Clinical trials: Neurofibroma
Plexiform Neurofibroma
Evaluation of TQ-B3234 Capsules in Patients With Symptomatic, Non-Surgical Type 1 Neurofibromatosis-Associated Plexiform Neurofibromas
This study aims to demonstrate that in subjects with symptomatic, inoperable plexiform neurofibromas associated with neurofibromatosis type 1, TQ-B3234 capsules significantly improve the objective response rate at Week 24…
- Trial name
- NCT07407803
- Testing
- TQ-B3234 capsules
- Where
- 29 sites in China
- Volunteers
- 177 people
- Sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Sarcoma, Synovial Sarcoma
Sorafenib and Dacarbazine in Soft Tissue Sarcoma
The purpose of this study is to find out what effects, good and/or bad, the combination of sorafenib and dacarbazine has on sarcoma. Recurrent sarcoma is difficult to treat.
- Conditions
- Sarcoma, Synovial Sarcoma, Leiomyosarcoma, Malignant Peripheral Nerve Sheath Tumor
- Trial name
- NCT00837148
- Testing
- Sorafenib and Dacarbazine
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma.
- Conditions
- Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease, Cystic Fibrosis, Fragile X Syndrome
- Trial name
- DANNIgene (NCT06147414)
- Testing
- Blood sample
- Where
- France
- Volunteers
- 550 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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.
Adult Alveolar Soft Part Sarcoma, Adult Angiosarcoma
Vismodegib and Gamma-Secretase/Notch Signalling Pathway Inhibitor RO4929097 in Treating Patients With Advanced or Metastatic Sarcoma
This randomized phase I/II clinical trial is studying the side effects and best dose of gamma-secretase/notch signalling pathway inhibitor RO4929097 when given together with vismodegib and to see how well they work in treating…
- Conditions
- Adult Alveolar Soft Part Sarcoma, Adult Angiosarcoma, Adult Desmoplastic Small Round Cell Tumor, Adult Epithelioid Hemangioendothelioma
- Trial name
- NCT01154452
- Testing
- Gamma-Secretase Inhibitor RO4929097, Laboratory Biomarker Analysis, Pharmacological Study
- Where
- United States
- Volunteers
- 78 people
- Sponsor
- National Cancer Institute (NCI)
Sarcoma
Doxorubicin With or Without Ifosfamide and Pegfilgrastim in Treating Patients With Locally Advanced or Metastatic Soft Tissue Sarcoma
RATIONALE: Drugs used in chemotherapy such as doxorubicin and ifosfamide use different ways to stop tumor cells from dividing so they stop growing or die.
- Trial name
- NCT00061984
- Testing
- pegfilgrastim, doxorubicin hydrochloride, ifosfamide
- Where
- 41 sites in Austria, Belgium and 9 more
- Volunteers
- 455 people
- Sponsor
- European Organisation for Research and Treatment of Cancer - EORTC
Neurofibromatosis 2
89Zr-Bevacizumab PET/CT Imaging in NF2 Patients
Bevacizumab can be an effective treatment for individuals with NF2 and improve different nerve functions (like hearing, tinnitus or balance problems) and the quality of life of NF2 patients.
- Trial name
- NCT05685836
- Testing
- Bevacizumab Zirconium Zr-89
- Where
- Netherlands
- Volunteers
- 15 people
- Sponsor
- Leiden University Medical Center
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
Neurofibromatosis Type 1
Effects of Physical Training on Bone and Muscle Quality, Muscle Strength, and Motor Coordination in Children With NF1
A physical training program will improve quality of life, participation in physical activity, motor coordination, muscle strength, and bone and muscle strength in children with neurofibromatosis type 1.
- Trial name
- NCT01058330
- Testing
- Plyometric training program
- Where
- 2 sites in United States
- Volunteers
- 36 people
- Sponsor
- Shriners Hospitals for Children
Neurofibromatosis 1, Plexiform Neurofibroma
FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas
A study to evaluate the efficacy of FCN-159 in adult patients with symptomatic, inoperable neurofibromatosis type 1-related plexiform neurofibromas.
- Conditions
- Neurofibromatosis 1, Plexiform Neurofibroma, NF1
- Trial name
- NCT05913037
- Testing
- Test group (Group A): FCN-159 8 mg, orally, once daily;
- Where
- 15 sites in China
- Volunteers
- 167 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Sarcoma, Malignant Peripheral Nerve Sheath Tumors
PLX3397 Plus Sirolimus in Unresectable Sarcoma and Malignant Peripheral Nerve Sheath Tumors
The purpose of this study is to determine if treatment with PLX3397 and Sirolimus will be tolerated and result in shrinking of the cancer or stopping the cancer from growing.
- Trial name
- PLX3397 (NCT02584647)
- Testing
- PLX3397, sirolimus
- Where
- 5 sites in United States
- Volunteers
- 39 people
- Sponsor
- Gulam Manji
Neurofibromatosis 1
Tailoring an Online Platform to Promote Evidence-Based Care for Adults With Neurofibromatosis 1 and Low Health Literacy
This decentralized, randomized study seeks to assess the feasibility, acceptability, and preliminary effectiveness of two approaches to assisting Neurofibromatosis 1 (NF1) patients with low health literacy improve their…
- Trial name
- NCT07233408
- Testing
- NF1 Educational Videos, Peer Navigator, Letters about NF1 Care
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Massachusetts General Hospital
Neurofibromatosis 1, Plexiform Neurofibroma (PN)
Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas
A global study to demonstrate the effectiveness of selumetinib in participants with NF1 who have symptomatic, inoperable plexiform neurofibromas.
- Trial name
- KOMET (NCT04924608)
- Testing
- Selumetinib
- Where
- 34 sites in United States, Australia and 11 more
- Volunteers
- 145 people
- Sponsor
- AstraZeneca
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 1, Cutaneous Neurofibroma
Use of Topical Liquid Diclofenac Following Laser Microporation of Cutaneous Neurofibromas in Patients With NF1
This is an open, controlled, prospective, proof-of-concept study, in 7 patients presenting NF1 and cutaneous neurofibromas. This study will include three treatment visits to the study center and three follow-up visits.
- Trial name
- NCT03090971
- Testing
- Diclofenac Sodium, Saline Solution
- Where
- Brazil
- Volunteers
- 7 people
- Sponsor
- Fundação Educacional Serra dos Órgãos
Adult Pleomorphic Rhabdomyosarcoma, AJCC Grade 2 Sarcoma
Evaluation of Chest CT Versus Chest X-Ray for Lung Surveillance After Curative-Intent Resection of High-Risk Truncal-Extremity Soft Tissue Sarcoma
This phase III trial compares chest computed tomography (CT) to chest x-ray (CXR) for lung surveillance after curative-intent resection of high-risk truncal-extremity soft tissue sarcoma.
- Conditions
- Adult Pleomorphic Rhabdomyosarcoma, AJCC Grade 2 Sarcoma, AJCC Grade 3 Sarcoma, Alveolar Soft Part Sarcoma
- Trial name
- NCT06526897
- Testing
- Biopsy, Chest Computed Tomography, Chest Radiography
- Volunteers
- 1,582 people
- Sponsor
- ECOG-ACRIN Cancer Research Group
Neurofibromatosis 1, Social Skills
Pilot Randomized Control Trial of Telehealth Group for Improving Peer Relationships (PEERS) in NF1
This is a pilot randomized control trial of the UCLA PEERS protocol delivered via Telehealth with teens with neurofibromatosis type 1 whose parents report that they have difficulty making and keeping friends.
- Trial name
- NCT04860362
- Testing
- PEERS
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- University of Wisconsin, Milwaukee
Glioma
Glioma Developmental and HyperActive Ras Tumor (DHART) Board
This study will collect medical records, scan results, and complete surveys to create a registry about people with a neurofibromatosis type 1-associated brain tumor (NF1-associated glioma).
- Trial name
- NCT05489783
- Where
- 7 sites in United States
- Volunteers
- 50 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Neurofibromatosis Type 1, Neurofibroma
Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis Type 1: A Qualitative Study
Background: Neurofibromatosis 1 (NF1) is a disease that causes tumors to grow along the nerves. These include plexiform neurofibromas (pNF) and cutaneous neurofibromas (cNF). Both pNF and cNF can be visible to other people.
- Trial name
- NCT06880991
- Testing
- Arm 1
- Where
- United States
- Volunteers
- 110 people
- Sponsor
- National Cancer Institute (NCI)
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