A Phase 1 interventional study of B7-H3.CD28Z.CART and Fludarabine in Central Nervous System Neoplasms, Brain Tumor and Brain Tumor, Recurrent, sponsored by Robbie Majzner. Recruiting at 2 sites in United States. Open to participants aged 2 Years to 21 Years. Per ClinicalTrials.gov, last updated 2026-09-03.
Sponsored by Robbie Majzner · Phase 1, Interventional, and Treatment
The purpose of this research study is to test the safety and effectiveness of a cell therapy at different doses for children and young adults with recurrent or progressive brain tumors. Recurrent/recurred means a tumor that has gone away and then came back. This cell therapy is called B7- H3.CD28Z.CART, referred to as B7-H3 CAR T cells. B7-H3 is a protein that is over-expressed on many tumor cells, making it a good target for cancer cell therapy.
The names of the study investigational therapies involved in this study are:
This is a single-institution, Phase 1/1b, open-label study to test the safety and effectiveness of a cell therapy at different doses for children and young adults with recurrent or progressive brain tumors. Recurrent/recurred means a tumor that has gone away and then came back. This cell therapy is called B7- H3.CD28Z.CART, referred to as B7-H3 CAR T cells. B7-H3 is a protein that is over-expressed on many tumor cells, making it a good target for cancer cell therapy.
The U.S. Food and Drug Administration (FDA) has not approved B7-H3 CAR T cells as a treatment for any disease.
Fludarabine and cyclophosphamide are standard lymphodepleting chemotherapy medications that are being used in this study to prepare the body for cell therapy. They are approved by the U.S. Food \& Drug Administration (FDA) for this purpose and are not intended to be treatment for recurrent or progressive brain tumors.
The structure of the study will be by dose-escalation using a modified 3+3 design in two risk strata (standard risk, high risk), followed by a two-stage Phase 1b expansion at the recommended Phase 2 dose.
Participation in this study is expected to last up to 15 years.
It is expected that about 70 people will participate in this study.
66 studies on the registry are indexed under Pinealoma; 11 are open to participants now.
This study's planned enrollment of 70 is above the median of 26 across 43 interventional studies indexed under Pinealoma.
Browse Pinealoma studies →Robbie Majzner is the lead sponsor of 2 studies on the registry; 2 are open to participants now.
Compared within Pinealoma, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Central Nervous System Neoplasms. 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.
Pre-screening Inclusion Criteria:
Participants must have histologically and/or molecularly confirmed CNS embryonal tumor (see eligible tumor types below), OR ependymoma that is recurrent/progressive following standard of care treatment.
--Eligible CNS embryonal tumor types include:
Inclusion Criteria:
Participants must have histologically and/or molecularly confirmed CNS embryonal tumor (see eligible tumor types below), OR ependymoma that is recurrent/progressive following standard of care treatment.
--Eligible CNS embryonal tumor types include:
Disease status: Participants must have evaluable disease in the central nervous system to be eligible. Evaluable disease includes either measurable OR non-, defined as follows:
--Measurable disease (contrast-enhancing or non-enhancing tumor)
At least two times (in both perpendicular diameters) the MRI slice thickness, plus the interslice gap
--Non-measurable disease (tumor that is too small to be accurately measured)
Participants must meet the following washouts prior to enrollment:
Radiation therapy - Participants must have had their last fraction of:
---Craniospinal irradiation, whole brain radiation therapy, or radiation therapy to >50% of the pelvis or spine >28 days prior to enrollment
---Focal irradiation (small port) >14 days prior to enrollment
discretion.
Participants must have adequate organ function, as defined below
--Adequate bone marrow function
Adequate renal function defined as creatinine within normal limits for age OR (as estimated by Cockcroft Gault Equation for participants ≥ 18yo and Bedside Schwartz for participants \<18yo) ≥70mL/min
Maximum Serum Creatinine mg/DL ---6 months to 1 year Male 0.5 Female 0.5 ---1 to \< 2 years Male 0.6 Female 0.6 ---2 to \< 6 years Male 0.8 Female 0.8
13 years to \< 16 years Male 1.5 Female 1.4
Adequate hepatic function
Adequate cardiac function
-- ≥50% or fractional shortening ≥28%, measured by echocardiography
Adequate pulmonary function
Adequate neurologic function
Exclusion Criteria:
Participants with bulky tumor are ineligible. Bulky tumor is defined as:
Concurrent illness
Concomitant medications
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.
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.
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.
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
Cancer that has spread to the leptomeninges, the thin layers covering the brain and spinal cord, and the fluid between them. It is not the same as brain metastases.
Criteria often treat it separately, and many studies leave it out even where treated brain metastases are allowed.
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.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
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.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
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 protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
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.
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.
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.
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.
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 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.
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
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.
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 condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
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.
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.
Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.
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.
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.
Measurable disease
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Leptomeningeal disease
Cancer that has spread to the leptomeninges, the thin layers covering the brain and spinal cord, and the fluid between them. It is not the same as brain metastases.
Criteria often treat it separately, and many studies leave it out even where treated brain metastases are allowed.
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.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
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.
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.
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.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (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.
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.
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.
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.
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.
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.
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.
CTCAE grade
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
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.
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.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
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.
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.
Live vaccines
Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will be enrolled in a staggered, sequential fashion using a modified 3 + 3 dose escalation design, assigning participants to one of three intravenous dose levels to determine the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) of IV B7-H3.CD28Z.CART. Dose escalation will occur per dose-limiting toxicity rules. * Baseline visit * Apheresis to obtain T cells * Days -4, -3, -2: predetermined doses of Fludarabine and Cyclophosphamide 1x daily * Day 0: B7-H3 CAR T cell infusion 1x daily * 2 doses of B7-H3 CAR T cell infusion Intracerebroventricular (ICV) Infusion every 28 days until 8 doses total according to clinical response * Follow up every 3 months after last infusion up until 2 years * Long term follow annually for an additional 13 years
Biological: B7-H3.CD28Z.CART · Drug: Fludarabine · Drug: Cyclophosphamide
Participants will be enrolled in a staggered, sequential fashion using a modified 3 + 3 dose escalation design, assigning participants to one of three intravenous dose levels to determine the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) of IV B7-H3.CD28Z.CART. Dose escalation will occur per dose-limiting toxicity rules. * Baseline visit * Apheresis to obtain T cells * Days -4, -3, -2: predetermined doses of Fludarabine and Cyclophosphamide 1x daily * Day 0: B7-H3 CAR T cell infusion 1x daily * 2 doses of B7-H3 CAR T cell infusion Intracerebroventricular (ICV) Infusion every 28 days until 8 doses total according to clinical response * Follow up every 3 months after last infusion up until 2 years * Long term follow annually for an additional 13 years
Biological: B7-H3.CD28Z.CART · Drug: Fludarabine · Drug: Cyclophosphamide
Dose expansion will occur per dose-limiting toxicity rules. * Baseline visit * Apheresis to obtain T cells * Days -4, -3, -2: predetermined doses of Fludarabine and Cyclophosphamide 1x daily * Day 0: B7-H3 CAR T cell infusion 1x daily * 2 doses of B7-H3 CAR T cell infusion Intracerebroventricular (ICV) Infusion every 28 days until 8 doses total according to clinical response * Follow up every 3 months after last infusion up until 2 years * Long term follow annually for an additional 13 years
Biological: B7-H3.CD28Z.CART · Drug: Fludarabine · Drug: Cyclophosphamide
Dose expansion will occur per dose-limiting toxicity rules. * Baseline visit * Apheresis to obtain T cells * Days -4, -3, -2: predetermined doses of Fludarabine and Cyclophosphamide 1x daily * Day 0: B7-H3 CAR T cell infusion 1x daily * 2 doses of B7-H3 CAR T cell infusion Intracerebroventricular (ICV) Infusion every 28 days until 8 doses total according to clinical response * Follow up every 3 months after last infusion up until 2 years * Long term follow annually for an additional 13 years
Biological: B7-H3.CD28Z.CART · Drug: Fludarabine · Drug: Cyclophosphamide
Autologous CAR T cells targeting B7-H3 (CD276), administered intravenously (into the vein) per protocol.
Lymphodepleting chemotherapy administered intravenously per institutional standards.
Lymphodepleting chemotherapy administered intravenously per institutional standards.
Manufacturing Success Rate of Autologous B7-H3.CD28Z CAR T Cells
Each participant's product will be tested for the following criteria: cell viability ≥ 70%; cell number within ± 20% of the planned dose; CD3+ T cells ≥ 80% of leukocytes; CAR-positive cells ≥ 10% of CD3+ T cells; endotoxin ≤ 5 EU/kg; mycoplasma not detected; vector copy number (VCN) per transduced cell ≤ 10; replication-competent retrovirus (RCR) not detected; and sterility confirmed as "No Growth to Date" (NGTD) after a minimum of 5 days in culture. A participant will be classified as a manufacturing success if the final product satisfies all release criteria. If any criterion is not met, the participant will be classified as a manufacturing failure. The manufacturing success rate is defined as the proportion of participants classified as a success.
Time frame: Participants will receive the CART cell infusion on Day 0.
Maximum Tolerated Dose (MTD) of B7-H3.CD28Z.CART Cells
The MTD is defined as the highest dose level of B7-H3.CD28Z.CART cells at which the rate of dose-limiting toxicity (DLT) is considered acceptable according to the modified 3+3 rules. Additional details are provided in Protocol Section 13.1. Definition of DLT is outlined in protocol. The definition of DLT uses NCI's Common Terminology Criteria for Adverse Events (CTCAEv6.0).
Time frame: 28 days
Recommended Phase 2 Dose (RP2D) of B7-H3.CD28Z.CART Cells
The recommended phase 2 dose (RP2D) is the dose of B7-H3.CD28Z.CAR T cells selected for Phase 2 based on Phase 1 results, considering safety, tolerability, manufacturing feasibility, and observed clinical activity, and may be at or below the Maximum Tolerated Dose (MTD) or Maximum Administered Dose (MAD).
Time frame: 28 days
Number of Participants Experience Dose-Limiting Toxicity (DLT) during Dose Escalation
Definition of DLT is outlined in protocol. The definition of DLT uses NCI's Common Terminology Criteria for Adverse Events (CTCAEv6.0).
Time frame: 28 years
Number of Participants Experience Dose-Limiting Toxicity (DLT) at Maximum Tolerated Dose (MTD) / Recommend Phase 2 Dose (RP2D)
Definition of DLT is outlined in protocol. The definition of DLT uses NCI's Common Terminology Criteria for Adverse Events (CTCAEv6.0).
Time frame: 28 days
Number of Participants with Two Additional ICV B7-H3.CD28Z.CAR T Infusions
The number of participants in the High-Risk Stratum who receive two additional intracerebroventricular (ICV) infusions of B7-H3.CD28Z.CAR T cells following the initial intravenous(IV) infusion.
Time frame: From Day 0 (initial IV infusion) through Day 56 (third ICV infusion)
Adverse Events of Special Interest (AESIs) Rate
AESIs rate is defined as the proportion of participants who experience AESIs during treatment period. AESIs is defined in protocol section 7.3. Adverse events will be summarized and graded according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 6.0.
Time frame: From Day 0 (initial IV infusion) through Day 56 (third ICV infusion) plus a 28-day follow-up period, for a total of 74 days.
Complete Response Rate (CRR) at Day 28
CRR is defined as the proportion of participants who achieve complete response (CR) assessed by Response Assessment in Pediatric Neuro-Oncology (RAPNO) response guidelines for medulloblastoma and ependymoma. Immune-related response criteria (irRC) as defined in RANO 2.0 may be used to interpret responses potentially influenced by immune effects.
Time frame: Day 28
Partial Response Rate (PRR) at Day 28
PRR is defined as the proportion of participants who achieve partial response (PR) assessed by Response Assessment in Pediatric Neuro-Oncology (RAPNO) response guidelines for medulloblastoma and ependymoma. Immune-related response criteria (irRC) as defined in RANO 2.0 may be used to interpret responses potentially influenced by immune effects.
Time frame: Day 28
Stable Disease Rate (SDR) at Day 28
SDR is defined as the proportion of participants who achieve stable disease (SD) assessed by Response Assessment in Pediatric Neuro-Oncology (RAPNO) response guidelines for medulloblastoma and ependymoma. Immune-related response criteria (irRC) as defined in RANO 2.0 may be used to interpret responses potentially influenced by immune effects.
Time frame: Day 28
3 Month Complete Response Rate (CRR3)
CRR3 is defined as the proportion of participants who achieve complete response (CR) at 3 months, which assessed by Response Assessment in Pediatric Neuro-Oncology (RAPNO) response guidelines for medulloblastoma and ependymoma. Immune-related response criteria (irRC) as defined in RANO 2.0 may be used to interpret responses potentially influenced by immune effects.
Time frame: 3 months
3 Month Partial Response Rate (PRR3)
PRR3 is defined as the proportion of participants who achieve partial response (PR) at 3 months, which assessed by Response Assessment in Pediatric Neuro-Oncology (RAPNO) response guidelines for medulloblastoma and ependymoma. Immune-related response criteria (irRC) as defined in RANO 2.0 may be used to interpret responses potentially influenced by immune effects.
Time frame: 3 months
3 Month Stable Disease Rate (SDR3)
SDR3 is defined as the proportion of participants who achieve stable disease (SD) at 3 months, which assessed by Response Assessment in Pediatric Neuro-Oncology (RAPNO) response guidelines for medulloblastoma and ependymoma. Immune-related response criteria (irRC) as defined in RANO 2.0 may be used to interpret responses potentially influenced by immune effects.
Time frame: 3 months
Best Overall Response Rate (BORR)
BORR is defined as the proportion of participants who achieve a best overall response of complete response (CR), partial response (PR), or stable disease (SD) from the start of treatment until progressive disease (PD) or recurrence, taking as reference for progression the smallest measurements recorded since treatment initiation. Per Pediatric Neuro-Oncology (RAPNO) response guidelines, PD is defined as meeting any of the following criteria: a ≥25% increase in the sum of perpendicular diameters of measurable lesions or clear progression of non-measurable disease on MRI (brain/spine), appearance of any new lesion (biopsy confirmation if feasible), conversion of CSF cytology from negative to positive, clinical deterioration not attributable to other causes, or new extra-CNS disease.
Time frame: Treatment duration may be up to 224 days, and disease evaluations will be performed at Day 28 (±4 days), at Months 2 and 3 (±1 week), and every 3 months thereafter through Month 24 (±4 weeks) after the end of treatment.
Median Duration of Response (DOR)
DOR, estimated using the Kaplan-Meier method, is measured from the time when the criteria for complete response (CR) or partial response (PR) are first met until the first documented date of recurrent or progressive disease (PD) or death from any cause, using as reference for progression the smallest measurements recorded since treatment initiation. Participants without events are censored at the date of their last disease evaluation. Per Pediatric Neuro-Oncology (RAPNO) response guidelines, PD is defined as meeting any of the following criteria: a ≥25% increase in the sum of perpendicular diameters of measurable lesions or clear progression of non-measurable disease on MRI (brain/spine), appearance of any new lesion (biopsy confirmation if feasible), conversion of CSF cytology from negative to positive, clinical deterioration not attributable to other causes, or new extra-CNS disease.
Time frame: Treatment duration may be up to 224 days, and disease evaluations will be performed at Day 28 (±4 days), at Months 2 and 3 (±1 week), and every 3 months thereafter through Month 24 (±4 weeks) after the end of treatment.
2 Year Progression-Free Survival (PFS2)
PFS2 is the percent probability estimate at 2 years based on the Kaplan-Meier method. PFS is defined as the time from infusion of B7-H3.CD28Z.CART to the first occurrence of an event (progression, relapse, or death due to any cause). Participants alive without an event are censored at date of last disease evaluation. Per Pediatric Neuro-Oncology (RAPNO) response guidelines, PD is defined as meeting any of the following criteria: a ≥25% increase in the sum of perpendicular diameters of measurable lesions or clear progression of non-measurable disease on MRI (brain/spine), appearance of any new lesion (biopsy confirmation if feasible), conversion of CSF cytology from negative to positive, clinical deterioration not attributable to other causes, or new extra-CNS disease.
Time frame: 2 years
Median Overall Survival (OS)
OS based on Kaplan-Meier method is defined as the time from infusion of B7-H3.CD28Z.CART to death due to any cause, or censored at date last known alive.
Time frame: 15 years
Plan to share: Yes — The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: [contact information for Sponsor Investigator or designee]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
Supporting information: Study protocol, Statistical analysis plan
Time frame: Data can be shared no earlier than 1 year following the date of publication
Access criteria: Contact the Belfer Office for Dana-Farber Innovations (BODFI) at innovation@dfci.harvard.edu
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Start is estimated for Sep 2026; primary completion for Aug 2030, 47 months later.
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Robbie Majzner