Clinical trials: Neurofibromatosis Type 1 (NF1)
Neurofibromatosis 1, Plexiform Neurofibroma
Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1
FCN-159 is a highly active MEK1/2 inhibitor that was designed, synthesized and screened on the basis of the structure of trametinib.
- Conditions
- Neurofibromatosis 1, Plexiform Neurofibroma, NF1
- Trial name
- NCT04954001
- Testing
- FCN-159
- Where
- 14 sites in United States, China and 1 more
- Volunteers
- 160 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Neurofibromatosis Type 1, Pediatric Brain Tumor
Neurofibromatosis Type 1 Brain Tumor Genetic Risk
This study will analyze DNA samples to determine associations between maternal and offspring genetic factors and pediatric brain tumor development in children with Neurofibromatosis Type 1.
- Trial name
- NCT01707836
- Where
- United States
- Volunteers
- 176 people
- Sponsor
- Washington University School of Medicine
Neurofibromatosis Type 2
Doxycycline in Cutaneous Schwannoma (NF2)
In this research study the investigators want to learn more about an alternate, local treatment for skin schwannomas. Specifically, local doxycycline intra-tumoral injection will be performed as a potential treatment for…
- Trial name
- NCT05521048
- Testing
- Doxycycline Injection [Doxy]
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Massachusetts Eye and Ear Infirmary
Neurofibromatosis 1, Neurofibroma, Plexiform
Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1(NF1) on Clinical Treatment Trials
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.
- Trial name
- NCT03531814
- Testing
- Medication Event Monitoring System (MEMS^TM), Questionnaires
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- National Cancer Institute (NCI)
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 (NF1)
Vitamin D Supplementation for Adults With Neurofibromatosis Type 1 (NF1)
This clinical trial is conducted by one of 4 locations; University of British Columbia (Vancouver, CA), University of Utah (Salt Lake City, UT, USA), University of Cincinnati (Cincinnati, OH, USA), and University of Hamburg…
- Trial name
- VitDBoneNF1 (NCT01968590)
- Testing
- Cholecalciferol
- Where
- 3 sites in United States, Canada
- Volunteers
- 26 people
- Sponsor
- University of Utah
Advanced Unresectable Melanoma, Metastatic Melanoma
Mirdametinib in Patients With Advanced NF1-mutant Melanoma
The goal of this clinical trial is to evaluate clinical efficacy of mirdametinib in patients with advanced NF1-mutant melanoma whose disease has progressed while on or after previous immunotherapy.
- Trial name
- NCT07237100
- Testing
- Mirdametinib
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Kevin Kim, MD
Neoplasms, Nerve Tissue, Neurofibromatosis 1
Mitogen Activated Protein Kinase Kinase (MEK1/2) Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in People With Neurofibromatosis Type 1 (NF1) Mutated Gastrointestinal Stromal Tumors (GIST)
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.
- Conditions
- Neoplasms, Nerve Tissue, Neurofibromatosis 1, Heredodegenerative Disorders, Nervous System, Peripheral Nervous System Diseases
- Trial name
- NCT03109301
- Testing
- Selumetinib (AZD6244 hyd sulfate) 50mg/dose, Selumetinib (AZD6244 hyd sulfate) 25mg/m2
- Sponsor
- National Cancer Institute (NCI)
Hypertension, Neurofibromatosis 1
Clinical Hypnosis and Home Blood Pressure Monitoring in Children With Neurofibromatosis Type 1
This study has two primary objectives. The first is to determine if it is feasible and reliable for children and families with a diagnosis of Neurofibromatosis Type 1 (NF1) to use of blood pressure (BP) monitor at home.
- Conditions
- Hypertension, Neurofibromatosis 1, Blood Pressure
- Trial name
- NCT05196854
- Testing
- Group 1. Standard Home Blood Pressure Measurement, Group 2. Hypnosis script prior to home blood pressure measurement
- Where
- Australia
- Volunteers
- 54 people
- Sponsor
- Murdoch Childrens Research Institute
Neurofibromatosis Type 1, Cutaneous Neurofibromas
Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1
The purpose of this research study is to determine if ranibizumab can prevent the growth of neurofibromas. We will also be collecting extra blood and serum samples to help us learn more about NF1.
- Trial name
- NCT00657202
- Testing
- Ranibizumab
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Massachusetts General Hospital
Neurofibromatosis 1
Dosing and Tolerability of Deoxycholic Acid vs. Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutaneous Neurofibromas
This study will evaluate the tolerability and effectiveness of two treatments in Neurofibromatosis Type 1 Cutaneous Neurofibromas. These treatments are: Kybella and Asclera injection.
- Trial name
- NCT06120036
- Testing
- Kybella, Asclera
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Massachusetts General Hospital
Low Grade Glioma (LGG) of Brain With BRAF Aberration, High Grade Glioma (HGG) of the Brain With BRAF Aberration
A Trial of Dabrafenib, Trametinib and Hydroxychloroquine for Patients With Recurrent LGG or HGG With a BRAF Aberration
This phase I/II trial is designed to study the side effects, best dose and efficacy of adding hydroxychloroquine to dabrafenib and/or trametinib in children with low grade or high grade brain tumors previously treated with…
- Conditions
- Low Grade Glioma (LGG) of Brain With BRAF Aberration, High Grade Glioma (HGG) of the Brain With BRAF Aberration, Low Grade Glioma of Brain With Neurofibromatosis Type 1
- Trial name
- NCT04201457
- Testing
- Dabrafenib, Trametinib, Hydroxychloroquine
- Where
- 15 sites in United States
- Volunteers
- 57 people
- Sponsor
- Pediatric Brain Tumor Consortium
NF1, Neurofibromatosis
Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults
This study, "A Phase II Study of Cabozantinib (XL l84) for Plexiform Neurofibromas in Subjects with Neurofibromatosis Type I in Children and Adults diagnosed with Neurofibromatosis Type 1 (NF1) and have a type of tumor called a…
- Conditions
- NF1, Neurofibromatosis, Plexiform Neurofibromas
- Trial name
- NF105-CABO (NCT02101736)
- Testing
- Cabozantinib
- Where
- 12 sites in United States
- Volunteers
- 45 people
- Sponsor
- University of Alabama at Birmingham
NF1, Low-grade Glioma
A Phase II Trial of Poly-ICLC for Low-Grade Gliomas
This is a phase II, prospective, longitudinal, multi-center trial of poly-ICLC (Hiltonol ®) treatment for progressive low-grade gliomas in pediatric patients with NF1.
- Trial name
- NF111 (NCT04544007)
- Testing
- Poly ICLC
- Where
- 12 sites in United States
- Volunteers
- 3 people
- Sponsor
- University of Alabama at Birmingham
Neurofibromatosis 1, Malignant Peripheral Nerve Sheath Tumor (MPNST) of Soft Tissue (Diagnosis)
Multi-parametric Biomarker Development to Predict Malignant Conversion in Patients With Neurofibromatosis Type 1
The goal of this prospective observational study is to learn about the utility of imaging and clinical features in patients with Neurofibromatosis type 1 categorized as high risk for the development of malignant peripheral nerve…
- Conditions
- Neurofibromatosis 1, Malignant Peripheral Nerve Sheath Tumor (MPNST) of Soft Tissue (Diagnosis), Atypical Neurofibroma
- Trial name
- NCT05677594
- Testing
- Whole Body Magnetic Resonance Imaging
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- Johns Hopkins University
Scoliosis; Idiopathic, Infantile, Scoliosis; Juvenile
Physical Activity, Motor Competence, Pulmonary Function, and Quality of Life in Children With Severe Spinal Disease
Observation study about physical activity, motor competence, pulmonary function, and health related quality of life in children, surgically treated for early onset scoliosis
- Conditions
- Scoliosis; Idiopathic, Infantile, Scoliosis; Juvenile, Scoliosis Neuromuscular, Scoliosis in Neurofibromatosis
- Trial name
- NCT03494829
- Where
- Norway
- Volunteers
- 50 people
- Sponsor
- Oslo University Hospital
Neurofibromatosis and Malignant Peripheral Nerve Sheath Tumors
A Clinical Trial to Evaluate the Safety and Efficacy of AL2846 Capsules in Chinese Patients With Type I Neurofibromatosis
AL2846 is a multi-target receptor tyrosine kinase inhibitor. The purpose of this study is to evaluate the safety and efficacy of AL2846 capsules in Chinese patients with type I neurofibromatosis (NF1) (neurofibromas and malignant…
- Trial name
- NCT05011019
- Testing
- AL2846 capsules
- Where
- China
- Volunteers
- 22 people
- Sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Neurofibromatosis 1
Trial to Evaluate the Safety of Lovastatin in Individuals With Neurofibromatosis Type I (NF1)
Neurofibromatosis type I (NF1) is a genetic disorder that affects approximately 1 in 3500 individuals. Half of people with NF1 inherit the condition from a parent, and half have a new occurrence of the condition.
- Trial name
- NF1 (NCT00352599)
- Testing
- Lovastatin
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- University of California, Los Angeles
Neurofibromatosis Type 1
Reading Disability in Children With NF1
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.
- Trial name
- NCT02397967
- Testing
- Neuropsychological assessments
- Where
- 6 sites in France
- Volunteers
- 181 people, healthy volunteers welcome
- Sponsor
- University Hospital, Toulouse
Neurofibromatosis Type 1
Prevalence of Constitutional Mismatch-repair Deficiency Among Suspected Neurofibromatosis Type 1/Legius Syndrome Children Without a Malignancy and Without a NF1 or SPRED1 Mutation
Frequency of constitutional mismatch-repair deficiency among suspected neurofibromatosis type 1 patients without a NF1 mutation Constitutional mismatch repair deficiency (CMMRD) is a rare inherited condition.
- Trial name
- CMMRD in NF1 (NCT03757247)
- Volunteers
- 670 people
- Sponsor
- Medical University Innsbruck