Clinical trials: Neurofibromatosis
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
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
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
Malignant Peripheral Nerve Sheath Tumors, MPNST
SARC016: Study of Everolimus With Bevacizumab to Treat Refractory Malignant Peripheral Nerve Sheath Tumors
To determine the clinical response rate of everolimus in combination with bevacizumab for patients with chemotherapy refractory sporadic or neurofibromatosis type 1 (NF1) associated malignant peripheral nerve sheath tumor…
- Conditions
- Malignant Peripheral Nerve Sheath Tumors, MPNST, Sarcoma
- Trial name
- NCT01661283
- Testing
- everolimus, bevacizumab
- Where
- 12 sites in United States
- Volunteers
- 25 people
- Sponsor
- Sarcoma Alliance for Research through Collaboration
Solid Malignancy, Solid Tumor
Everolimus in Patients With Advanced Solid Malignancies With TSC1, TSC2, NF1, NF2, or STK11 Mutations
The purpose of this research study is to look at participants with solid tumor malignancies and specific mutations respond to treatment with everolimus.
- Trial name
- NCT02352844
- Testing
- Everolimus
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Washington University School of Medicine
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
CNS Cancer, Meningioma
Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable Meningioma
The purpose of this study is to find out what effects, good and/or bad, sunitinib has on patients and their tumors. At this time, no drugs are routinely used to treat meningioma, hemangioblastoma or hemangiopericytoma.
- Conditions
- CNS Cancer, Meningioma, Intracranial Hemangiopericytoma, Hemangioblastoma
- Trial name
- NCT00589784
- Testing
- Sunitinib
- Where
- 6 sites in United States
- Volunteers
- 50 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
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)
Developmental Cochlear Nerve Deficiency, Acquired Cochlear Nerve Deficiency
Auditory Brainstem Implantation in Young Children
To purpose of this feasibility study is to demonstrate the safety and efficacy of the Nucleus 24 Multichannel Auditory Brainstem Implant (ABI, Cochlear Corp, Sydney, AUS) in children without the diagnosis of neurofibromatosis…
- Conditions
- Developmental Cochlear Nerve Deficiency, Acquired Cochlear Nerve Deficiency, Cochlear Aplasia, Post-meningitis Cochlear Ossification
- Trial name
- NCT01904448
- Testing
- Auditory Brainstem Implantation in Children
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- University of North Carolina, Chapel Hill
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 Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1
The specific aim of this study is to determine whether Lovastatin ™ significantly improves visual spatial learning and/or sustained attention in children with NF1.
- Trial name
- STARS (NCT00853580)
- Testing
- Lovastatin ™
- Where
- 12 sites in United States, Australia
- Volunteers
- 146 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
Acoustic Schwannoma, Adult Anaplastic Meningioma
Bevacizumab in Treating Patients With Recurrent or Progressive Meningiomas
RATIONALE: Monoclonal antibodies, such as bevacizumab, can block tumor growth in different ways. Some block the ability of tumor cells to grow and spread.
- Conditions
- Acoustic Schwannoma, Adult Anaplastic Meningioma, Adult Ependymoma, Adult Grade I Meningioma
- Trial name
- NCT01125046
- Testing
- bevacizumab
- Where
- 5 sites in United States
- Volunteers
- 50 people
- Sponsor
- Northwestern University
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, 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
Phase II Study of Gleevec/Imatinib Mesylate (STI-571, NCS 716051) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas
THe primary objective is to estimate the response rate at 6 months to Gleevec® in patients with plexiform neurofibromas
- Trial name
- NCT01673009
- Testing
- Gleevec
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Indiana University
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
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
Neurofibromatosis 1
Antioxidant Therapy With N-acetylcysteine for 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
- DoDNAC (NCT04481048)
- Testing
- N-Acetyl cysteine
- Where
- United States
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
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 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