Jul 1st, 2022 - The purpose of this protocol is to study the natural history of spondyloarthritis (SpA) in children and adults. Spondyloarthritis encompasses a spectrum of immune-mediated inflammatory diseases that exhibit overlapping features, but differ from other types of inflammatory arthritis in genetic predisposition, pathogenesis, and outcome. Ankylosing spondylitis (AS), the most common form of SpA, fr...

Jun 21st, 2022 - The hypothesis is that the addition of daratumumab administered SC to standard MM regimens will improve responses compared to response data observed in completed phase 3 studies without daratumumab. Disease evaluations will include measurements of myeloma proteins, bone marrow examinations, skeletal surveys, assessment of extramedullary plasmacytomas, and measurements of serum calcium corrected...

Jul 1st, 2022 - Between 80 and 100 allogeneic stem cell transplants (allo-HSCT) are performed every year at the NIH to treat a variety of malignant and nonmalignant conditions. The current transplant protocols at the NIH focus on research regarding the response of the underlying disease, the development of graft versus host disease (GVHD) as well as the feasibility and safety of a variety of transplant strateg...

Jul 1st, 2022 - Title: Electrical Impedance Myography: Natural History Studies in Neuromuscular Disorders and Healthy Volunteers Study Description: This is a natural history study examining the utility of electrical impedance myography (EIM) device in the evaluation of neuromuscular disorders. This is a novel device so the initial step was to establish reproducibility of the data and expected normative values ...

Jul 1st, 2022 - Obesity leads to an increased risk of cardiovascular risk factors and death from cardiovascular disease. Therefore, interventions that slow or reverse the obesity epidemic are essential. Community-based interventions can reach those most at risk for obesity and obesity-related cardiovascular risk factors. Interventions based on community-based participatory research (CBPR) principles develop in...

Jul 1st, 2022 - Background: Endocrine neoplasms are among the fastest growing tumors in incidence in the United States. Between 1995 and 2005, the incidence of thyroid carcinoma has increased 98 percent. Tumors of the thyroid, parathyroid, adrenal gland and gastrointestinal-pancreatic neuroendocrine tumors are among some of the most difficult tumors to clinically and histopathological distinguish as benign or ...

Jul 1st, 2022 - PRIMARY OBJECTIVES: I. To determine whether the addition of erlotinib (erlotinib hydrochloride) to gemcitabine (gemcitabine hydrochloride) adjuvant chemotherapy shows a signal for improved survival as compared to gemcitabine alone following R0 or R1 resection of head of pancreas adenocarcinoma (including adenocarcinoma of the head, neck, and uncinate process). (Phase II-R) II. To determine whet...

Jul 1st, 2022 - Herpes simplex virus 2 (HSV-2) causes genital herpes and increases the risk of acquisition and transmission of HIV. An HSV-2 vaccine is not available. We will study a replication-defective HSV-2 vaccine, HSV529, that is deleted for 2 essential viral proteins, that can infect, but not replicate in normal cells. The goals of the study are to determine (a) the safety of HSV529 vaccine in persons w...

Jul 1st, 2022 - Viral infections in the normal host are usually self-limited as the innate and acquired immune systems mount successful antiviral responses. However, in some instances, apparently immunocompetent persons manifest infections with viruses that would otherwise be observed only in severely immunocompromised hosts. For example, cases of herpes simplex virus (HSV) encephalitis, esophagitis orgastriti...

Jul 1st, 2022 - Combination antiretroviral therapy (ART) has dramatically improved survival in individuals with human immunodeficiency virus type1 (HIV-1) infection. Despite recent development of more potent regimens with fewer toxicities and lower pill burden, there remains a subpopulation of subjects who fail to achieve and maintain viral suppression while on treatment. Factors known to contribute to virolog...

Jul 1st, 2022 - Over the past two decades, the number of subjects with cardiometabolic diseases (CMD) such as atherosclerotic cardiovascular disease (CVD), dyslipidemia, insulin resistance and diabetes have been rising. Characterizing these disease states reveals that inflammation is a common feature of CMD; however, mechanistic links between inflammation and these disease states in humans remain poorly unders...

Jul 1st, 2022 - Objectives The purpose of this protocol is to improve understanding of the pathophysiology of movement disorders by performing small behavioral, electrophysiological and neuroimaging sub-studies. This will allow identifying dysfunction of the central nervous system that causes behavioral abnormalities seen in movement disorder patients. This will also help to determine potential diagnostic or t...

Jul 1st, 2022 - Background: Recent advances and insights into the molecular pathogenesis of cancer have led to the development of novel molecular and biologic targeted therapies for the treatment of advanced cancer patients. A critical challenge in extending these studies involves the identification and validation of new therapeutic targets for future cancer therapies. As Surgical Oncologists at the NCI, we ha...

Jul 1st, 2022 - This multicenter prospective study will evaluate the safety and efficacy of Avastin (bevacizumab) in routine clinical practice in patients with advanced/metastatic epithelial ovarian cancer, fallopian tube cancer or primary peritoneal cancer. Data will be collected from eligible patients until death, withdrawal of consent, loss to follow-up, or study closure.

Jul 1st, 2022 - The goal of this protocol is to explore changes to microbial communities during the course of hospitalcare, and to determine the extent to which these alterations affect whether hospitalized patients become colonized with healthcare-associated organisms. The study enables collection of clinical specimens and associated medical data to evaluate the natural history, epidemiology, and genomics of ...

Jul 1st, 2022 - Mutations in GATA2, a critical hematopoietic transcription factor, underlie a complex congenital disorder characterized by immunodeficiency, bone marrow failure, and lymphatic/vascular dysfunction. Patients with GATA2 deficiency may suffer from a striking variety of diseases including severe and recurrent infections, myelodysplasia/leukemia, pulmonary alveolar proteinosis, lymphedema, sensorine...

Jul 1st, 2022 - The proposed study aims to test the effect of the degree of perceived uncertainty on coping efficacy among parents of children with undiagnosed medical conditions. There are many dimensions to uncertainty when there is no identified cause for a condition that affects one s child. They include illness identity, management, longevity and life planning, the meaning of the child s condition for the...

Jul 1st, 2022 - Objective The primary objective of this study is to characterize the natural history of disease in patients who carry a repeat expansion in the C9ORF72 gene, which causes amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The secondary objective is to assess whether candidate biomarkers correlate with disease progression. Study population 62 persons with a documented repeat ...

Jul 1st, 2022 - The purpose of this study is to evaluate the hemoglobin response (efficacy), safety, and tolerability of orally administered AKB-6548 in participants with Chronic Kidney Disease (pre-dialysis) with anemia with dosing for 20 weeks.

Jul 1st, 2022 - OUTLINE: CONDITIONING REGIMEN: Patients undergo total body irradiation (TBI) twice daily (BID) on days -10 to -7, receive thiotepa intravenously (IV) over 4 hours on days -6 and -5 and fludarabine phosphate IV over 30 minutes on days -6 to -2. TRANSPLANT: Patients undergo CD34+ enriched, CD45RA+ T cell-depleted allogeneic PBSCT on day 0. POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive tacro...