Clinical trials: Neurofibromatosis Type I
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 1, Plexiform Neurofibroma
Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas
Based on the clinical activity of both selumetinib and cabozantinib as monotherapies in clinical trials, the demonstrated activity of these agents in reduced doses in preclinical studies, and the non-overlapping toxicity…
- Trial name
- NF113 (NCT06502171)
- Testing
- Cabozantinib Oral Tablet, Selumetinib Oral Capsule
- Where
- 8 sites in United States
- Volunteers
- 30 people
- Sponsor
- Girish Dhall, MD
Low Grade Astrocytoma, Low Grade Glioma
A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma
This phase III trial compares the effect of selumetinib versus the standard of care treatment with carboplatin and vincristine (CV) in treating patients with newly diagnosed or previously untreated low-grade glioma (LGG) that…
- Conditions
- Low Grade Astrocytoma, Low Grade Glioma, Metastatic Low Grade Astrocytoma, Metastatic Low Grade Glioma
- Trial name
- NCT04166409
- Testing
- Biospecimen Collection, Carboplatin, Echocardiography Test
- Where
- 132 sites in United States, Canada and 1 more
- Volunteers
- 170 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis Type I, Plexiform Neurofibroma
Sorafenib to Treat Children and Young Adults With Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas
Background: Patients with neurofibromatosis type 1 are at increased risk of developing tumors called plexiform neurofibromas (PN) that arise from nerves.
- Trial name
- NCT00727233
- Testing
- Nexavar (BAY 43-9006) (Sorafenib), Toxicity, Pharmacokinetics, Pharmacodynamics
- Where
- 2 sites in United States
- Volunteers
- 9 people
- Sponsor
- National Cancer Institute (NCI)
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 1, Malignant Peripheral Nerve Sheath Tumor (MPNST) of Soft Tissue (Diagnosis)
Multi-parametric Biomarker Development to Predict Malignant Conversion in Patients With Neurofibromatosis Type 1
The goal of this prospective observational study is to learn about the utility of imaging and clinical features in patients with Neurofibromatosis type 1 categorized as high risk for the development of malignant peripheral nerve…
- Conditions
- Neurofibromatosis 1, Malignant Peripheral Nerve Sheath Tumor (MPNST) of Soft Tissue (Diagnosis), Atypical Neurofibroma
- Trial name
- NCT05677594
- Testing
- Whole Body Magnetic Resonance Imaging
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- Johns Hopkins University
Neurofibromatosis Type 1, Plexiform Neurofibromas
Selumetinib in Chinese Paediatric With Post-operative NF1-PNs, PhaseⅡ, Double-Blinded, Placebo-Controlled Study
This is a phase II, multicenter, randomised, parallel, double-blind, placebo-controlled study assessing the efficacy and safety of the MEKi selumetinib compared with placebo in Chinese paediatric participants with post-operative…
- Conditions
- Neurofibromatosis Type 1, Plexiform Neurofibromas, Post-operative
- Trial name
- PROTECT (NCT05825365)
- Testing
- Selumetinib
- Sponsor
- AstraZeneca
Neurofibromatosis Type 1, Precancerous Condition
Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform Neurofibromas
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining methotrexate with vinblastine may be effective treatment for neurofibromatosis type 1 associated…
- Trial name
- NCT00030264
- Testing
- Methotrexate, Vinblastine
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Children's Hospital of Philadelphia
Melanoma
MElanoma Research Lymph Node Prediction Implementation National_001
MERLIN_001 is a prospective registry study of a primary melanoma gene-signature to predict sentinel node (SN) status and to determine its prognostic value for more accurate staging of SN-negative melanoma patients.
- Trial name
- MERLIN_001 (NCT04759781)
- Where
- 9 sites in United States
- Volunteers
- 1,820 people
- Sponsor
- SkylineDx
Metastatic Malignant Peripheral Nerve Sheath Tumor, Neurofibromatosis Type 1
Vaccine Therapy in Treating Patients with Malignant Peripheral Nerve Sheath Tumor That is Recurrent or Cannot Be Removed by Surgery
This phase I trial studies the side effects and the best dose of a vaccine therapy in treating patients with malignant peripheral nerve sheath tumor that cannot be removed by surgery (unresectable) or has come back after a period…
- Conditions
- Metastatic Malignant Peripheral Nerve Sheath Tumor, Neurofibromatosis Type 1, Recurrent Malignant Peripheral Nerve Sheath Tumor
- Trial name
- NCT02700230
- Testing
- Computed Tomography, Laboratory Biomarker Analysis, Oncolytic Measles Virus Encoding Thyroidal Sodium Iodide Symporter
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Mayo Clinic
Neurofibroma
Non-interventional Study of Patients With PN NF1 Starting Selumetinib in Russia
The purpose of this study is to evaluate the effectiveness, safety of selumetinib and patient's parameters of symptomatic inoperable PN NF1 in real clinical practice in Russia
- Trial name
- NCT05891847
- Where
- 5 sites in Russia
- Volunteers
- 150 people
- Sponsor
- AstraZeneca
Neurofibromatosis 1, Diagnoses Disease
Study About Annoucement of the Diagnosis of Neurofibromatosis 1 in de Novo Forms
Neurofibromatosis type 1 (NF1) is one of the most common autosomal dominant genetic disorders. The aim of our study was to evaluate post-traumatic stress disorder (PTSD) in patients and their families following the disclosure of…
- Conditions
- Neurofibromatosis 1, Diagnoses Disease, Post Traumatic Stress Disorder
- Trial name
- NF1 (NCT03298438)
- Where
- France
- Volunteers
- 31 people
- Sponsor
- University Hospital, Brest
Neurofibromatosis 2
An Implant for Hearing Loss Due to Removal of Neurofibromatosis 2 Tumors
This is a study to gather some information on the safety and efficacy of the penetrating auditory brainstem implant (PABI) in patients with neurofibromatosis type 2.
- Trial name
- NCT00030043
- Testing
- Penetrating auditory brainstem implant
- Where
- 3 sites in United States
- Volunteers
- 10 people
- Sponsor
- FDA Office of Orphan Products Development
Neurofibromatosis Type 1
A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1
The trial will be an open label, single arm, phase 2 study to assess the tolerability and efficacy of HLX-1502 in participants with NF1 that are 16 years or older in age with progressive and/or symptomatic PN.
- Trial name
- INSPIRE-NF1 (NCT06541847)
- Testing
- HLX-1502
- Where
- 14 sites in United States
- Volunteers
- 25 people
- Sponsor
- Healx Limited
Neurofibromatosis Type 1
How Neurofibromatosis Type 1 (NF1) Affects Schoolwork and Self-Esteem
The study will seek to determine whether there is a corresponding deficit in school performance for teenagers with NF1. Secondly, the study seeks to explore the issue of self-esteem in teenagers with NF1.
- Trial name
- NCT01626846
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- John Jay High School
Neurofibromatosis, Neurofibromas
Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas
This is a second Pilot Study to determine the efficacy of Gleevec® in neurofibromatosis (NF1) patients with plexiform neurofibromas using new response assessment modalities with the secondary goals of assessing Gleevec toxicity…
- Trial name
- 0908-09 (NCT01140360)
- Testing
- Gleevec
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Kent Robertson
Cancer
Immunotherapy Based on Antigen-specific Immune Effector Cells Targeting Neurofibromatosis or Schwannomatosis
The primary objective of this study is to verify the safety of antigen-specific T cells (CAR-T) and engineered immune effector cytotoxic T cells (EIE) modified by immunoregulatory genes and immune modified dendritic cell vaccine…
- Trial name
- NCT04085159
- Testing
- Antigen-specific T cells CART/CTL and DCvac
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Prader-Willi Syndrome, PWS-like Syndrome
GROWing Up With Rare GENEtic Syndromes
Introduction Rare complex syndromes Patients with complex genetic syndromes, by definition, have combined medical problems affecting multiple organ systems, and intellectual disability is often part of the syndrome.
- Conditions
- Prader-Willi Syndrome, PWS-like Syndrome, Silver Russel Syndrome, Congenital Hypopituitarism
- Trial name
- GROW UR GENES (NCT04463316)
- Testing
- Retrospective file studies
- Where
- Netherlands
- Volunteers
- 600 people
- Sponsor
- dr. Laura C. G. de Graaff-Herder
Neurofibromatosis Type 1, Brain Tumor
Feasibility and Clinically Application of Magnetic Resonance Fingerprinting
This study will look at the feasibility of using magnetic resonance fingerprinting (MRF) in children, adolescents and young adults (AYA) with and without brain tumors.
- Conditions
- Neurofibromatosis Type 1, Brain Tumor, Glioma
- Trial name
- NCT02387840
- Testing
- Magnetic Resonance Imaging, Magnetic Resonance Fingerprinting
- Where
- United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Case Comprehensive Cancer Center
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.