SuspendedPhase 1

Testing a New Immune Cell Therapy, GD2-Targeted Modified T-cells (GD2CART), in Children, Adolescents, and Young Adults With Relapsed/Refractory Osteosarcoma and Neuroblastoma, The GD2-CAR PERSIST Trial

Stopped: Other - review of safety data

United States67 participantsStarted 2022-01-25

Plain-language summary

This phase I trial investigates the side effects and determines the best dose of an immune cell therapy called GD2CART, as well as how well it works in treating patients with osteosarcoma or neuroblastoma that has come back (relapsed) or does not respond to treatment (refractory). T cells are infection fighting blood cells that can kill tumor cells. The T cells given in this trial will come from the patient and will have a new gene put in them that makes them able to recognize GD2, a protein on the surface of tumor cells. These GD2-specific T cells may help the body's immune system identify and kill GD2 positive tumor cells.

Who can participate

Age range

40 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.

Inclusion Criteria: * Must have histologically confirmed neuroblastoma or osteosarcoma that is recurrent or refractory and for which standard curative measures do not exist or are no longer effective. Must have histologic verification of their disease at diagnosis or at relapse * Patients with osteosarcoma in the dose escalation cohort, must have evaluable or measurable disease at enrollment * Patients with osteosarcoma in the expansion cohort must have measurable disease by Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) at enrollment * Patients with neuroblastoma in the dose escalation or dose the expansion cohort must have: * Prior progressive disease OR refractory disease present since diagnosis AND at least one of the following: * Evidence of tumor in the bone marrow (BM) * At least one metaiodobenzylguanidine (MIBG)-avid soft tissue or skeletal site * For MIBG-nonavid disease, at least one FDG-PET- positive soft tissue or skeletal site plus past histologic confirmation * Progressive disease is defined as any disease progression occurring at any time after the diagnosis of high-risk neuroblastoma. Refractory disease is defined as an incomplete response of high-risk neuroblastoma to all treatments but without disease progression * Must be \< 40 years of age * There is no limit to the number of prior treatment regimens. The following washout periods prior to leukapheresis apply to patients undergoing leukapheresis on this study. If…

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

Feasibility of producing GD2-CAR-expressing autologous T-lymphocytes (GD2CART) cells

Timeframe: Up to day 28 days after cell infusion

Incidence of adverse events (AEs)

Timeframe: Up to 15 years

Maximum tolerated dose

Timeframe: Up to day 28 days after cell infusion

Best response to GD2CART cells

Timeframe: Up to day 28 days after cell infusion

Trial details

NCT IDNCT04539366

SponsorNational Cancer Institute (NCI)

Sponsor typeNIH

Study typeINTERVENTIONAL

Primary completion2040-12-31

View on ClinicalTrials.gov More Recurrent Childhood Neuroblastoma trials

Plain-language summary

Who can participate

Age range

40 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.

What they're measuring

Feasibility of producing GD2-CAR-expressing autologous T-lymphocytes (GD2CART) cells

Timeframe: Up to day 28 days after cell infusion

Incidence of adverse events (AEs)

Timeframe: Up to 15 years

Maximum tolerated dose

Timeframe: Up to day 28 days after cell infusion

Best response to GD2CART cells

Timeframe: Up to day 28 days after cell infusion