In short
Clinical trials are studying AUTOLOGOUS T CELLS TRANSDUCED WITH A RETROVIRAL VECTOR ENCODING A CHIMERIC ANTIGEN RECEPTOR AGAINST GD2, EX-VIVO EXPANDED in children and young adults with hard-to-treat cancers. These studies mainly aim to check safety, find the best dose, and look for signs of benefit in relapsed or refractory central nervous system tumors and GD2-positive neuroblastoma or other GD2-positive tumors.
Key points
- Clinical trials are studying AUTOLOGOUS T CELLS TRANSDUCED WITH A RETROVIRAL VECTOR ENCODING A CHIMERIC ANTIGEN RECEPTOR AGAINST GD2, EX-VIVO EXPANDED in children and young adults with hard-to-treat cancers. One trial is Phase 1 in relapsed or refractory malignant central nervous system tumors, and another is Phase 1/2 in high-risk and/or relapsed or refractory neuroblastoma and other GD2-positive tumors. The main goals are to assess safety, find dose-limiting toxicity, and determine the maximum tolerated or recommended dose. The Phase 1/2 study also plans to test efficacy at the optimal dose. Both trials are authorised and are focused on patients whose cancer has come back or not responded well to prior treatment.
Trial overview
These studies are testing AUTOLOGOUS T CELLS TRANSDUCED WITH A RETROVIRAL VECTOR ENCODING A CHIMERIC ANTIGEN RECEPTOR AGAINST GD2, EX-VIVO EXPANDED in children and young adults with cancers that are hard to treat.12
The two trials are both interventional studies, which means the research team gives a treatment and then measures what happens.12
Both trials are currently Authorised.12
Who is being studied
One trial includes pediatric and young adult patients with relapsed/refractory malignant central nervous system tumors.1
The other trial includes pediatric patients with high-risk and/or relapsed/refractory neuroblastoma and other GD2-positive tumors.2
In simple terms, these are patients whose cancer has either come back after treatment or has not responded well to treatment.12
Trial phases and design
The first study is a Phase 1 trial with 27 planned participants.1
The second study is a Phase 1/2 trial with 54 planned participants.2
Phase 1 studies mainly look at safety and dose, while Phase 1/2 studies begin with safety and dose finding and then also look more closely at whether the treatment may help.12
In the second study, the Phase II part is aimed at testing efficacy at the optimal dose found in Phase I, including patients with extremely high-risk neuroblastoma.2
Main endpoints
The main goal in both studies is to evaluate safety of the infusion and to find the dose-limiting toxicity (DLT), which means side effects serious enough to limit treatment.12
The first trial also aims to establish the maximum tolerated dose (MTD) and the recommended dose (RD) of the cellular product.1
The second trial has the same early safety goals and then moves on to test efficacy at the dose chosen from Phase I.2
Safety is measured with toxicity grading systems, including the Common Terminology Criteria for Adverse Events, and the first trial specifically mentions both systemic and neurological toxicity.1
Trial summary
- The first study tests patients with relapsed or refractory malignant central nervous system tumors and focuses on safety, DLT, MTD, and RD.1
- The second study tests pediatric patients with high-risk and/or relapsed or refractory neuroblastoma and other GD2-positive tumors, with safety first and efficacy later in the study.2
- Both studies use intravenous treatment and are designed to learn which dose is best tolerated.12
- Both studies are early-phase trials, so they are not yet focused on proving long-term benefit.12
Key terms explained
Intravenous use means the treatment is given through a vein.12
Escalating/de-escalating doses means the study may increase or decrease the dose to find the safest level.12
CTC AE stands for Common Terminology Criteria for Adverse Events, a standard way to grade side effects in trials.12
Neurological toxicity means harmful effects on the brain, nerves, or nervous system.1
Extremely high-risk neuroblastoma refers to a form of neuroblastoma with a greater chance of coming back after treatment.2