View clinical trials related to Neurofibroma.
Filter by:This study will evaluate the tolerability and effectiveness of two treatments in Neurofibromatosis Type 1 Cutaneous Neurofibromas. These treatments are: Kybella and Asclera injection. Each patient will have a treatment and a control site.
The purpose of this study is to find out whether avutometinib is a safe treatment for advanced or recurrent solid tumor cancers in children and young adults. Researchers will look for the highest dose of avutometinib that is safe and cause few or mild side effects.
A study to evaluate the efficacy of FCN-159 in adult patients with symptomatic, inoperable neurofibromatosis type 1-related plexiform neurofibromas.
Neurofibromatosis type 1 is a common genetic disease with a broad spectrum of clinical manifestations in multiple organs of the body. This project will study the (dys)function of mitochondria in patients with neurofibromatosis through multiple collections of blood samples from patients and people not afflicted by neurofibromatosis (control group). This study will evaluate how the function of mitochondria changes with time and if medications and supplements can influence the function of the mitochondria. Patients will also answer questions regarding symptoms like fatigue and pain.
The purpose of this study is to evaluate the effectiveness, safety of selumetinib and patient's parameters of symptomatic inoperable PN NF1 in real clinical practice in Russia
This is a phase II, multicenter, randomised, parallel, double-blind, placebo-controlled study assessing the efficacy and safety of the MEKi selumetinib compared with placebo in Chinese paediatric participants with post-operative NF1-associated PNs.
Some parents of children living with a visible difference can experience heightened stress due to the associated challenges of this. Parent's views of the child's visible difference and their responses to the child are important. Mindful parenting approaches have been found to reduce stress or distress for parents of children with disabilities, physical health problems and skin conditions. This study will be completed with a small number of participants (around six to 12). Participants will be parents or carers of a child living with a visible difference aged four to 16 years, who are experiencing stress. Parents or carers will complete an online mindful parenting intervention (called Two Hearts) including video content, audio files and a workbook, over six weeks. Over the twelve-week study period participants will also provide the following information which will be compared over time: - Complete four group support sessions during the intervention via videoconferencing - Complete questionnaires at four timepoints - Provide information about their use of the intervention materials and home practice weekly - Answer two questions daily via text message about parenting stress levels We hope to learn about the initial effects of the mindful parenting programme for parents or carers of children living with a visible difference. We also hope to learn whether parents or carers find completing an online programme possible and practical. Finally, we hope to learn what parents' or carers' views are of the online programme and whether this type of intervention in online format would be helpful for other parents or carers.
Bevacizumab can be an effective treatment for individuals with NF2 and improve different nerve functions (like hearing, tinnitus or balance problems) and the quality of life of NF2 patients. However, bevacizumab is not effective in all patients or all tumors, at the cost of moderate toxicity and considerable financial burden. Therefore, this observational study will validate an imaging biomarker method to predict bevacizumab efficacy in order to avoid adverse effects and high costs in non-responders to bevacizumab treatment. Patients will undergo standard-of-care treatment with the sole addition of a pre-treatment 89Zr-Bevacizumab PET/CT-scan. Per standard-of-care bevacizumab therapy is administered every three weeks for six months. To monitor treatment effect, follow-up is performed at 3-month intervals.
The purpose of this study is to understand treatment patterns and assess long-term effectiveness and safety outcomes associated with selumetinib treatment as well as to explore clinical and non-clinical factors affecting those outcomes in participants with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PNs) who were aged 2 to 18 years at the time selumetinib was started in a real-world setting.
The goal of this prospective observational study is to learn about the utility of imaging and clinical features in patients with Neurofibromatosis type 1 categorized as high risk for the development of malignant peripheral nerve sheath tumors. The main objectives are: - To evaluate the prevalence, multi-parametric imaging features of distinct nodular lesions ("DNLs") and natural history in people with NF1 with clinical and genetic features deemed "high-risk" for malignancy. - To assess the relationship between individual clinical, genetic and imaging factors that have been suggested to be risk factors for malignant peripheral nerve sheath tumors (MPNST) and the confirmation of atypical neurofibromas (aNF)/ atypical neurofibromatous neoplasm of unknown biologic potential (ANNUBP) or MPNST on pathology. In this research study, the participants will be asked to undergo whole body MRI, provide blood sample and clinical evaluation annually.