Clinical trials: Neurofibromatosis Type I
Neurofibromatosis Type 1, Neurofibroma
Natural History and Biology of Skin Neurofibromas in Neurofibromatosis Type 1
This study will explore the growth of dermal neurofibromas (skin tumors) in patients with neurofibromatosis type 1 (NF1). Investigators will try to learn: 1) how fast (or slow) these benign tumors grow in NF1, 2) how often new…
- Trial name
- NCT00314119
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- National Cancer Institute (NCI)
Vestibular Schwannoma, Meningioma
Exploratory Evaluation of AR-42 Histone Deacetylase Inhibitor in the Treatment of Vestibular Schwannoma and Meningioma
This will be a multi-center, proof of concept phase 0 study to assess the suppression of p-AKT in Vestibular Schwannoma (VS) and meningiomas by AR-42 in adult patients undergoing tumor resection.
- Conditions
- Vestibular Schwannoma, Meningioma, Acoustic Neuroma, Neurofibromatosis Type 2
- Trial name
- NCT02282917
- Testing
- AR-42
- Where
- 4 sites in United States
- Volunteers
- 7 people
- Sponsor
- Massachusetts Eye and Ear Infirmary
Neurofibromatosis Type 2, Vestibular Schwannoma
Innovative Trial for Understanding the Impact of Targeted Therapies in NF2-Related Schwannomatosis (INTUITT-NF2)
This is a multi-arm phase II platform-basket screening study designed to test multiple experimental therapies simultaneously in patients with NF2-related schwannomatosis (NF2-SWN, formerly known as neurofibromatosis type 2) with…
- Conditions
- Neurofibromatosis Type 2, Vestibular Schwannoma, Non-vestibular Schwannoma, Meningioma
- Trial name
- INTUITT-NF2 (NCT04374305)
- Testing
- Brigatinib, Neratinib, Retifanlimab
- Where
- 6 sites in United States
- Volunteers
- 109 people
- Sponsor
- Scott R. Plotkin, MD, PhD
NF1 Mutation, Neurofibroma Plexiform
PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas
The main purpose of this clinical trial is to test PAS-004 in people with at least one symptomatic plexiform neurofibroma due to Neurofibromatosis Type 1 (NF1).
- Conditions
- NF1 Mutation, Neurofibroma Plexiform, Neurofibroma, Plexiform, Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs)
- Trial name
- NCT06961565
- Testing
- PAS-004 Tablets, PAS-004 Capsule
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 56 people
- Sponsor
- Pasithea Therapeutics Corp.
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 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)
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 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
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 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 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)
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 Type 1-Associated Plexiform Neurofibromas, Histiocytic Neoplasm
Individual Patient Compassionate Use of Mirdametinib
This program is being offered on a patient by patient basis and will require company, Institutional Review Board/Independent Ethics Committee, and applicable competent authority approval.
- Conditions
- Neurofibromatosis Type 1-Associated Plexiform Neurofibromas, Histiocytic Neoplasm, Other MAP-K Pathway Driven Diseases
- Trial name
- NCT05028166
- Testing
- Mirdametinib (MEK Inhibitor)
- Where
- 2 sites in United States, Germany
- Sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
This phase I/II trial studies the side effects and the best dose of selumetinib and how well it works in treating or re-treating young patients with low grade glioma that has come back (recurrent) or does not respond to treatment…
- Conditions
- Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma, Recurrent Neurofibromatosis Type 1, Recurrent Visual Pathway Glioma
- Trial name
- NCT01089101
- Testing
- Biospecimen Collection, Selumetinib
- Where
- 17 sites in United States
- Volunteers
- 217 people
- Sponsor
- National Cancer Institute (NCI)
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
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
Neurofibromatosis Type 1
Prevalence, Clinical Characteristics, Progression, and Management of Neurofibromatosis Type 1 in Egypt (NF1-Egy)
Neurofibromatosis type 1 (NF1), a genetic disorder, results from NF1 gene mutations with nearly complete penetrance (1). NF1 is considered common as a rare disease; it has a birth incidence of approximately one every 3000 and a…
- Trial name
- PRECIOUS Egypt (NCT07221331)
- Where
- 9 sites in Egypt
- Volunteers
- 200 people
- Sponsor
- AstraZeneca
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