Clinical trials: Neurofibromatoses
Sturge-Weber Syndrome, Neurocutaneous Syndromes
Neurocutaneous Syndromes in Children: Prevalence and Clinical Characteristics
This cross-sectional study aims to determine the prevalence, clinical characteristics, and management patterns of neurocutaneous syndromes among children attending the Pediatric Neurology Unit at Assiut University.
- Conditions
- Sturge-Weber Syndrome, Neurocutaneous Syndromes, Neurofibromatoses, Tuberous Sclerosis
- Trial name
- NCT07843823
- Volunteers
- 100 people
- Sponsor
- Assiut University
Neurofibromatosis 1, NF1
Reliability of Functional Outcome Measures in Neurofibromatosis 1
Neurofibromatosis 1 (NF1) is a common inherited condition that affects the skin, bone and nervous system. The complications of NF1 are widespread and can lead to varied difficulties dependent on the location of neurofibromas and…
- Trial name
- NCT02479360
- Where
- United Kingdom
- Volunteers
- 49 people
- Sponsor
- Guy's and St Thomas' NHS Foundation Trust
Neurofibromatosis Type 1
PASS of Paediatric Patients Initiating Selumetinib
Neurofibromatosis type 1 (NF1) is a rare, autosomal dominant genetic disorder that is caused by germline mutations in the NF1 tumour suppressor gene, which encodes the tumour suppressor protein neurofibromin 1.
- Trial name
- NCT05388370
- Where
- 46 sites in Austria, France and 8 more
- Volunteers
- 124 people
- Sponsor
- AstraZeneca
Neurofibromatosis 1, Plexiform Neurofibroma
Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1
Plexiform neurofibromas (PN) are known to cause significant morbidity in children with NF1. The recent FDA approval for selumetinib in children 2 years and older with inoperable symptomatic PN was based on the finding that…
- Trial name
- NF114 (NCT06188741)
- Testing
- Selumetinib
- Where
- 15 sites in United States
- Volunteers
- 200 people
- Sponsor
- University of Alabama at Birmingham
Whole Body Imaging, Magnetic Resonance Imaging
Whole Body Magnetic Resonance Imaging With Diffusion Weighted Imaging : Potential Role in Neurofibromatosis
Whole body MRI will be performed in patients with neurofibromatosis Type 1 PURPOSE 1: To determine the total tumor load (neurofibroma) and to diagnose plexiform neurofibromas or malignant peripheral nerve sheath tumors.
- Conditions
- Whole Body Imaging, Magnetic Resonance Imaging, Neurofibromatosis 1, Diffusion Magnetic Resonance Imaging
- Trial name
- NEFIMAR (NCT01777451)
- Testing
- Additional imaging or surgery
- Where
- Belgium
- Volunteers
- 30 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Neurofibromatosis Type 2, Vestibular Schwannoma
Innovative Trial for Understanding the Impact of Targeted Therapies in NF2-Related Schwannomatosis (INTUITT-NF2)
This is a multi-arm phase II platform-basket screening study designed to test multiple experimental therapies simultaneously in patients with NF2-related schwannomatosis (NF2-SWN, formerly known as neurofibromatosis type 2) with…
- Conditions
- Neurofibromatosis Type 2, Vestibular Schwannoma, Non-vestibular Schwannoma, Meningioma
- Trial name
- INTUITT-NF2 (NCT04374305)
- Testing
- Brigatinib, Neratinib, Retifanlimab
- Where
- 6 sites in United States
- Volunteers
- 109 people
- Sponsor
- Scott R. Plotkin, MD, PhD
Inherited Epidermolysis Bullosa, Ichthyosis
FARD (RaDiCo Cohort) (RaDiCo-FARD)
The goal of this observational study is to conduct a prospective assessment of the individual Burden of 9 rare skin diseases to assess disability in the broadest sense of the term (psychological, social, economic and physical)…
- Conditions
- Inherited Epidermolysis Bullosa, Ichthyosis, Ectodermal Dysplasia, Incontinentia Pigmenti
- Trial name
- FARD (NCT05954416)
- Where
- 15 sites in France
- Volunteers
- 900 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Developmental Cochlear Nerve Deficiency, Acquired Cochlear Nerve Deficiency
Auditory Brainstem Implantation in Young Children
To purpose of this feasibility study is to demonstrate the safety and efficacy of the Nucleus 24 Multichannel Auditory Brainstem Implant (ABI, Cochlear Corp, Sydney, AUS) in children without the diagnosis of neurofibromatosis…
- Conditions
- Developmental Cochlear Nerve Deficiency, Acquired Cochlear Nerve Deficiency, Cochlear Aplasia, Post-meningitis Cochlear Ossification
- Trial name
- NCT01904448
- Testing
- Auditory Brainstem Implantation in Children
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- University of North Carolina, Chapel Hill
Neurofibromatosis Type 1
Reliability of Functional Outcome Measures in Neurofibromatosis 1: Test- Retest
To examine the test-retest reliability (how stable the results are when the same participants, whose symptoms have remained stable, are assessed on 2 different occasions, 14 days apart) of the 10 meter walk test, the timed up and…
- Trial name
- NCT05186870
- Testing
- Outcome measures
- Where
- United Kingdom
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Guy's and St Thomas' NHS Foundation Trust
Neurofibromatosis Type 1
A Study to Evaluate the Feasibility of a Physiologic Biomarker to Assess Pain and Other Sensory Problems Using Pupillometry in Participants With Neurofibromatosis Type 1 (NF1)
Background: Neurofibromatosis type 1 (NF1) is a genetic condition that causes tumors to grow along the nerves in the skin, brain, and other parts of the body.
- Trial name
- NCT06507748
- Testing
- AlgometRx Nociometer(R)
- Where
- 2 sites in United States
- Volunteers
- 70 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatoses
Resiliency Training for Adolescent Neurofibromatosis Patients Via Videoconferencing With Skype
The aim of this research is to adapt the NF-specific adult version of the Skype 3RP for use with adolescents, and to test its feasibility, acceptability, and preliminary effect in improving quality of life, and in decreasing…
- Trial name
- NCT02387177
- Testing
- Stress Management Group 1, Stress Management Group 2
- Where
- United States
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Neurofibromatosis Type 1
Vision, Attention and Reading in Neurofibromatosis Type 1 (NF1) Children
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.
- Trial name
- NCT03518710
- Testing
- Neuropsychological evaluation, Evaluation of the reading assistance technique
- Where
- France
- Volunteers
- 15 people
- Sponsor
- University Hospital, Toulouse
Neurofibromatosis
Clinical and Demographic Characteristics of Adult p Atients With NEurofibromatosis in RUSsia
Clinical and Demographic Characteristics of Adult Patients with NEurofibromatosis in RUSsia (NEREUS)
- Trial name
- NEREUS (NCT07088991)
- Where
- 16 sites in Russia
- Volunteers
- 200 people
- Sponsor
- AstraZeneca
Neurofibromatosis Type 1
Analysis of Peripheral Nerve Sheath Tumors (PNSTs) in Neurofibromatosis Type 1 (NF1) Patients
Neurofibromatosis type 1 (NF1) is a frequent, autosomal dominant disorder caused by heterozygous mutations (intragenic or microdeletion) of the NF1 tumor suppressor gene (chr.17q11.2).
- Trial name
- NCT01218139
- Where
- Belgium
- Volunteers
- 50 people
- Sponsor
- KU Leuven
Neurofibromatosis 1
Medical Treatment of "High-Risk" Neurofibromas
Patients with neurofibromatosis type 1 (NF1) commonly develop non-cancerous tumors called plexiform neurofibromas. These tumors can be defined as "high-risk" when they result in severe pain, physical disability, organ dysfunction…
- Trial name
- NCT00846430
- Testing
- Peg-Interferon alpha-2b, Celecoxib (Celebrex), Temozolomide (temodar)
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Corewell Health West
Neurofibroma
Non-interventional Study of Patients With PN NF1 Starting Selumetinib in Russia
The purpose of this study is to evaluate the effectiveness, safety of selumetinib and patient's parameters of symptomatic inoperable PN NF1 in real clinical practice in Russia
- Trial name
- NCT05891847
- Where
- 5 sites in Russia
- Volunteers
- 150 people
- Sponsor
- AstraZeneca
Neurofibromatosis Type 1, Reading Disabilities
Neurobiology and Treatment of Reading Disability in NF-1
The goal of this trial is to determine if children with neurofibromatosis type 1 who have reading disabilities respond the same way-both behaviorally and neurobiologically-to specialized treatment programs as children with…
- Trial name
- NCT00624234
- Testing
- Tutoring Program I, Tutoring Program II
- Where
- United States
- Volunteers
- 184 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University
Neurofibromatosis
Phase II Study of Gleevec/Imatinib Mesylate (STI-571, NCS 716051) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas
THe primary objective is to estimate the response rate at 6 months to Gleevec® in patients with plexiform neurofibromas
- Trial name
- NCT01673009
- Testing
- Gleevec
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Indiana University
Low Grade Astrocytoma, Low Grade Glioma
A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma
This phase III trial compares the effect of selumetinib versus the standard of care treatment with carboplatin and vincristine (CV) in treating patients with newly diagnosed or previously untreated low-grade glioma (LGG) that…
- Conditions
- Low Grade Astrocytoma, Low Grade Glioma, Metastatic Low Grade Astrocytoma, Metastatic Low Grade Glioma
- Trial name
- NCT04166409
- Testing
- Biospecimen Collection, Carboplatin, Echocardiography Test
- Where
- 132 sites in United States, Canada and 1 more
- Volunteers
- 170 people
- Sponsor
- National Cancer Institute (NCI)
Leukemia, Juvenile Myelomonocytic, JMML
A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia
This clinical trial will test the safety and efficacy of combining trametinib and azacitidine in patients with juvenile myelomonocytic leukemia (JMML).
- Conditions
- Leukemia, Juvenile Myelomonocytic, JMML, JCML, Neurofibromatosis 1
- Trial name
- NCT05849662
- Testing
- Trametinib, Azacitidine, Fludarabine
- Where
- 21 sites in United States
- Volunteers
- 58 people
- Sponsor
- Therapeutic Advances in Childhood Leukemia Consortium