Neurofibromatosis – European Clinical Trials Information Network

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

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 Crizotinib for Treating Severe Neurofibromatosis Type 2 in Children Not Eligible for Surgery or Radiotherapy

Wed, 29 Apr 2026 15:00:13 +0000

This clinical trial is focused on studying a condition called Neurofibromatosis type 2 (NF-2), which is a genetic disorder that can lead to the growth of tumors in the nervous system. These tumors are usually non-cancerous but can cause serious health issues, especially in children. The trial is testing a medication called Crizotinib, which is taken as a capsule. The purpose of the study is to evaluate how effective Crizotinib is in treating children with severe NF-2 who cannot undergo surgery or radiotherapy.

Participants in the study will take Crizotinib orally, and the study will monitor changes in the size of the tumors using imaging techniques. The study aims to see if the tumors stabilize or shrink over time. The treatment period can last up to 60 days, and the study will continue to observe the effects for at least six months after the treatment ends.

The main goal is to determine if Crizotinib can help manage the tumors in children with NF-2, potentially improving their quality of life and extending their survival. The study is non-commercial and non-randomized, meaning it is focused on understanding the treatment’s effects without comparing it to other treatments or placebos. The trial is expected to conclude by the end of 2026.

Study on Trametinib for Patients with Neurofibromatosis Type 1 and Symptomatic Plexiform Neurofibromas

Wed, 29 Apr 2026 14:59:46 +0000

This clinical trial is focused on studying a condition called Neurofibromatosis type 1, which is a genetic disorder that often leads to the development of tumors along nerves in the skin, brain, and other parts of the body. The trial is specifically looking at a type of tumor known as plexiform neurofibromas, which can cause significant symptoms and complications. The treatment being tested in this study is a medication called Trametinib, also known by its code name GSK1120212B. Trametinib is taken in the form of film-coated tablets.

The purpose of the study is to determine if Trametinib can help reduce the size of these tumors. Participants in the study will take the medication orally, and the effects will be monitored over time. The study will use MRI scans to measure any changes in the size of the tumors. The trial will also assess other factors such as pain levels and any side effects experienced by participants.

The study is expected to continue until early 2027. Participants will be closely monitored throughout the trial to ensure their safety and to gather data on the effectiveness of Trametinib in treating plexiform neurofibromas associated with Neurofibromatosis type 1. This research aims to provide valuable insights into potential new treatments for this challenging condition.

Imaging Study with 89Zr-Bevacizumab for Predicting Treatment Effects in Patients with NF2-Related Schwannomatosis

Wed, 29 Apr 2026 14:58:50 +0000

This clinical trial is focused on studying a condition known as Neurofibromatosis Type 2 (NF2), which is a genetic disorder that causes tumors to grow on nerves, particularly affecting the nerves responsible for hearing. The study is specifically looking at a type of tumor called vestibular schwannomas, which can lead to hearing loss. The treatment being investigated is called 89Zr-Bevacizumab, which is a special form of a drug that is used to help visualize these tumors using a type of imaging called PET/CT scan. This imaging technique combines two methods: positron emission tomography (PET) and computed tomography (CT), to provide detailed pictures of the inside of the body.

The purpose of the study is to see if the 89Zr-Bevacizumab PET/CT scan can predict how well patients with NF2 will respond to treatment with Bevacizumab, a medication that is used to treat these tumors. During the study, participants will undergo the PET/CT scan before starting their treatment with Bevacizumab. The researchers will then monitor the participants to see if there is a correlation between the scan results and improvements in hearing or changes in the size of the tumors.

Throughout the study, the researchers will also look at other factors such as how the treatment affects the patients’ overall well-being, balance, and function of other nerves. The study aims to provide valuable information that could help improve the management of NF2 and potentially enhance the quality of life for those affected by this condition.

Study on the Effect of Food on Selumetinib in Adolescents with Neurofibromatosis Type 1 Plexiform Neurofibromas

Wed, 29 Apr 2026 14:57:42 +0000

This clinical trial is focused on studying a condition called Neurofibromatosis Type 1 (NF1), which is a genetic disorder that often leads to the development of tumors along nerves in the skin, brain, and other parts of the body. Specifically, the study is looking at a type of tumor known as plexiform neurofibromas (PN), which are complex nerve tumors that can cause various health issues. The treatment being tested in this study is a medication called Selumetinib, also known by its code name AZD6244. This medication is taken in the form of hard capsules and is being evaluated for its effects when taken with food.

The purpose of the study is to understand how a low-fat meal affects the way Selumetinib is processed in the body and its impact on the stomach and intestines. The study involves adolescent children who have NF1-related plexiform neurofibromas. Participants will take part in different treatment periods where they will receive multiple doses of Selumetinib. During these periods, the study will assess how the medication is absorbed and tolerated by the body, especially when taken with or without food.

Throughout the study, participants will be monitored for any side effects, and their overall health will be assessed through various tests, including blood tests and heart function tests. The study aims to gather information on the safety and tolerability of Selumetinib in young patients with NF1-related plexiform neurofibromas, providing valuable insights into how the medication can be used effectively in managing this condition.

Study on Selumetinib for Children Aged 1 to 6 with Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas

Wed, 29 Apr 2026 14:29:33 +0000

This clinical trial is focused on studying a condition called Neurofibromatosis Type 1 (NF1), which is a genetic disorder that can cause tumors to grow on nerves. Specifically, the study is looking at a type of tumor known as plexiform neurofibromas (PN), which are often difficult to remove with surgery. The trial is testing a medication called Selumetinib, also known by its code name AZD6244. This medication is being studied in a granule form that is suitable for children. The purpose of the study is to understand how the body processes Selumetinib, as well as to evaluate its safety and how well it is tolerated by young children.

The study involves children aged 1 to 7 years who have symptomatic, inoperable plexiform neurofibromas. Participants will receive the Selumetinib granule formulation orally, and researchers will monitor how the medication is absorbed and processed in the body. The study will also assess the safety of the medication by observing any side effects and conducting various health checks, including physical exams and imaging tests like MRI scans. Parents will also provide feedback on how easy it is for their children to take the medication.

This trial is designed to gather important information over several years, with an estimated completion date in 2028. The findings from this study could help improve treatment options for children with NF1-related plexiform neurofibromas, providing insights into the effectiveness and safety of Selumetinib in this young age group.

Study on Selumetinib for Adults with Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas

Wed, 29 Apr 2026 14:29:15 +0000

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.