In short
Clinical trials are investigating Selumetinib in several patient groups, mainly people with neurofibromatosis type 1 (NF1) and plexiform neurofibromas, and also some cancer studies. These trials look at safety, how well treatment works, and how the body handles the medicine. They include adults, young children, and people with advanced cancer.
Key points
- Clinical trials of Selumetinib are mainly focused on NF1-related symptomatic, inoperable plexiform neurofibromas, with studies in adults and young children. The data also includes trials in metastatic breast cancer and advanced non-small cell lung cancer. The trials range from Phase 1 to Phase 3, so they study safety, pharmacokinetics, and treatment effect at different stages. Main endpoints include objective response rate, progression-free survival, and measures of safety and tolerability. Some studies compare Selumetinib with placebo, while others test specific formulations or treatment settings. Overall, the trial program is aimed at different patient groups to learn how Selumetinib performs in real research settings.
Clinical trial overview
The trial data shows that Selumetinib is being studied in different research settings, mainly for people with neurofibromatosis type 1 (NF1) and plexiform neurofibromas, which are nerve tumors linked to NF1. Other trials include people with metastatic breast cancer and advanced non-small cell lung cancer.
These are interventional studies, meaning the researchers assign a study treatment and then measure outcomes such as tumor response, survival without disease worsening, safety, and how the body handles the treatment.
Trials in NF1-related plexiform neurofibromas
Two trials focus on NF1-related symptomatic, inoperable plexiform neurofibromas, one in adults and one in younger children. “Symptomatic” means the tumor is causing problems, and “inoperable” means it cannot be safely removed by surgery.
NCT04924608 is a Phase 3 study in 145 adults with NF1 and symptomatic, inoperable plexiform neurofibromas. It compares Selumetinib with placebo, which is a look-alike treatment used for comparison, and the main outcome is objective response rate using volumetric MRI analysis and REiNS criteria. This means the study checks how many tumors shrink or respond based on MRI measurements of tumor volume.
NCT05309668, also called SPRINKLE, is a Phase 1 study in 36 children aged 1 to under 7 years with NF1-related symptomatic, inoperable plexiform neurofibromas. It studies a granule formulation of Selumetinib and looks at pharmacokinetics, safety, and tolerability. The trial measures exposure after a single dose using AUC0-12 and tracks safety with lab tests, physical exams, weight, vital signs, ECG, ECHO, eye exams, MRI or X-ray, and performance status.
NCT05101148 is another Phase 1 study in 20 adolescent children with NF1-related plexiform neurofibromas. It looks at the effect of food on Selumetinib capsule pharmacokinetics and gastrointestinal tolerability, including whether a low-fat meal changes drug exposure after multiple doses.
Trials in other cancers
The trial list also includes a Phase 2 study in metastatic breast cancer, identified as 2024-513934-40-00, with 400 participants. The main outcome is progression-free survival, which means the study checks how long the cancer stays from getting worse. Selumetinib is listed among the study interventions, together with other treatments in the trial.
NCT03944772 is a Phase 2 study in 99 people with advanced non-small cell lung cancer and EGFR mutations who became resistant to osimertinib. The main outcome is objective response rate using RECIST 1.1, a standard system for measuring how much a tumor shrinks or grows in solid tumors. Selumetinib appears as one of the listed study treatments in this multi-treatment trial.
What the trials measure
Across these studies, the most important endpoints are objective response rate, progression-free survival, pharmacokinetics, and safety. Objective response rate tells researchers how many participants have a meaningful tumor response. Progression-free survival measures how long the disease does not get worse.
Pharmacokinetics, often shortened to PK, describes how the body absorbs and processes the treatment. Safety and tolerability look at side effects and whether participants can stay on the study treatment.
Study phases and who can join
The Selumetinib trial data includes Phase 1, Phase 2, and Phase 3 studies. Phase 1 studies usually focus on safety, tolerability, and PK, while Phase 2 and Phase 3 studies focus more on how well the treatment works.
The target groups are different across trials: adults with NF1, children aged 1 to under 7 years, adolescent children, people with metastatic breast cancer, and people with advanced non-small cell lung cancer. This shows that Selumetinib is being studied in both rare tumor settings and some advanced cancer settings.
Key points for patients
Some studies compare Selumetinib with placebo, while others test different formulations such as capsules or granules. This means the research is not only about whether the treatment works, but also about which form works best for specific age groups and study needs.
Overall, the trial program is designed to learn about treatment effect, safety, and how Selumetinib behaves in the body across different patient groups.