Clinical trials: Neurofibromatoses
Neurofibromatosis 1, Neurofibroma, Plexiform
Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas
Background: Neurofibromatosis Type 1 (NF1) is an autosomal dominant, progressive genetic disorder characterized by diverse clinical manifestations.
- Trial name
- NCT00076102
- Testing
- Pirfenidone
- Where
- 15 sites in United States
- Volunteers
- 36 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis Type 1, Malignant Peripheral Nerve Sheath Tumor
Natural History Study of Patients With Neurofibromatosis Type I
Background: Neurofibromatosis Type 1 (NF1) is a genetic disorder in which patients are at increased risk of developing tumors (usually non-cancerous) of the central and peripheral nervous system.
- Conditions
- Neurofibromatosis Type 1, Malignant Peripheral Nerve Sheath Tumor, Plexiform Neurofibroma, Optic Glioma
- Trial name
- NCT00924196
- Where
- United States
- Volunteers
- 259 people
- Sponsor
- National Cancer Institute (NCI)
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 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
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
Neurofibromatoses
Phase I Photodynamic Therapy (PDT) for Benign Dermal Neurofibromas (NF1)
GENERAL OBJECTIVE The general objective is to assess the safety and efficacy of photodynamic therapy (PDT) in the treatment of neurofibromatosis 1 (NF1) tumors in the skin.
- Trial name
- NCT01682811
- Testing
- Part 1 Levulan injection, Part 1 Levulan surface application, Part 1 Levulan surface application twice
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Harry T Whelan, MD
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)
Bilateral Hearing Loss for Causes Other Than Tumors
Auditory Brainstem Implant (ABI) in Pediatric Non-Neurofibromatosis Type 2 Subjects
The purpose of this research study is to determine whether Auditory Brainstem Implant (ABI) can improve hearing in children who are deaf and cannot receive a cochlear implants.
- Trial name
- ABI (NCT01864291)
- Testing
- Nucleus 24 (discontinued) and ABI541 Auditory Brainstem Implants (ABI)
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Massachusetts Eye and Ear Infirmary
Neurofibromatosis Type I, Tuberous Sclerosis Complex
The Cognitive Variability in NF1 and TSC Monozygotic Twins
Both Neurofibromatosis type 1 (NF1) and Tuberous Sclerosis Complex (TSC) are highly heterogeneous diseases. Cognitive features seem to vary widely even between family members carrying the same mutation.
- Trial name
- COVANTT (NCT02436746)
- Where
- Netherlands
- Volunteers
- 116 people
- Sponsor
- Erasmus Medical Center
NEUROFIBROMATOSIS 1
Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II
The investigators wish to determine the time to disease progression for benign neurofibromas treated with Levulan Kerastick topical photosensitizer and red light photodynamic therapy (PDT) in patients with neurofibromatosis type…
- Trial name
- NCT02728388
- Testing
- aminolevulinic acid
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Donald Basel
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
Neurofibromatosis 2
PTC299 for Treatment of Neurofibromatosis Type 2
Formation of new blood vessels (angiogenesis) is important for tumor growth in neurofibromatosis type 2 (NF2). It is known that tumors make a protein called vascular endothelial growth factor (VEGF) and there are higher levels of…
- Trial name
- NCT00911248
- Testing
- PTC299
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- PTC Therapeutics
Neurofibromatosis Type 1, Learning Disability
Interventions for Reading Disabilities in NF1
Neurofibromatosis Type 1 (NF1) is a common genetic disorder that is associated with a four times greater risk of learning disabilities, including reading disabilities, and a deficiency of neurofibromin - a protein important in a…
- Conditions
- Neurofibromatosis Type 1, Learning Disability, Reading Disability, NF1
- Trial name
- NCT02964884
- Testing
- Lovastatin, reading tutoring intervention
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University
Brain Tumor, Neurofibromatoses
Quality Improvement Project - "My Logbook! - I Know my Way Around!"; ("Mein Logbuch - Ich Kenne Mich Aus!")
In this study, the main goal is to implement and evaluate a novel, evidence-based psycho-educative program for children in oncological care. Patients are provided with booklets tailored to each specific stage of their treatment.
- Conditions
- Brain Tumor, Neurofibromatoses, Sarcoma, Leukemia
- Trial name
- NCT04474678
- Testing
- "My Logbook! - I know my way around!"
- Where
- 22 sites in Austria, Germany and 2 more
- Volunteers
- 240 people
- Sponsor
- Medical University of Vienna
Neurofibromatosis Type 1-Associated Plexiform Neurofibromas
A Study of Mirdametinib in Infants and Toddlers With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
The purpose of this study is to determine the safety, pharmacokinetic (PK), and tolerability of multiple treatment cycles of oral mirdametinib in infants and toddlers (0 to less than [<] 24 months of age) with measurable…
- Trial name
- NCT07850635
- Testing
- Mirdametinib
- Where
- Germany
- Volunteers
- 6 people
- Sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
Neurofibromatosis Type 2, Neuroma, Acoustic
Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma
The purpose of the study is to determine if RAD001 treatment will shrink or slow the growth of the vestibular schwannoma(s) in Neurofibromatosis 2 (NF2) patients.
- Trial name
- NCT01345136
- Testing
- RAD001, everolimus
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Jonsson Comprehensive Cancer Center
NF1 Mutation, Neurofibroma Plexiform
PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas
The main purpose of this clinical trial is to test PAS-004 in people with at least one symptomatic plexiform neurofibroma due to Neurofibromatosis Type 1 (NF1).
- Conditions
- NF1 Mutation, Neurofibroma Plexiform, Neurofibroma, Plexiform, Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs)
- Trial name
- NCT06961565
- Testing
- PAS-004 Tablets, PAS-004 Capsule
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 56 people
- Sponsor
- Pasithea Therapeutics Corp.
Neurofibromatosis Type 1
Multi-center Project: Spinal Abnormalities in Neurofibromatosis Type1 (NF1) Patients
We propose to establish a multi-center study to investigate the outcome of scoliosis and spinal abnormalities in patients with NF1.
- Trial name
- NCT00667836
- Where
- United States
- Volunteers
- 300 people
- Sponsor
- Shriners Hospitals for Children
Neurofibroma, Neurofibroma, Plexiform
Evaluation of Percutaneous Cryotherapy in the Treatment of Plexiform Neurofibromas and Unresectable Neurofibromas in Neurofibromatosis Type 1
Neurofibromatosis type 1 (NF1) is an autosomal dominant disease affecting chromosome 17. It is manifested by a neurogenic tumor proliferation that forms cutaneous, subcutaneous or deep neurofibromas.
- Trial name
- CryoNF1 (NCT05199376)
- Testing
- Cryotherapy
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Centre Leon Berard
Neurofibromatosis 1
Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas
Background: NF1 is a genetic disease that causes tumors called atypical neurofibromas. These tumors, which arise from nerves, can cause serious medical problems. The only treatment is surgery.
- Trial name
- NCT04750928
- Testing
- Abemaciclib
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- National Cancer Institute (NCI)