Clinical trials: Neurofibromatosis Type I
Neurofibromatosis 1, Plexiform Neurofibroma
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.
- Conditions
- Neurofibromatosis 1, Plexiform Neurofibroma, NF1
- Trial name
- NCT04954001
- Testing
- FCN-159
- Where
- 14 sites in United States, China and 1 more
- Volunteers
- 160 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Neurofibromatosis 1, Plexiform Neurofibroma
A Retrospective Study on Epidemiological Characteristics of Chinese NF1 Patients in Real World (PROMISE)
Background/Rationale: Neurofibromatosis type 1 (NF1) affects about 1 in every 3000 people worldwide. Globally, 30~50% NF1 patients will develop plexiform neurofibromas (PNs), which grow rapidly in early childhood and can cause…
- Trial name
- NCT06379230
- Testing
- This study is purely descriptive study.
- Where
- 5 sites in China
- Volunteers
- 2,000 people
- Sponsor
- Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University
Glioma
Everolimus for Children With NF1 Chemotherapy-Refractory Radiographic Progressive Low Grade Gliomas
The purpose of this research study is to learn if the study drug RAD001 can shrink or slow the growth of low-grade gliomas in children with Neurofibromatosis type 1 (NF1). Additionally, the safety of RAD001 will be studied.
- Trial name
- NFC-RAD001 (NCT01158651)
- Testing
- RAD001 (Everolimus)
- Where
- 12 sites in United States
- Volunteers
- 23 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
Neurofibromatosis Type 1
Ophthalmological Screening and Follow-up of Optic Pathway Gliomas in Children With Neurofibromatosis Type 1.
The goal of this project is to get more insight into the (neuro)ophthalmological characteristics of children with neurofibromatosis type 1.
- Trial name
- NCT02078401
- Where
- Belgium
- Volunteers
- 200 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Neurofibromatosis Type 1, Tumors of the Central Nervous System
Stem Cells in NF1 Patients With Tumors of the Central Nervous System
Objectives 1. Establish an induced pluripotent stem cell (iPSC) bank for phenotypically well-characterized patients with NF1. 2.
- Trial name
- NCT03332030
- Testing
- Collection of Stem Cells
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- Roger Packer
Neurofibromatosis 1
Non-invasive Stimulation in Neurofibromatosis Type 1
Intellectual impairments are a significant cause of morbidity in children with birth defects along with long term implication on academic and occupational functioning.
- Trial name
- tDCS is NF1 (NCT03310996)
- Testing
- Transcranial direct current stimulation
- Where
- United Kingdom
- Volunteers
- 16 people
- Sponsor
- University of Manchester
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
Brain and Central Nervous System Tumors, Neurofibromatosis Type 1
Vinblastine and Carboplatin in Treating Young Patients With Newly Diagnosed or Recurrent Low-Grade Glioma
RATIONALE: Drugs used in chemotherapy, such as vinblastine and carboplatin, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00352495
- Testing
- carboplatin, vinblastine sulfate
- Where
- 21 sites in United States, Canada
- Volunteers
- 26 people
- Sponsor
- Children's Oncology Group
Low-grade Glioma, Plexiform Neurofibroma
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.
- Conditions
- Low-grade Glioma, Plexiform Neurofibroma, Central Nervous System Glioma
- Trial name
- NCT03363217
- Testing
- Trametinib
- Where
- 7 sites in Canada
- Volunteers
- 114 people
- Sponsor
- St. Justine's Hospital
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
This phase II trial studies how well trametinib works in treating patients with juvenile myelomonocytic leukemia that has come back (relapsed) or does not respond to treatment (refractory).
- Trial name
- NCT03190915
- Testing
- Bone Marrow Aspiration and Biopsy, Trametinib
- Where
- 57 sites in United States
- Volunteers
- 10 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatoses, Neurofibromatosis 1
Resiliency Training for Adults With Neurofibromatosis Via Live Videoconferencing
The aims of this study are to compare the effect and durability of two stress and symptom management programs tailored for patients with neurofibromatosis on quality of life and psychosocial functioning.
- Conditions
- Neurofibromatoses, Neurofibromatosis 1, Neurofibromatosis 2, Schwannomatosis
- Trial name
- NCT03406208
- Testing
- Stress and Symptom Management Program 1, Stress and Symptom Management Program 2
- Where
- United States
- Volunteers
- 228 people
- Sponsor
- Massachusetts General Hospital
Neurofibromatosis Type 1
Molecular Aspects of Preimplantation Genetic Diagnosis for NF1
Neurofibromatosis 1 (NF1) is a multisystem disorders characterized by skin abnormalities, such as café-au-lait spots and neurofibromas, learning disabilities, skeletal anomalies and vascular complications.
- Trial name
- NF1 (NCT05149469)
- Where
- France
- Volunteers
- 60 people
- Sponsor
- University Hospital, Strasbourg, France
Parents of Children With Cancer, Parents of Children With NF1
Relationship Stressors in Parents of Children With Cancer or Neurofibromatosis Type 1 (NF1)
Background: Serious illnesses like cancer or Neurofibromatosis Type 1 (NF1), can cause high levels of stress in a family. When a child is diagnosed with cancer or NF1, parents face numerous stressors, each of which can strain…
- Conditions
- Parents of Children With Cancer, Parents of Children With NF1, Parents of Children With Neurofibromatosis Type I
- Trial name
- NCT01702922
- Where
- 3 sites in United States, Canada
- Volunteers
- 262 people
- Sponsor
- National Cancer Institute (NCI)
NF2 Deficiency
Gene Therapy for Neurofibromatosis Type 2 (NF2) with ST002
This is an open-label, single-arm, dose-escalation pilot study to evaluate the safety, tolerability and preliminary efficacy of ST002 in the treatment of patients with NF2 mutation-related solid tumors.
- Trial name
- NCT06834438
- Testing
- ST002
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Cancer Institute and Hospital, Chinese Academy of Medical Sciences
Neurofibromatosis Type 1
Hypotonia and Neurofibromatosis Type 1 (NF1) Glioma
Currently, optic pathway gliomas (OPG) are detected based on abnormal findings made during annual ophthalmologic exams. However, because these exams are annual, it is possible for healthcare providers to miss the point at which a…
- Trial name
- NCT02584413
- Testing
- Magnetic resonance imaging, Gadolinium contrast
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Washington University School of Medicine
Neurofibromatosis Type 1, Plexiform Neurofibroma
Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas
This is a phase II open label study that will evaluate children ≥ 1 year of age and adults with neurofibromatosis type 1 (NF1) and plexiform neurofibromas treated with the MEK inhibitor, binimetinib.
- Trial name
- NF108-BINI (NCT03231306)
- Testing
- Binimetinib
- Where
- 22 sites in United States
- Volunteers
- 45 people
- Sponsor
- University of Alabama at Birmingham
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
Malignant Peripheral Nerve Sheath Tumors
Imatinib Mesylate Treatment of Patients With Malignant Peripheral Nerve Sheath Tumors
This study assesses the safety and efficacy of imatinib mesylate treatment of patients with malignant peripheral nerve sheath tumors
- Trial name
- NCT00427583
- Testing
- imatinib mesylate
- Where
- 2 sites in Germany
- Volunteers
- 11 people
- Sponsor
- Novartis Pharmaceuticals
Cutaneous Neurofibroma
Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas
This study will evaluate the tolerability and effectiveness of three FDA-approved treatments in Neurofibromatosis Type 1 Cutaneous Neurofibromas. These treatments are: a 1064nm laser, a 755nm laser, and a Kybella injection.
- Trial name
- NCT04730583
- Testing
- Kybella, 1064nm laser, 755nm Alexandrite Laser
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Massachusetts General Hospital
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