Education and Training

  • The PRONTO Study, a Global Phase 2b Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis

    This is a global, multicenter, Phase 2b, randomized, double-blind, placebo-controlled, two-arm, parallel-group efficacy and safety study of NEOD001 as a single agent administered intravenously in adults with AL amyloidosis who had a hematologic response to previous treatment for their amyloidosis (e.g., chemotherapy, autologous stem cell transplant [ASCT]) and have persistent cardiac dysfunction.

    Investigator

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  • Quantification of Upper Extremity Hypertonia

    Dr. Sanger and the research team want to learn about upper extremity hypertonia (stiffness) in children and young adults with cerebral palsy. Specifically, they want to learn about ways to measure the stiffness in the joints of hypertonic arms. The information the research team will collect includes any physical exams or computer generated data about your arm movements.

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  • To Evaluate the Safety and Efficacy for GORE TAG Thoracic Endoprosthesis in the Treatment of Thoracic Aortic Disease

    PURPOSE OF RESEARCH:

    Endovascular stent-graft repair of aortic pathologies is a minimally-invasive alternative to open surgery that may decrease morbidity and mortality, particularly in high risk patients. Optimal patient selection, based on pathology and anatomy, is being defined. Technically successful implantation requires adequate assessment of pathology and anatomy, and development and execution of novel and delicate procedures that resolve the pathology while minimizing morbidity and mortality.

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  • SARS-COV-2 Screening in Dialysis Facilities

    Patients receiving dialysis are one of the highest risk groups for serious illness with SARS-CoV-2 infection. In addition to the inherent risks of travel to and dialysis within indoor facilities, patients receiving dialysis are more likely to be older, non-white, from disadvantaged backgrounds, and have impaired immune responses to viral infections and vaccinations. Universal testing offered at hemodialysis facilities could shield this vulnerable population from exposure, enable early identification and treatment for those affected, and reduce transmission to other patients and family members. In this pragmatic cluster randomized controlled trial as part of NIH RADx-UP Consortium, we will randomize 62 US Renal Care facilities with an estimated 2480 patients to static versus dynamic universal screening testing strategies. Static universal screening will involve offering patients SARS-CoV-2 screening tests every two weeks; the dynamic universal screening strategy will vary the frequency of testing from once every week to once every four weeks, depending on community COVID-19 case rates. We hypothesize that patients dialyzing at facilities randomized to a dynamic testing frequency responsive to community case rates will have higher test acceptability (primary outcome), experience lower rates of COVID-19 death and hospitalization, and report better experience-of-care metrics.

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  • Trial of Strategies to Communicate Genetic Information to Different Ethnic Subpopulations

    The goal of this study is to better understand how clinicians give genetic risk information to patients from multi-ethnic groups and how patients understand this information and remember it and act upon it. In addition investigators want to know how to better communicate with patients about complex health issues across the health literacy divide and communication gap that exists between doctors and their patients.

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  • RRx-001 in Lung Cancer, Ovarian Cancer and Neuroendocrine Tumors Prior to Re-administration of Platinum Based Doublet Regimens (QUADRUPLE THREAT)

    This study is designed to explore the potential of the epigenetic agent RRx-001 to sensitize patients who previously received and now have failed a platinum based doublet regimen. RRx-001 is administered with autologous blood once weekly followed by or in combination with reintroduction of platinum-based doublet therapy.

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  • QUILT-3.026: AMG 655 in Combination With AMG 479 in Advanced, Refractory Solid Tumors

    This is a multi-center, 2-part phase 1b/2 study of AMG 655 in combination with AMG 479 to be conducted in the United States and Spain.

    Part 1 is a dose escalation segment to identify a dose of AMG 655 in combination with AMG 479 that is safe and tolerable.

    Part 2 will evaluate the safety and estimate the efficacy of AMG 655 at the dose selected in Part 1 in combination with AMG 479 for the treatment of patients with advanced NSCLC (non-squamous histology; squamous histology), CRC, pancreatic cancer, ovarian cancer, and sarcoma.

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  • The PLATFORM Study: Prospective LongitudinAl Trial of FFRct: Outcome and Resource IMpacts)

    The objective of the PLATFORM Study is to compare clinical outcomes, resource utilization, and quality of life (QOL) of FFRCT-guided evaluation versus standard practice evaluation in patients with suspected CAD in order to further inform patients, health care providers, and other stakeholders about which technologies are most effective and efficient in the diagnosis of CAD

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  • Transcatheter Mitral Valve Replacement With the Medtronic Intrepid™ TMVR System in Patients With Severe Symptomatic Mitral Regurgitation.

    Multi-center, global, prospective, non-randomized, interventional, pre-market trial. All subjects enrolled with receive the study device.

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  • Ruxolitinib Phosphate in Treating Patients With Chronic Neutrophilic Leukemia or Atypical Chronic Myeloid Leukemia

    This phase II trial studies how well ruxolitinib phosphate works in treating patients with chronic neutrophilic leukemia (CNL) or atypical chronic myeloid leukemia (aCML). Ruxolitinib phosphate may stop the growth of cancer cells by blocking some of the enzymes needed for cells to reproduce. This trial also studies the genetic makeup of patients. Certain genes in cancer cells may determine how the cancer grows or spreads and how it may respond to different drugs. Studying how the genes associated with CNL and aCML respond to the study drug may help doctors learn more about CNL and aCML and improve the treatment for these diseases.

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  • Radiation Therapy, Paclitaxel, and Carboplatin With or Without Trastuzumab in Treating Patients With Esophageal Cancer

    This randomized phase III trial studies how well radiation therapy, paclitaxel, and carboplatin with or without trastuzumab work in treating patients with esophageal cancer. Radiation therapy uses high-energy x-rays to kill tumor cells. Drugs used in chemotherapy, such as paclitaxel and carboplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Monoclonal antibodies, such as trastuzumab, may interfere with the ability of tumor cells to grow and spread. It is not yet known whether giving radiation therapy and combination chemotherapy together with or without trastuzumab is more effective in treating esophageal cancer.

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  • Study to Intervene With Nutrition for Gastroparesis

    The study is a self-controlled study in which we will assess the nutritional effects of the Kate Farm Peptide 1.5 nutritional Formula in patients with gastroparesis, relative to their pre-enrollment nutritional diet regimen.

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  • The Pediatric Artificial Pancreas (PEDAP) Trial of Control-IQ Technology in Young Children in Type 1 Diabetes

    The purpose of this study is to learn whether an investigational automated insulin delivery system ("study system") for young children (2 yo to less than 6 yo) with type 1 diabetes can safely improve blood glucose (sometimes called blood sugar) control.

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  • The IDEA Study (Individualized Decisions for Endocrine Therapy Alone)

    This study will collect rates of local/regional recurrence in select patients who do not receive radiation treatment after lumpectomy surgery. These women must be postmenopausal; have hormone receptor-positive, Her2-negative tumors; have Oncotype-DX RS less than or equal to 18; and plan to receive endocrine therapy. In this way, this study seeks to collect prospective data supporting the idea that this is a population at sufficiently low risk of local/regional recurrence that omission of adjuvant radiation might be a reasonable option.

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  • Relacorilant in Combination With Nab-Paclitaxel in Advanced, Platinum-Resistant, High-Grade Epithelial Ovarian, Primary Peritoneal, or Fallopian-Tube Cancer

    The primary objective of this study is to evaluate progression-free survival (PFS) by blinded independent central review (BICR) in patients treated with intermittent regimen of relacorilant in combination with nab-paclitaxel compared with patients treated with nab-paclitaxel monotherapy.

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  • Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990

    Wiskott - Aldrich syndrome (WAS) is a rare serious medical condition that causes problems both with the immune system and with easy bruising and bleeding. The immune abnormalities cause patients with WAS to be very susceptible to infections. Depending on the specific type of primary immune deficiency diseases, there are effective treatments, including antibiotics, cellular therapy and gene therapy, but studies of large numbers of patients are needed to determine the full range of causes, natural history, or the best methods of treatment for long term success.

    This multicenter study combines retrospective, prospective and cross-sectional analyses of the transplant experiences for patients with WAS who have already received HCT since 1990, or who will undergo Hematopoietic cell transplant (HCT) during the study period. The retrospective and prospective portions of the study will address the impact of a number of pre and post-transplant factors on post-transplant disease correction and ultimate benefit from HCT and the cross-sectional portion of the study will assess the benefit of HCT 2 years post-HCT in consenting surviving patients.

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  • Talazoparib + Enzalutamide vs. Enzalutamide Monotherapy in mCRPC

    This study compares rPFS in men with mCRPC treated with talazoparib plus enzalutamide vs. enzalutamide after confirmation of the starting dose of talazoparib in combination with enzalutamide.

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  • Post-operative Adjuvant Treatment for HPV-positive Tumours (PATHOS)

    The main objectives of the PATHOS study are:

    To assess whether swallowing function can be improved following transoral resection of HPV-positive OPSCC, by reducing the intensity of adjuvant treatment protocols. The aim is to personalise treatment, based on disease biology (HPV status and pathology findings), to optimise patient outcomes.

    To demonstrate the non-inferiority of reducing the intensity of adjuvant treatment protocols in terms of overall survival in the reduced intensity treatment arms.

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  • Radiation Therapy, Amifostine, and Chemotherapy in Treating Young Patients With Newly Diagnosed Nasopharyngeal Cancer

    This phase III trial is studying how well radiation therapy, amifostine, and chemotherapy work in treating young patients with newly diagnosed nasopharyngeal cancer. Radiation therapy uses high-energy x-rays to kill tumor cells. Drugs, such as amifostine, may protect normal cells from the side effects of radiation therapy. Drugs used in chemotherapy, such as cisplatin and fluorouracil, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving radiation therapy together with amifostine and chemotherapy may kill more tumor cells.

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  • The Combination of Venetoclax and Obinutuzumab in People With Chronic Lymphocytic Leukemia (CLL)

    This study will help researchers collect more information about how effective the combination of venetoclax and obinutuzumab is in treating CLL in people who have not received a previous treatment for their cancer.

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