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Neurofibromatosis Clinical Trials

A listing of Neurofibromatosis medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

RESULTS

Found (43) clinical trials

This registry will collect information to help doctors and researchers better understand the range of medical and social problems experienced by children and adults with NF1. Volunteers will complete a 30 minute questionnaire and will be asked to update their information yearly. There are no direct benefits to the volunteers. ...

Phase

Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (PNs)

Background Neurofibromatosis 1 (NF1) is a genetic disease with multiple clinical manifestations, including plexiform neurofibromas (PN) that can cause pain and may significantly impact daily functioning and quality of life (QOL). Patient-reported outcomes (PROs) are useful in trials for conditions that are disabling and chronic like NF1, where symptom reduction ...

Phase N/A

Dexmedetomidine and Propofol for Pediatric MRI Sedation

The purpose of this study is to compare the results of combining two anesthetic medications (dexmedetomidine and propofol) in low doses with a standard dose of a single drug that is commonly used to provide sedation/anesthesia for MRI studies in young children (propofol). Recent studies and the FDA have raised ...

Phase

Natural History Study of Patients With Neurofibromatosis Type I

BACKGROUND Neurofibromatosis Type 1 (NF1) is an autosomal dominant, progressive genetic disorder characterized by diverse clinical manifestations. Patients with NF1 have an increased risk of developing tumors of the central and peripheral nervous system including plexiform neurofibromas (PN), dermal neurofibromas, optic pathway tumors, brain tumors, malignant peripheral nerve sheath tumors ...

Phase N/A

Non-invasive Stimulation in Neurofibromatosis Type 1

16 children between the age of 11-16 years will be recruited through Manchester Centre of Genomic Medicine NF1 Database. The participants will be randomly assigned to 2 groups- active and sham treatment based on a computer generated random allocation list. The assessors will remain blind to treatment allocation. On day ...

Phase N/A

Vitamin D Supplementation for Adults With Neurofibromatosis Type 1 (NF1)

Individuals with NF1 and vitamin D insufficiency will be supplemented with 2 different doses of cholecalciferol (vitamin D) to see if their loss of bone mineral density is lessened over time. It is important to study vitamins as carefully as one would study medications, even though vitamins are generally safer ...

Phase

Trametinib in Patients With Advanced Neurofibromatosis Type 1 (NF1)-Mutant Non-small Cell Lung Cancer

This is a phase II, single-arm, open-label, multicenter clinical trial evaluating the efficacy and safety of trametinib monotherapy in patients with advanced non-squamous NSCLC whose tumors non-synonymous NF1 mutations and are KRAS wildtype. Eligible patients must have documented disease progression either during or after treatment with most recent therapy and ...

Phase

Trial of Selumetinib in Patients With Neurofibromatosis Type II Related Tumors

This is a Phase 2 trial to assess the hearing response rate and radiographic response of VS in children and young adults with NF2 who are treated with selumetinib. Dosing will be based on age: For patients with NF2 who are 3 to < 18 years of age, dosing will ...

Phase

Acceptance and Commitment Training for Adolescents and Young Adults With Neurofibromatosis Type 1 Plexiform Neurofibromas and Chronic Pain

BACKGROUND Neurofibromatosis type 1 (NF1) is a genetic disorder that affects approximately 1 in 3,500 individuals. A number of common clinical manifestations, including plexiform neurofibromas, can cause frequent and significant pain and impact quality of life. Often, NF1-related pain is not well-controlled with medication and many of the medications cause ...

Phase

Synaptic Plasticity and Cognitive Function in RASopathies

The project is targeting cognitive impairment, one of the main health problems of patients with RAS pathway disorders. The aim of this study is to translate findings of animal studies to humans. This has been done by the applicants successfully for Lovastatin in Nf1. This result will be transferred to ...

Phase