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Found 10 clinical trials
  • 25 Oct, 2022
  • 26 locations
A Phase I/II, Single-Arm, Open Label Study to Evaluate the Pharmacokinetics, Safety/Tolerability and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With Neurofibromatosis Type 1 (NF1) Related Symptomatic, Inoperable Plexiform Neurofibromas (PN) (SPRINKLE) (SPRINKLE)

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. The study will inform the benefit risk profile of the granule formulation in children aged ≥ 1 to < 7 …

  • 13 Oct, 2022
  • 7 locations
Pegintron to Treat Plexiform Neurofibromas in Children and Young Adults

inoperable plexiform neurofibroma that has the potential to cause significant morbidity. Design: Patients will be enrolled on one of three strata depending on disease status and age

  • 07 Nov, 2020
  • 1 location
HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas

This is a Multi-center, Open-label, Single-arm Phase II Study to Evaluate the Efficacy and Safety of HL-085 in the treatment of Adult Participants with Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas(PN)

measurable disease
nf1 mutation
  • 28 Apr, 2022
  • 1 location
Trametinib for Pediatric Neuro-oncology Patients With Refractory Tumor and Activation of the MAPK/ERK Pathway.

This is a phase 2, open-label, interventional clinical trial that will study the response rate of pediatric glioma and plexiform neurofibroma (PN) to oral administration of trametinib. Patients

serum pregnancy test
brain tumor
biologic agent
platelet count
colony stimulating factor
  • 09 May, 2022
  • 6 locations
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. FCN-159 is an orally available and highly potent selective inhibitor of MEK1/2, which is expected to be a targeted therapy for the treatment of advanced solid tumors and neurofibromatosis …

solid tumors
  • 07 Oct, 2022
  • 7 locations
Intermittent Dosing Of Selumetinib In Childhood NF1 Associated Tumours (INSPECT)

Phase I and II study of the MEK inhibitor Selumetinib given twice daily on 5 out of 7 days in children with NF1 and inoperable plexiform neurofibromas or progressive/relapsed optic pathway gliomas. This study will test the early and late toxicities of selumetinib when it is given in this intermittent …

measurable disease
ejection fraction
gilbert disease
renal function
  • 27 Feb, 2022
  • 1 location
Phase I Trial of Turalio(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN)

Background Some people with cancer have solid tumors. Others have refractory leukemia. This may not go away after treatment. Researchers want to see if a drug called Turalio(R) can shrink tumors or stop them from growing. Objectives To find the highest safe dose and side effects of Turalio(R). To see …

measurable disease
ewing's sarcoma
malignant peripheral nerve sheath tumor
neutrophil count
  • 06 Apr, 2023
  • 1 location
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. This study aims to improve questionnaires that measure their pain, daily living, and physical functioning. Objectives To examine and improve questionnaires about daily living for people with NF1 and pNFs. Eligibility …

Accepts healthy volunteers
  • 11 Apr, 2023
  • 1 location
Developing Biomarkers of Plexiform Tumor Burden in Patients With Neurofibromatosis-Type 1

process such as progress of a disease or treatment. Individuals with this diagnosis are at an elevated risk of developing a type of tumor called a plexiform neurofibroma. Currently, detecting the risk

  • 15 Mar, 2022
  • 1 location