Active clinical trials for "Neurofibromatoses"

Intellectual impairments are a significant cause of morbidity in children with birth defects along with long term implication on academic and occupational functioning. Long lasting functional changes in the brain occur when children learn new things or memorise new information. Enhancing this and learning is a key objective inneurodevelopment and neuro-rehabilitation. This is pilot study aimed at testing a experimental neuroscience technique, Transcranial Direct Current Stimulation (tCDS) on the cognitive functioning of the brain in children with Neurofibromatosis type 1 (NF1). tDCS is an established research tool and has the potential for ameliorating the cognitive impairments associated with NF1. There is a growing interest in the use of tDCS in children but to our knowledge there have been no reported studies using tDCS intervention in NF1. 16 children aged 11-16 years will be recruited through the Manchester Centre of Genomic Medicine NF1 database. Participants will be randomised to receive active or sham tDCS. The treatment will be delivered for 20 minutes for 3 days. In the experimental group a 1mA current will be applied for 20 mins ; in sham tDCS the electrodes will be placed in an identical spot but the current is ramped down to 30 seconds to prevent stimulation. The aim of the study is to look into the acceptability and feasibility of using tDCS intervention within the NF1 group, obtain pilot data on the effect of tDCS on EEG (Electroencephalogram), cognitive and behavioural measures.

This study tests the efficacy of an 8-week, Skype-based, group resiliency training intervention (The Relaxation Response Resiliency Program) for improving psychological stress in patients with Neurofibromatosis. A control group will receive a general health education curriculum. The investigators hypothesize that patients will improve on measures of psychological stress as a result of the resiliency program. A substudy (Unique Protocol ID: 2013P002605b) has been approved to test this study on a sub-population: patients with NF2 who are hard of hearing.

The aims of this study are to compare the effect and durability of two stress and symptom management programs tailored for patients with neurofibromatosis on quality of life and psychosocial functioning.

The primary objective of this study is to verify the safety of antigen-specific T cells (CAR-T) and engineered immune effector cytotoxic T cells (EIE) modified by immunoregulatory genes and immune modified dendritic cell vaccine (DCvac) in the treatment of neurofibromatosis or schwannoma.

The purpose of this study is to determine whether multiple cutaneous neurofibromas in patients with neurofibromatosis type 1 can be removed with an erbium-YAG-laser.

This is an open trial of the UCLA PEERS protocol delivered via Telehealth with teens with neurofibromatosis type 1 whose parents report that they have difficulty making and keeping friends.

A national, multicenter, randomized, transverse clinical trial, estimating the existence of phonological deficits in children with NF1 children compared with control children without NF1 with the same reading level.

Background:<TAB> - Many people with neurofibromatosis type 1 (NF1) get tumors of the nervous system. Finding malignant tumors early is important for removing them. Researchers want to find ways of doing this with scans and genetic testing. Objectives: - To learn more about neurofibromatosis type 1. Eligibility: - People age 10 and older with NF1 who have a benign tumor or have had a malignant one. Design: Participants will be screened in another study with medical history, physical exam, and urine and blood tests. They will have a magnetic resonance imaging (MRI) scan. MRI: Participants will lie on a table that slides into a metal cylinder. They will be in the scanner for 60 90 minutes, lying still for 15 minutes at a time. Participants will get earplugs for the loud sounds. They will get a contrast agent (dye) through a thin plastic tube (catheter) inserted in an arm vein. As part of their regular care, participants will have: FDG-PET/CT scan. They will get radioactive glucose (sugar) through a catheter in an arm vein. [18F]-FLT-PET/CT scan. This is like the FDG scan but with a different radioactive chemical. Biopsy. A piece of tumor tissue is removed with a needle. A piece of tissue from a previous biopsy may also be studied. Participants may have genetic testing. Blood will be taken. It will be tested along with biopsy samples. Researchers will explain the risks and procedures. They may notify participants if testing shows health problems. After this study, participants will continue their regular cancer care.

Objectives 1. Establish an induced pluripotent stem cell (iPSC) bank for phenotypically well-characterized patients with NF1. 2. Develop isogenic NF1 wild-type (NF1+/+), NF1 heterozygous (NF1+/-) and NF1 homozygous (NF1-/-) iPSC lines from individual patients using CRISPR/CAS9 technology. 3. Differentiate and characterize disease-relevant brain cells such as excitatory and inhibitory neurons, astrocytes and oligodendrocytes from patient-specific iPSC lines. 4. Screen and identify the drug(s) that can reverse or alleviate the disease phenotypes.

This is a pilot randomized control trial of the UCLA PEERS protocol delivered via Telehealth with teens with neurofibromatosis type 1 whose parents report that they have difficulty making and keeping friends.