Clinical trials: Neurofibromatoses
Neurofibromatosis Type 1, Tibial Dysplasia
Neurofibromatosis Type 1 (NF1) and Tibial Dysplasia
The study is a multicenter four-year outcome study of the natural history of tibial dysplasia in patients with NF1 and selected patients without NF1.
- Trial name
- NF1TD (NCT00303368)
- Where
- United States
- Volunteers
- 395 people
- Sponsor
- Shriners Hospitals for Children
Schwannoma, Vestibular, Neurofibromatosis 2
NF2 Natural History Consortium
OBJECTIVES: I. Define the growth rates and clinical course of NF2-related tumors in patients with neurofibromatosis type 2. Associate growth rate with physical function.
- Conditions
- Schwannoma, Vestibular, Neurofibromatosis 2, Meningioma
- Trial name
- NCT00004483
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- House Ear 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
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
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)
Neurofibromatosis 1
Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in 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
- NF1NAC (NCT04481035)
- Testing
- N-acetylcysteine (NAC)
- Where
- United States
- Volunteers
- 5 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Neurofibromatoses, Neurofibromatosis 1
Resiliency Training for Adults With Neurofibromatosis Via Live Videoconferencing
The aims of this study are to compare the effect and durability of two stress and symptom management programs tailored for patients with neurofibromatosis on quality of life and psychosocial functioning.
- Conditions
- Neurofibromatoses, Neurofibromatosis 1, Neurofibromatosis 2, Schwannomatosis
- Trial name
- NCT03406208
- Testing
- Stress and Symptom Management Program 1, Stress and Symptom Management Program 2
- Where
- United States
- Volunteers
- 228 people
- Sponsor
- Massachusetts General Hospital
Neurofibromatosis 1
iCanCope With NF: Innovating an Efficacious Digital Self-management and Transitional Care Program for Adolescents With Neurofibromatosis
Neurofibromatosis Type 1 (NF1) often significantly impairs the mental health, social life, self-esteem, and quality of life of adolescent patients. These impacts can be made worse with the experience of NF1-related pain.
- Trial name
- NCT07077408
- Testing
- iCanCope-NF Youth app, iCanCope-NF Youth app + CM
- Volunteers
- 60 people
- Sponsor
- The Hospital for Sick Children
Neurofibromatosis 1
Tailoring an Online Platform to Promote Evidence-Based Care for Adults With Neurofibromatosis 1 and Low Health Literacy
This decentralized, randomized study seeks to assess the feasibility, acceptability, and preliminary effectiveness of two approaches to assisting Neurofibromatosis 1 (NF1) patients with low health literacy improve their…
- Trial name
- NCT07233408
- Testing
- NF1 Educational Videos, Peer Navigator, Letters about NF1 Care
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Massachusetts General Hospital
Neurofibromatosis 2, Dermatology/Skin - Other
Cutaneous and Mucosal Manifestations of Neurofribromatosis Type 2 in Children Under 15
.Neurofibromatosis type 2 is an inherently autosomal dominant genetic disease, but cases of mosaicism or de novo mutation are not uncommon. the prevalence is estimated at 1 / 60,000.
- Trial name
- NCT03893643
- Testing
- no intervention
- Where
- France
- Volunteers
- 1,000 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
CNS Cancer, Meningioma
Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable Meningioma
The purpose of this study is to find out what effects, good and/or bad, sunitinib has on patients and their tumors. At this time, no drugs are routinely used to treat meningioma, hemangioblastoma or hemangiopericytoma.
- Conditions
- CNS Cancer, Meningioma, Intracranial Hemangiopericytoma, Hemangioblastoma
- Trial name
- NCT00589784
- Testing
- Sunitinib
- Where
- 6 sites in United States
- Volunteers
- 50 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Neurofibromatosis Type 1, Plexiform Neurofibromas
US Selumetinib Registry
The purpose of this study is to understand treatment patterns and assess long-term effectiveness and safety outcomes associated with selumetinib treatment as well as to explore clinical and non-clinical factors affecting those…
- Trial name
- NCT05683678
- Where
- 10 sites in United States
- Volunteers
- 37 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Vestibular Schwannoma, Acoustic Neuroma
Study of Aspirin in Patients With Vestibular Schwannoma
This is a phase II prospective, randomized, double-blind, longitudinal study evaluating whether the administration of aspirin can delay or slow tumor growth and maintain or improve hearing in VS patients.
- Conditions
- Vestibular Schwannoma, Acoustic Neuroma, Neurofibromatosis 2
- Trial name
- NCT03079999
- Testing
- Aspirin
- Where
- 5 sites in United States
- Volunteers
- 97 people
- Sponsor
- Massachusetts Eye and Ear Infirmary
Neurofibromatosis Type 1, Brain Tumor
Feasibility and Clinically Application of Magnetic Resonance Fingerprinting
This study will look at the feasibility of using magnetic resonance fingerprinting (MRF) in children, adolescents and young adults (AYA) with and without brain tumors.
- Conditions
- Neurofibromatosis Type 1, Brain Tumor, Glioma
- Trial name
- NCT02387840
- Testing
- Magnetic Resonance Imaging, Magnetic Resonance Fingerprinting
- Where
- United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Case Comprehensive Cancer Center
Plexiform Neurofibromas
Study of Imatinib Mesylate in Neurofibromatosis Type I Patients Aged 2 to 21 With Plexiform Neurofibromas
This phase II trial will test the hypothesis that inhibition of c-kit signalling pathways in pediatric patients with Neurofibromatosis Type I(NF-1) and progressing plexiform neurofibroma will result in objective reduction and/or…
- Trial name
- NCT02177825
- Testing
- Imatinib Mesylate
- Where
- Canada
- Volunteers
- 5 people
- Sponsor
- St. Justine's Hospital
NF1, Congenital Pseudarthrosis of Tibia
A Study of INFUSE Bone Graft (BMP-2) in the Treatment of Tibial Pseudarthrosis in Neurofibromatosis Type 1 (NF1)
The current study proposes adding BMP-2 (INFUSE), an anabolic agent, at the surgical site of TPA (tibial pseudarthrosis) repair in children with NF1, compared to a control group of patients treated surgically without BMP-2.
- Trial name
- NF107-BMP2 (NCT02718131)
- Testing
- INFUSE Bone Graft (BMP-2), Control Group
- Where
- 16 sites in United States, Australia
- Volunteers
- 5 people
- Sponsor
- University of Alabama at Birmingham
Neurofibromatosis Type 1, Precancerous Condition
Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas
RATIONALE: Chemoprevention therapy is the use of certain drugs to try to prevent the development or recurrence of cancer. Pirfenidone may slow the growth or prevent further development of plexiform neurofibromas.
- Trial name
- NCT00053937
- Testing
- pirfenidone
- Where
- 15 sites in United States
- Sponsor
- National Cancer Institute (NCI)
Neurofibromas, Cutaneous, Neurofibromatosis 1
Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs)
The goal of this clinical trial is to evaluate the tolerability and effectiveness of multiple treatments of an FDA-approved drug in those with Neurofibromatosis Type 1 (NF1) Cutaneous Neurofibromas (cNFs).
- Trial name
- NCT06300502
- Testing
- Kybella
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Massachusetts General Hospital
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
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)