Education and Training

  • Erlotinib and Momelotinib for the Treatment of Epidermal Growth Factor Receptor (EGFR) Mutated EGFR Tyrosine Kinase Inhibitor (TKI) Naive Metastatic Non-Small Cell Lung Cancer (NSCLC)

    This study will evaluate the safety, preliminary efficacy, and pharmacokinetics (PK) of momelotinib (MMB) and erlotinib, as well as define the maximum tolerated dose (MTD) of momelotinib (MMB) combined with erlotinib in adults with epidermal growth factor receptor (EGFR)-mutated, EGFR tyrosine kinase inhibitor (TKI) naive metastatic non-small cell lung cancer (NSCLC). Participants will be sequentially enrolled to receive progressively increasing doses of momelotinib (MMB) in combination with erlotinib. Escalation of momelotinib (MMB) doses will proceed to the MTD, defined as the highest tested dose associated with dose-limiting toxicities (DLT) during the first 28 days of combined erlotinib and momelotinib (MMB) treatment. There will be four dose levels and each treatment cycle will consist of 28 days.

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  • Long-term Lung Function and Disease Progression in Children With Early Onset Primary Ciliary Dyskinesia Lung Disease

    Primary ciliary dyskinesia (PCD), also known as Kartagener syndrome, is a genetic disorder of the cilia, which are microscopic hair-like cells. Cilia work to keep the respiratory system clean by moving mucus that contains debris to the large airways, where it can be coughed out. People with PCD have cilia that do not move properly and therefore are not effective in cleaning the respiratory system. This study will determine when PCD starts and how it changes over time, specifically in terms of how well the lungs work, what germs grow in lung secretions, and how the lungs look on computed tomography (CT) scans.

    Investigators

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  • Patient Functioning and Well-being, Economic, and Clinical Impact of Hemophilia A and Its Treatment

    The purpose of the study is to improve the understanding of key patient reported outcomes such as quality of life as well as clinical outcomes in hemophilia A, in a global real world setting.

    Investigator

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  • Evaluation of the Use of Apple Watch Features for Identification of Cardiac Arrhythmias

    This post-marketing study is conducted to characterize the ability of Apple Watch rhythm analysis software to identify Atrial Fibrillation (AF) and facilitate subsequent clinical evaluation among users who contact AppleCare.

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  • Long-term Safety of Dasatinib in Patients With Chronic Myelogenous Leukemia or Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia

    This study assesses the long-term safety and tolerability of dasatinib administered to patients with chronic myelogenous leukemia or Philadelphia chromosome positive acute lymphoblastic leukemia and experienced clinical benefit from treatment with dasatinib or imatinib in previous protocols.

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  • GTSCOPE - To Evaluate the Natural Progression of Dry Age-related Macular Degeneration (AMD)

    An observational study to evaluate the natural progression of dry AMD in genetically defined subjects

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  • ExAblate UF V2 System for the Treatment of Symptomatic Uterine Fibroids

    The purpose of this study is to evaluate the safety and ablation efficacy of the ExAblate UF V2 System when treating symptomatic uterine fibroids.

    The ExAblate System is a medical device that involves a focused ultrasound system and an MRI scanner. ExAblate delivers a pulse of focused ultrasound energy, or sonication, to the targeted tissue. In this particular study, the targeted tissue is uterine fibroids. Each sonication is used to heat small spots in the fibroid much like a magnifying glass can be used to focus light to heat a spot. The heat created kills a portion of the fibroid with the goal of decreasing or eliminating uterine fibroid-related symptoms. Repeated sonications are performed until the entire fibroid is treated or the treated volume is determined to be appropriate.

    The ExAblate system is commercially approved in the United States to treat symptomatic uterine fibroids.

    The ExAblate UF V2 System is an experimental device and is being investigated in this study. While similar to the commercial system, the ExAblate UF V2 device includes the following major changes, among others, which are intended to improve device performance and safety:

    - Up and down movement of the ultrasound transducer, in an attempt to improve fibroid treatment by moving the ultrasound focal point within the targeted fibroid.

    - Ultrasound energy can be turned off for a specific area in an attempt to minimize amount of energy passing through sensitive areas of the body.

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  • Denosumab in Treating Patients With Recurrent or Refractory Osteosarcoma

    This phase II trial studies how well denosumab works in treating patients with osteosarcoma that has come back (recurrent) or does not respond to treatment (refractory). Immunotherapy with monoclonal antibodies, such as denosumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.

    Investigator

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  • Functional Magnetic Resonance Imaging (MRI) of Hypnosis and Mindfulness Meditation

    Hypnotic trance and mindfulness meditation have both been shown to have benefits for physical health. The current study seeks to determine if there are distinct patterns of brain activity that correlate with hypnotic trance, mindfulness meditation or both, and to relate these patterns to measurable markers of physical well-being. Precise neuroimaging of heightened attentional states will guide future researchers and practitioners toward more effective techniques of mind/body control.

    Investigator

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  • Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

    This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).

    Investigator

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  • Preventing HIV Among Native Americans Through the Treatment PTSD & Substance Use

    Investigators will conduct a two-arm, comparative effectiveness randomized controlled trial of two culturally adapted, empirically based programs (EBP) - Narrative Exposure Therapy (NET) vs. Motivational Interviewing with Skills Training (MIST) in terms of lowering HIV sexual-risk behaviors (HSB) for American Indian / Alaska Native (AIAN) men and women. NET addresses Posttraumatic Stress Disorder (PTSD) as a pathway to preventing substance use disorders (SUD) and HSB whereas MIST addresses substance misuse as a way of preventing SUD and HSB.

    Investigator

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  • Pioglitazone in Patients With Mood Disorders

    The purpose of this study is to see how an insulin sensitizing medication, Pioglitazone, can cause changes in mood in some depressed patients. Study participants receive assessment of their cognitive and metabolic functioning.

    If they meet criteria, they will be asked to take either Pioglitazone or a placebo for a 90-day trial. Participants will undergo an Oral Glucose Tolerance Test to measure fasting insulin and glucose levels, as well as routine blood testing.

    The investigators hope to quantify the role of Pioglitazone in patients with mood disorders and compare the values to those previously obtained in a healthy age-matched control population. The investigators also hope to examine the association between IR and cognitive performance and clinical course of depression in patients with mood disorders.

    Investigator

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  • Efficacy and Safety Study of Adjunctive Troriluzole in Obsessive Compulsive Disorder

    The study's purpose is to evaluate the efficacy and safety of troriluzole as adjunctive therapy compared to placebo in subjects with Obsessive Compulsive Disorder (OCD)

    Investigator

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  • Liraglutide and a Calorie Restricted Diet Augments Weight Loss and Decreases Risk of Type 2 Diabetes and CVD.

    The goal of this study is to evaluate the hypothesis that the addition of liraglutide, a long-acting glucagon-like peptide 1 (GLP-1) analogue, to a calorie-restricted diet will lead to greater weight loss than will a calorie-restricted diet alone in subjects who are older (50 to 60 years of age), overweight/obese, and prediabetic. These individuals have been selected for study because they are at greatly increased risk to develop type 2 diabetes (2DM) and cardiovascular disease (CVD), and it is hypothesized that the addition of liraglutide to a calorie-restricted diet will significantly decrease risk of these adverse outcomes.

    There is considerable evidence that GLP-I compounds, including liraglutide, improve glycemic control in patients with manifest 2DM. However, there is relatively little information as to the potential utility of these compounds in nondiabetic individual at greatly increased risk of 2DM and CVD. This research proposal is aimed at providing some of this information by quantifying the effects of liraglutide, a long-acting GLP-1 analogue, on weight loss, insulin secretion, insulin action, and multiple CVD risk factors in a very high risk group-older, overweight/obese, prediabetic individuals. Furthermore, by using specific methods, not surrogate estimates, and avoiding the confounding effects of glucotoxicity, it will be possible to gain new insights into the effects of GLP-1 on insulin secretion and insulin action.

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  • 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

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  • Language Development in Preterm Children

    This research investigates the ways in which preterm birth affects how very young children learn to speak and understand language, and how older children gain efficiency in language processing. The investigators observe how children at different ages learn new words and comprehend familiar words, how they communicate effectively with others, and how they use both linguistic and non-linguistic skills in problem-solving. All of the activities in the investigators' studies are designed to be age-appropriate and fun for children.

    Investigator

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  • Optimizing Fidelity to Family-Based Treatment for Adolescent Anorexia Nervosa

    This study will use a data base of archived therapy sessions of family therapy for adolescent anorexia nervosa to determine the role of fidelity to treatment and outcome. In addition, it will develop a novel, more efficient way to train therapists in family therapy for adolescent anorexia nervosa and examine if it is feasible to conduct a trial comparing this novel training to standard, more intensive training.

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  • International Consortium for Multimodality Phenotyping in Adults With Non-compaction

    Non-compaction cardiomyopathy (NCCM) is a heterogeneous, poorly understood disorder characterized by a prominent inner layer of loose myocardial tissue, and associated with heart failure, stroke, severe rhythm irregularities and death. For a growing population diagnosed with NCCM there is a need for better risk stratification to appropriately allocate (or safely withhold) these impactful preventive measures. The goal of this international consortium is to improve care of patients with non-compaction cardiomyopathy. We hypothesize that comprehensive analysis of clinical, genetic, structural and functional information will improve risk stratification. In addition, we hypothesize that detailed structural analysis will allow for differentiation of pathological and benign patterns of non-compaction. In a large cohort of adult patients with suspected NCCM we will perform in-depth phenotyping, including clinical information, pedigree data, genetics, echocardiography and MRI, and follow patients for up to 3 years. We will apply machine-learning based analytics to develop predictive models and compare their performance to currently used models and treatment criteria. Secondly, in a subset of patients we will perform high-resolution cardiac CT for detailed structural characterization of the myocardial wall. We will investigate associations between myocardial structure and regional contractile function, as assessed by echo and MRI. The aim of this proposal is to identify a structural signature associated with pathological non-compaction and improve developed risk prediction models. Discovery of pathological structural signatures through innovative imaging techniques, in relation to myocardial contractility, will advance our understanding of NCCM.

    Investigator

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  • Efficacy and Safety Study of Desmoteplase to Treat Acute Ischemic Stroke (DIAS-4)

    The purpose of the study is to determine whether desmoteplase is effective and safe in the treatment of patients with acute ischaemic stroke when given within 3 to 9 hours from onset of stroke symptoms.

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  • Exablate Blood-Brain Barrier Disruption for the Treatment of rGBM in Subjects Undergoing Carboplatin Monotherapy

    The purpose of this study is to evaluate the safety and feasibility of the Exablate Model 4000 Type 2 system when used as a tool to open the blood-brain-barrier (BBB) in subjects with recurrent glioblastoma (rGBM) undergoing carboplatin monotherapy.

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