Clinical trials: Neurofibromatoses
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
Cutaneous Neurofibromas
First Clinical Study of Erbium - Yttrium Aluminium Garnet (YAG) Laser Vaporization of Cutaneous Neurofibromas
The purpose of this study is to determine whether multiple cutaneous neurofibromas in patients with neurofibromatosis type 1 can be removed with an erbium-YAG-laser.
- Trial name
- NCT00921037
- Testing
- Erbium-YAG laser vaporization
- Where
- Austria
- Volunteers
- 12 people
- Sponsor
- Medical University of Vienna
Plexiform Neurofibroma
Study of PEG-Intron for Plexiform Neurofibromas
This study is for slow growing tumors called plexiform neurofibromas (PNF) which are a relatively common problem in people with neurofibromatosis type 1 (NF1).
- Trial name
- NCT00396019
- Testing
- PEG-Intron
- Where
- United States
- Volunteers
- 86 people
- Sponsor
- University of Pittsburgh
Neurofibromatosis Type 1
Gait in Young Children With NF1
The objective of this study is to develop office-based tools to quantify gait in young children with NF1 that reflect overall gross motor impairment and predict future gross motor difficulties.
- Trial name
- NCT07564622
- Volunteers
- 56 people
- Sponsor
- NYU Langone Health
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
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
Identification of Pre-Malignant Lesions In Pediatric Patients With Neurofibromatosis Type 1 Using Novel Magnetic Resonance Imaging Techniques Paired With Artificial Intelligence
This is a single arm pilot trial of a novel whole-body Magnetic Resonance Imaging paired with artificial intelligence intervention, to evaluate feasibility defined as scan-rescan reliability, and to estimate the positive…
- Trial name
- NCT04763109
- Testing
- Whole-body Magnetic Resonance Imaging
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Nicole Baca
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
Neurofibromatosis 1, Social Skills
Open Trial of Telehealth Group for Improving Peer Relationships (PEERS) in NF1
This is an open 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
- NCT04856514
- Testing
- PEERS
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- University of Wisconsin, Milwaukee
Neurofibromatosis 1, Plexiform Neurofibromas
Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)
Background: People with neurofibromatosis 1 (NF1) who have plexiform neurofibromas (pNFs) can have pain that affects their daily lives.
- Trial name
- NCT02544022
- Where
- 6 sites in United States
- Volunteers
- 476 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis 1
Systematically Assessing Changes in Plexiform Neurofibroma Related Disfigurement From Photographs of Subjects With Neurofibromatosis Type 1 on a Phase 2 Clinical Trial
Background: People with Neurofibromatosis type 1 (NF1) have an increased risk of developing plexiform neurofibromas (PNs). PNs are tumors that form in the tissue. They can form anywhere in the body.
- Trial name
- NCT04879160
- Where
- United States
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis Type 1, Legius Syndrome
Study of Disease Severity in Adults With Neurofibromatosis Type 1 (NF1)
This study may identify genes that predict the seriousness of neurofibromatosis type 1 (NF1). Finding these genes may explain why some people with NF1 have more medical problems than others.
- Trial name
- NCT00111384
- Where
- United States
- Volunteers
- 313 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis Type 2, NF2
Selumetinib for NF2-Related Schwannomatosis
The goal of this clinical trial is to evaluate the efficacy and safety of the MEK1/2 inhibitor selumetinib in treating patients with neurofibromatosis type 2-related schwannomatosis (NF2-SWN), including both adults and children…
- Conditions
- Neurofibromatosis Type 2, NF2, NF2-related Schwannomatosis, Meningioma
- Trial name
- ASSIST (NCT07707947)
- Testing
- Selumetinib
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Beijing Tiantan Hospital
Neurofibromatosis Type 1
Neuropsychological Impairment and Quality of Life in Neurofibromatosis Type 1
The main objective of the study is to investigate the determinants of the quality of life in children and adults with Neurofibromatosis type 1 (NF1) and more particularly the specific impact of neuropsychological deficits.
- Trial name
- NF1-QDV (NCT01851135)
- Testing
- Neuropsychological examination and quality of life measures
- Where
- 2 sites in France
- Volunteers
- 286 people, healthy volunteers welcome
- Sponsor
- Nantes University Hospital
Neurofibromatosis, Neurofibromas
Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas
This is a second Pilot Study to determine the efficacy of Gleevec® in neurofibromatosis (NF1) patients with plexiform neurofibromas using new response assessment modalities with the secondary goals of assessing Gleevec toxicity…
- Trial name
- 0908-09 (NCT01140360)
- Testing
- Gleevec
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Kent Robertson
Neurofibromatosis
Acceptance and Commitment Therapy for Adolescents and Young Adults With Neurofibromatosis and Chronic Pain
Background: Neurofibromatosis type 1 (NF1) is a genetic disorder that can cause frequent and significant pain. This pain can have a severe impact on a person s quality of life.
- Trial name
- NCT01633008
- Testing
- ACT Workshop
- Where
- United States
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Cancer
Immunotherapy Based on Antigen-specific Immune Effector Cells Targeting Neurofibromatosis or Schwannomatosis
The primary objective of this study is to verify the safety of antigen-specific T cells (CAR-T) and engineered immune effector cytotoxic T cells (EIE) modified by immunoregulatory genes and immune modified dendritic cell vaccine…
- Trial name
- NCT04085159
- Testing
- Antigen-specific T cells CART/CTL and DCvac
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Low-grade Gliomas
Tarceva/Rapamycin for Children With Low-grade Gliomas With or Without Neurofibromatosis Type 1 (NF1)
This study will evaluate the feasibility of combining two drugs, Tarceva (an anti-EGFR agent), and Rapamycin (an mTOR inhibitor), in children with progressive low-grade gliomas who have failed initial conventional treatment.
- Trial name
- NCT00901849
- Testing
- Tarceva and Rapamycin
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Roger Packer
Headache, Tumor
Dexmedetomidine and Propofol for Pediatric MRI Sedation
The purpose of this study is to compare the results of combining two anesthetic medications (dexmedetomidine and propofol) in low doses with a standard dose of a single drug that is commonly used to provide sedation/anesthesia…
- Conditions
- Headache, Tumor, Seizure Disorder, Neurofibromatoses
- Trial name
- NCT03513757
- Testing
- propofol, Dexmedetomidine, Glycopyrrolate
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Medical College of Wisconsin
Neurofibromatosis 1, Plexiform Neurofibroma
Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas
Based on the clinical activity of both selumetinib and cabozantinib as monotherapies in clinical trials, the demonstrated activity of these agents in reduced doses in preclinical studies, and the non-overlapping toxicity…
- Trial name
- NF113 (NCT06502171)
- Testing
- Cabozantinib Oral Tablet, Selumetinib Oral Capsule
- Where
- 8 sites in United States
- Volunteers
- 30 people
- Sponsor
- Girish Dhall, MD