Clinical trials: Neurofibromatosis Type 1
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
This phase II trial studies how well trametinib works in treating patients with juvenile myelomonocytic leukemia that has come back (relapsed) or does not respond to treatment (refractory).
- Trial name
- NCT03190915
- Testing
- Bone Marrow Aspiration and Biopsy, Trametinib
- Where
- 57 sites in United States
- Volunteers
- 10 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis Type 1
A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas
Treatment Overview This phase II study will evaluate the activity of sirolimus in children and adults with NF1 and inoperable plexiform neurofibromas that have the potential to cause significant morbidity.
- Trial name
- Protocol 102 (NCT00634270)
- Testing
- Sirolimus
- Where
- 9 sites in United States
- Volunteers
- 58 people
- Sponsor
- University of Alabama at Birmingham
NF1, Neurofibromatosis
Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults
This study, "A Phase II Study of Cabozantinib (XL l84) for Plexiform Neurofibromas in Subjects with Neurofibromatosis Type I in Children and Adults diagnosed with Neurofibromatosis Type 1 (NF1) and have a type of tumor called a…
- Conditions
- NF1, Neurofibromatosis, Plexiform Neurofibromas
- Trial name
- NF105-CABO (NCT02101736)
- Testing
- Cabozantinib
- Where
- 12 sites in United States
- Volunteers
- 45 people
- Sponsor
- University of Alabama at Birmingham
Neurofibromatosis 2, Vestibular Schwannoma
Lapatinib Study for Children and Adults With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors
The purpose of this study is to determine if Lapatinib has any effect on tumors found in patients with Neurofibromatosis Type 2 (NF2). NF2 is a condition that mainly affects the skin and nervous system.
- Trial name
- NCT00973739
- Testing
- Lapatinib
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- NYU Langone Health
Neurofibromatosis Type II
Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2
This trial studies whether Everolimus is efficacious in treating neurofibromatosis 2.
- Trial name
- NCT01419639
- Testing
- Everolimus (RAD001) , Afinitor®
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- NYU Langone Health
Neurofibromatosis 1, Neurofibroma, Plexiform
Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas
Background: Neurofibromatosis Type 1 (NF1) is an autosomal dominant, progressive genetic disorder characterized by diverse clinical manifestations.
- Trial name
- NCT00076102
- Testing
- Pirfenidone
- Where
- 15 sites in United States
- Volunteers
- 36 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis 1
Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T
This trial is evaluating the use of oral Everolimus to determine if there is a reduction in the size of the disfiguring cutaneous lesions in patients with Neurofibromatosis 1 over a 6 month period.
- Trial name
- DCLNF1 (NCT02332902)
- Testing
- Everolimus
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- The University of Texas Health Science Center, Houston
Non-small Cell Lung Cancer
Trametinib in Patients With Advanced Neurofibromatosis Type 1 (NF1)-Mutant Non-small Cell Lung Cancer
Phase II trial to evaluate trametinib in patients with locally advanced non-squamous, non-small cell lung cancer (NSCLC) whose tumors harbor a non-synonymous NF-1 mutation, with progressive disease on at least one prior line of…
- Trial name
- NCT03232892
- Testing
- Trametinib
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of California, San Francisco
Age-Related Macular Degeneration
Study of Safety and Efficacy of Brolucizumab 6 mg Dosed Every 4 Weeks Compared to Aflibercept 2 mg Dosed Every 4 Weeks in Patients With Retinal Fluid Despite Frequent Anti-VEGF Injections
This clinical study was designed to compare the safety and efficacy of brolucizumab 6 mg dosed every 4 weeks to aflibercept 2 mg dosed every 4 weeks in those neovascular age-related macular degeneration (nAMD) patients with…
- Trial name
- MERLIN (NCT03710564)
- Testing
- Brolucizumab, Aflibercept
- Where
- 61 sites in United States, Puerto Rico
- Volunteers
- 535 people
- Sponsor
- Novartis Pharmaceuticals
Neurofibromatosis 1, Neurofibroma, Plexiform
Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1(NF1) on Clinical Treatment Trials
Background: Neurofibromatosis type 1 (NF1) is a genetic disorder. It has a broad variety of effects on the body. Up to half of people with NF1 get plexiform neurofibromas (PNs). These are benign tumors.
- Trial name
- NCT03531814
- Testing
- Medication Event Monitoring System (MEMS^TM), Questionnaires
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis 2, Meningioma
AZD2014 In NF2 Patients With Progressive or Symptomatic Meningiomas
The goal of this clinical research study is to learn if the study drug AZD2014 can shrink growing or symptomatic meningiomas.
- Trial name
- NCT02831257
- Testing
- AZD2014
- Where
- 2 sites in United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Vestibular Schwannoma, Meningioma
Exploratory Evaluation of AR-42 Histone Deacetylase Inhibitor in the Treatment of Vestibular Schwannoma and Meningioma
This will be a multi-center, proof of concept phase 0 study to assess the suppression of p-AKT in Vestibular Schwannoma (VS) and meningiomas by AR-42 in adult patients undergoing tumor resection.
- Conditions
- Vestibular Schwannoma, Meningioma, Acoustic Neuroma, Neurofibromatosis Type 2
- Trial name
- NCT02282917
- Testing
- AR-42
- Where
- 4 sites in United States
- Volunteers
- 7 people
- Sponsor
- Massachusetts Eye and Ear Infirmary
Malignant Peripheral Nerve Sheath Tumors, Neurofibromatosis 1
SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors
To determine the clinical benefit rate of selumetinib in combination with sirolimus in patients with unresectable or metastatic neurofibromatosis type 1 (NF1) associated or sporadic MPNST.
- Trial name
- NCT03433183
- Testing
- Selumetinib, Sirolimus
- Where
- 5 sites in United States
- Volunteers
- 21 people
- Sponsor
- Sarcoma Alliance for Research through Collaboration
Neurofibromatosis Type 1, Plexiform Neurofibromas
Acceptance and Commitment Training for Adolescents and Young Adults With Neurofibromatosis Type 1, Plexiform Neurofibromas, and Chronic Pain
Background: People with neurofibromatosis type I (NF1) and plexiform neurofibroma (PN) tumors often have chronic pain that his hard to control.
- Trial name
- NCT02471339
- Testing
- Acceptance and Commitment Therapy (ACT)
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- National Cancer Institute (NCI)
Cutaneous Neurofibroma, Neurofibromatosis 1
NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)
This is a randomized, double-blind, vehicle-controlled, parallel group dose response study evaluating the safety and effectiveness of 2 concentrations of NFX-179 gel in participants with cNF.
- Trial name
- NCT05005845
- Testing
- NFX-179 gel, Vehicle gel
- Where
- 23 sites in United States
- Volunteers
- 199 people
- Sponsor
- NFlection Therapeutics, Inc.
Neurofibromatosis Type 2, Vestibular Schwannomas
Study of Axitinib in Patients With Neurofibromatosis Type 2 and Progressive Vestibular Schwannomas
The purpose of this study is to determine if the study drug, AXITINIB, has any effect on tumors found in patients with Neurofibromatosis Type 2 (NF2).
- Trial name
- NCT02129647
- Testing
- Axitinib
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- NYU Langone Health
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, 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.
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)