Table of Contents
- Clinical trial overview
- Trials in NF1-related plexiform neurofibromas
- Trials in other cancers
- What the trials measure
- Study phases and who can join
- Key points for patients
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.[1][2] Other trials include people with metastatic breast cancer and advanced non-small cell lung cancer.[3][4]
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.[1][2][3][4][5]
Trials in NF1-related plexiform neurofibromas
Two trials focus on NF1-related symptomatic, inoperable plexiform neurofibromas, one in adults and one in younger children.[1][2] “Symptomatic” means the tumor is causing problems, and “inoperable” means it cannot be safely removed by surgery.[1][2]
NCT04924608 is a Phase 3 study in 145 adults with NF1 and symptomatic, inoperable plexiform neurofibromas.[1] 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.[1] This means the study checks how many tumors shrink or respond based on MRI measurements of tumor volume.[1]
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.[2] It studies a granule formulation of Selumetinib and looks at pharmacokinetics, safety, and tolerability.[2] 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.[2]
NCT05101148 is another Phase 1 study in 20 adolescent children with NF1-related plexiform neurofibromas.[5] 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.[5]
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.[3] The main outcome is progression-free survival, which means the study checks how long the cancer stays from getting worse.[3] Selumetinib is listed among the study interventions, together with other treatments in the trial.[3]
NCT03944772 is a Phase 2 study in 99 people with advanced non-small cell lung cancer and EGFR mutations who became resistant to osimertinib.[4] 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.[4] Selumetinib appears as one of the listed study treatments in this multi-treatment trial.[4]
What the trials measure
Across these studies, the most important endpoints are objective response rate, progression-free survival, pharmacokinetics, and safety.[1][2][3][4][5] Objective response rate tells researchers how many participants have a meaningful tumor response.[1][4] Progression-free survival measures how long the disease does not get worse.[3]
Pharmacokinetics, often shortened to PK, describes how the body absorbs and processes the treatment.[2][5] Safety and tolerability look at side effects and whether participants can stay on the study treatment.[2]
Study phases and who can join
The Selumetinib trial data includes Phase 1, Phase 2, and Phase 3 studies.[1][2][3][4][5] 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.[2][3][4]
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.[1][2][3][4][5] This shows that Selumetinib is being studied in both rare tumor settings and some advanced cancer settings.[1][3][4]
Key points for patients
Some studies compare Selumetinib with placebo, while others test different formulations such as capsules or granules.[1][2][5] 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.[2][5]
Overall, the trial program is designed to learn about treatment effect, safety, and how Selumetinib behaves in the body across different patient groups.[1][2][3][4][5]
