• HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

    This study will evaluate the efficacy and safety of vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) compared to placebo in patients with ATTR amyloidosis with cardiomyopathy.

    Investigator

    Now accepting new patients View Details

  • Neoadjuvant Chemotherapy With or Without Second-Look Surgery Followed by Radiation Therapy With or Without Peripheral Stem Cell Transplantation in Treating Patients With Intracranial Germ Cell Tumors

    RATIONALE: Drugs used in chemotherapy work in different ways to stop tumor cells from dividing so they stop growing or die. Giving a chemotherapy drug before surgery may shrink the tumor so that it is no longer present by conventional imaging and tumor markers from serum and cerebrospinal fluid. Radiation therapy uses high-energy x-rays to damage tumor cells. Peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells. Combining different types of therapy may kill more tumor cells.

    PURPOSE: This Phase II trial is studying how well neoadjuvant chemotherapy with or without surgery and with or without high dose chemotherapy and peripheral stem cell transplantation, can increase response rates prior to radiation therapy and increase progression free and overall surviving patients with newly diagnosed intracranial germ cell tumors.

    Investigator

    Not accepting patients at this time View Details

  • Inducing Graft Tolerance in HLA Haplotype Matched Related and 3 Ag Matched Unrelated Living Donor Kidney Transplantation

    This research study is to determine if donor blood stem cells given after living, related, HLA antigen (Ag) haplotype match or living, unrelated donor kidney transplantation. Minimal HLA antigen matching will include matching of 2 HLA antigens that can be either HLA A, B, and /or DR. This research will change the immune system such that immunosuppressive drugs can be completely withdrawn or reduced to minimal dose without kidney rejection.

    Investigators

    Now accepting new patients View Details

  • Non-Myeloablative Allogeneic Transplant for Myelodysplastic Syndromes and Myeloproliferative Disorders

    To improve survival outcomes for patients with MDS and MPD with a nonmyeloablative allogeneic hematopoietic cell transplant.

    Investigators

    Not accepting patients at this time View Details

  • Neural Signatures of Parkinson's Disease

    The purpose of this study is to provide objective measurements of abnormal movements of the body in correlation with neural activity of the brain and track how these change over time. This may allow for the development of objective evaluation of the neural activity causing abnormal movements, which may lead to the ability of the DBS system to stimulate the brain by sensing the abnormal neural activity that is causing abnormal movements.

    Investigator

    Not accepting patients at this time View Details

  • High-Dose Combination Chemotherapy and Radiation Therapy in Treating Patients With Newly Diagnosed Metastatic Rhabdomyosarcoma or Ectomesenchymoma

    RATIONALE: Drugs used in chemotherapy, such as vincristine, irinotecan, ifosfamide, etoposide, doxorubicin, cyclophosphamide, and dactinomycin, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Radiation therapy uses high-energy x-rays to kill tumor cells. Giving high-dose combination chemotherapy together with radiation therapy may kill more tumor cells.

    PURPOSE: This phase III trial is studying how well giving high-dose combination chemotherapy together with radiation therapy works in treating patients with newly diagnosed metastatic rhabdomyosarcoma or ectomesenchymoma.

    Investigator

    Not accepting patients at this time View Details

  • Ph II of Capecitabine, Carboplatin & Bevacizumab for Gastroesophageal Junction & Gastric Carcinoma

    To investigate bevacizumab in combination with carboplatin and capecitabine for patients with unresectable or metastatic GEJ or gastric cancers. We hope that by adding bevacizumab to standard chemotherapy for this patient population we will improve Progression Free Survival by 90% over historical controls.

    Investigator

    Not accepting patients at this time View Details

  • MAGE A10ᶜ⁷⁹⁶T for Advanced NSCLC

    This first time in human study is intended for men and women at least 18 years of age who have advanced lung cancer which has grown or returned after being treated. In particular, it is a study for subjects who have a blood test positive for HLA-A*02:01 and/or HLA-A*02:06 and a tumor test positive for MAGE A10 protein expression (protein or gene). This trial is a dose escalation trial that will evaluate 3 doses of transduced cells administered after a lymphodepleting chemotherapy regimen using a 3+3 dose escalation design .The study will take the subject's T cells, which are a natural type of immune cell in the blood, and send them to a laboratory to be modified. The changed T cells used in this study will be the subject's own T cells that have been genetically changed with the aim of attacking and destroying cancer cells.

    When the MAGE A10ᶜ⁷⁹⁶T cells are available, subjects will receive lymphodepleting chemotherapy with cyclophosphamide and fludarabine, followed by the T cell infusion. The purpose of this study is to test the safety of genetically changed T cells and find out what effects, if any, they have in subjects with lung cancer. The study will evaluate three different cell dose levels in order to find out the target cell dose. Once the target cell dose is determined, additional subjects will be enrolled to further test the safety and effects at this cell dose.

    Subjects will be seen frequently by the Study Physician right after receiving their T cells back and up to first 6 months. After that, subjects will be seen every three months. Subjects will be seen every 6 months by their Study Physician for the first 5 years after the T cell infusion. If the T cells are found in the blood at five years, then the subjects will continue to be seen once a year until the T cells are no longer found in the blood for a maximum of 15 years. If the T cells are no longer found in the blood at 5 years, then the subject will be contacted by the Study Physician for the next 10 years. Subjects who have a confirmed response or clinical benefit ≥4 weeks after the first T-cell infusion and whose tumor continues to express the appropriate antigen target may be eligible for a second infusion. All subjects, completing or withdrawing from the Interventional Phase of the study, will enter a 15-year long-term follow-up phase for observation of delayed adverse events. All subjects will continue to be followed for overall survival during the long-term follow-up phase.

    Investigators

    Now accepting new patients View Details

  • Molecular Analysis of Thoracic Malignancies

    A research study to learn about the biologic features of cancer development, growth, and spread. We are studying components of blood, tumor tissue, normal tissue, and other fluids, such as urine, cerebrospinal fluid, abdominal or chest fluid in patients with cancer. Our analyses of blood, tissue, and/or fluids may lead to improved diagnosis and treatment of cancer by the identification of markers that predict clinical outcome, markers that predict response to specific therapies, and the identification of targets for new therapies.

    Investigators

    Now accepting new patients View Details

  • Neural Links Between OCD and Anorexia

    Individuals with anorexia nervosa (AN) have long been observed to demonstrate symptoms in common with obsessive-compulsive disorder (OCD), in particular, an obsessive fear of normal weight leading to dangerous food restriction, as well as many compulsive rituals about food. Both AN and OCD are seriously handicapping and often resistant to conventional therapies. Given that the two conditions often co-occur and are associated with still unknown genetic risk factors, the aim of this project is to identify their shared and distinct patterns of brain activity.

    The investigators propose to use functional magnetic resonance imaging to compare brain response among adolescents with AN, OCD, and age-matched healthy individuals. Specifically, this study will investigate function of distinct brain circuits related to core aspects of these related disorders. The investigators use three tasks related to set shifting, global vs. local processing, and reward. Based on evidence of deficits in cognitive flexibility and ability to change behavior, the investigators hypothesize that adolescents with AN and with OCD will show hypoactivity of frontostriatal circuitry during cognitive tasks, and adolescents with AN will show hyperactivity in limbic regions in a reward task.

    This study is the first to directly compare brain activation patterns using functional neuroimaging in AN and OCD. The goal is to determine how abnormal brain activity relates to symptom formation, what accounts for shared characteristics amongst these disorders, and whether deficits in specific circuitry underlie their unique defining features. The study of shared and unique elements of functional brain circuitry reflects a new, emerging approach to the classification of psychiatric illness, one based on identifying unique combinations of biological risk factors that link related conditions. This approach is widely believed to be a critical step forward in developing more brain-relevant targeted strategies for preventative interventions.

    Investigator

    Not accepting patients at this time View Details

  • Hoarding Disorder Treatment With Virtual Reality

    This study explores whether a facilitated peer support group called Buried in Treasures (BIT) and a virtual reality decluttering practice can help individuals with clutter challenges.

    Investigator

    Not accepting patients at this time View Details

  • Open-label Extension Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis

    The rationale for this study is to provide additional treatment with NEOD001 for subjects who complete Study NEOD001-001, and to continue to evaluate long term safety and tolerability. All subjects in the current NEOD001 trials are being dosed at 24 mg/kg, which will be continued in this study.

    Investigator

    Not accepting patients at this time View Details

  • Hyperpolarized Carbon C 13 Pyruvate Magnetic Resonance Spectroscopic Imaging in Detecting Lactate and Bicarbonate in Participants With Central Nervous System Tumors

    This early phase I trial studies how well hyperpolarized carbon C 13 pyruvate magnetic resonance imaging works in detecting lactate and bicarbonate in participants with central nervous system tumors. Hyperpolarized carbon C 13 pyruvate magnetic resonance imaging may be used to measure the metabolic state of malignant brain tumors.

    Investigators

    Not accepting patients at this time View Details

  • Nanatinostat Plus Valganciclovir in Patients With Advanced EBV+ Solid Tumors, and in Combination With Pembrolizumab in EBV+ RM-NPC

    This study will evaluate the safety and efficacy of nanatinostat in combination with valganciclovir in patients with relapsed/refractory EBV-positive solid tumors and in combination with pembrolizumab in patients with recurrent/metastatic nasopharyngeal carcinoma

    Investigator

    Not accepting patients at this time View Details

  • Immunobiology of Cancer

    To learn whether or not an Interferon defect in cell signaling, recently discovered in immune cells from melanoma patients as well as breast cancer patients, is common to all cancers.

    Investigator

    Not accepting patients at this time View Details

  • Neuroblastoma Precision Trial

    This proposal sets forth the platform for a Precision Medicine clinical trial through the New Approaches to Neuroblastoma Therapy (NANT) consortium. The plan is to utilize NANT's established multi-institutional infrastructure and Translational Genomics Research Institute GEM sequencing platform for acquisition and gene panel sequencing of relapsed biological specimens in relapsed/refractory neuroblastoma (rNB) including those obtained from the bone, bone marrow or soft tissue. Our primary aim is to identify subgroups of rNB patients who have potentially targetable genetic (ALK, MAPK pathway, Metabolic-related genes) and/or immunologic (tumor-associated macrophage infiltration and/or programmed death ligand [PD-L1] expression) biomarkers in rNB. Additional potential novel biomarkers will also be evaluated and reported in this cohort of patients.

    Investigator

    Not accepting patients at this time View Details

  • Nivolumab After Combined Modality Therapy in Treating Patients With High Risk Stage II-IIIB Anal Cancer

    This phase III trial investigates how well nivolumab after combined modality therapy works in treating patients with high risk stage II-IIIB anal cancer. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.

    Investigator

    Not accepting patients at this time View Details

  • High-Field MRI Iron-Based Contrast-Enhanced Characterization of Multiple Sclerosis and Demyelinating Diseases

    Feraheme (ferumoxytol) is FDA-approved for iron supplementation and is composed of iron oxide nanoparticles classified among the ultra-small superparamagnetic iron oxides (USPIO). In this project we hypothesize that Feraheme could become a sensitive and specific marker of active inflammation in multiple sclerosis. We will explore this hypothesis taking advantage of ultra high field strength (7T) MRI to further increase the effectiveness of the contrast agent Feraheme at revealing inflammatory activity.

    Investigator

    Not accepting patients at this time View Details

  • Ibrutinib in Treating Patients With Advanced Systemic Mastocytosis

    This phase 2 trial studies ibrutinib to see how well it works in treating patients with systemic (affecting the entire body) mastocytosis that has spread to other parts of the body and usually cannot be cured or controlled with treatment (advanced). Systemic mastocytosis is a disease in which too many mast cells (a type of immune system cell) are found throughout the body. Mast cells give off chemicals such as histamine that can cause flushing (a hot, red face), itching, abdominal cramps, muscle pain, nausea, vomiting, diarrhea, low blood pressure, and shock. Ibrutinib may stop the growth of mast cells by blocking some of the enzymes needed for cell growth.

    Not accepting patients at this time View Details

  • Pivotal Response Treatment for Individuals With Intellectual Disabilities

    The investigators will assess the efficacy of Pivotal Response Treatment (PRT) in the treatment of communication deficits in children with intellectual disabilities. By collecting information about parent and child functioning before and after PRT, The investigators will be able to determine whether the intervention is effective in improving child communication and reducing parent stress.

    Investigator

    Not accepting patients at this time View Details

Our research team includes physicians, residents, medical students, research assistants, and volunteers. Our research topics include medical imaging, device validation,  mobile application development, and pharmaceutical trials.  

Some of the Neuro-Opthalmic concerns we investigate include Multiple Sclerosis, Optic Neuritis, IIH, and ICP.