Clinical trials: Plexiform Neurofibroma
Neurofibromatosis Type 1
Selumetinib Paediatric NF1 Japan Study
This is a phase I open label study designed to evaluate the safety, tolerability, PK and efficacy of selumetinib in Japanese paediatric patients with neurofibromatosis type 1 and inoperable and symptomatic plexiform neurofibroma.
- Trial name
- NCT04495127
- Testing
- Selumetinib
- Where
- 3 sites in Japan
- Volunteers
- 12 people
- Sponsor
- AstraZeneca
Neurofibromatosis Type 1, Plexiform Neurofibroma
Use of RAD001 as Monotherapy in the Treatment of Neurofibromatosis 1 Related Internal Plexiform Neurofibromas
Background: Patients with the genetic disorder neurofibromatosis Type 1 (NF1) are at increased risk of developing tumors of the central and peripheral nervous system. These include plexiform neurofibromas.
- Conditions
- Neurofibromatosis Type 1, Plexiform Neurofibroma, Neurofibromatoses
- Trial name
- NFitor (NCT01412892)
- Testing
- RAD001: Everolimus
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Neurofibromatosis 1, Neurofibroma
Whole Body MRI to Identify Atypical Neurofibromas in Patients With NF1
This study is being conducted to determine if Whole Body MRI (WBMRI) can be used to identify Atypical Neurofibromas (ANF) in Neurofibromatosis Type 1 (NF1) patients with high tumor burden.
- Conditions
- Neurofibromatosis 1, Neurofibroma, Atypical Neurofibroma, Atypical Neurofibromatosis
- Trial name
- NCT03820778
- Testing
- Whole Body MRI
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Children's National Research Institute
Plexiform Neurofibromas
Study of Imatinib Mesylate in Neurofibromatosis Type I Patients Aged 2 to 21 With Plexiform Neurofibromas
This phase II trial will test the hypothesis that inhibition of c-kit signalling pathways in pediatric patients with Neurofibromatosis Type I(NF-1) and progressing plexiform neurofibroma will result in objective reduction and/or…
- Trial name
- NCT02177825
- Testing
- Imatinib Mesylate
- Where
- Canada
- Volunteers
- 5 people
- Sponsor
- St. Justine's Hospital
Neurofibromatosis 1, Neurofibroma, Plexiform
Real-World Treatment Study of Koselugo (Selumetinib)
As part of a post-approval commitment, the Korean health authority requests a study to characterize safety and effectiveness in patients treated with Koselugo (Selumetinib), an oral selective inhibitor of MAPK kinase (MEK) 1 and…
- Trial name
- NCT06360406
- Where
- 12 sites in South Korea
- Volunteers
- 200 people
- Sponsor
- AstraZeneca
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
Neurofibromatosis Type 1
Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN
This study is designed to define a dosing regimen and assess the pharmacokinetics(PK) and safety of the granule formulation; the study will also include descriptive analyses of exploratory efficacy endpoints.
- Trial name
- SPRINKLE (NCT05309668)
- Testing
- Selumetinib granule formulation, Selumetinib capsule formulation
- Where
- 16 sites in United States, Germany and 5 more
- Volunteers
- 36 people
- Sponsor
- AstraZeneca
Neoplasms, Nerve Tissue, Neurofibromatosis 1
Mitogen Activated Protein Kinase Kinase (MEK1/2) Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in People With Neurofibromatosis Type 1 (NF1) Mutated Gastrointestinal Stromal Tumors (GIST)
Background: Gastrointestinal stromal tumors (GIST) can cause serious medical problems. The only known treatment is surgery. But completely removing a GIST tumor with surgery is often not possible.
- Conditions
- Neoplasms, Nerve Tissue, Neurofibromatosis 1, Heredodegenerative Disorders, Nervous System, Peripheral Nervous System Diseases
- Trial name
- NCT03109301
- Testing
- Selumetinib (AZD6244 hyd sulfate) 50mg/dose, Selumetinib (AZD6244 hyd sulfate) 25mg/m2
- Sponsor
- National Cancer Institute (NCI)
Cancer
Study to Investigate Safety, Pharmacokinetic (PK), Pharmacodynamic (PD) and Clinical Activity of Trametinib in Subjects With Cancer or Plexiform Neurofibromas and Trametinib in Combination With Dabrafenib in Subjects With Cancers Harboring V600 Mutations
This was a 4-part (Part A, Part B, Part C and Part D), Phase I/IIa, multi-center, open label, study in pediatric subjects with refractory or recurrent tumors.
- Trial name
- NCT02124772
- Testing
- Trametinib, Dabrafenib
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 139 people
- Sponsor
- Novartis Pharmaceuticals
Neurofibromatosis Type 1, Precancerous Condition
Fludeoxyglucose F 18 Positron Emission Tomography and Magnetic Resonance Perfusion Imaging in Patients With Neurofibromatosis 1 and Plexiform Neurofibroma
RATIONALE: New imaging procedures such as fludeoxyglucose F 18 positron emission tomography (FDG-PET) and magnetic resonance (MR) perfusion imaging may improve the ability to detect disease progression, help doctors predict a…
- Trial name
- NCT00060008
- Testing
- fludeoxyglucose F 18, gadopentetate dimeglumine
- Where
- 2 sites in United States
- Volunteers
- 18 people
- Sponsor
- Children's Hospital of Philadelphia
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
Neurofibromatosis 1, Neurofibroma Plexiform
A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)
A Phase 1 Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Clinical Efficacy of Selumetinib, a Selective Mitogen Activated Protein Kinase Kinase (MEK) 1 Inhibitor, in Chinese Paediatric and Adult Subjects…
- Trial name
- NCT04590235
- Testing
- Selumetinib
- Where
- 2 sites in China
- Volunteers
- 32 people
- Sponsor
- AstraZeneca
Neurofibromatosis Type 1 and Growing or Symptomatic, Inoperable PN
MEK Inhibitor PD-0325901 Trial in Adolescents and Adults With NF1
This phase II open label study will evaluate adolescents (≥ 16 years of age) and adults with neurofibromatosis type-1 (NF1) and plexiform neurofibromas treated with the MEK inhibitor PD-0325901.
- Trial name
- MEK Inhibitor (NCT02096471)
- Testing
- PD-0325901
- Where
- 11 sites in United States
- Volunteers
- 19 people
- Sponsor
- University of Alabama at Birmingham
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
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)
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)
Neurofibroma, Plexiform
Study of Imatinib in Children With Neurofibromatosis and Airway Tumors
The purpose of this study is to look at a subset of plexiform neurofibromas and determine if the airway tumors are more sensitive to imatinib therapy. Funding Source - FDA OOPD
- Trial name
- NCT03688568
- Testing
- Imatinib Mesylate
- Where
- United States
- Sponsor
- Indiana University
Neurofibromatosis Type 1
Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Tolerability of Selumetinib in Adolescent Children With Neurofibromatosis Type 1 Related Plexiform Neurofibromas
This study in adolescent participants with NF1 who have inoperable PN is designed to evaluate the effect of a low fat meal on steady state selumetinib exposure; to assess the effect on GI tolerability when selumetinib is dosed…
- Trial name
- NCT05101148
- Testing
- Selumetinib
- Where
- 9 sites in United States, Poland and 2 more
- Volunteers
- 24 people
- Sponsor
- AstraZeneca
Neoplasm of Uncertain Malignant Potential, Unspecified Childhood Solid Tumor, Protocol Specific
PEG-Interferon Alfa-2b in Treating Young Patients With Plexiform Neurofibroma
RATIONALE: PEG-interferon alfa-2b may interfere with the growth of tumor cells. PURPOSE: This phase I trial is studying the side effects and best dose of PEG-interferon alfa-2b in treating young patients with plexiform…
- Trial name
- NCT00253474
- Testing
- PEG-interferon alfa-2a
- Where
- 4 sites in United States
- Volunteers
- 36 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
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.