Table of Contents
- Trial overview
- Who is being studied
- Treatment plan in the trial
- Trial phase and design
- What the trial measures
- What this may mean for patients
Trial overview
This article covers one authorised interventional study of ALLOGENEIC PERIPHERAL BLOOD-DERIVED HAEMATOPOIETIC STEM CELLS, TCR ALPHA/BETA+ CD19+ DEPLETED in high-risk neuroblastoma.[1] The trial is testing a treatment approach for patients whose disease has relapsed or is progressing after first-line treatment.[1]
Who is being studied
The study population includes patients affected by high-risk neuroblastoma that has relapsed or progressed after initial treatment.[1] Neuroblastoma is a cancer that starts in nerve tissue, and high-risk disease means it is more serious and harder to treat.
Treatment plan in the trial
The trial evaluates haploidentical stem cell transplantation followed by immunotherapy.[1] Haploidentical means the donor is a partial match, not a full match.
The brief summary says the study uses transplantation with alfa-beta-CD19+ depletion, then donor NK cells infusion and anti-GD2 monoclonal antibody treatment.[1] The intervention list also includes several medicines given by vein as part of the study plan, including Rixathon, Aplo-TCSE TCRaß+CD19+ depleto, allogeneic peripheral blood-derived NK cells CD3- CD56+, Qarziba, Melfalan Tillomed, TEPADINA, Fludarabina Accord, and Grafalon.[1]
Trial phase and design
This is a Phase 2 trial.[1] Phase 2 studies are usually done to see whether a treatment appears to work and to keep tracking safety in a smaller group of patients.
The planned enrollment is 19 participants.[1] The trial status is Authorised.[1]
What the trial measures
The primary outcome is Overall Survival (OS).[1] Overall survival means how long patients live after starting the study treatment.
The brief summary states that the main purpose is to evaluate the safety and efficacy of the treatment plan.[1] Efficacy means how well the treatment works against the disease.
What this may mean for patients
For patients and families, this trial is looking at a complex treatment plan for a very serious cancer that came back or got worse after standard treatment.[1] The study is not about routine care; it is designed to learn whether this transplant-plus-immunotherapy approach can help this group of patients.
The source data do not provide full details about all eligibility rules, visit schedule, or secondary outcomes.[1] They do show that the study is focused on a narrow patient group and on survival as the main result.
