Neurofibroma – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Tue, 02 Jun 2026 12:23:16 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Neurofibroma – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 A study of mirdametinib for infants and toddlers under 24 months of age with neurofibromatosis type 1 associated plexiform neurofibromas https://clinicaltrials.eu/trial/a-study-of-mirdametinib-for-infants-and-toddlers-under-24-months-of-age-with-neurofibromatosis-type-1-associated-plexiform-neurofibromas/ Wed, 29 Apr 2026 15:07:55 +0000 https://clinicaltrials.eu/trial/a-study-of-mirdametinib-for-infants-and-toddlers-under-24-months-of-age-with-neurofibromatosis-type-1-associated-plexiform-neurofibromas/ This study involves infants and toddlers under 24 months of age who have Neurofibromatosis Type 1 associated with Plexiform Neurofibromas. Neurofibromatosis Type 1 is a genetic condition that causes tumors to grow along nerves in the body. Plexiform Neurofibromas are a type of tumor that can grow in multiple areas and may cause problems depending on where they are located, such as near important blood vessels, airways, or nerves. These tumors can cause symptoms like pain, loss of function, or visible changes in appearance, or they may not cause symptoms but are in locations where they could lead to serious problems. The treatment being tested in this study is Mirdametinib, also known by its code name PD-0325901, which is given as a dispersible tablet taken by mouth.

The purpose of this study is to evaluate the safety, how well the body handles the medication, and how the medication works in very young children with these tumors. The study will look at whether the medication causes any side effects, changes in laboratory test results, vital signs, heart function measured by tests called electrocardiograms and echocardiogram, and eye examinations. The study will also measure the amount of medication in the blood to understand how it is processed by the body. Additionally, the study will examine whether the tumors shrink by at least 20 percent compared to their size at the start, using imaging scans analyzed by independent reviewers.

During the study, children will receive Mirdametinib for up to 36 months, and they will be monitored regularly through physical examinations, blood tests, imaging scans using MRI, and assessments of quality of life and pain levels using age-appropriate scales. The study will also track how long any tumor shrinkage lasts and whether there are improvements in the child’s comfort and daily functioning. The medication dose can be up to 8 milligrams per day, with a maximum total dose of 288 milligrams during the treatment period.

]]> Study on Selumetinib for Adults with Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas https://clinicaltrials.eu/trial/56721/ Wed, 29 Apr 2026 14:29:15 +0000 https://clinicaltrials.eu/trial/56721/ This study focuses on a condition known as Neurofibromatosis Type 1 (NF1), which can cause non-cancerous tumors called plexiform neurofibromas. These tumors are symptomatic and cannot be operated on, making them a challenge to treat. The investigation will explore the effectiveness and safety of a drug named Selumetinib, taken in capsule form, compared to a placebo. The goal of the research is to evaluate how well Selumetinib works in treating these tumors.

Participants in the study will receive either Selumetinib or a placebo without knowing which one they are taking to ensure impartiality in the results. The focus will be on monitoring changes in the size and symptoms of the tumors and other related health aspects. Researchers will use a type of imaging called volumetric MRI to assess the effectiveness of the treatment by measuring any changes in tumor size.

Through this approach, the study aims to determine whether Selumetinib can help manage NF1-related plexiform neurofibromas by reducing tumor size and alleviating symptoms. The results will help understand if Selumetinib is a viable treatment for this condition.

]]>