Clinical trials: Neurofibromatosis 1
Neurofibromatosis Type 1, Legius Syndrome
Study of Disease Severity in Adults With Neurofibromatosis Type 1 (NF1)
This study may identify genes that predict the seriousness of neurofibromatosis type 1 (NF1). Finding these genes may explain why some people with NF1 have more medical problems than others.
- Trial name
- NCT00111384
- Where
- United States
- Volunteers
- 313 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis Type 1
Identification of Pre-Malignant Lesions In Pediatric Patients With Neurofibromatosis Type 1 Using Novel Magnetic Resonance Imaging Techniques Paired With Artificial Intelligence
This is a single arm pilot trial of a novel whole-body Magnetic Resonance Imaging paired with artificial intelligence intervention, to evaluate feasibility defined as scan-rescan reliability, and to estimate the positive…
- Trial name
- NCT04763109
- Testing
- Whole-body Magnetic Resonance Imaging
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Nicole Baca
Hematologic Malignancy, Acute Leukemia
MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies
This is a phase II, open-label, prospective study of T cell receptor alpha/beta depletion (TCR α/β TCD) peripheral blood stem cell (PBSC) transplantation for children and adults with hematological malignancies.
- Conditions
- Hematologic Malignancy, Acute Leukemia, Remission, Acute Myeloid Leukemia
- Trial name
- NCT05735717
- Testing
- Fludarabine, Busulfan, Melphalan
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
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.
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.
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
RAS Mutation, NF1 Mutation
PAS-004 in Patients With Advanced Solid Tumors
The main purpose of this clinical trial is to test PAS-004 in people with advanced solid tumors with rat sarcoma virus (RAS), neurofibromatosis type I (NF1), or rapidly accelerated fibrosarcoma (RAF) mutations.
- Conditions
- RAS Mutation, NF1 Mutation, RAF Mutation, Advanced Solid Tumors
- Trial name
- NCT06299839
- Testing
- PAS-004 Capsules, PAS-004 Tablets
- Where
- 7 sites in United States, Bulgaria and 1 more
- Volunteers
- 48 people
- Sponsor
- Pasithea Therapeutics Corp.
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.
Low-grade Gliomas
Tarceva/Rapamycin for Children With Low-grade Gliomas With or Without Neurofibromatosis Type 1 (NF1)
This study will evaluate the feasibility of combining two drugs, Tarceva (an anti-EGFR agent), and Rapamycin (an mTOR inhibitor), in children with progressive low-grade gliomas who have failed initial conventional treatment.
- Trial name
- NCT00901849
- Testing
- Tarceva and Rapamycin
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Roger Packer
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
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