Clinical trials: Neurofibromatosis 1, active
Neurofibromatosis Type 1, Neurofibromatosis 1
Natural History Study of Cutaneous Neurofibromas in People With NF1
People diagnosed with NF1 may develop cutaneous neurofibromas, also known as cNFs. These benign tumors can cause discomfort and affect a person's quality of life.
- Conditions
- Neurofibromatosis Type 1, Neurofibromatosis 1, Neurofibromatosis (Nonmalignant), Cutaneous Neurofibroma
- Trial name
- cNF Natural Hx (NCT05581511)
- Testing
- Evaluation of the natural history of cutaneous neurofibromas
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- Johns Hopkins University
Neurofibromatosis 1, Neurofibromatosis 2
The NF Registry Sponsored by the Children's Tumor Foundation
The NF Registry is a database of patient-reported symptoms, treatments, and experiences with their neurofibromatosis disease. It is a contact registry to relay clinical trial opportunities to targeted patient subgroups, and to…
- Conditions
- Neurofibromatosis 1, Neurofibromatosis 2, Schwannomatosis, NF2-related Schwannomatosis
- Trial name
- NCT01885767
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- The Children's Tumor Foundation
Neurofibromatosis Type 1 (NF1)
Observational Study of the Real-world Effectiveness and Safety of Selumetinib Treating Adult Patients With Neurofibromatosis Type 1 (NF1) Who Have Symptomatic, Inoperable Plexiform Neurofibroma (PN)
This is a prospective, multicentre, observational registry study with primary data collection designed to evaluate the real-world effectiveness and safety of selumetinib in adult patients with NF1 who have symptomatic, inoperable…
- Trial name
- PROCEED (NCT07888517)
- Where
- 26 sites in China
- Volunteers
- 800 people
- Sponsor
- AstraZeneca
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma.
- Conditions
- Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease, Cystic Fibrosis, Fragile X Syndrome
- Trial name
- DANNIgene (NCT06147414)
- Testing
- Blood sample
- Where
- France
- Volunteers
- 550 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Neurofibromatosis Type 1-Associated Plexiform Neurofibromas
A Study of Mirdametinib in Infants and Toddlers With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
The purpose of this study is to determine the safety, pharmacokinetic (PK), and tolerability of multiple treatment cycles of oral mirdametinib in infants and toddlers (0 to less than [<] 24 months of age) with measurable…
- Trial name
- NCT07850635
- Testing
- Mirdametinib
- Where
- Germany
- Volunteers
- 6 people
- Sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
Neurofibromatosis
Natural History Study of Patients With Neurofibromatosis Type 2
Objective With this prospective natural experiment trial on neurofibromatosis type 2 (NF2) study, we hope to understand the factors leading to tumor progression and neurological disease burden in NF2.
- Trial name
- NCT00598351
- Where
- United States
- Volunteers
- 269 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Neurofibromatosis Type 1, Malignant Peripheral Nerve Sheath Tumor
Natural History Study of Patients With Neurofibromatosis Type I
Background: Neurofibromatosis Type 1 (NF1) is a genetic disorder in which patients are at increased risk of developing tumors (usually non-cancerous) of the central and peripheral nervous system.
- Conditions
- Neurofibromatosis Type 1, Malignant Peripheral Nerve Sheath Tumor, Plexiform Neurofibroma, Optic Glioma
- Trial name
- NCT00924196
- Where
- United States
- Volunteers
- 259 people
- Sponsor
- National Cancer Institute (NCI)
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)
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 1
Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas
Background: NF1 is a genetic disease that causes tumors called atypical neurofibromas. These tumors, which arise from nerves, can cause serious medical problems. The only treatment is surgery.
- Trial name
- NCT04750928
- Testing
- Abemaciclib
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis 1, Noonan Syndrome
Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial
Background: RASopathies are a group of genetic diseases that affect a child s development. They cause physical, cognitive, and behavioral symptoms. Caring for a child with a RASopathy can be stressful.
- Conditions
- Neurofibromatosis 1, Noonan Syndrome, Legius Syndrome, Cardiofaciocutaneous Syndrome
- Trial name
- NCT05361811
- Testing
- Waitlist, ACT Intervention
- Where
- United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis Type 2
Doxycycline in Cutaneous Schwannoma (NF2)
In this research study the investigators want to learn more about an alternate, local treatment for skin schwannomas. Specifically, local doxycycline intra-tumoral injection will be performed as a potential treatment for…
- Trial name
- NCT05521048
- Testing
- Doxycycline Injection [Doxy]
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Massachusetts Eye and Ear Infirmary
Neurofibromatosis Type 1
A Study to Evaluate the Feasibility of a Physiologic Biomarker to Assess Pain and Other Sensory Problems Using Pupillometry in Participants With Neurofibromatosis Type 1 (NF1)
Background: Neurofibromatosis type 1 (NF1) is a genetic condition that causes tumors to grow along the nerves in the skin, brain, and other parts of the body.
- Trial name
- NCT06507748
- Testing
- AlgometRx Nociometer(R)
- Where
- 2 sites in United States
- Volunteers
- 70 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis Type 1, Neurofibroma
Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis Type 1: A Qualitative Study
Background: Neurofibromatosis 1 (NF1) is a disease that causes tumors to grow along the nerves. These include plexiform neurofibromas (pNF) and cutaneous neurofibromas (cNF). Both pNF and cNF can be visible to other people.
- Trial name
- NCT06880991
- Testing
- Arm 1
- Where
- United States
- Volunteers
- 110 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
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)
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 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
Sturge-Weber Syndrome, Neurocutaneous Syndromes
Neurocutaneous Syndromes in Children: Prevalence and Clinical Characteristics
This cross-sectional study aims to determine the prevalence, clinical characteristics, and management patterns of neurocutaneous syndromes among children attending the Pediatric Neurology Unit at Assiut University.
- Conditions
- Sturge-Weber Syndrome, Neurocutaneous Syndromes, Neurofibromatoses, Tuberous Sclerosis
- Trial name
- NCT07843823
- Volunteers
- 100 people
- Sponsor
- Assiut University