Table of contents
- Trial overview
- Who is being studied
- Trial phases and design
- Main endpoints
- Trial summary
- Key terms explained
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.[1][2]
The two trials are both interventional studies, which means the research team gives a treatment and then measures what happens.[1][2]
Both trials are currently Authorised.[1][2]
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.[1][2]
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.[1][2]
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.[1][2]
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.[1][2]
- Both studies are early-phase trials, so they are not yet focused on proving long-term benefit.[1][2]
Key terms explained
Intravenous use means the treatment is given through a vein.[1][2]
Escalating/de-escalating doses means the study may increase or decrease the dose to find the safest level.[1][2]
CTC AE stands for Common Terminology Criteria for Adverse Events, a standard way to grade side effects in trials.[1][2]
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]
