Table of Contents
- Clinical trials overview
- Pediatric solid tumors study
- Mastocytosis studies
- Trial phases and endpoints
- Who participated
- Study status
Clinical trials overview
These studies investigate Avapritinib in different groups of patients with cancer-related conditions and mastocytosis.[1][2][3]
All three trials are interventional studies, which means researchers gave a study treatment and measured what happened next.[1][2][3]
Pediatric solid tumors study
NCT04773782 studied pediatric patients with solid tumors dependent on KIT or PDGFRA signaling.[1] This was a Phase 1 trial with 37 enrolled patients and it is completed.[1]
The study had two main parts.[1] Part 1 looked for the recommended dose for Part 2 and checked safety and tolerability, which means how well patients could handle the treatment.[1] Part 2 looked at anti-tumor activity, which means whether the tumors responded to treatment.[1]
The main endpoints were dose-limiting toxicity, adverse events, and objective response rate.[1] The tumor response was measured with RECIST version 1.1 or RANO, depending on the tumor type.[1]
Mastocytosis studies
NCT03731260 studied patients with indolent systemic mastocytosis (ISM), a form of systemic mastocytosis that was listed in the trial data as the condition studied.[2] This was a Phase 2 study with 251 enrolled patients and an authorised status.[2]
The trial had three parts.[2] Part 1 aimed to determine the RP2D, which means the recommended Phase 2 dose.[2] Part 2 compared the mean change in ISM-SAF TSS from baseline to C7D1 against placebo, so it measured symptom changes over time.[2] Part 3 assessed the long-term safety and efficacy of Avapritinib.[2]
The rollover study 2024-514969-18-00 included adults who had taken part in a previous Avapritinib study and studied systemic mastocytosis.[3] This was a Phase 3 interventional study with 60 enrolled patients and an authorised status.[3]
Its main objective was long-term safety, measured by serious adverse events, adverse events of special interest, and adverse events that led to stopping treatment.[3]
Trial phases and endpoints
A Phase 1 trial usually focuses on dose finding and early safety, and that is how the pediatric study was designed.[1]
A Phase 2 trial usually looks more closely at whether a treatment may help while still tracking safety, which matches the ISM study.[2]
A Phase 3 trial often follows earlier studies and looks at longer-term outcomes in a larger or more specific group, which fits the rollover mastocytosis study.[3]
The key endpoints across these studies were recommended dose, safety, symptom change, tumor response, and long-term treatment effects.[1][2][3]
Who participated
The pediatric study included children with solid tumors dependent on KIT or PDGFRA signaling.[1]
The ISM study included patients with indolent systemic mastocytosis.[2]
The rollover study included adults who had already joined a previous Avapritinib study and had systemic mastocytosis.[3]
Study status
One trial, NCT04773782, is completed.[1]
The other two studies, NCT03731260 and 2024-514969-18-00, are authorised.[2][3]
