Active — Not RecruitingPhase 1

HER2-specific Chimeric Antigen Receptor (CAR) T Cells for Children With Ependymoma

United States, Canada50 participantsStarted 2022-07-27

Plain-language summary

This is a Phase I study to evaluate the safety profile of a type of immune therapy called HER2 CAR T cells (short for HER2 chimeric antigen receptor T cells). In addition to looking for side effects, we will study how well this treatment works against a brain tumor called ependymoma that has come back after treatment (recurrent) or has not responded well to treatment (progressive) in children. The HER2 CAR T cells used in this trial are made from the patient's own blood. A new gene, called the HER2 CAR, will be inserted into patient's T cells to allow them recognize a protein on the tumor called HER2. These HER2-specific CAR T cells may be able to target and kill ependymoma tumors that express HER2. This research is also studying how doable it is to provide this type of CAR T cell treatment to children being treated at different hospitals.

Who can participate

Age range

1 Year – 22 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Exclusion criteria

. Tumor
. Performance Score
. Prior Therapy
. Organ Function
. Peripheral absolute neutrophil count (ANC) \> 1.0 x 109 cells/L
. Platelet count ≥ 75 100 x 109 cells/L (unsupported, defined as no platelet transfusion within 4 days)
. Hemoglobin ≥ 8 g/dL (may receive red blood cell transfusions)
. Total bilirubin ≤ 1.5 x institutional upper limit of normal (ULN) for age

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Number of Subjects with Dose-Limiting Toxicity (DLT) in Phase I Arm

Timeframe: Up to 42 days following the first CAR T cell infusion

Number of Subjects with Dose-Limiting Toxicity (DLT) in Surgical Arm

Timeframe: Up to 42 days following the first CAR T cell infusion

Percentage of Subjects whose Treatment Delivery Meets Feasibility Criteria

Timeframe: Approximately 3 months after enrollment for treatment

Trial details

NCT IDNCT04903080

SponsorChildren's Oncology Group

Sponsor typeNETWORK

Study typeINTERVENTIONAL

Primary completion2033-03-31

View on ClinicalTrials.gov More Ependymoma trials

Plain-language summary

Who can participate

Age range

1 Year – 22 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Exclusion criteria

. Tumor
. Performance Score
. Prior Therapy
. Organ Function
. Peripheral absolute neutrophil count (ANC) \> 1.0 x 109 cells/L
. Platelet count ≥ 75 100 x 109 cells/L (unsupported, defined as no platelet transfusion within 4 days)
. Hemoglobin ≥ 8 g/dL (may receive red blood cell transfusions)
. Total bilirubin ≤ 1.5 x institutional upper limit of normal (ULN) for age

What they're measuring

Number of Subjects with Dose-Limiting Toxicity (DLT) in Phase I Arm

Timeframe: Up to 42 days following the first CAR T cell infusion

Number of Subjects with Dose-Limiting Toxicity (DLT) in Surgical Arm

Timeframe: Up to 42 days following the first CAR T cell infusion

Percentage of Subjects whose Treatment Delivery Meets Feasibility Criteria

Timeframe: Approximately 3 months after enrollment for treatment