 6 | 21 |
| Male | 3 | 4 | 3 | 1 | 0 | 12 | 0 | 1 | 9 | 33 |
| Ethnicity (NIH/OMB)(Participants) | GROUP 1: Cohort 1, Dose Level 1, 3 x10^5 Transduced T Cells/kg (+Or-20%) | GROUP 2: Cohort 1, Dose Level 2, 1x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 3: Cohort 1, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 4: Cohort 1A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 5: Cohort 1B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 6: Cohort 2A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 7: Cohort 2B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20% | GROUP 8: Cohort 2C, Dose Level 3, 3 x 10^6 Transduced T-cells/kg (+Or- 20%) | Enrolled and Not Assigned to a Treatment Assignment Code; Not Treated | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| Hispanic or Latino | 1 | 2 | 2 | 0 | 0 | 12 | 0 | 1 | 6 | 24 |
| Not Hispanic or Latino | 3 | 2 | 3 | 2 | 1 | 9 | 1 | 0 | 7 | 28 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 2 | 2 |
| Race (NIH/OMB)(Participants) | GROUP 1: Cohort 1, Dose Level 1, 3 x10^5 Transduced T Cells/kg (+Or-20%) | GROUP 2: Cohort 1, Dose Level 2, 1x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 3: Cohort 1, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 4: Cohort 1A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 5: Cohort 1B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 6: Cohort 2A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 7: Cohort 2B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20% | GROUP 8: Cohort 2C, Dose Level 3, 3 x 10^6 Transduced T-cells/kg (+Or- 20%) | Enrolled and Not Assigned to a Treatment Assignment Code; Not Treated | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 1 |
| Asian | 1 | 0 | 0 | 0 | 0 | 3 | 0 | 0 | 1 | 5 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 1 | 1 | 0 | 1 | 0 | 0 | 1 | 4 |
| White | 2 | 2 | 3 | 1 | 1 | 11 | 1 | 1 | 9 | 31 |
| More than one race | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 1 | 2 | 1 | 0 | 0 | 6 | 0 | 0 | 3 | 13 |
| Region of Enrollment(participants) | GROUP 1: Cohort 1, Dose Level 1, 3 x10^5 Transduced T Cells/kg (+Or-20%) | GROUP 2: Cohort 1, Dose Level 2, 1x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 3: Cohort 1, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 4: Cohort 1A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 5: Cohort 1B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 6: Cohort 2A, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20%) | GROUP 7: Cohort 2B, Dose Level 3, 3 x10^6 Transduced T Cells/kg (+Or-20% | GROUP 8: Cohort 2C, Dose Level 3, 3 x 10^6 Transduced T-cells/kg (+Or- 20%) | Enrolled and Not Assigned to a Treatment Assignment Code; Not Treated | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| United States | 4 | 4 | 5 | 2 | 1 | 21 | 1 | 1 | 15 | 54 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — All individual participant data (IPD) recorded in the medical record will be shared with intramural investigators upon request.
Supporting information: Study protocol, Statistical analysis plan, Informed consent form
Time frame: Clinical data available during the study and indefinitely.
Access criteria: Clinical data will be made available via subscription to Biomedical Translational Research Information System (BTRIS) and with the permission of the study principal investigator (PI).
Ran from Mar 2018 to primary completion in Jul 2024, 75 months.
Results were first posted in Jun 2025, 10 months after primary completion.
Of 117 non-industry phase 1 interventional studies under Lymphoma, B-Cell, half reached primary completion within 54 months of starting (the median), counting those still running; a quarter within 37 and three quarters within 75. This study's 75 months is above that.
Counting every sponsor, half of 207 studies reached it within 51 months; a quarter within 37 and three quarters within 78.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in May 2025. You cannot join it, but the record below documents what was studied.
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