Clinical trials: NF1
Neurofibromatosis 1, Neurofibromatosis Type 1
AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors
Background: Plexiform neurofibromas are tumors that grow in and around nerves. The only way to treat them is with surgery. Some of these tumors cannot be completely removed.
- Conditions
- Neurofibromatosis 1, Neurofibromatosis Type 1, NF1, Neurofibroma, Plexiform
- Trial name
- NCT01362803
- Testing
- AZD6244
- Where
- 4 sites in United States
- Volunteers
- 99 people
- Sponsor
- National Cancer Institute (NCI)
LGCMN, Large and Giant Congenital Melanocytic Nevi
Mirdametinib to Reduce the Size of Large/Giant Congenital Melanocytic Nevi in Children, Adolescents and Young Adults
Background: Large and giant congenital melanocytic nevi (LGCMN) are dark birthmarks (moles). They can grow to 8 inches or more across.
- Conditions
- LGCMN, Large and Giant Congenital Melanocytic Nevi, NF1, Solid Tumor
- Trial name
- NCT07891286
- Testing
- Mirdametinib
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis 1 (NF1), Executive Function (Cognition)
Cognitive Intervention for Children and Adolescents With NF1
Psychological interventions have been shown to be effective for managing cognitive and mental health challenges in different patient populations.
- Conditions
- Neurofibromatosis 1 (NF1), Executive Function (Cognition), Cognitive Intervention
- Trial name
- NCT07869914
- Testing
- Unstuck and On Target!
- Volunteers
- 60 people
- Sponsor
- The Hospital for Sick Children
Plexiform Neurofibroma, Neurofibromatosis Type 1 (NF1)
MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
This study evaluates mirdametinib (PD-0325901) in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive mirdametinib (PD-0325901).
- Trial name
- ReNeu (NCT03962543)
- Testing
- Mirdametinib (PD-0325901) oral capsule or dispersible tablet
- Where
- 43 sites in United States
- Volunteers
- 114 people
- Sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
Neurofibromatosis 1 (NF1)
Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma
This is a phase 2, open label, parallel multi-arm study of mirdametinib in combination with radiation in participants with recurrent sporadic glioblastoma (GBM) harboring NF1 alterations (Cohort 1); participants with NF1 with a…
- Trial name
- NF118 (NCT07521657)
- Testing
- mirdametinib with radiation, mirdametinib
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- University of Alabama at Birmingham
Neurofibromatosis Type 1 (NF1), NF2-related Schwannomatosis
How Can the Experience of Hospital Outpatient Departments be Improved?
This project aims to improve outpatient departments by working with patients and staff to make these spaces more inclusive for people with sensory challenges (it focuses on people with nerve tumour conditions as they are more…
- Trial name
- NCT07818213
- Testing
- Qualitative data collection activities
- Where
- United Kingdom
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- King's College London
Neurofibromatosis 1, NF1
Developing Biomarkers of Plexiform Tumor Burden in Patients With Neurofibromatosis-Type 1
The purpose of this study is to identify tumor biomarkers in individuals with Neurofibromatosis type 1 (NF1). Biomarkers are signals that the investigator can measure that tell us about a process such as progress of a disease or…
- Conditions
- Neurofibromatosis 1, NF1, Neurofibromatosis Type 1
- Trial name
- NCT05238909
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
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
Refractory Cancer, CNS Tumors
A Study of Avutometinib for People With Solid Tumor Cancers
The purpose of this study is to find out whether avutometinib is a safe treatment for advanced or recurrent solid tumor cancers in children and young adults.
- Conditions
- Refractory Cancer, CNS Tumors, CNS Tumor, Adult, CNS Tumor, Childhood
- Trial name
- NCT06104488
- Testing
- Avutometinib
- Where
- 2 sites in United States
- Volunteers
- 3 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Hematologic Malignancy, Acute Leukemia
MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies
This is a phase II, open-label, prospective study of T cell receptor alpha/beta depletion (TCR α/β TCD) peripheral blood stem cell (PBSC) transplantation for children and adults with hematological malignancies.
- Conditions
- Hematologic Malignancy, Acute Leukemia, Remission, Acute Myeloid Leukemia
- Trial name
- NCT05735717
- Testing
- Fludarabine, Busulfan, Melphalan
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Neurocutaneous Syndromes, Neurofibromatosis Type 1 (NF1)
Precision Medicine for Neurocutaneous Syndromes in Western China
The goal of this observational study (retrospective multicenter cohort study) is to learn if precision medicine approaches-including genetic testing, targeted drugs, and coordinated care from multiple specialists-can improve…
- Conditions
- Neurocutaneous Syndromes, Neurofibromatosis Type 1 (NF1), Tuberous Sclerosis Complex (TSC), Sturge-Weber Syndrome (SWS)
- Trial name
- NCT07327164
- Where
- China
- Volunteers
- 1,200 people
- Sponsor
- West China Hospital
Neurofibromatosis 1, Plexiform Neurofibroma
Follow-up Study to Evaluate the Safety and Efficacy of FCN-159 in Pediatric Participants With Neurofibromatosis Type 1
FCN-159 (Luvometinib Tablets), an orally available and highly potent selective inhibitor of MEK1/2,demonstrated good tolerability and exhibited notable anti-tumor activity in pediatric pts with NF1-related PN in study…
- Conditions
- Neurofibromatosis 1, Plexiform Neurofibroma, NF1
- Trial name
- NCT07024394
- Testing
- Luvometinib Tablets
- Where
- China
- Volunteers
- 65 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
NF1, Low-grade Glioma
A Phase II Trial of Poly-ICLC for Low-Grade Gliomas
This is a phase II, prospective, longitudinal, multi-center trial of poly-ICLC (Hiltonol ®) treatment for progressive low-grade gliomas in pediatric patients with NF1.
- Trial name
- NF111 (NCT04544007)
- Testing
- Poly ICLC
- Where
- 12 sites in United States
- Volunteers
- 3 people
- Sponsor
- University of Alabama at Birmingham
Neurofibromatosis 1, Plexiform Neurofibroma
Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1
FCN-159 is a highly active MEK1/2 inhibitor that was designed, synthesized and screened on the basis of the structure of trametinib.
- Conditions
- Neurofibromatosis 1, Plexiform Neurofibroma, NF1
- Trial name
- NCT04954001
- Testing
- FCN-159
- Where
- 14 sites in United States, China and 1 more
- Volunteers
- 160 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
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
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 1 and 2 (NF1 and NF2)
A Single Center Pilot Study to Assess the Intra-observer Reliability of Measuring Muscle Strength Using a Hand Held Dynamometer in Children and Adults With Neurofibromatosis Type 1 (NF1) and Type 2 (NF2)
Patients with clinically confirmed neurofibromatosis type 1 (NF1) or neurofibromatosis type 2 (NF2) or a known neurofibromatosis (NF) mutation aged 5 years and above will be eligible to participate and will be recruited from the…
- Trial name
- NCT03672838
- Testing
- hand-held dynamometer
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- NYU Langone Health
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)
Neurofibromatosis, NF1
Study of Sutent®/Sunitinib (SU11248) in Subjects With NF-1 Plexiform Neurofibromas
This is a pilot study to determine if adults and children with neurofibromatosis type 1 who have plexiform tumors given Sutent® respond to this drug therapy.
- Conditions
- Neurofibromatosis, NF1, Plexiform Neurofibromas
- Trial name
- NCT01402817
- Testing
- Sutent®/Sunitinib
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Indiana University
neurofibromatosis1 (NF1), Recurrent or Progressive Optic Pathway Gliomas (OPG)
Sorafenib in Children and Young Adults With Recurrent or Progressive Low-Grade Astrocytomas
The purpose of this study is to determine if a drug called sorafenib can shrink LGA tumors (low-grade astrocytomas) in children and adults.
- Conditions
- neurofibromatosis1 (NF1), Recurrent or Progressive Optic Pathway Gliomas (OPG), Recurrent or Progressive Low-grade Glioma
- Trial name
- NCT01338857
- Testing
- Sorafenib (Nexavar)
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- NYU Langone Health