View clinical trials related to Neurofibromatosis 1.
Filter by: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.
The purpose of this study is to look at a subset of plexiform neurofibromas and determine if the airway tumors are more sensitive to imatinib therapy. Funding Source - FDA OOPD
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.
Background: Gastrointestinal stromal tumors (GIST) can cause serious medical problems. The only known treatment is surgery. But completely removing a GIST tumor with surgery is often not possible. Researchers want to see if a new drug, selumetinib, can help treat these tumors. Objective: To find out if selumetinib shrinks or slows the growth of GIST tumors and to see its side effects. Eligibility: People ages 3 and over who have one or more GIST tumors and may have neurofibromatosis type I (also called NF1). Their NF1 GIST has shown some growth or cannot be completely removed with surgery. Design: Participants will be screened with heart and eye tests and scans. Participants will be told what foods and medicines they cannot take during the study. Participants will keep a diary of the medicine they take during the study. Participants will take selumetinib capsules twice daily on an empty stomach for 28 days in a row. This is 1 cycle. During the cycles, participants will have study visits. These may include: Medical history Physical exam Blood and urine tests Heart tests Scans of their tumors Eye exam Positron emission tomography scan. They will be get radioactive glucose an IV line. They will lie quietly in a darkened room for 50-60 minutes then have the scan. Participants will answer questions about how they are feeling. Participants can stay in the study until they have bad side effects or their tumor grows. After finishing treatment, participants will be watched for side effects for 30 days.