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Neurofibromatoses clinical trials

View clinical trials related to Neurofibromatoses.

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NCT ID: NCT05825365 Withdrawn - Clinical trials for Neurofibromatosis Type 1

Selumetinib in Chinese Paediatric With Post-operative NF1-PNs, Phaseâ…¡, Double-Blinded, Placebo-Controlled Study

PROTECT
Start date: August 31, 2023
Phase: Phase 2
Study type: Interventional

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.

NCT ID: NCT03688568 Withdrawn - Clinical trials for Neurofibroma, Plexiform

Study of Imatinib in Children With Neurofibromatosis and Airway Tumors

Start date: September 1, 2018
Phase: Phase 2
Study type: Interventional

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

NCT ID: NCT03332030 Withdrawn - Clinical trials for Neurofibromatosis Type 1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NCT ID: NCT03109301 Withdrawn - Neurofibromatosis 1 Clinical Trials

Mitogen Activated Protein Kinase Kinase (MEK1/2) Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in People With Neurofibromatosis Type 1 (NF1) Mutated Gastrointestinal Stromal Tumors (GIST)

Start date: April 7, 2017
Phase: Phase 2
Study type: Interventional

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.

NCT ID: NCT01567787 Withdrawn - Neurofibroma Clinical Trials

Proton Radiation Therapy for Spinal Tumors

NF01
Start date: June 2013
Phase: N/A
Study type: Interventional

The purpose of this study is to determine if Proton Therapy can provide effective and safe treatment for Malignant Peripheral Nerve Sheath Tumors of the spine and Neurofibromas of the spine.