Clinical trials: Neurofibromatosis 1
Neurofibromatosis Type 1, Plexiform Neurofibroma
Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas
This is a phase II open label study that will evaluate children ≥ 1 year of age and adults with neurofibromatosis type 1 (NF1) and plexiform neurofibromas treated with the MEK inhibitor, binimetinib.
- Trial name
- NF108-BINI (NCT03231306)
- Testing
- Binimetinib
- Where
- 22 sites in United States
- Volunteers
- 45 people
- Sponsor
- University of Alabama at Birmingham
Neurofibromatosis 2, Progressive Vestibular Schwannoma (VS)
Phase 2 Clinical Trial of Crizotinib for Children and Adults With Neurofibromatosis Type 2 and Progressive Vestibular Schwannomas
Subjects with Neurofibromatosis Type 2 (NF2) and progressive vestibular schwannoma (VS) will be treated with crizotinib administered orally.
- Trial name
- NF110 (NCT04283669)
- Testing
- Crizotinib
- Where
- 15 sites in United States
- Volunteers
- 12 people
- Sponsor
- University of Alabama at Birmingham
Neurofibromatosis Type 2, Progressive Vestibular Schwannomas
Phase 2 Study of Bevacizumab in Children and Young Adults With NF 2 and Progressive Vestibular Schwannomas
To determine the hearing response rate at 24 weeks after treatment with bevacizumab for symptomatic vestibular schwannomas (VS) in children and young adults with Neurofibromatosis Type 2 (NF 2).
- Trial name
- NCT01767792
- Testing
- Bevacizumab
- Where
- 12 sites in United States
- Volunteers
- 22 people
- Sponsor
- University of Alabama at Birmingham
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
Neurofibromatosis 1, Chronic Pain
Innovation in the Treatment of Persistent Pain in Adults With NF1: Implementation of the iCanCope Mobile Application- Clinical Trial
To demonstrate the initial feasibility and determine preliminary impact on clinical outcomes of the iCanCope-NF program in a pilot RCT.
- Trial name
- NCT04561765
- Testing
- iCanCope, iCanCope+Contingency Management
- Where
- United States
- Volunteers
- 122 people
- Sponsor
- Yale University
Neurofibromatosis Type 1
Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN
This study is designed to define a dosing regimen and assess the pharmacokinetics(PK) and safety of the granule formulation; the study will also include descriptive analyses of exploratory efficacy endpoints.
- Trial name
- SPRINKLE (NCT05309668)
- Testing
- Selumetinib granule formulation, Selumetinib capsule formulation
- Where
- 16 sites in United States, Germany and 5 more
- Volunteers
- 36 people
- Sponsor
- AstraZeneca
Neurofibromatosis 1
Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1
Children with neurofibromatosis type 1 (NF1) commonly suffer from the effects of cognitive, behavioral, and motor impairments. At present there is no specific treatment for this NF1 complication.
- Trial name
- NF1NAC (NCT04481035)
- Testing
- N-acetylcysteine (NAC)
- Where
- United States
- Volunteers
- 5 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Neurofibromatosis Type 1, Recurrent Childhood Pilocytic Astrocytoma
Low-Dose or High-Dose Lenalidomide in Treating Younger Patients With Recurrent, Refractory, or Progressive Pilocytic Astrocytoma or Optic Pathway Glioma
This randomized phase II trial studies how well low-dose lenalidomide works compared with high-dose lenalidomide in treating younger patients with juvenile pilocytic astrocytomas or optic nerve pathway gliomas that have come back…
- Conditions
- Neurofibromatosis Type 1, Recurrent Childhood Pilocytic Astrocytoma, Recurrent Childhood Visual Pathway Glioma
- Trial name
- NCT01553149
- Testing
- Lenalidomide, Pharmacological Study
- Where
- 126 sites in United States, Australia and 2 more
- Volunteers
- 75 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis 2, Vestibular Schwannoma
Trial of Selumetinib in Patients With Neurofibromatosis Type II Related Tumors
In this research study the researchers want to learn more about the effects (both good and bad) the study drug selumetinib has on participants with neurofibromatosis type II (NF2) related tumor.
- Conditions
- Neurofibromatosis 2, Vestibular Schwannoma, Meningioma, Ependymoma
- Trial name
- SEL-TH-1601 (NCT03095248)
- Testing
- Selumetinib
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Advanced Malignant Neoplasm, Castleman Disease
Bevacizumab and Temsirolimus Alone or in Combination With Valproic Acid or Cetuximab in Treating Patients With Advanced or Metastatic Malignancy or Other Benign Disease
This phase I trial studies the side effects and best dose of bevacizumab and temsirolimus alone or in combination with valproic acid or cetuximab in treating patients with a malignancy that has spread to other places in the body…
- Conditions
- Advanced Malignant Neoplasm, Castleman Disease, Digestive System Carcinoma, Erdheim-Chester Disease
- Trial name
- NCT01552434
- Testing
- Bevacizumab, Cetuximab, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 116 people
- Sponsor
- M.D. Anderson Cancer Center
NF1, Low-grade Glioma
A Phase II Trial of Poly-ICLC for Low-Grade Gliomas
This is a phase II, prospective, longitudinal, multi-center trial of poly-ICLC (Hiltonol ®) treatment for progressive low-grade gliomas in pediatric patients with NF1.
- Trial name
- NF111 (NCT04544007)
- Testing
- Poly ICLC
- Where
- 12 sites in United States
- Volunteers
- 3 people
- Sponsor
- University of Alabama at Birmingham
Neurofibromatosis, NF1
Study of Sutent®/Sunitinib (SU11248) in Subjects With NF-1 Plexiform Neurofibromas
This is a pilot study to determine if adults and children with neurofibromatosis type 1 who have plexiform tumors given Sutent® respond to this drug therapy.
- Conditions
- Neurofibromatosis, NF1, Plexiform Neurofibromas
- Trial name
- NCT01402817
- Testing
- Sutent®/Sunitinib
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Indiana University
Neurofibromatosis Type 1, Sarcoma
Combination Chemotherapy in Treating Patients With Stage III or Stage IV Malignant Peripheral Nerve Sheath Tumors
RATIONALE: Drugs used in chemotherapy, such as doxorubicin, ifosfamide, and etoposide, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00304083
- Testing
- filgrastim, doxorubicin hydrochloride, etoposide
- Where
- 17 sites in United States
- Volunteers
- 48 people
- Sponsor
- Sarcoma Alliance for Research through Collaboration
Neurofibromatosis Type 1, Plexiform Neurofibroma
AZD2171 in Treating Patients With Neurofibromatosis Type 1 and Plexiform Neurofibroma and/or Neurofibroma Near the Spine
This phase II trial is studying how well AZD2171 works in treating patients with neurofibromatosis type 1 and plexiform neurofibroma and/or neurofibroma near the spine.
- Conditions
- Neurofibromatosis Type 1, Plexiform Neurofibroma, Spinal Cord Neurofibroma
- Trial name
- NCT00326872
- Testing
- Cediranib Maleate
- Where
- 9 sites in United States
- Volunteers
- 26 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis Type 1, Brain Tumor
Feasibility and Clinically Application of Magnetic Resonance Fingerprinting
This study will look at the feasibility of using magnetic resonance fingerprinting (MRF) in children, adolescents and young adults (AYA) with and without brain tumors.
- Conditions
- Neurofibromatosis Type 1, Brain Tumor, Glioma
- Trial name
- NCT02387840
- Testing
- Magnetic Resonance Imaging, Magnetic Resonance Fingerprinting
- Where
- United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Case Comprehensive Cancer Center
Bilateral Hearing Loss for Causes Other Than Tumors
Auditory Brainstem Implant (ABI) in Adult Non-Neurofibromatosis Type 2 Subjects
The purpose of this research study is to determine whether Auditory Brainstem Implant (ABI) can improve hearing in persons who are deaf in both ears and are not candidates for cochlear implants.
- Trial name
- NCT01736267
- Testing
- Nucleus 24 (discontinued) and ABI541 Auditory Brainstem Implants (ABI)
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Massachusetts Eye and Ear Infirmary
Glioma
Everolimus for Children With NF1 Chemotherapy-Refractory Radiographic Progressive Low Grade Gliomas
The purpose of this research study is to learn if the study drug RAD001 can shrink or slow the growth of low-grade gliomas in children with Neurofibromatosis type 1 (NF1). Additionally, the safety of RAD001 will be studied.
- Trial name
- NFC-RAD001 (NCT01158651)
- Testing
- RAD001 (Everolimus)
- Where
- 12 sites in United States
- Volunteers
- 23 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
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, NF1
Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas
The purpose of this Pilot Study is to determine if NF1 patients with plexiform neurofibromas treated with Tasgina® respond to therapy.
- Conditions
- Neurofibromatosis, NF1, Neurofibromas
- Trial name
- NCT01275586
- Testing
- Tasigna
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Indiana University
Bilateral Hearing Loss for Causes Other Than Tumors
Auditory Brainstem Implant (ABI) in Pediatric Non-Neurofibromatosis Type 2 Subjects
The purpose of this research study is to determine whether Auditory Brainstem Implant (ABI) can improve hearing in children who are deaf and cannot receive a cochlear implants.
- Trial name
- ABI (NCT01864291)
- Testing
- Nucleus 24 (discontinued) and ABI541 Auditory Brainstem Implants (ABI)
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Massachusetts Eye and Ear Infirmary