Clinical trials: Neurofibromatosis
Bilateral Cochlear Aplasia, Bilateral Cochlear Nerve Deficiency
Feasibility Study of Auditory Brainstem Implant in Young Children
Current treatment options for bilateral profoundly deaf children, diagnosed with inner ear anatomical abnormalities are limited, and in the case of absent cochleas, non-existent.
- Conditions
- Bilateral Cochlear Aplasia, Bilateral Cochlear Nerve Deficiency, Bilateral Cochlear Ossification Secondary to Meningitis
- Trial name
- NCT01850225
- Testing
- Auditory brainstem implant
- Where
- United States
- Sponsor
- House Research Institute
Neurofibromatosis 1
Antioxidant Therapy With N-acetylcysteine for Children With Neurofibromatosis Type 1
Children with neurofibromatosis type 1 (NF1) commonly suffer from the effects of cognitive, behavioral, and motor impairments. At present, there is no specific treatment for this NF1 complication.
- Trial name
- DoDNAC (NCT04481048)
- Testing
- N-Acetyl cysteine
- Where
- United States
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
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
Age-Related Macular Degeneration
Study of Safety and Efficacy of Brolucizumab 6 mg Dosed Every 4 Weeks Compared to Aflibercept 2 mg Dosed Every 4 Weeks in Patients With Retinal Fluid Despite Frequent Anti-VEGF Injections
This clinical study was designed to compare the safety and efficacy of brolucizumab 6 mg dosed every 4 weeks to aflibercept 2 mg dosed every 4 weeks in those neovascular age-related macular degeneration (nAMD) patients with…
- Trial name
- MERLIN (NCT03710564)
- Testing
- Brolucizumab, Aflibercept
- Where
- 61 sites in United States, Puerto Rico
- Volunteers
- 535 people
- Sponsor
- Novartis Pharmaceuticals
Neurofibroma, Plexiform
Observational Study to Evaluate the Effect and Safety of Selumetinib in Pediatric Patients With NF1-PNs
This is a prospective, multicenter, observational study of Chinese pediatric NF1-PN patients treated with selumetinib. The study will be conducted at approximately 12 centers in China and will include approximately 80-100…
- Trial name
- PEDIA (NCT06175637)
- Where
- 25 sites in China
- Volunteers
- 409 people
- Sponsor
- AstraZeneca
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
Neurofibroma, Plexiform, Neurofibromatosis Type I
R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas
This study will examine whether the experimental drug R115777 (Tipifarnib) can shrink or slow the growth of plexiform neurofibromas in children and young adults with neurofibromatosis type 1 (NF1) and determine what side effects…
- Trial name
- NCT00021541
- Testing
- tipifarnib
- Where
- 12 sites in United States, Germany
- Volunteers
- 62 people
- Sponsor
- National Cancer Institute (NCI)
Acute Leukemia, Adenomatous Polyposis
Familial Investigations of Childhood Cancer Predisposition
NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll.
- Conditions
- Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML
- Trial name
- SJFAMILY (NCT03050268)
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- St. Jude Children's Research Hospital
RAS Mutation, NF1 Mutation
PAS-004 in Patients With Advanced Solid Tumors
The main purpose of this clinical trial is to test PAS-004 in people with advanced solid tumors with rat sarcoma virus (RAS), neurofibromatosis type I (NF1), or rapidly accelerated fibrosarcoma (RAF) mutations.
- Conditions
- RAS Mutation, NF1 Mutation, RAF Mutation, Advanced Solid Tumors
- Trial name
- NCT06299839
- Testing
- PAS-004 Capsules, PAS-004 Tablets
- Where
- 5 sites in United States, Bulgaria and 1 more
- Volunteers
- 83 people
- Sponsor
- Pasithea Therapeutics Corp.
Neurofibromatosis 1, Nerve Sheath Neoplasms
Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)
Background: Neurofibromatosis type 1 (NF1) is a genetic disease that can cause many symptoms. About half of people with NF1 will develop benign (noncancerous) tumors along nerves in the skin, brain, and other parts of the body.
- Trial name
- NCT06222203
- Where
- United States
- Volunteers
- 225 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis 1
Clinical Study on the Treatment of Type I Neurofibromatosis With Smeitinib Hydrosulfate Capsule
This study focused on patients with type I neurofibromatosis, who currently lack effective drug therapy and have a high recurrence rate after surgical resection.
- Trial name
- NCT06620354
- Testing
- Selumetinib
- Volunteers
- 33 people
- Sponsor
- Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
Neurofibromatosis 1
Evaluating Genetic Modifiers of Cutaneous Neurofibromas in Adults With Neurofibromatosis Type 1
The main goal of this protocol is to develop a well-phenotyped genetic biobank to identify genetic variants associated with the heterogeneous clinical presentations of Neurofibromatosis Type 1 (NF1).
- Trial name
- NCT04941027
- Where
- 2 sites in United States
- Volunteers
- 1,046 people
- Sponsor
- Stanford University
Parenting, Alopecia
Mindful Parenting - A Single Case Experimental Design Study
Some parents of children living with a visible difference can experience heightened stress due to the associated challenges of this. Parent's views of the child's visible difference and their responses to the child are important.
- Conditions
- Parenting, Alopecia, Skin Condition, Limb Difference
- Trial name
- NCT05779527
- Testing
- Mindful Parenting Programme
- Where
- United Kingdom
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Cardiff University
Neurofibromatosis 1, Social Skills
Pilot Randomized Control Trial of Telehealth Group for Improving Peer Relationships (PEERS) in NF1
This is a pilot randomized control trial of the UCLA PEERS protocol delivered via Telehealth with teens with neurofibromatosis type 1 whose parents report that they have difficulty making and keeping friends.
- Trial name
- NCT04860362
- Testing
- PEERS
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- University of Wisconsin, Milwaukee
Neurofibromatosis 1
A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1
The goal of this fully decentralized, randomized controlled trial is to compare the efficacy of two educational interventions for individuals with Neurofibromatosis 1 (NF1).
- Trial name
- NCT06262113
- Testing
- Letters about NF1 Care (Content Type 1), Letters about NF1 Care (Content Type 2)
- Where
- United States
- Volunteers
- 294 people
- Sponsor
- Massachusetts General Hospital
Neurofibromatosis Type 1
Selumetinib Paediatric NF1 Japan Study
This is a phase I open label study designed to evaluate the safety, tolerability, PK and efficacy of selumetinib in Japanese paediatric patients with neurofibromatosis type 1 and inoperable and symptomatic plexiform neurofibroma.
- Trial name
- NCT04495127
- Testing
- Selumetinib
- Where
- 3 sites in Japan
- Volunteers
- 12 people
- Sponsor
- AstraZeneca
NF2-related Schwannomatosis, NF2
Luvometinib in Combination With Serplulimab for NF2-Related Tumors
This is an investigator-initiated, exploratory, multicenter, open-label, single-arm clinical trial and a substudy of the Platform Research for Innovative Medicines in NF2-SWN (PRIME-NF2).
- Conditions
- NF2-related Schwannomatosis, NF2, Neurofibromatosis Type 2, Vestibular Schwannoma
- Trial name
- NCT07708285
- Testing
- Luvometinib, Serplulimab
- Where
- 5 sites in China
- Volunteers
- 30 people
- Sponsor
- Beijing Tiantan Hospital
Neurofibromatosis-1, Optic Glioma
PET/MRI in CNS and Extra-CNS Tumors of Patients With Neurofibromatosis-1 (NF1)
This prospective pilot study is designed to provide preliminary data on the use of Fluorodeoxyglucose Positron Emission Tomography-Magnetic Resonance Imaging (FDG-PET-MRI) in patients with neurofibromatosis-1 (NF1) associated…
- Conditions
- Neurofibromatosis-1, Optic Glioma, Plexiform Neurofibroma
- Trial name
- NCT01800032
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
Neurofibromatosis 1, Plexiform Neurofibroma (PN)
Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas
A global study to demonstrate the effectiveness of selumetinib in participants with NF1 who have symptomatic, inoperable plexiform neurofibromas.
- Trial name
- KOMET (NCT04924608)
- Testing
- Selumetinib
- Where
- 34 sites in United States, Australia and 11 more
- Volunteers
- 145 people
- Sponsor
- AstraZeneca
Neurofibromatosis 1
Evaluating Mitochondrial Dysfunction in Patients With Neurofibromatosis Type 1
Neurofibromatosis type 1 is a common genetic disease with a broad spectrum of clinical manifestations in multiple organs of the body.
- Trial name
- NCT05912400
- Testing
- Blood draw, FACIT-F and Pain Scales
- Where
- United States
- Volunteers
- 55 people, healthy volunteers welcome
- Sponsor
- University of Arkansas