Clinical trials: Neurofibromatosis Type 1 (NF1)
Profound Bilateral Deafness Due to, Bilateral Cochlear Aplasia
A Safety Study of the Auditory Brainstem Implant for Pediatric Profoundly Deaf Patients
Current treatment options for bilateral profoundly deaf children, diagnosed with inner ear anatomical abnormalities, are limited and, in the case of absent cochleas, non-existent.
- Conditions
- Profound Bilateral Deafness Due to, Bilateral Cochlear Aplasia, Bilateral Cochlear Nerve Deficiency, Bilateral Cochlear Ossification Secondary to Meningitis
- Trial name
- NCT02102256
- Testing
- Auditory Brainstem Implant
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- Laurie Eisenberg
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
Dosing and Tolerability of Deoxycholic Acid vs. Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutaneous Neurofibromas
This study will evaluate the tolerability and effectiveness of two treatments in Neurofibromatosis Type 1 Cutaneous Neurofibromas. These treatments are: Kybella and Asclera injection.
- Trial name
- NCT06120036
- Testing
- Kybella, Asclera
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Massachusetts General Hospital
Bilateral Cochlear Aplasia, Bilateral Cochlear Nerve Deficiency
Feasibility Study of Auditory Brainstem Implant in Young Children
Current treatment options for bilateral profoundly deaf children, diagnosed with inner ear anatomical abnormalities are limited, and in the case of absent cochleas, non-existent.
- Conditions
- Bilateral Cochlear Aplasia, Bilateral Cochlear Nerve Deficiency, Bilateral Cochlear Ossification Secondary to Meningitis
- Trial name
- NCT01850225
- Testing
- Auditory brainstem implant
- Where
- United States
- Sponsor
- House Research Institute
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 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
Neurofibromatosis Type 2, Vestibular Schwannoma
Platform Research for Innovative Medicines in NF2-SWN (PRIME-NF2)
This is an adaptive platform-basket trial that aims to evaluate the safety and efficacy of multiple novel agents and combination therapies in patients with NF2-related schwannomatosis (NF2-SWN).
- Conditions
- Neurofibromatosis Type 2, Vestibular Schwannoma, Non-vestibular Schwannoma, Meningioma
- Trial name
- PRIME-NF2 (NCT07713745)
- Testing
- Selumetinib 25mg/m2, Luvometinib, Serplulimab
- Where
- 5 sites in China
- Volunteers
- 200 people
- Sponsor
- Beijing Tiantan Hospital
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)
Neurofibromatosis 1, Cutaneous Neurofibroma
NFX-179 Topical Gel Treatment in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)
This study will enroll and treat subjects with cutaneous neurofibromas with NFX-179, a topical study drug. Eligible subjects will receive treatment for 28 days and be observed by a study doctor for approximately 56 days.
- Trial name
- NCT04435665
- Testing
- NFX-179 Gel, Vehicle Gel
- Where
- 6 sites in United States
- Volunteers
- 48 people
- Sponsor
- NFlection Therapeutics, Inc.
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, Neurofibromatosis Type 1
MicroRNAs in Patients With Neurofibromatosis Type 1
MicroRNAs are small molecules which have recently been discovered in cells. They are known to be responsible for the normal development of cells and when they are disrupted can contribute to the development of cancer.
- Trial name
- NCT01595139
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Neurofibromatosis Type 1-Associated Plexiform Neurofibromas
A Study of Mirdametinib in Infants and Toddlers With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
The purpose of this study is to determine the safety, pharmacokinetic (PK), and tolerability of multiple treatment cycles of oral mirdametinib in infants and toddlers (0 to less than [<] 24 months of age) with measurable…
- Trial name
- NCT07850635
- Testing
- Mirdametinib
- Where
- Germany
- Volunteers
- 6 people
- Sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
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
Vestibular Schwannoma, Neurofibromatosis Type 2
Bevacizumab for Symptomatic Vestibular Schwannoma in Neurofibromatosis Type 2 (NF2)
People who have neurofibromatosis type 2 (NF2) can have tumors that grow on the auditory nerves and cause hearing loss. These tumors are called vestibular schwannomas (VSs), or acoustic neuromas.
- Trial name
- NCT01207687
- Testing
- bevacizumab, laboratory biomarker analysis, quality-of-life assessment
- Where
- 3 sites in United States
- Volunteers
- 14 people
- Sponsor
- National Cancer Institute (NCI)