Clinical trials: Neurofibromatosis Type 1
RAS Mutation, Neurofibromatosis 1
RASopathy Biorepository
The RASopathies are a group of developmental disorders caused by genetic changes in the genes that compose the Ras/mitogen activated protein kinase (MAPK) pathway. New RASopathies are being diagnosed frequently.
- Conditions
- RAS Mutation, Neurofibromatosis 1, Noonan Syndrome, Noonan Syndrome With Multiple Lentigines
- Trial name
- NCT04395495
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Cutaneous Neurofibromas, Neurofibromatosis Type 1
Targeting the Mechanisms Underlying Cutaneous Neurofibroma Formation in NF1: A Clinical Translational Approach.
The purpose of the research study is intended to use specimens (such as tissue) and medical information in the Laboratory of Musculoskeletal Oncology at the Van Andel Research Institute for laboratory research in Grand Rapids…
- Conditions
- Cutaneous Neurofibromas, Neurofibromatosis Type 1, Plexiform Neurofibromas
- Trial name
- NCT02777775
- Testing
- Skin Biopsy
- Where
- United States
- Volunteers
- 71 people, healthy volunteers welcome
- Sponsor
- Corewell Health West
Plexiform Neurofibromas, NF1
Study of Plexiform Neurofibromas in Neurofibromatosis Type 1
Background: -Plexiform neurofibromas in patients with NF1 are a significant cause of morbidity but little is known about the natural history of these lesions.
- Trial name
- NCT00006435
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- National Cancer Institute (NCI)
Sickle Cell Disease, Solid Tumor
Validating Pain Scales in Children and Young Adults
Background: Assessing pain levels is important to improve treatments for different illnesses. Most pain rating scales are used to determine pain levels in adults. Pain is also a common symptom among children who have cancer.
- Conditions
- Sickle Cell Disease, Solid Tumor, Leukemia, Neurofibromatosis
- Trial name
- NCT01639950
- Where
- United States
- Volunteers
- 252 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis 2, Meningioma
AZD2014 In NF2 Patients With Progressive or Symptomatic Meningiomas
The goal of this clinical research study is to learn if the study drug AZD2014 can shrink growing or symptomatic meningiomas.
- Trial name
- NCT02831257
- Testing
- AZD2014
- Where
- 2 sites in United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Solid Tumor
PF-07284892 in Participants With Advanced Solid Tumors
The purpose of this first-in-patient, open label study is to determine the maximum tolerated dose and/or recommended dose for further study of PF-07284892 as a single agent and in combination with lorlatinib, encorafenib and…
- Trial name
- NCT04800822
- Testing
- PF-07284892, lorlatinib, binimetinib
- Where
- 11 sites in United States
- Volunteers
- 53 people
- Sponsor
- Pfizer
Bilateral Hearing Loss for Causes Other Than Tumors
Auditory Brainstem Implant (ABI) in Adult Non-Neurofibromatosis Type 2 Subjects
The purpose of this research study is to determine whether Auditory Brainstem Implant (ABI) can improve hearing in persons who are deaf in both ears and are not candidates for cochlear implants.
- Trial name
- NCT01736267
- Testing
- Nucleus 24 (discontinued) and ABI541 Auditory Brainstem Implants (ABI)
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Massachusetts Eye and Ear Infirmary
Neurofibromatosis Type 1, Plexiform Neurofibromas
Acceptance and Commitment Training for Adolescents and Young Adults With Neurofibromatosis Type 1, Plexiform Neurofibromas, and Chronic Pain
Background: People with neurofibromatosis type I (NF1) and plexiform neurofibroma (PN) tumors often have chronic pain that his hard to control.
- Trial name
- NCT02471339
- Testing
- Acceptance and Commitment Therapy (ACT)
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- National Cancer Institute (NCI)
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
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
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
Diffuse Astrocytoma, Anaplastic Astrocytoma
Pediatric Long-Term Follow-up and Rollover Study
A roll-over study to assess long-term effect in pediatric patients treated with dabrafenib and/or trametinib.
- Conditions
- Diffuse Astrocytoma, Anaplastic Astrocytoma, Astrocytoma, Oligodendroglioma, Childhood
- Trial name
- NCT03975829
- Testing
- dabrafenib, trametinib
- Where
- 54 sites in United States, Argentina and 17 more
- Volunteers
- 163 people
- Sponsor
- Novartis Pharmaceuticals
Ewing Sarcoma, Osteosarcoma
Eligibility Screening for a NCI Pediatric Oncology Branch Research Study
Patients who are being considered for participation in a NCI Pediatric Oncology Branch research study will be screened for eligibility under this protocol.
- Conditions
- Ewing Sarcoma, Osteosarcoma, Neuroblastoma, Acute Lymphoblastic Leukemia
- Trial name
- NCT00026780
- Where
- United States
- Volunteers
- 1,720 people
- Sponsor
- National Cancer Institute (NCI)
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)