Clinical trials: Neurofibromatoses
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 2, Vestibular Schwannoma
WES of NF2-associated in Comparison to Sporadic Vestibular Schwannomas - Correlation With Clinical Data
Whole exome sequencing (WES) of 50 sporadic and 50 Neurofibromatosis Type2 (NF2)-associated vestibularis schwannomas (VS) in children and young adults.
- Conditions
- Neurofibromatosis Type 2, Vestibular Schwannoma, Acoustic Neuroma
- Trial name
- NF2 (NCT03210285)
- Testing
- Whole exome sequencing
- Where
- Germany
- Volunteers
- 70 people
- Sponsor
- University Hospital Tuebingen
Neurofibromatosis 1, Plexiform Neurofibroma
Follow-up Study to Evaluate the Safety and Efficacy of FCN-159 in Pediatric Participants With Neurofibromatosis Type 1
FCN-159 (Luvometinib Tablets), an orally available and highly potent selective inhibitor of MEK1/2,demonstrated good tolerability and exhibited notable anti-tumor activity in pediatric pts with NF1-related PN in study…
- Conditions
- Neurofibromatosis 1, Plexiform Neurofibroma, NF1
- Trial name
- NCT07024394
- Testing
- Luvometinib Tablets
- Where
- China
- Volunteers
- 65 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
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.
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
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
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
Cancer
Study to Investigate Safety, Pharmacokinetic (PK), Pharmacodynamic (PD) and Clinical Activity of Trametinib in Subjects With Cancer or Plexiform Neurofibromas and Trametinib in Combination With Dabrafenib in Subjects With Cancers Harboring V600 Mutations
This was a 4-part (Part A, Part B, Part C and Part D), Phase I/IIa, multi-center, open label, study in pediatric subjects with refractory or recurrent tumors.
- Trial name
- NCT02124772
- Testing
- Trametinib, Dabrafenib
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 139 people
- Sponsor
- Novartis Pharmaceuticals
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
Parenting, Alopecia
Mindful Parenting - A Single Case Experimental Design Study
Some parents of children living with a visible difference can experience heightened stress due to the associated challenges of this. Parent's views of the child's visible difference and their responses to the child are important.
- Conditions
- Parenting, Alopecia, Skin Condition, Limb Difference
- Trial name
- NCT05779527
- Testing
- Mindful Parenting Programme
- Where
- United Kingdom
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Cardiff University
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)