Clinical trials: Neurofibromatosis Type 1
Acute Leukemia, Adenomatous Polyposis
Familial Investigations of Childhood Cancer Predisposition
NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll.
- Conditions
- Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML
- Trial name
- SJFAMILY (NCT03050268)
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- St. Jude Children's Research Hospital
Neurofibromatosis 1, Nerve Sheath Neoplasms
Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)
Background: Neurofibromatosis type 1 (NF1) is a genetic disease that can cause many symptoms. About half of people with NF1 will develop benign (noncancerous) tumors along nerves in the skin, brain, and other parts of the body.
- Trial name
- NCT06222203
- Where
- United States
- Volunteers
- 225 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis 1
Clinical Study on the Treatment of Type I Neurofibromatosis With Smeitinib Hydrosulfate Capsule
This study focused on patients with type I neurofibromatosis, who currently lack effective drug therapy and have a high recurrence rate after surgical resection.
- Trial name
- NCT06620354
- Testing
- Selumetinib
- Volunteers
- 33 people
- Sponsor
- Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
Neurofibromatosis 1
Evaluating Genetic Modifiers of Cutaneous Neurofibromas in Adults With Neurofibromatosis Type 1
The main goal of this protocol is to develop a well-phenotyped genetic biobank to identify genetic variants associated with the heterogeneous clinical presentations of Neurofibromatosis Type 1 (NF1).
- Trial name
- NCT04941027
- Where
- 2 sites in United States
- Volunteers
- 1,046 people
- Sponsor
- Stanford University
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
Neurofibromatosis 1
A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1
The goal of this fully decentralized, randomized controlled trial is to compare the efficacy of two educational interventions for individuals with Neurofibromatosis 1 (NF1).
- Trial name
- NCT06262113
- Testing
- Letters about NF1 Care (Content Type 1), Letters about NF1 Care (Content Type 2)
- Where
- United States
- Volunteers
- 294 people
- Sponsor
- Massachusetts General Hospital
Neurofibromatosis Type 1
Selumetinib Paediatric NF1 Japan Study
This is a phase I open label study designed to evaluate the safety, tolerability, PK and efficacy of selumetinib in Japanese paediatric patients with neurofibromatosis type 1 and inoperable and symptomatic plexiform neurofibroma.
- Trial name
- NCT04495127
- Testing
- Selumetinib
- Where
- 3 sites in Japan
- Volunteers
- 12 people
- Sponsor
- AstraZeneca
NF2-related Schwannomatosis, NF2
Luvometinib in Combination With Serplulimab for NF2-Related Tumors
This is an investigator-initiated, exploratory, multicenter, open-label, single-arm clinical trial and a substudy of the Platform Research for Innovative Medicines in NF2-SWN (PRIME-NF2).
- Conditions
- NF2-related Schwannomatosis, NF2, Neurofibromatosis Type 2, Vestibular Schwannoma
- Trial name
- NCT07708285
- Testing
- Luvometinib, Serplulimab
- Where
- 5 sites in China
- Volunteers
- 30 people
- Sponsor
- Beijing Tiantan 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 1
Evaluating Mitochondrial Dysfunction in Patients With Neurofibromatosis Type 1
Neurofibromatosis type 1 is a common genetic disease with a broad spectrum of clinical manifestations in multiple organs of the body.
- Trial name
- NCT05912400
- Testing
- Blood draw, FACIT-F and Pain Scales
- Where
- United States
- Volunteers
- 55 people, healthy volunteers welcome
- Sponsor
- University of Arkansas
Neurofibromatosis 2
Reliability of Functional Outcome Measures in Neurofibromatosis 2
Neurofibromatosis 2 (NF2) is a rare inherited condition. Those with NF2 usually present with tumours (schwannomas) in their inner ear, or vestibular organ, with common symptoms including problems with hearing, balance, dizziness…
- Trial name
- NCT03617276
- Where
- United Kingdom
- Volunteers
- 30 people
- Sponsor
- Guy's and St Thomas' NHS Foundation Trust
NF1, Congenital Pseudarthrosis of Tibia
A Study of INFUSE Bone Graft (BMP-2) in the Treatment of Tibial Pseudarthrosis in Neurofibromatosis Type 1 (NF1)
The current study proposes adding BMP-2 (INFUSE), an anabolic agent, at the surgical site of TPA (tibial pseudarthrosis) repair in children with NF1, compared to a control group of patients treated surgically without BMP-2.
- Trial name
- NF107-BMP2 (NCT02718131)
- Testing
- INFUSE Bone Graft (BMP-2), Control Group
- Where
- 16 sites in United States, Australia
- Volunteers
- 5 people
- Sponsor
- University of Alabama at Birmingham
Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs), Healthy Participants
A Study to Evaluate Bioavailability and Food Effect of Selumetinib (AZD6244) in Healthy Male Participants
This study will evaluate bioavailability and food effect of selumetinib (AZD6244) in healthy male participants. A total of 24 healthy male participants will be included to ensure at least 20 evaluable participants.
- Trial name
- NCT03649165
- Testing
- Treatment A, Treatment B, Treatment C
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
NF2-related Schwannomatosis
Assessment of Patient Experience With Auto-Captioning Glasses in NF2-Related-Schwannomatosis
The goal of this clinical trial is to investigate the usability of auto-captioning glasses for adults diagnosed with NF2-related schwannomatosis and loss of hearing.
- Trial name
- NCT07420751
- Testing
- Auto-captioning Glasses
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Massachusetts General Hospital
Neurofibromatosis Type 1
NF1-Attention: Study of Children With Neurofibromatosis Type 1 Treated by Methylphenidate
Neurofibromatosis type 1 (NF1) is an autosomal dominant disorder with an estimated prevalence of 1/2190 to 1/6711. Attention deficit hyperactivity disorder (ADHD) has been reported to be common in NF1.
- Trial name
- NCT00169611
- Testing
- methylphenidate
- Where
- France
- Volunteers
- 80 people
- Sponsor
- Hospices Civils de Lyon
Neurofibromatosis Type 1 (NF1)
Vitamin D Supplementation for Adults With Neurofibromatosis Type 1 (NF1)
This clinical trial is conducted by one of 4 locations; University of British Columbia (Vancouver, CA), University of Utah (Salt Lake City, UT, USA), University of Cincinnati (Cincinnati, OH, USA), and University of Hamburg…
- Trial name
- VitDBoneNF1 (NCT01968590)
- Testing
- Cholecalciferol
- Where
- 3 sites in United States, Canada
- Volunteers
- 26 people
- Sponsor
- University of Utah
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
Neurofibromatosis Type 1, Precancerous Condition
Fludeoxyglucose F 18 Positron Emission Tomography and Magnetic Resonance Perfusion Imaging in Patients With Neurofibromatosis 1 and Plexiform Neurofibroma
RATIONALE: New imaging procedures such as fludeoxyglucose F 18 positron emission tomography (FDG-PET) and magnetic resonance (MR) perfusion imaging may improve the ability to detect disease progression, help doctors predict a…
- Trial name
- NCT00060008
- Testing
- fludeoxyglucose F 18, gadopentetate dimeglumine
- Where
- 2 sites in United States
- Volunteers
- 18 people
- Sponsor
- Children's Hospital of Philadelphia
Neurofibromatosis 1, Neurofibroma, Plexiform
Real-World Treatment Study of Koselugo (Selumetinib)
As part of a post-approval commitment, the Korean health authority requests a study to characterize safety and effectiveness in patients treated with Koselugo (Selumetinib), an oral selective inhibitor of MAPK kinase (MEK) 1 and…
- Trial name
- NCT06360406
- Where
- 12 sites in South Korea
- Volunteers
- 200 people
- Sponsor
- AstraZeneca
Neurofibromatosis 1
Analysis of Plasma for Diagnosis and Follow-up of Neurofibromatosis Type 1
The purpose of this study is to find blood plasma based biomarkers of disease progression in neurofibromatosis type 1 (NF1). NF1 is associated with the development of benign cutaneous tumors as well as a variety of malignancies.
- Trial name
- NCT02680431
- Testing
- Blood sample
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Juha Peltonen