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Neurofibromatosis 1 clinical trials

View clinical trials related to Neurofibromatosis 1.

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NCT ID: NCT03531814 Completed - Neurofibromatosis 1 Clinical Trials

Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1(NF1) on Clinical Treatment Trials

Start date: October 23, 2018
Phase: N/A
Study type: Interventional

Background: Neurofibromatosis type 1 (NF1) is a genetic disorder. It has a broad variety of effects on the body. Up to half of people with NF1 get plexiform neurofibromas (PNs). These are benign tumors. But they can have serious effects like pain and disfigurement. To treat PNs, a person may have to take medicine every day for a long period of time. Researchers think that it will be important for people to take the medicine regularly for it to work. They want to study how well people with NF1 follow their treatment plan for PNs. Objective: To study how often people with neurofibromatosis type 1 take medicine that has been prescribed to them for treating plexiform neurofibromas. Eligibility: People ages 3-59 already enrolled in an NF1 clinical trial Design: Participants will need access to the internet to do the study activities. Parents or caregivers will do some study activities for child participants. Participants will complete 5 questionnaires. They will take about 20 minutes total. The topics will be: Demographic data Recent life events How much pain interferes with daily life Ability to focus and pay attention to tasks Emotional distress or depression Participants will mark down every time they take a dose of the medicine in their clinical trial. They will use a form the researchers give them. The pill bottles they get in their trial will have a chip in the cap that will record when it is opened. Participants will keep a daily diary of their medicine. Their pills will be counted at clinical trial visits. Participants may have more short questionnaires. They may have interviews by phone or video.

NCT ID: NCT03518710 Not yet recruiting - Clinical trials for Neurofibromatosis Type 1

Vision, Attention and Reading in Neurofibromatosis Type 1 (NF1) Children

Start date: August 2018
Phase: N/A
Study type: Interventional

The present project will therefore focus upon those processes related to visual attention and perceptual abilities and on their potential to explain reading behavior and reading problems in NF1. The main objective of this study is to clarify the specificity and heterogeneity of reading profiles and the causes of its disturbance in NF1. In particular, this project allow the investigators to study more precisely the relations between perceptual, oculomotor and visuo-attentional skills in NF1 children and reading abilities. In addition, a new oculomotor/perceptual reading aid for NF1 children will be evaluated. The investigators believe that the early intervention for perceptual, visuo-attentional or oculomotor problems may promote academic skill development.

NCT ID: NCT03433183 Completed - Neurofibromatosis 1 Clinical Trials

SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors

Start date: October 2, 2019
Phase: Phase 2
Study type: Interventional

To determine the clinical benefit rate of selumetinib in combination with sirolimus in patients with unresectable or metastatic neurofibromatosis type 1 (NF1) associated or sporadic MPNST.

NCT ID: NCT03406208 Recruiting - Neurofibromatosis 1 Clinical Trials

Resiliency Training for Adults With Neurofibromatosis Via Live Videoconferencing

Start date: October 1, 2017
Phase: N/A
Study type: Interventional

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.

NCT ID: NCT03332030 Withdrawn - Clinical trials for Neurofibromatosis Type 1

Stem Cells in NF1 Patients With Tumors of the Central Nervous System

Start date: November 27, 2015
Phase:
Study type: Observational

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.

NCT ID: NCT03326388 Completed - Clinical trials for Neurofibromatosis Type 1

Intermittent Dosing Of Selumetinib In Childhood NF1 Associated Tumours

INSPECT
Start date: September 26, 2019
Phase: Phase 1/Phase 2
Study type: Interventional

Phase I and II study of the MEK inhibitor Selumetinib given twice daily on 5 out of 7 days in children with NF1 and inoperable plexiform neurofibromas or progressive/relapsed optic pathway gliomas. This study will test the early and late toxicities of selumetinib when it is given in this intermittent schedule (in 5 out of 7 days) and will also test the effectiveness of the drug in reducing the size of plexiform neurofibromas and optic pathway gliomas in children with NF1. It will also test the effectiveness of the drug in improving the participants function in day to day life.

NCT ID: NCT03310996 Completed - Neurofibromatosis 1 Clinical Trials

Non-invasive Stimulation in Neurofibromatosis Type 1

tDCS is NF1
Start date: October 13, 2017
Phase: N/A
Study type: Interventional

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.

NCT ID: NCT03298438 Completed - Clinical trials for Post Traumatic Stress Disorder

Study About Annoucement of the Diagnosis of Neurofibromatosis 1 in de Novo Forms

NF1
Start date: July 15, 2016
Phase: N/A
Study type: Observational

Neurofibromatosis type 1 (NF1) is one of the most common autosomal dominant genetic disorders. The aim of our study was to evaluate post-traumatic stress disorder (PTSD) in patients and their families following the disclosure of sporadic NF1. Diagnosis of NF1 was retained according to NIH criteria, familial forms were excluded. The French version of the Impact of Event Scale-Revised was used for the diagnosis of PTSD.

NCT ID: NCT03259633 Approved for marketing - Clinical trials for NF type1 With Inoperable Plexiform Neurofibromas

An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1

Start date: n/a
Phase:
Study type: Expanded Access

This will be an open-label, single-arm, multicenter intermediate access protocol which provides treatment access to selumetinib for eligible patients with neurofibromatosis type 1 (NF1) who have inoperable, progressive/symptomatic plexiform neurofibromas (PN) without any alternative therapeutic options. All patients will continue to receive drug while they are deriving clinical benefit. Approximately 100 patients in the US will be treated as part of this protocol

NCT ID: NCT03231306 Active, not recruiting - Clinical trials for Neurofibromatosis Type 1

Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas

NF108-BINI
Start date: November 28, 2017
Phase: Phase 2
Study type: Interventional

This is a phase II open label study that will evaluate children ≥ 1 year of age and adults with neurofibromatosis type 1 (NF1) and plexiform neurofibromas treated with the MEK inhibitor, binimetinib. The primary objective is to determine if there is an adequate level of disease responsiveness to binimetinib in children and adults with NF1 and inoperable plexiform neurofibromas. The objective response to binimetinib is defined as ≥ 20% decrease in tumor volume reduction by 12 courses.