Clinical trials: Neurofibromatosis 1
Grade 1 Glioma, Mixed Glio-neuronal Tumors
Pediatric Low Grade Glioma - MEKinhibitor TRIal vs Chemotherapy
Pediatric low-grade glioma (PLGG) is a heterogeneous group of WHO grade I and II brain tumors, associated with a 10-year overall survival of 90%.
- Conditions
- Grade 1 Glioma, Mixed Glio-neuronal Tumors, Pleomorphic Xanthoastrocytoma
- Trial name
- PLGG - MEKTRIC (NCT05180825)
- Testing
- Trametinib, Vinblastine
- Where
- 25 sites in France
- Volunteers
- 134 people
- Sponsor
- University Hospital, Strasbourg, France
Neurofibromatosis Type 2
Efficacy and Safety of REC-2282 in Patients With Progressive Neurofibromatosis Type 2 (NF2) Mutated Meningiomas
This is a two-staged, Phase 2/3, randomized, multi-center study to investigate the efficacy and safety of REC-2282 in participants with progressive NF2 mutated meningiomas.
- Trial name
- POPLAR-NF2 (NCT05130866)
- Testing
- REC-2282
- Where
- 15 sites in United States, United Kingdom
- Volunteers
- 25 people
- Sponsor
- Recursion Pharmaceuticals Inc.
Neurofibromatosis 1
Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T
This trial is evaluating the use of oral Everolimus to determine if there is a reduction in the size of the disfiguring cutaneous lesions in patients with Neurofibromatosis 1 over a 6 month period.
- Trial name
- DCLNF1 (NCT02332902)
- Testing
- Everolimus
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- The University of Texas Health Science Center, Houston
Neurofibromatosis Type 1, Cutaneous Neurofibromas
Topical Imiquimod 5% Cream for Treatment of Cutaneous Neurofibromas in Adults With Neurofibromatosis 1
The purpose of this study is to determine if imiquimod cream can reverse the growth of neurofibromas. Imiquimod is a skin cream that works by stimulating the body's immune system to respond to tumors.
- Trial name
- NCT00865644
- Testing
- Imiquimod 5% Cream
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Massachusetts General Hospital
Neurofibromatosis 1 (NF1), Plexiform Neurofibromas (PN)
MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas
Background: Neurofibromatosis type 1 (NF1) is a disorder that can cause plexiform neurofibromas (PNs). These are tumors that grow along nerves. Some PNs cause serious health problems.
- Trial name
- NCT02407405
- Testing
- Selumetinib
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- National Cancer Institute (NCI)
Cutaneous Neurofibroma
DPCP to Treat Cutaneous Neurofibromas Associated With NF1
Neurofibromatosis type 1 (NF1) is the most common genetic tumor predisposition syndrome, affecting up to 1 in 2500 individuals. Cutaneous neurofibromas are benign with self-limited growth; however, tumor burden may be excessive…
- Trial name
- NCT05438290
- Testing
- DPCP
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Nicholas Gulati
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.
Autism Spectrum Disorder, Neurofibromatosis 1
From Molecules to Cognition: Inhibitory Mechanisms in ASD and NF1
This study aims to investigate synaptic physiology and behavioral inhibition in patients with NF1 and ASD and to answer whether inhibitory deficits at these levels are modulated by lovastatin.
- Trial name
- ASD/NF1inhib (NCT03826940)
- Testing
- Lovastatin 60 MG
- Where
- Portugal
- Volunteers
- 16 people
- Sponsor
- University of Coimbra
Neurofibromatosis 2
Resiliency Training for Patients With NF2 Via Videoconferencing With Skype
The aim of this study is to determine, whether the 3RP is feasible, accepted and efficacious and durable when delivered via Skype to patients with neurofibromatosis type 2 (NF2) who are deaf or have severe hearing loss using…
- Trial name
- NCT02811718
- Testing
- Stress Management Group 1, Stress Management Group 2
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- Massachusetts General Hospital
Neurofibromatosis 1
Mechanism of Action of Transcranial Direct Current Stimulation in Neurofibromatosis Type 1
Intellectual impairment, particularly working memory deficits are a significant cause of morbidity in children with Neurofibromatosis type (NF1) with long-term implications on academic and occupational functioning.
- Trial name
- NCT04991428
- Testing
- Transcranial direct current stimulation
- Where
- United Kingdom
- Volunteers
- 31 people
- Sponsor
- University of Manchester
Cutaneous Neurofibroma, Neurofibromatosis 1
NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)
This is a randomized, double-blind, vehicle-controlled, parallel group dose response study evaluating the safety and effectiveness of 2 concentrations of NFX-179 gel in participants with cNF.
- Trial name
- NCT05005845
- Testing
- NFX-179 gel, Vehicle gel
- Where
- 23 sites in United States
- Volunteers
- 199 people
- Sponsor
- NFlection Therapeutics, Inc.
Vestibular Schwannoma, Neurofibromatosis Type 2
Icotinib Study for Patients With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors
1)Preliminarily evaluate the treatment effect of Icotinib Hydrochloride Tablets on NF2; 2)Preliminarily evaluate the safety and the patient's tolerance of the treatment of Icotinib; 3)Provide an objective basis for an enlarged…
- Trial name
- Icotinib (NCT02934256)
- Testing
- Icotinib
- Where
- 2 sites in China
- Volunteers
- 10 people
- Sponsor
- Li Peng
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
Neurofibromatosis Type 1
Function of the Pigment Epithelium in Patients With Type 1 Neurofibromatosis
The objective of this study is to study the function of the pigment epithelium in patients with neurofibromatosis type 1 using electro-oculogram to confirm abnormally high values reported in previous studies, but also to…
- Trial name
- NEF-1 (NCT04153344)
- Testing
- Electro-oculogram, Full-field electroretinogram
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Neurofibromatosis Type 1, Von Recklinghausen Disease
Adaptation and Quality of Life Among Adults With Neurofibromatosis Type I
This study aims to understand predictors of adaptation and quality of life among adults affected with neurofibromatosis type 1 (NF1) and autosomal dominant neurocutaneous condition.
- Conditions
- Neurofibromatosis Type 1, Von Recklinghausen Disease, NF1
- Trial name
- NCT00684398
- Where
- United States
- Volunteers
- 645 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Neurofibromatosis
A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I
The purpose of this tria is to evaluate the safety and efficacy of long-term treatment with NPC-12G gel to patients with neurofibromatosis type I.
- Trial name
- NCT04461886
- Testing
- NPC-12G gel
- Where
- 4 sites in Japan
- Volunteers
- 100 people
- Sponsor
- Nobelpharma
Neurofibromatosis Type 1
A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1
The specific aim of this study is to determine whether Lovastatin ™ significantly improves visual spatial learning and/or sustained attention in children with NF1.
- Trial name
- STARS (NCT00853580)
- Testing
- Lovastatin ™
- Where
- 12 sites in United States, Australia
- Volunteers
- 146 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
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
Inherited Epidermolysis Bullosa, Ichthyosis
FARD (RaDiCo Cohort) (RaDiCo-FARD)
The goal of this observational study is to conduct a prospective assessment of the individual Burden of 9 rare skin diseases to assess disability in the broadest sense of the term (psychological, social, economic and physical)…
- Conditions
- Inherited Epidermolysis Bullosa, Ichthyosis, Ectodermal Dysplasia, Incontinentia Pigmenti
- Trial name
- FARD (NCT05954416)
- Where
- 15 sites in France
- Volunteers
- 900 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Neurofibromatosis 1, Child
Treatment of NF1-related Plexiform Neurofibroma With Trametinib
This trial, Treatment of NF1-related plexiform neurofibroma with trametinib; a single arm,open-label study with the goals of volumetric partial remission and pain relief (EudraCT 2018-001846-32, Sponsor protocol number BUS2018-1…
- Conditions
- Neurofibromatosis 1, Child, Neurofibroma, Plexiform
- Trial name
- plexifpc (NCT03741101)
- Testing
- Trametinib
- Where
- Sweden
- Volunteers
- 15 people
- Sponsor
- Region Skane