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About 1,050,810 results

Natural History and Development of Spondyloarthritis
https://clinicaltrials.gov/ct2/show/NCT01422694

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...

A Study to Evaluate Subcutaneous Daratumumab in Combination With Standard Multiple Myeloma Treatment Regimens
https://clinicaltrials.gov/ct2/show/NCT03412565

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...

Effects of Sucralose on Drug Absorption and Metabolism (The SweetMeds Study)
https://clinicaltrials.gov/ct2/show/NCT03407079

Jul 1st, 2022 - Background: Consumption of non-nutritive sweeteners (NNS) has dramatically increased worldwide and is more prevalent in women than men. Similarly, obesity rates have continued to rise, most notably in minorities. Since NNS consumption has been linked to obesity, we propose studying NNS effects specifically in minority women. NNS are frequently consumed in combination with prescription medicatio...

Study of People With Generalized Arterial Calcification of Infancy (GACI) or Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2)
https://clinicaltrials.gov/ct2/show/NCT03478839

Jul 1st, 2022 - This is a natural history study of patients with Generalized Arterial Calcification of Infancy (GACI) or Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2). GACI is an ultra-rare disorder with an estimated birth prevalence of around 1 in 200,000. GACI is characterized by extensive arterial calcifications, arterial stenosis, myointimal proliferation and periarticular calcifications. Ind...

Investigating Epilepsy: Screening and Evaluation
https://clinicaltrials.gov/ct2/show/NCT03478852

Jul 1st, 2022 - Study Description: This protocol is designed to serve as a screening protocol that provides standard evaluation and treatment for patients with epilepsy. Clinical data collected through this protocol will be used in other NIH epilepsy-related research to screen for eligibility in the respective protocols and may also be used for descriptive and/or correlative research through this protocol...

Vitamin A Palmitate Supplementation in Patients With Reticular Pseudodrusen (RPD) and Delayed Dark Adaptation
https://clinicaltrials.gov/ct2/show/NCT03478878

Jul 1st, 2022 - Objective: The objective of this study is to investigate the potential efficacy and safety of vitamin A palmitate dosing in improving dark adaptation in participants with reticular pseudodrusen (RPD) and abnormal dark adaptation. Study Population: The first cohort consists of seven participants with RPD who meet the eligibility criteria. The second cohort will consist of five participants with ...

Early Diagnosis of Sickle Acute Chest Syndrome Using a Combination of Plasma Bimarkers and Chest Imaging
https://clinicaltrials.gov/ct2/show/NCT03478917

Jul 1st, 2022 - Painful vasoocclusive crisis (VOC) is the most frequent acute clinical manifestation of sickle cell disease (SCD), frequently necessitating emergency department visits and/or hospitalization. Approximately 10-30% of patients admitted to the hospital with a VOC will go on to develop acute chest syndrome (ACS), a complication of SCD resulting in significant morbidity and mortality. While ACS rela...

Vitamin A Palmitate Supplementation in People With Age-Related Macular Degeneration (and Without Reticular Pseudodrusen) and Delayed Dark Adaptation
https://clinicaltrials.gov/ct2/show/NCT03478865

Jul 1st, 2022 - Objective: The objective of this study is to investigate the potential efficacy and safety of vitamin A palmitate dosing in improving dark adaptation in participants with age-related macular degeneration (AMD) and abnormal dark adaptation. Study Population: The first cohort consists of five participants with AMD who meet the eligibility criteria. The second cohort will consist of five participa...

Precision Diagnosis Directing HDACi and TKI Target Therapy for Adult Ph-like ALL
https://clinicaltrials.gov/ct2/show/NCT03564470

Jul 1st, 2022 - Ph-like, or BCR-ABL1-like lymphoblastic leukemia is neoplasm of lymphoblasts committed to the B-cell lineage that lack the BCR-ABL1 translocation but show a pattern of the expression very similar to that seen in BCR-ABL1-positive ALL. Chidamide is a novel oral HDACi with promising activity in non-Hodgkin lymphoma (NHL). Based on the pediatric-inspired, PEG-L-asparaginase-intensified and MRD-dir...

Regorafenib in Indian Patients With Metastatic Colorectal Cancer (mCRC).
https://clinicaltrials.gov/ct2/show/NCT03564938

Jul 1st, 2022 - This is a Phase IV, single-arm, prospective, open-label, multicenter, interventional study to evaluate safety and efficacy of regorafenib in patients with mCRC who have been previously treated with fluoropyrimidine , oxaliplatin-, and irinotecan based chemotherapy, an anti-VEGF therapy, and, if RAS wild type, an anti-EGFR therapy.

High Image Matrix CT Reconstruction
https://clinicaltrials.gov/ct2/show/NCT02610465

Jul 1st, 2022 - Computed Tomography (CT) images the body. Different methods of creating CT pictures are avilable on a research basis that enables higher resolution imaging using the same aquisition. The purpose of this research study is to learn wheteher these new high resolution images derived from the original CT scan provide similar or perhaps more information than the current standard clinical method.

Lifestyle Interventions for Expectant Mothers (LIFE-Moms Phoenix) - Phoenix
https://clinicaltrials.gov/ct2/show/NCT01812694

Jul 1st, 2022 - LIFE-Moms (Phoenix center) is a randomized clinical trial in overweight and obese pregnant women that will test the effects of an intensive lifestyle intervention on gestational weight gain, control of maternal hyperglycemia, and post-partum return to pre-pregnancy weight. The Phoenix center will be conducted in collaboration between the Phoenix Indian Medical Center (PIMC) nurse midwifery and ...

Evaluation of Measurable Residual Disease in Patients With Acute Myeloid Leukemia as Surrogate Endpoint for Survival
https://clinicaltrials.gov/ct2/show/NCT03549351

Jul 1st, 2022 - Acute myeloid leukemia is a genetically and phenotypically heterogeneous disorder with an incidence of 3 to 4 per 100 000 men and women per year and a median age at diagnosis of about 70 years. Prognosis, especially in older patients, has remained very poor. In patients considered suitable for intensive chemotherapy, the combination of an anthracycline and cytarabine remains the standard of car...

CD19/CD22 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory CD19/CD22-expressing B Cell Malignancies
https://clinicaltrials.gov/ct2/show/NCT03448393

Jul 1st, 2022 - Background: Acute lymphoblastic leukemia (ALL) accounts for approximately 25% of childhood cancer. Survival rates have improved, but outcomes for some subgroups, including infants and young adults remain poor, and survival for patients who relapse is < 50%, despite allogeneic stem cell transplant following second remission. CD19 immune escape has been observed by several groups following CD19-C...

Rod and Cone Mediated Function in Retinal Disease
https://clinicaltrials.gov/ct2/show/NCT02617966

Jul 1st, 2022 - Objective: The objective of this protocol is to investigate local changes in rod and cone photoreceptor function across the retina in healthy volunteers and participants with retinal disease. Study Population: Up to 120 healthy volunteers and 250 participants, age five or older, with retinal disease. Design: This single-center, observational, case-control study will be comprised of three relate...

M7824 and Topotecan or Temozolomide in Relapsed Small Cell Lung Cancers
https://clinicaltrials.gov/ct2/show/NCT03554473

Jul 1st, 2022 - Background Small cell lung cancer (SCLC) is an aggressive cancer with a poor prognosis. Although highly responsive to chemotherapy initially, SCLC relapses quickly and becomes refractory to treatment within a few months. Extrapulmonary small cell cancers are extremely rare and management of systemic disease with chemotherapy is patterned after the approach used in SCLC. The inability to destroy...

Customized Registry Tool for Tracking Adherence to Clinical Guidelines for Head and Neck Cancers
https://clinicaltrials.gov/ct2/show/NCT03546322

Jul 1st, 2022 - The investigators aim to implement and evaluate a health information technology (HIT) registry at an urban, public delivery system. The investigators will not assign specific interventions to the study participants, but rather observationally assess the effect of the HIT registry, designed to support routine medical care, on participants. The investigators will use systems engineering methodolo...

Study of Sacituzumab Govitecan-hziy in Metastatic Urothelial Cancer
https://clinicaltrials.gov/ct2/show/NCT03547973

Jul 1st, 2022 - The objective of this study is to evaluate the efficacy and safety of sacituzumab govitecan-hziy in participants with metastatic urothelial cancer (mUC).

Extracorporeal Elimination of Cytokines Following Abdominal-thoracic Esophagectomy
https://clinicaltrials.gov/ct2/show/NCT02606799

Jul 1st, 2022 - Radical esophagectomy combined with extensive lymphadenectomy for esophageal cancer is one of the most invasive surgical procedures. Even with progress made in surgical technique and postoperative management the rate of short- and long term complications remains high. The surgical trauma invariably causes liberation and activation of inflammatory mediators and danger associated molecular patter...

Vascular Disease Discovery Protocol
https://clinicaltrials.gov/ct2/show/NCT03538639

Jul 1st, 2022 - The NIH provides a unique opportunity for research scientists to participate in the bench-to-bedside process, which is essential for the full implementation of translational medicine programs. The study of rare monogenetic and undiagnosed diseases is a complex bedside-to-bench-to-bedside endeavor. Recent studies show that rare genetic variants are more likely than common variants to have delete...

Just-In-Time Adaptive Interventions for Addictive Behaviors
https://clinicaltrials.gov/ct2/show/NCT03538652

Jul 1st, 2022 - BACKGROUND: Our research group has made extensive use of ecological momentary assessment (EMA) to understand people s daily experiences with opioid craving, use, and lapse. We have also administered clinic-based psychotherapies such as cognitive-behavioral therapy (CBT), in which clients learn to avoid, escape, resolve, or reframe problems (such as stress) that can trigger lapses to drug use. C...

Improving the HIV PrEP Cascade Using an Intervention for Healthcare Providers
https://clinicaltrials.gov/ct2/show/NCT03532191

Jul 1st, 2022 - Despite data indicating nearly 80,000 individuals starting HIV pre-exposure prophylaxis (PrEP) by the end of 2015, the Centers for Disease Control and Prevention (CDC) estimates that there are over 1.2 million adults with a substantial risk for HIV acquisition. Therefore, only 6% of these individuals are receiving PrEP. One of the barriers to PrEP implementation is lack of healthcare provider (...

Investigating Vector-Borne Determinants of Aedes Transmitted Arboviral Infections in Cambodia: An Observational Longitudinal Cohort Study in Children
https://clinicaltrials.gov/ct2/show/NCT03534245

Jul 1st, 2022 - Mosquito-borne viruses continue to cause significant global morbidity and mortality, particularly in Southeast Asia. When mosquitoes deliver the virus into the skin of humans while probing for a blood meal, they deposit also saliva, which contains a myriad of pharmacologically active compounds that modulate the host immune system. Most vaccines against vector-borne diseases under development ig...

VRC 609 Study: A Phase I, Open-Label, Dose-Escalation Study of the Safety and Pharmacokinetics of a Human Monoclonal Antibody, VRC-HIVMAB091-00-AB (N6LS), Administered Intravenously or Subcutaneous...
https://clinicaltrials.gov/ct2/show/NCT03538626

Jul 1st, 2022 - Study Design: This is the first study in healthy adults of the N6LS monoclonal antibody (MAb). It is a dose-escalation study to examine safety, tolerability, dose, and pharmacokinetics (PK) of N6LS administered intravenously (IV) and subcutaneously (SC) to healthy adults. For SC administration, N6LS will be administered alone or coadministered with the permeation enhancer rHuPH20 enzyme. Primar...

Feasibility and Preliminary Efficacy of a Mindfulness-based Intervention for Children and Young Adults With High Grade or High-Risk Cancer and Their Caregivers
https://clinicaltrials.gov/ct2/show/NCT03538587

Jul 1st, 2022 - Background Children and young adults diagnosed with a high-grade or high-risk cancer (e.g., diffuse intrinsic pontine glioma, glioblastoma multiforme, relapsed-refractory leukemia, refractory metastatic sarcomas) face a poor prognosis given limited curative options. Recent research has indicated that this population of patients and their parents experience elevated stress and poorer health-rela...

A Study of Fluzoparib(SHR-3162)in BRCA1/2-mutant Relapsed Ovarian Cancer
https://clinicaltrials.gov/ct2/show/NCT03509636

Jul 1st, 2022 - Fluzoparib (SHR-3162) is an orally available, small molecule inhibitor of poly-adenosine diphosphate [ADP] ribose polymerase (PARP) 1/2 being developed for treatment of BRCA1/2-mutant solid tumor. The tolerability, safety and PK of fluzoparib has been evaluated in Phase 1 study. An oral formulation is the focus of current development efforts.

Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1 (NF1) on Clinical Treatment Trials
https://clinicaltrials.gov/ct2/show/NCT03531814

Jul 1st, 2022 - Background: Neurofibromatosis type 1 (NF1) is a genetic disorder that affects approximately 1 in 3,500 individuals and is associated with a broad variety of symptoms and physical findings. Plexiform neurofibromas (PN) are histologically benign tumors which occur in 25-50% of patients with NF1 and can lead to significant morbidity. Oral therapeutic options for the treatment of plexiform neurofib...

The Vitreous Proteome and Inflammatory Mediators in Ocular Inflammatory Disease
https://clinicaltrials.gov/ct2/show/NCT00331331

Jul 1st, 2022 - Newer therapies to treat many ocular disorders including uveitis are being considered and studied. We do not have a clear picture as to what lymphokines/chemokines are present in the vitreous or anterior chamber fluid of patients with active intraocular inflammatory disease as well as other disorders with a proposed inflammatory component such as age-related macular degeneration (AMD) and diabe...

Myalgic Encephalomyelitis Chronic Fatigue at the National Institutes of Health
https://clinicaltrials.gov/ct2/show/NCT02669212

Jul 1st, 2022 - Objective: The primary objective is to explore the clinical and biological phenotypes of post-infectious myalgic encephalomyelitis/chronic fatigue syndrome (PI-ME/CFS). The secondary objective is to explore the pathophysiology of fatigue and post-exertional malaise (PEM). Study population: Up to 206 persons will be enrolled as part of this protocol. Up to 150 persons aged 18-60 will be part of ...

Brain Amyloid- Retention During Wakefulness and Following Emergence From Sleep in Healthy People
https://clinicaltrials.gov/ct2/show/NCT02669225

Jul 1st, 2022 - Objective: To assess if there are differences in [18F]florbetaben uptake following the first 120 minutes of its injection (reflecting amyloid-beta or Ab load and/or docked Ab) in subjects during rested wakefulness (RW) after normal sleep compared to wakefulness after 24 hrs of sleep deprivation (SD). Specifically, we hypothesize that during RW after a normal night s sleep there will be less [18...

A Study Combining Eribulin Mesylate With Avelumab in Cisplatin Ineligible Metastatic Urothelial Cell Cancer Patients
https://clinicaltrials.gov/ct2/show/NCT03502681

Jul 1st, 2022 - Dose Escalation Plan: A standard "3+3" design will be used to determine the MTD of eribulin with avelumab. The maximum tolerated dose is the dose of eribulin combined with avelumab with dose limiting toxicity of 0-1 of 6 patients in the first cycle of combination therapy. After the MTD has been determined, an additional 12 patients will be enrolled in an expansion cohort at the MTD to evaluate ...

Establishment of a Perinatal Database and a Bank of Biological Materials
https://clinicaltrials.gov/ct2/show/NCT00339235

Jul 1st, 2022 - The aim of this project is to establish a clinical database and a bank of biological materials which will be used to improve the pathophysiologic understanding of the mechanisms underlying various pregnancy diseases. The US-Mexico Reproductive Health Research Development Workshop, sponsored by the NIH, recommended that the setting up of "tissue, blood, and placental banks from human and relevan...

Collection and Distribution of Samples From Healthy Donors for In Vitro Research at the NCI-Frederick
https://clinicaltrials.gov/ct2/show/NCT00339911

Jul 1st, 2022 - Background -NCI-Frederick scientists performing in vitro studies involving human specimens have a need for a reliable and consistent source of samples which protects the donor from research risks. Objective -The purpose of this protocol is to establish a centralized repository for the collection and distribution of samples (either blood, buccal mucosal cells, semen, urine, or nail clippings) fr...

Prospective Studies of the Natural History of Diabetes Mellitus and Its Complications in the Gila River Indian Community
https://clinicaltrials.gov/ct2/show/NCT00339482

Jul 1st, 2022 - This project is a continuation of an extensive longitudinal population-based epidemiological investigation of the etiology and determinants of type 2 diabetes (and its complications) and formerly arthritis. The original baseline observations were made on the residents of the Gila Indian Reservation, predominantly Pima Indians, in 1965. We attempt to reexamine the population at two-yearly interv...

Physicians' Understanding of Human Genetic Variation
https://clinicaltrials.gov/ct2/show/NCT00339924

Jul 1st, 2022 - In recognition of the emerging social and scientific research related to assessing the complex relationships between self-identified race, ancestral origin and the genetic components of diseases, it is important to study physicians and other health professionals knowledge of human genetic variation and how they interpret and apply race, ethnicity and human genetic variation in their clinical de...

Prostate Lung Colorectal and Ovarian (PLCO) Cancer Screening Trial
https://clinicaltrials.gov/ct2/show/NCT00339495

Jul 1st, 2022 - The Division of Cancer Preventionl (DCP, formerly DCPC), under extramural contracts to 10 U.S. clinical centers, is evaluating the effectiveness of screening for prostate, lung, colorectal and ovarian cancer (The PLCO Trial). In 1996 the NCI Executive Committee approved the expansion of the PLCO Trial to collect additional materials and to conduct additional studies. About, 74,000 men and 74,00...

Alvelestat (MPH966), an Oral Neutrophil Elastase Inhibitor, in Bronchiolitis Obliterans Syndrome After Allogeneic Hematopoietic Stem Cell Transplantation
https://clinicaltrials.gov/ct2/show/NCT02669251

Jul 1st, 2022 - Background: Chronic graft-versus-host disease (cGVHD) is the leading cause of non-relapse morbidity and mortality in persons after allogeneic hematopoietic stem cell transplantation (SCT). Bronchiolitis obliterans syndrome (BOS) is a complication of cGVHD associated with a high morbidity and mortality, and treatment options are limited. Neutrophil elastase (NE) is a protease released by neutrop...

Molecular Basis of Human Phagocyte Interactions With Bacterial Pathogens
https://clinicaltrials.gov/ct2/show/NCT00339287

Jul 1st, 2022 - Human phagocytic cells such as polymorphonuclear leukocytes (neutrophils or PMNs) are readily mobilized to sites of infection and ingest microorganisms by a process known as phagocytosis. The combined effects of reactive oxygen species (ROS) and antimicrobial peptides released into forming bacterial phagosomes kill most ingested bacteria. However, many bacterial pathogens have devised means to ...

Genome Expression in Lymphoma, Leukemia and Multiple Myeloma
https://clinicaltrials.gov/ct2/show/NCT00339963

Jul 1st, 2022 - Current diagnosis of the lymphoid malignancies relies upon the morphological appearance of the cancer cells supplemented by a few molecular markers. Within a diagnostic category, the clinical courses and responses of patients to therapy are variable, suggesting that the existing diagnostic categories may harbor more than one disease entity. Recent genomic technologies allow a comprehensive mole...

Natural History, Genetics, and Pathophysiology of Systemic Juvenile Idiopathic Arthritis, Adult-Onset Still's Disease, and Related Conditions
https://clinicaltrials.gov/ct2/show/NCT03510442

Jul 1st, 2022 - The purpose of this protocol is to study the natural history, genetics and pathophysiology of systemic juvenile idiopathic arthritis (sJIA), adult-onset Still s disease (AOSD) and related inflammatory conditions. One of seven subtypes of juvenile idiopathic arthritis (JIA), sJIA contributes disproportionately to the morbidity and mortality of JIA and is considered by many to be the most severe ...

Impact of Weekly Administration of Rifapentine and Isoniazid on Steady State Pharmacokinetics of Tenofovir Alafenamide in Healthy Volunteers (YODA)
https://clinicaltrials.gov/ct2/show/NCT03510468

Jul 1st, 2022 - Rifapentine (RPT) is a long-acting rifamycin that can be used weekly with isoniazid (INH) as a first-line regimen in the treatment of latent tuberculosis infection (LTBI). Although this regimen offers several potential benefits, the use of weekly RPT plus INH is limited in adults infected with human immunodeficiency virus (HIV) on antiretroviral therapy (ART) due to lack of drug interaction dat...

Tool to Assess Psycho-Social-Spiritual Healing: Cognitive Interviewing
https://clinicaltrials.gov/ct2/show/NCT02664402

Jul 1st, 2022 - Background: We identify healing in the context of chronic or life-threatening illness as a patient-reported outcome consisting of growth or benefit in psychological, social and/or spiritual dimensions representing improvement well above the patient s pre-morbidity baseline. This positive outcome often occurs despite substantial suffering during the illness, even in terminal cases. Current liter...

Immunogenicity of the 9-Valent Human Papillomavirus Recombinant Vaccine in People With Idiopathic CD4 T Cell Lymphocytopenia
https://clinicaltrials.gov/ct2/show/NCT03519464

Jul 1st, 2022 - As observed in other immunocompromised individuals with selective or combined T cell deficits, the prevalence and morbidity of human papillomavirus (HPV)-related disease is increased in patients with idiopathic CD4 T cell lymphocytopenia (ICL). The high burden and aggressive clinical course of HPV-associated disease in patients with ICL requires the development of effective preventive measures ...

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
https://clinicaltrials.gov/ct2/show/NCT03520647

Jul 1st, 2022 - Severe aplastic anemia (SAA),myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) are life-threatening bone marrow disorders. For SAA patients, long term survival can be achieved with immunosuppressive treatment. However, of those patients treated with immunosuppressive therapy, one quarter to one third will not respond, and about 50% of responders will relapse. Althoug...

A Response Modulation Hypothesis of Socioemotional Processing Associated With Alcohol Use Disorder
https://clinicaltrials.gov/ct2/show/NCT03535129

Jul 1st, 2022 - Objective The purpose of this protocol is to understand the mechanism whereby neural processes of socioemotional cognition associated with alcohol use disorders lead to negative drinking consequences. This study is a two-stage procedure to both provide evidence of a response modulation deficit associated with socioemotioal processing in individuals with alcohol use disorder and investigate how ...

People With CHC Who Achieved a Sustained Virological Response Following Therapy With Direct Acting Antiviral Agents
https://clinicaltrials.gov/ct2/show/NCT03520660

Jul 1st, 2022 - We intend to enroll up to 350 subjects with chronic hepatitis C virus (HCV) infection. Subjects will be recruited from two sources: Phase I: treatment naive or experienced who have failed a prior treatment (including DAA-experienced) who are willing to undergo a pre-treatment liver biopsy. Subjects yet to achieve an SVR will receive 12 weeks of therapy with sofosbuvir/velapatasvir (Epclusa ) fi...

Liver Fibrosis in HIV-Infected Patients With Elevated Liver Enzymes on Antiretroviral Therapy
https://clinicaltrials.gov/ct2/show/NCT00326482

Jul 1st, 2022 - At present, there are no clear guidelines as to when antiretroviral therapy for human immunodeficiency virus (HIV) infection should be stopped in the setting of elevated liver enzymes. In large part, this is due to a limited understanding of the natural history of antiretroviral-related hepatotoxicity. Although several antiretrovirals have been reported to cause fatal acute hepatitis, more ofte...

Natural History of the Collagen-Related Disorder Osteogenesis Imperfecta and Genotype Phenotype Correlation
https://clinicaltrials.gov/ct2/show/NCT03575221

Jul 1st, 2022 - Description: This is a longitudinal study of the natural history of the collagen-related disorder osteogenesis imperfecta (OI), that includes enrolling new patients under the age of 12 years, along with an extended data collection from adult patients on whom previous childhood data was collected at the NIH. Objectives: Primary Objectives: 1) Identify and monitor longitudinal functional outcomes...

Sample Collection From Healthy Volunteers for Assay Optimization
https://clinicaltrials.gov/ct2/show/NCT03538600

Jul 1st, 2022 - The Laboratory of Immune System Biology plays a major role in fostering the growth of systems biology efforts across the National Institutes of Health, in large measure through its development of new tools for high-throughput data generation and complex systems modeling. The lab s experimental component requires ongoing assay development and optimization, which depend on the availability of hum...

Palbociclib in Treating Patients With Relapsed or Refractory Rb Positive Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Activating Alterations in Cell Cycle Genes (A Ped...
https://clinicaltrials.gov/ct2/show/NCT03526250

Jul 1st, 2022 - PRIMARY OBJECTIVE: I. To determine the objective response rate (ORR; complete response + partial response) in pediatric patients treated with palbociclib with advanced solid tumors (including central nervous system [CNS] tumors), non-Hodgkin lymphomas or histiocytic disorders that harbor activating genetic alterations in cell cycle genes. SECONDARY OBJECTIVES: I. To estimate the progression fre...

The DETECT Study: Discovery and Evaluation of Testing for Endometrial Cancer in Tampons
https://clinicaltrials.gov/ct2/show/NCT03538665

Jul 1st, 2022 - BACKGROUND: Endometrial cancer is the most common and second deadliest gynecological cancer in women in the United States, with over 63,000 new cases and 11,000 deaths expected to occur in 2018. Unlike most cancers, endometrial cancer incidence and mortality are increasing, due to increases in risk factors such as obesity and population aging. Racial disparities in endometrial cancer incidence ...

Reducing Inequities in Care of Hypertension, Lifestyle Improvement for Everyone (RICH LIFE Project)
https://clinicaltrials.gov/ct2/show/NCT02674464

Jul 1st, 2022 - The investigators refined research aim is to determine if a clinic-based collaborative care team, including a community health worker (CHW) to deliver community-based contextualized care, reduces disparities in blood pressure control rates, lowers cardiovascular disease (CVD) risk, and improves outcomes among patients with hypertension and other common comorbid conditions when compared to stand...

Biospecimen Procurement for Genitourinary Malignancies Branch Immunotherapy Protocols
https://clinicaltrials.gov/ct2/show/NCT02682667

Jul 1st, 2022 - Background: -Immunotherapy is a promising approach to the treatment of cancer. GMB investigators are studying immunotherapy for the treatment of diverse malignant and premalignant conditions. These studies require the collection of biospecimens for research purposes. Objectives: To collect and bank blood, apheresis products, tumor, body fluids, and other biospecimens from patients with cancer o...

20-Week Repeat Oral Dose Study of AKB-6548 in Participants With Chronic Kidney Disease and Anemia
https://clinicaltrials.gov/ct2/show/NCT01906489

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.

Natural History Study of GATA2 Deficiency and Related Disorders
https://clinicaltrials.gov/ct2/show/NCT01905826

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...

The Role of Uncertainty in Coping: The Experience of Parents of Children With Undiagnosed Medical Conditions
https://clinicaltrials.gov/ct2/show/NCT01905865

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...

Natural History and Biomarkers of Amyotrophic Lateral Sclerosis and Frontotemporal Dementia Caused by the C9ORF72 Gene Mutation
https://clinicaltrials.gov/ct2/show/NCT01925196

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 ...

Heart Health Study in Washington D.C. to Develop a Community-Based Behavioral Weight Loss Intervention
https://clinicaltrials.gov/ct2/show/NCT01927783

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...

An Interventional Study of Avastin (Bevacizumab) in Patients With Advanced/Metastatic Epithelial Ovarian Cancer, Fallopian Tube Cancer or Primary Peritoneal Cancer
https://clinicaltrials.gov/ct2/show/NCT01932125

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.

Study of the Human Microbiome in Clinical Center Patients
https://clinicaltrials.gov/ct2/show/NCT01933620

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 ...

Links Between Inflammation and Cardiometabolic Diseases
https://clinicaltrials.gov/ct2/show/NCT01934660

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...

Obtaining Solid Tumor Tissue From People Having Biopsy or Surgery for Certain Types of Cancer
https://clinicaltrials.gov/ct2/show/NCT01915225

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...

Study of the Safety of a Particular Herpes Vaccine in Adults With or Without Herpes Infection
https://clinicaltrials.gov/ct2/show/NCT01915212

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...

Mechanisms of Diabetic Kidney Disease in American Indians
https://clinicaltrials.gov/ct2/show/NCT01878045

Jul 1st, 2022 - The purpose of this protocol is to examine the long-term impact of treatment with the angiotensin receptor blocker losartan on progression of diabetic kidney disease in American Indians with type 2 diabetes. Data from this protocol will also be used to guide the search for candidate biomarkers of diabetic end-organ damage in serum and urine and to identify mechanisms of diabetic kidney disease ...

Study to Investigate CSL112 in Subjects With Acute Coronary Syndrome
https://clinicaltrials.gov/ct2/show/NCT03473223

Jul 1st, 2022 - This is a phase 3, multicenter, double-blind, randomized, placebo-controlled, parallel-group study to evaluate the efficacy and safety of CSL112 on reducing the risk of major adverse CV events [MACE - cardiovascular (CV) death, myocardial infarction (MI), and stroke] in subjects with acute coronary syndrome (ACS) diagnosed with either ST-segment elevation myocardial infarction (STEMI) or non-ST...

A Study of SHR-1210 in Combination With Capecitabine + Oxaliplatin or Apatinib in Treatment of Advanced Gastric Cancer
https://clinicaltrials.gov/ct2/show/NCT03472365

Jul 1st, 2022 - Approximately 110 participants will be assigned to SHR-1210 + capecitabine + oxaliplatin combination therapy (Cohort 1), or SHR-1210 + apatinib combination therapy (Cohort 2).

Impact of Photobiomodulation (PBM) on Biomarkers of Alzheimer's Disease
https://clinicaltrials.gov/ct2/show/NCT03405662

Jul 1st, 2022 - Alzheimer's disease (AD), the most common form of dementia, is characterized by the loss of higher brain function such as memory, problem-solving abilities, and language. Photobiomodulation (PBM) describes a kind of light therapy that uses red or near-infrared light to stimulate, heal, regenerate, and protect tissue that has either been injured, is degenerating, or else is at risk of dying. The...

Multimodal Neuroimaging Genetic Biomarkers of Nicotine AddictionSeverity
https://clinicaltrials.gov/ct2/show/NCT01867411

Jul 1st, 2022 - Objective: To develop a neuroimaging/genetic/epigenetic biomarker of nicotine dependence severity that may be useful in predicting success in smoking cessation and in development of new smoking cessation treatment targets. Further, by evaluating those who vape nicotine, findings will determine whether biomarkers differ between those who smoke or vape, allowing for the development of cessation s...

Expansion of Childhood Relationship Study to Young Adult Romantic Relationships
https://clinicaltrials.gov/ct2/show/NCT01866852

Jul 1st, 2022 - The purpose of this study is to expand our ongoing longitudinal study (study 88-CH-32) by gathering information about romantic partnerships from both the longitudinal study s now young adult target children, who were 5 months old when the study began and are now as old as 23 years of age, and their significant others. Because the target children are approaching the age when they form lasting ro...

Raltegravir for HAM/TSP
https://clinicaltrials.gov/ct2/show/NCT01867320

Jul 1st, 2022 - Objective: In this pilot study, we wish to determine the effects of Raltegravir, a clinically approved HIV-1 integrase inhibitor, on HTLV-1 proviral load (PVL) in patients with HTLV-1 associated myelopathy / tropical spastic paraparesis (HAM/TSP). We will also provide safety and tolerability information on Raltegravir use in this condition and examine the correlation of immune activation marker...

Enzalutamide With or Without Vaccine Therapy for Advanced Prostate Cancer
https://clinicaltrials.gov/ct2/show/NCT01867333

Jul 1st, 2022 - Background: Enzalutamide is a well-tolerated, modern androgen receptor antagonist (ARA) with more enhanced anti-tumor activity compared to previous ARAs. Phase III trial has demonstrated a 4.8 month improvement in survival and a 37% risk reduction in death in metastatic castration resistant prostate cancer (mCRPC) patients who have had previous docetaxel. PROSTVAC is a therapeutic cancer vaccin...

NK White Blood Cells and Interleukin in Children and Young Adults With Advanced Solid Tumors
https://clinicaltrials.gov/ct2/show/NCT01875601

Jul 1st, 2022 - BACKGROUND: Despite progress, some children and young adults with solid tumors still experience poor survival. Activated NK cells potently kill autologous pediatric solid tumors, and clinical grade procedures are available to generate large numbers of activated NK cells for adoptive cell therapy. OBJECTIVES: Primary objectives are: 1) to assess the feasibility of harvesting and expanding activa...

Thinking and Memory Problems in People With HIV
https://clinicaltrials.gov/ct2/show/NCT01875588

Jul 1st, 2022 - The natural history of neurocognitive impairment in human immunodeficiency virus (HIV)-infected individuals remains poorly understood. While the advent of highly active antiretroviral therapy (HAART) has led to a decreased incidence of the most severe form of HIV associated neurocognitive disorders (HAND), HIV-associated dementia, it does not appear to have impacted overall prevalence of HAND. ...

Pyruvate Kinase Deficiency Global Longitudinal Registry
https://clinicaltrials.gov/ct2/show/NCT03481738

Jul 1st, 2022 - Data will be submitted to the Registry via electronic case report forms (eCRFs). Relevant datasets, such as historical trial data, claims, medical records, or central lab data will be electronically integrated into the Registry or Registry reporting data sets. Participants of all ages with a confirmed diagnosis of PK deficiency via genetic testing will be eligible to participate in this Registr...

Patient Perspectives and Doctor Visits
https://clinicaltrials.gov/ct2/show/NCT01888913

Jul 1st, 2022 - Background: - We are studying people s psychological experiences and how these relate to the way people respond have when they see information communicated in different ways. We are testing multiple research questions in this research. Objectives: - To learn about how to design future research studies. Also to learn about how to best give people information about health topics. Eligibility: - W...

Brentuximab Vedotin and Nivolumab With or Without Ipilimumab in Treating Patients With Relapsed or Refractory Hodgkin Lymphoma
https://clinicaltrials.gov/ct2/show/NCT01896999

Jul 1st, 2022 - PRIMARY OBJECTIVES: I. To determine the maximum tolerated dose (MTD) and dose limiting toxicities (DLT) of the combinations of brentuximab vedotin and ipilimumab, brentuximab vedotin and nivolumab, and brentuximab vedotin, ipilimumab, and nivolumab. (Phase I) II. To evaluate the complete response (CR) rate for the regimens of brentuximab vedotin and nivolumab compared to brentuximab vedotin, ip...

Identifying Risk for Diabetes and Heart Disease in Women
https://clinicaltrials.gov/ct2/show/NCT01809288

Jul 1st, 2022 - African-American women experience disproportionately high rates of conditions related to insulin resistance, specifically type 2 diabetes (T2D) and cardiovascular disease (CVD). In Africa, T2D and CVD have also become leading causes of death for women. Early recognition of risk and the implementation of preventive measures could improve both quality of life and longevity as well as decrease hea...

The Effects of Castration on the Pharmacokinetics of Zolpidem After Single Dose Administration In Men With Prostate Cancer Undergoing Androgen Deprivation Therapy Compared to Normal Healthy Females
https://clinicaltrials.gov/ct2/show/NCT03436745

Jul 1st, 2022 - Background: Zolpidem is currently approved for the treatment of patients with insomnia. Women reported experiencing an increased incidence of adverse effects than men, resulting in a reduction of the recommended dose of zolpidem for women. Zolpidem metabolism is affected by both age and gender; the recommended dose for the elderly and female populations is 5mg daily. Subsequent studies have sho...

A Study Osimertinib in Patients With Stage 4 Non-small Cell Lung Cancer With Uncommon EGFR Mutations
https://clinicaltrials.gov/ct2/show/NCT03434418

Jul 1st, 2022 - This is a research study to find out if a drug called, osimertinib, is safe and effective in treating advanced Non-Small Cell Lung Cancer (NSCLC) by targeting the treatment of epidermal growth factor receptor (EGFR) mutation exon 18 G719X, exon 20 S7681, or exon 21 L861Q. Patients on the study will not have had previous tyrosine kinase inhibitor (TKI) treatment. Patients who have one of the fol...

Near-infrared Spectroscopy and Electroencephalography to Assess Cortical Activation During Motor Tasks in Infants and Toddlers With and Without Cerebral Palsy
https://clinicaltrials.gov/ct2/show/NCT03434080

Jul 1st, 2022 - OBJECTIVE Portable neural imaging during functional tasks is now possible utilizing noninvasive near-infrared spectroscopy (NIRS) which identifies areas of brain activity by measuring blood flow dynamics and electroencephalography (EEG) which measures electrical activity on the cortical surface. Use of these technologies for studying movement is rapidly increasing; however, investigations in ch...

Safety Study of Cord Blood Units for Stem Cell Transplants
https://clinicaltrials.gov/ct2/show/NCT01861093

Jul 1st, 2022 - Background The requirement for licensure of human cell and blood products became part of the FDA final rules for Human Cells, Tissues, and Tissue-Based Products (HCT/Ps) (March 29, 2004 and May 25, 2005) in an effort to prevent transmission of communicable disease, minimize contamination and preserved integrity and function during processing, outline safety and effectiveness requirements for ce...

Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations
https://clinicaltrials.gov/ct2/show/NCT01861106

Jul 1st, 2022 - Background: Genetic and sporadic mutations on one allele of the GATA2 gene lead to a syndrome termed MonoMAC. MonoMAC is characterized by: 1) infections with Mycobacterium avium complex (MAC) and other opportunistic infections, 2) deficiency of monocytes, B-lymphocytes, and Natural Killer (NK) cells in the peripheral blood, and 3) progression to myelodysplastic syndrome (MDS), chronic myelomono...

Cerebrospinal Fluid Collection and Repository Storage
https://clinicaltrials.gov/ct2/show/NCT01862107

Jul 1st, 2022 - In this study we are planning to establish a repository of cerebrospinal fluid (CSF), serum, and plasma. We are planning to collect a small amount of additional CSF and blood on subjects who are already going to have a lumbar puncture as part of their research or clinical care at the National Institutes of Health (NIH). This will allow us to study whether there is an association between neurolo...

Muscle Strength Loss and Its Effect on Knee Cap Motion in Volunteers With Anterior Knee Pain
https://clinicaltrials.gov/ct2/show/NCT01862731

Jul 1st, 2022 - Chronic idiopathic patellofemoral pain, a potential precursor to osteoarthritis, is one of the most common problems of the knee. It is characterized by anterior knee pain that is aggravated by deep knee flexion, prolonged sitting, and repetitive flexion/extension. The most widely accepted theory in regards to the source of this pain is that a force imbalance around the knee leads to static pate...

PRISM Study-Pruritus Relief Through Itch Scratch Modulation
https://clinicaltrials.gov/ct2/show/NCT03497975

Jul 1st, 2022 - This is a randomized, double-blinded, placebo-controlled, 2-arm study with an open label extension period following double-blind treatment, to investigate the anti-pruritic efficacy and safety of Nalbuphine ER tablets. Subjects will be randomized to NAL ER (2-week titration followed by 162 mg twice daily [BID] for 12 weeks) or matching placebo (14 weeks duration), with the primary endpoint eval...

Tooth and Tissue Sample Collection During Wisdom Teeth Removal
https://clinicaltrials.gov/ct2/show/NCT01805869

Jul 1st, 2022 - Objective The objective of this protocol is to evaluate and provide treatment for subjects who have a clinical indication for the routine extraction of dental third molar wisdom teeth with the aim to allow investigators to maintain their expertise in dental clinical care, gain additional knowledge about oral health and to utilize surgical waste collected from these patients for research studies...

Perception, Sensation, Cognition and Action in Humans
https://clinicaltrials.gov/ct2/show/NCT03407066

Jul 1st, 2022 - Objective: The experiments covered by this protocol aim to uncover basic knowledge about the normal brain mechanisms that give rise to perception and cognition. The protocol encompasses sub-studies in healthy volunteers ( participants ) to uncover behaviors and their physiological basis. The protocol includes only non- or minimally-invasive techniques with minimal risk, including psychophysics,...

Spontaneous Haematomas of Psoas Occurring in Intensive Care
https://clinicaltrials.gov/ct2/show/NCT03452631

Jul 1st, 2022 - Psoas hematomas are a rare and potentially lethal pathology whose incidence in resuscitation is unknown. The pathophysiology of this pathology is not known to date.

Evaluation of Effectiveness and Safety of Aortic Valve Replacement in Routine Clinical Practice
https://clinicaltrials.gov/ct2/show/NCT03453567

Jul 1st, 2022 - This study evaluated effectiveness and safety of aortic valve replacement in real-world clinical practice.

Title: Effect of Opioid Receptor Modulation on Alcohol Self-Administration and Neural Response to Alcohol Cues in Heavy Drinkers: Role of OPRM1 Gene Variation
https://clinicaltrials.gov/ct2/show/NCT02639273

Jul 1st, 2022 - Previous studies have shown that the opioid receptor modulator nalmefene can reduce heavy drinking among alcoholics. Genetic variation at the micro-opioid receptor gene locus, OPRM1, specifically the A118G polymorphism, is associated with differential subjective responses to alcohol. Further, the A118G polymorphism has been shown to moderate the effect of opioid receptor modulators on alcohol c...

A Phase 1 Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ManNAc in Subjects With Primary Podocyte Diseases
https://clinicaltrials.gov/ct2/show/NCT02639260

Jul 1st, 2022 - Background. ManNAc (N-acetyl D-mannosamine) is an uncharged monosaccharide that is the biologic precursor of N-acetyl neuraminic acid (Neu5Ac, sialic acid). Sialic acids are the negatively charged, terminal monosaccharides of carbohydrate chains that are attached to glycoproteins and glycolipids (glycans). Most glycans serve cellular signaling functions, and frequently appear on the cell surfac...

rCSP/AP10-602 [GLA-LSQ] Vaccine Trial
https://clinicaltrials.gov/ct2/show/NCT03589794

Jul 1st, 2022 - This is a phase I, single-site, dose escalation study to evaluate the safety, immunogenicity, and efficacy of the recombinant circumsporozoite protein (rCSP) antigen malaria vaccine administered with and without AP 10-602 [Glucopyranosyl Lipid A (GLA) in liposome Quillaja saponaria 21 formulation (LSQ)]. The study population will consist of 65 healthy male and female adults aged 18 to 45 years ...

mHealth App for Engagement in Care Among Youth Living With HIV
https://clinicaltrials.gov/ct2/show/NCT03587857

Jul 1st, 2022 - WYZ is a modular, adaptive, and personalized intervention delivered via a mobile phone. It is grounded in the Information-Motivation-Behavioral Skills (IMB) model which has been valuable for understanding and guiding the development of interventions for complex health behaviors. WYZ was created in collaboration with 18-29 year-old YLWH using a Human-Centered Design (HCD) approach that emphasize...

Deferred or Immediate Stent Implantation Based on Microvascular Function in STEMI
https://clinicaltrials.gov/ct2/show/NCT03581513

Jul 1st, 2022 - Methodology: A multicenter, prospective, randomized, controlled clinical study enroll patients with ST-segment elevation myocardial infarction (STEMI) intended for PCI with stenting implantation. Eligible patients are randomly assigned to the immediate stenting group or delayed stenting group in a 1:1 ratio when residual stenosis >70% and TIMI grade 3 with angiography guidance. The index of mic...

Deep Brain Stimulation for Treatment Resistant Depression
https://clinicaltrials.gov/ct2/show/NCT00367003

Jul 1st, 2022 - Major Depression is one of the most common and costly of all psychiatric disorders. While depression can be effectively treated in the majority of patients by either medication or some form of evidence-based psychotherapy, up to 20% of patients fail to respond to standard interventions. For these patients, trial-and-error combinations of multiple medications and electroconvulsive therapy are of...

Technical and Translational Development of Cardiovascular Magnetic Resonance (CMR) Imaging
https://clinicaltrials.gov/ct2/show/NCT03581318

Jul 1st, 2022 - The purpose of this protocol is to develop and test new MRI techniques applicable to patients undergoing cardiovascular evaluation with the ultimate goal of translating the technical advances into improving clinical diagnosis and management of patients undergoing cardiovascular evaluation. Patients undergoing cardiovascular evaluation and healthy volunteers will be scanned under the protocol. W...

Oral Ifetroban to Treat Diffuse Cutaneous Systemic Sclerosis (SSc) or SSc-associated Pulmonary Arterial Hypertension
https://clinicaltrials.gov/ct2/show/NCT02682511

Jul 1st, 2022 - This study is a randomized, placebo-controlled, double-blind phase 2 trial of patients with dcSSc or SSc-PAH. Twenty participants with SSc-PAH and 14 participants with dcSSc will be randomized to receive either oral ifetroban daily or matching placebo. Study participants will be treated for 12 months, followed by a 30-day follow-up period. The study will test whether ifetroban is safe and stati...

Cholinergic Receptor Imaging in Dystonia
https://clinicaltrials.gov/ct2/show/NCT02689466

Jul 1st, 2022 - Dystonia is a heterogeneous group of movement disorders characterized by abnormal muscle contractions resulting in abnormal postures and movements. The spectrum of dystonia includes focal, segmental, multifocal, and generalized presentations with a broad range of age of onset. An example of a focal dystonia is cervical dystonia. The pathophysiology of dystonia is unclear, but prior neuroimaging...

Adoptive Cellular Immunotherapy for Progressive Multifocal Leukoencephalopathy With Ex Vivo Generated Polyomavirus-Specific T-Cells
https://clinicaltrials.gov/ct2/show/NCT02694783

Jul 1st, 2022 - Objective This pilot clinical trial will aim to determine the feasibility and potential toxicities associated with treating patients with progressive multifocal leukoencephalopathy (PML) with polyomavirus (PyV)-specific partially matched polyclonal allogeneic T cells. It will further provide initial efficacy data for this indication. Study Population Up to 18 subjects with definite PML, defined...

Clinical and Molecular Phenotyping in IBD
https://clinicaltrials.gov/ct2/show/NCT02694588

Jul 1st, 2022 - Inflammatory bowel disease (IBD) and psoriasis (Ps) are common, chronic, immune- mediated barrier diseases with shared inflammatory pathways. Current therapeutic interventions with anti-cytokine antibodies (TNF-α, IL-23/IL-12) reflect the intent to disrupt specific pathways of inflammatory immunopathology. Individual responses to biological treatment can be thereby be exploited in a systems bio...