Antioxidant
Antioxidant Therapy with N-acetylcysteine for Neurofibromatosis Type 1
This trial tests N-Acetyl Cysteine (NAC), a common supplement, to see if it can help children with neurofibromatosis type 1 (NF1) who have cognitive, behavioral, and motor issues. NAC works by reducing harmful substances in the brain, potentially improving behavior and motor skills. NAC is a precursor to glutathione (GSH) and has been studied for its neuroprotective and cognitive benefits in various conditions.
Behavioural Intervention
Educational Letters for Neurofibromatosis
This trial aims to compare two educational interventions for individuals with Neurofibromatosis 1 (NF1) to see which one helps more in getting proper health screenings for NF1 patients in regular doctor
Kinase Inhibitor
Selumetinib for Plexiform Neurofibroma
This trial is testing how well selumetinib works when taken with a low-fat meal in adolescents with NF1 who have tumors that can't be removed by surgery. The goal is to see if eating a low-fat meal affects how the body absorbs the medication and if it helps reduce stomach-related side effects. Selumetinib is being investigated for its effectiveness in treating NF1-associated tumors, with previous studies showing promising positive results in patients.
Procedure
AI-Enhanced MRI for Detecting Precancerous Lesions in Neurofibromatosis
This trial uses a full-body scan and smart computer software to monitor changes in pediatric patients with neurofibromatosis type 1. The MRI takes detailed images, and the AI analyzes them to track any changes over time.
Popular Filters
Trials for NF Patients
Dietary Supplement
Nutraceuticals for Neurofibromatosis
This trial involves taking curcumin and a special type of olive oil twice a day to help reduce inflammation and oxidative stress. It targets individuals who can tolerate this treatment for an extended period. Curcumin is a natural polyphenol known for its antioxidant and anti-inflammatory properties, and it has been studied in various contexts including metabolic syndrome, diabetes, and exercise-induced oxidative stress.
Behavioural Intervention
Acceptance and Commitment Therapy for Caregivers of Children with Genetic Syndromes
This trial will test whether Acceptance and Commitment Therapy (ACT) can help caregivers of children with a RASopathy better cope with parenting stress.
MEK1/2 inhibitor
FCN-159 for Neurofibromatosis
This trial is testing FCN-159, a new drug taken by mouth, for patients with advanced solid tumors and neurofibromatosis type 1. The drug works by blocking specific proteins that help cancer cells grow. This targeted approach aims to slow down or stop the progression of these diseases.
Kinase Inhibitor
Selumetinib for Plexiform Neurofibromas
This trial is testing a medication called selumetinib to see if it can help adults with a condition called NF1 who have tumors that cause symptoms and cannot be removed by surgery. The medication works by blocking signals that make the tumors grow.
Trials for Neurofibroma Patients
Dietary Supplement
Nutraceuticals for Neurofibromatosis
This trial involves taking curcumin and a special type of olive oil twice a day to help reduce inflammation and oxidative stress. It targets individuals who can tolerate this treatment for an extended period. Curcumin is a natural polyphenol known for its antioxidant and anti-inflammatory properties, and it has been studied in various contexts including metabolic syndrome, diabetes, and exercise-induced oxidative stress.
Behavioural Intervention
Acceptance and Commitment Therapy for Caregivers of Children with Genetic Syndromes
This trial will test whether Acceptance and Commitment Therapy (ACT) can help caregivers of children with a RASopathy better cope with parenting stress.
MEK1/2 inhibitor
FCN-159 for Neurofibromatosis
This trial is testing FCN-159, a new drug taken by mouth, for patients with advanced solid tumors and neurofibromatosis type 1. The drug works by blocking specific proteins that help cancer cells grow. This targeted approach aims to slow down or stop the progression of these diseases.
Kinase Inhibitor
Selumetinib for Plexiform Neurofibromas
This trial is testing a medication called selumetinib to see if it can help adults with a condition called NF1 who have tumors that cause symptoms and cannot be removed by surgery. The medication works by blocking signals that make the tumors grow.
Trials for NF1 Positive Patients
MEK1/2 inhibitor
FCN-159 for Neurofibromatosis
This trial is testing FCN-159, a new drug taken by mouth, for patients with advanced solid tumors and neurofibromatosis type 1. The drug works by blocking specific proteins that help cancer cells grow. This targeted approach aims to slow down or stop the progression of these diseases.
Kinase Inhibitor
Selumetinib for Plexiform Neurofibromas
This trial is testing a medication called selumetinib to see if it can help adults with a condition called NF1 who have tumors that cause symptoms and cannot be removed by surgery. The medication works by blocking signals that make the tumors grow.
CDK4/6 Inhibitor
Abemaciclib for Neurofibroma
This trial is testing a drug, abemaciclib, to see if it can help treat atypical neurofibromas, which are tumors that arise from nerves and can cause serious medical problems. The drug is taken orally, twice daily, in 28-day cycles.
Antioxidant
Antioxidant Therapy for Neurofibromatosis Type 1
This trial is testing a medication called NAC in children aged 8-16 with a condition called NF1. These children often have problems with movement and behavior, and there is no current treatment for these issues. NAC works by reducing harmful molecules in the brain, which may help improve these symptoms.
Trials With No Placebo
Dietary Supplement
Nutraceuticals for Neurofibromatosis
This trial involves taking curcumin and a special type of olive oil twice a day to help reduce inflammation and oxidative stress. It targets individuals who can tolerate this treatment for an extended period. Curcumin is a natural polyphenol known for its antioxidant and anti-inflammatory properties, and it has been studied in various contexts including metabolic syndrome, diabetes, and exercise-induced oxidative stress.
Behavioural Intervention
Acceptance and Commitment Therapy for Caregivers of Children with Genetic Syndromes
This trial will test whether Acceptance and Commitment Therapy (ACT) can help caregivers of children with a RASopathy better cope with parenting stress.
MEK1/2 inhibitor
FCN-159 for Neurofibromatosis
This trial is testing FCN-159, a new drug taken by mouth, for patients with advanced solid tumors and neurofibromatosis type 1. The drug works by blocking specific proteins that help cancer cells grow. This targeted approach aims to slow down or stop the progression of these diseases.
CDK4/6 Inhibitor
Abemaciclib for Neurofibroma
This trial is testing a drug, abemaciclib, to see if it can help treat atypical neurofibromas, which are tumors that arise from nerves and can cause serious medical problems. The drug is taken orally, twice daily, in 28-day cycles.
Frequently Asked Questions
Introduction to neurofibromatosis type 1
What are the top hospitals conducting neurofibromatosis type 1 research?
When it comes to clinical trials for neurofibromatosis type 1 (NF1), several hospitals across the United States are leading the way in research and treatment. One such institution is the National Institutes of Health Clinical Center located in Bethesda, Maryland. With six active NF1 trials and an impressive track record of 20 completed trials, this facility has been dedicated to advancing our understanding of NF1 since its first recorded trial in 2005. Similarly, Cincinnati Children's Hospital Medical Center in Ohio has also made significant contributions with four ongoing NF1 trials and a total of 21 completed trials dating back to their inaugural study in 2005.
Another notable player on this list is the National Institutes of Health Clinical Center situated at 9000 Rockville Pike in Bethesda, which currently has three active NF1 trials along with a commendable history of 21 previous studies that began as early as 2001. In Gainesville, at a research site yet to be specified further, two active clinical trials for NF1 are underway—their initial undertaking into exploring treatments for this condition started quite recently in2021.
Additionally proving itself as a prominent location for cutting-edge research is Children's Hospital Los Angeles (CHLA) based in sunny California. CHLA currently conducts two active clinical trials focused on NF1 while boasting an impressive thirteen past studies conducted since their establishment as pioneers within this field starting from2009.
These hospitals represent pillars within the medical community when it comes to enhancing our knowledge and developing potential therapies for individuals affected by neurofibromatosis type 1—a genetic disorder characterized by tumors growing on nerves throughout the body. The dedication and expertise demonstrated by these institutions provide hope not only for those living with NF1 but also propel us closer towards effective treatments and improved quality of life for patients worldwide who face similar challenges stemming from rare conditions alike
Which are the best cities for neurofibromatosis type 1 clinical trials?
When it comes to neurofibromatosis type 1 clinical trials, several cities emerge as leaders in research and development. Bethesda, Maryland takes the lead with 10 active trials investigating treatments like AZD6244, Selumetinib, and Turalio. Los Angeles, California follows closely behind with 5 ongoing studies focused on innovative approaches such as FCN-159 and Whole-body Magnetic Resonance Imaging. Cincinnati, Ohio also offers promising opportunities with 5 active trials exploring interventions like Mirdametinib (PD-0325901) oral capsule or dispersible tablet and N-Acetyl cysteine. These cities provide individuals affected by neurofibromatosis type 1 access to cutting-edge clinical trials that hold potential for advancing care and improving outcomes.
Which are the top treatments for neurofibromatosis type 1 being explored in clinical trials?
Exciting developments are underway in clinical trials for neurofibromatosis type 1, with several top treatments showing promise. Leading the pack is selumetinib, currently being explored in three active trials dedicated to this condition. Since its introduction in 2010, it has been involved in a total of 10 neurofibromatosis type 1 trials. Joining the lineup is curcumin and high phenolic extra virgin olive oil (HP-EVOO), making waves with one active trial and one all-time neurofibromatosis type 1 trial since its recent listing in 2022. Also on the radar is the ACT Intervention and FCN-159, both beginning their journey with one active trial and an all-time count of one neurofibromatosis type 1 trial each—first listed respectively in 2022 and 2021. These groundbreaking research endeavors bring hope for improved treatment options to those affected by this challenging condition.
What are the most recent clinical trials for neurofibromatosis type 1?
Exciting developments are underway in the field of neurofibromatosis type 1 (NF1), with recent clinical trials exploring potential treatment options. One such trial focuses on a regimen combining Fludarabine and Total Body Irradiation, aiming to assess its efficacy in managing NF1. Additionally, another study investigates the use of DPCP as a potential therapy for NF1 patients. Furthermore, researchers are examining the effects of curcumin and high phenolic extra virgin olive oil (HP-EVOO) on NF1 symptoms. Abemaciclib has also been evaluated in Phase 1 and Phase 2 trials to determine its effectiveness against NF1. Lastly, Selumetinib is being studied in a Phase 3 trial with hopes of providing valuable insights into its therapeutic potential for individuals with NF1. The outcomes from these diverse studies offer promising prospects for improved management and treatment of neurofibromatosis type 1 moving forward.
What neurofibromatosis type 1 clinical trials were recently completed?
Three recent clinical trials for neurofibromatosis type 1 (NF1) have reached completion, bringing new insights and potential treatments for this complex disorder. In October 2021, a trial sponsored by the Children's Tumor Foundation investigated the effectiveness of a targeted therapy in managing NF1-related plexiform neurofibromas. Another significant trial was completed in September 2021, led by the National Cancer Institute, examining an innovative treatment approach for optic pathway gliomas associated with NF1. Additionally, in January 2022, a study supported by Pfizer explored the safety and efficacy of a novel drug candidate specifically designed to address NF1-related tumors. These promising developments highlight ongoing efforts to improve outcomes for individuals affected by NF1 and advance our understanding of its management options.