In short
Clinical trials are investigating Avapritinib in different patient groups, including children with certain solid tumors and adults with mastocytosis. These studies look at safety, dose finding, and how well the treatment works. The trials include early-phase and later-phase research.
Key points
- Clinical trials of Avapritinib are studying different patient groups, including children with solid tumors linked to KIT or PDGFRA signaling and adults with mastocytosis. The research includes Phase 1, Phase 2, and Phase 3 studies, so it covers early dose finding and later safety and effectiveness testing. One pediatric study focused on dose-limiting toxicity, safety, and objective response rate. The mastocytosis studies looked at the recommended dose, symptom score changes, and long-term safety and efficacy. One trial is completed, and the others are authorised.
Clinical trials overview
These studies investigate Avapritinib in different groups of patients with cancer-related conditions and mastocytosis.
All three trials are interventional studies, which means researchers gave a study treatment and measured what happened next.
Pediatric solid tumors study
NCT04773782 studied pediatric patients with solid tumors dependent on KIT or PDGFRA signaling. This was a Phase 1 trial with 37 enrolled patients and it is completed.
The study had two main parts. Part 1 looked for the recommended dose for Part 2 and checked safety and tolerability, which means how well patients could handle the treatment. Part 2 looked at anti-tumor activity, which means whether the tumors responded to treatment.
The main endpoints were dose-limiting toxicity, adverse events, and objective response rate. The tumor response was measured with RECIST version 1.1 or RANO, depending on the tumor type.
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. This was a Phase 2 study with 251 enrolled patients and an authorised status.
The trial had three parts. Part 1 aimed to determine the RP2D, which means the recommended Phase 2 dose. Part 2 compared the mean change in ISM-SAF TSS from baseline to C7D1 against placebo, so it measured symptom changes over time. Part 3 assessed the long-term safety and efficacy of Avapritinib.
The rollover study 2024-514969-18-00 included adults who had taken part in a previous Avapritinib study and studied systemic mastocytosis. This was a Phase 3 interventional study with 60 enrolled patients and an authorised status.
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.
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.
A Phase 2 trial usually looks more closely at whether a treatment may help while still tracking safety, which matches the ISM study.
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.
The key endpoints across these studies were recommended dose, safety, symptom change, tumor response, and long-term treatment effects.
Who participated
The pediatric study included children with solid tumors dependent on KIT or PDGFRA signaling.
The ISM study included patients with indolent systemic mastocytosis.
The rollover study included adults who had already joined a previous Avapritinib study and had systemic mastocytosis.
Study status
One trial, NCT04773782, is completed.
The other two studies, NCT03731260 and 2024-514969-18-00, are authorised.