Clinical trials: Neurofibromatosis Type 1
Neurofibromatosis Type 2, Neuroma, Acoustic
Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma
The purpose of the study is to determine if RAD001 treatment will shrink or slow the growth of the vestibular schwannoma(s) in Neurofibromatosis 2 (NF2) patients.
- Trial name
- NCT01345136
- Testing
- RAD001, everolimus
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Jonsson Comprehensive Cancer Center
MPNST, NF1
Trial of Selumetinib and Bromodomain Inhibitor With Durvalumab for Sarcomas
A multi-institutional open-label phase 1/2 trial of selumetinib in combination with ZEN-3694 and durvalumab in refractory/unresectable sarcomas including MPNST.
- Conditions
- MPNST, NF1, Sarcoma
- Trial name
- NCT05253131
- Testing
- Selumetinib + ZEN-3694 ± Durvalumab
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- University of Alabama at Birmingham
Neurofibromatosis Type 1, Cutaneous Neurofibromas
Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1
The purpose of this research study is to determine if ranibizumab can prevent the growth of neurofibromas. We will also be collecting extra blood and serum samples to help us learn more about NF1.
- Trial name
- NCT00657202
- Testing
- Ranibizumab
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Massachusetts General Hospital
Diffuse Astrocytoma, Anaplastic Astrocytoma
Pediatric Long-Term Follow-up and Rollover Study
A roll-over study to assess long-term effect in pediatric patients treated with dabrafenib and/or trametinib.
- Conditions
- Diffuse Astrocytoma, Anaplastic Astrocytoma, Astrocytoma, Oligodendroglioma, Childhood
- Trial name
- NCT03975829
- Testing
- dabrafenib, trametinib
- Where
- 54 sites in United States, Argentina and 17 more
- Volunteers
- 163 people
- Sponsor
- Novartis Pharmaceuticals
Schwannoma, Vestibular, Neurofibromatosis 2
NF2 Natural History Consortium
OBJECTIVES: I. Define the growth rates and clinical course of NF2-related tumors in patients with neurofibromatosis type 2. Associate growth rate with physical function.
- Conditions
- Schwannoma, Vestibular, Neurofibromatosis 2, Meningioma
- Trial name
- NCT00004483
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- House Ear Institute
NF1, Cutaneous Neurofibroma
Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF).
This is a Phase 1/2a, open-label, non-randomized, multi-dose study of mirdametinib monotherapy in adults with NF1 and cNF. In both Phases of the study, participation in the study will comprise three periods: screening, treatment…
- Conditions
- NF1, Cutaneous Neurofibroma, Monotherapy
- Trial name
- Mirda (NCT06159166)
- Testing
- Mirdametinib
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Johns Hopkins University
Neurofibromatosis Type 1, Precancerous Condition
Combination Chemotherapy in Treating Patients With Neurofibromatosis and Progressive Plexiform Neurofibromas
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining methotrexate with vinblastine may be effective treatment for neurofibromatosis type 1 associated…
- Trial name
- NCT00030264
- Testing
- Methotrexate, Vinblastine
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Children's Hospital of Philadelphia
Neurofibromatosis 2
An Implant for Hearing Loss Due to Removal of Neurofibromatosis 2 Tumors
This is a study to gather some information on the safety and efficacy of the penetrating auditory brainstem implant (PABI) in patients with neurofibromatosis type 2.
- Trial name
- NCT00030043
- Testing
- Penetrating auditory brainstem implant
- Where
- 3 sites in United States
- Volunteers
- 10 people
- Sponsor
- FDA Office of Orphan Products Development
Neurofibromatosis Type 1, Muscle Weakness
Whole-body Vibration in Children With Neurofibromatosis Type 1
It is known that children with NF1 can have weak muscles and suffer from tiredness. It is also known that in similar conditions affecting children's muscles, standing on a vibration device for a few minutes each day can…
- Trial name
- NCT03888248
- Testing
- Whole-body vibration, Muscle strengthening exercises
- Where
- United Kingdom
- Volunteers
- 30 people
- Sponsor
- Manchester University NHS Foundation Trust
RAS Mutation, Neurofibromatosis 1
RASopathy Biorepository
The RASopathies are a group of developmental disorders caused by genetic changes in the genes that compose the Ras/mitogen activated protein kinase (MAPK) pathway. New RASopathies are being diagnosed frequently.
- Conditions
- RAS Mutation, Neurofibromatosis 1, Noonan Syndrome, Noonan Syndrome With Multiple Lentigines
- Trial name
- NCT04395495
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Neurocognitive Deficit, Mental Health Impairment
Intervention Effectiveness on the Neurocognitive Functioning of Children and Adolescents With Neurofibromatosis Type 1
Children with neurofibromatosis are more likely to have difficulties related to their psychological and neurocognitive functioning (e.g., more likely to have depression, have social difficulties, be diagnosed with ADHD).
- Conditions
- Neurocognitive Deficit, Mental Health Impairment, Neurofibromatosis 1
- Trial name
- OPTIMAL-SSI (NCT05377008)
- Testing
- OPTIMAL-SSI
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Virginia Commonwealth University
Neurofibromatosis 1
Systematically Assessing Changes in Plexiform Neurofibroma Related Disfigurement From Photographs of Subjects With Neurofibromatosis Type 1 on a Phase 2 Clinical Trial
Background: People with Neurofibromatosis type 1 (NF1) have an increased risk of developing plexiform neurofibromas (PNs). PNs are tumors that form in the tissue. They can form anywhere in the body.
- Trial name
- NCT04879160
- Where
- United States
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Plexiform Neurofibroma, Neurofibromatosis Type 1 (NF1)
MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
This study evaluates mirdametinib (PD-0325901) in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive mirdametinib (PD-0325901).
- Trial name
- ReNeu (NCT03962543)
- Testing
- Mirdametinib (PD-0325901) oral capsule or dispersible tablet
- Where
- 43 sites in United States
- Volunteers
- 114 people
- Sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
Neurofibroma
Non-interventional Study of Patients With PN NF1 Starting Selumetinib in Russia
The purpose of this study is to evaluate the effectiveness, safety of selumetinib and patient's parameters of symptomatic inoperable PN NF1 in real clinical practice in Russia
- Trial name
- NCT05891847
- Where
- 5 sites in Russia
- Volunteers
- 150 people
- Sponsor
- AstraZeneca
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, Neurofibromatosis Type 2
Endostatin Study for Patients With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors
1)Preliminarily evaluate the treatment effect of continuous vein injection of recombinant human endostatin on NF2; 2)Preliminarily evaluate the safety and the patient's tolerance of the treatment of endostatin; 3)Provide an…
- Trial name
- Endostatin (NCT02104323)
- Testing
- Endostatin
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Beijing Tiantan Hospital
Neurofibromatosis Type 1, Neurofibromatosis 1
Neurofibromatosis Type 1 Tumor Early Detection Study
The goal of this observational study is to determine if a liquid biopsy (i.e. blood test) is an effective clinical tool for monitoring the development of malignant peripheral nerve sheath tumor (MPNST) among adults (18 years and…
- Conditions
- Neurofibromatosis Type 1, Neurofibromatosis 1, Plexiform Neurofibroma, Plexiform Neurofibromas
- Trial name
- NF1-TED (NCT06515860)
- Testing
- Liquid biopsy for MPNST development
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- David Miller
Neurofibromatosis Type 1, Plexiform Neurofibromas
Selumetinib in Chinese Paediatric With Post-operative NF1-PNs, PhaseⅡ, Double-Blinded, Placebo-Controlled Study
This is a phase II, multicenter, randomised, parallel, double-blind, placebo-controlled study assessing the efficacy and safety of the MEKi selumetinib compared with placebo in Chinese paediatric participants with post-operative…
- Conditions
- Neurofibromatosis Type 1, Plexiform Neurofibromas, Post-operative
- Trial name
- PROTECT (NCT05825365)
- Testing
- Selumetinib
- Sponsor
- AstraZeneca
Neurofibromatosis 1
Efficacy of Skin Cooling in Reducing Pain Associated With Non-invasive Treatments of Neurofibromatosis Type 1 Cutaneous Neurofibromas
This study will evaluate the effectiveness of skin cooling in increasing tolerability of four treatments in Neurofibromatosis Type 1 Cutaneous Neurofibromas.
- Trial name
- NCT06132165
- Testing
- Deoxycholic Acid, Polidocanol, 1064nm Nd:YAG laser
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Massachusetts General Hospital
Neurofibromatosis Type 1, Learning Disability
Interventions for Reading Disabilities in NF1
Neurofibromatosis Type 1 (NF1) is a common genetic disorder that is associated with a four times greater risk of learning disabilities, including reading disabilities, and a deficiency of neurofibromin - a protein important in a…
- Conditions
- Neurofibromatosis Type 1, Learning Disability, Reading Disability, NF1
- Trial name
- NCT02964884
- Testing
- Lovastatin, reading tutoring intervention
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University