About 60,928 results

Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency

Oct 14th, 2022 - Background Mutations in the Dedicator of Cytokinesis-8 (DOCK8) gene are responsible for an immunodeficiency disease characterized by: severe cutaneous and sinopulmonary infections with bacterial organisms; extensive cutaneous viral infections with Herpes simplex, Herpes zoster, Molluscum contagiosum, and Human Papilloma Virus; a marked elevation in serum IgE levels and eosinophilia; homozygous ...

Fever Control Using External Cooling in Mechanically Ventilated Patients With Septic Shock

Oct 14th, 2022 - Sepsis is a common syndrome responsible for multiple organ failure. Septic shock, defined as sepsis with hyperlactatemia and cardiovascular failure requiring vasopressor infusion despite adequate fluid resuscitation has an extremely high mortality rate. Fever is a frequent disease process during sepsis. Fever increases oxygen consumption and can worsen imbalance between oxygen supply and oxygen...

Randomized Trial to Evaluate the Efficacy of Deploying Genetically-engineered Mosquitos to Control Dengue Burden in the City of Sao Jose do Rio Preto, Brazil

Oct 14th, 2022 - Study Design Introduction:This will be a parallel design cluster randomized controlled trial with 3 years (3 seasons) of follow-up from initial deployment of mosquitoes. The intervention will be routine control measures, as recommended by the Brazilian National Control Program (larvicide using PPF at individual house visits at least 4 times a year), in the presence or absence of the deployment ...

Testing the Combination of the Anti-cancer Drugs XL184 (Cabozantinib) and Nivolumab in Patients With Advanced Cancer and HIV

Oct 14th, 2022 - PRIMARY OBJECTIVES: I. To determine the safety of combined nivolumab and cabozantinib s-malate (XL184 [cabozantinib]) in human immunodeficiency virus (HIV) patients with advanced solid tumors. II. To determine the feasibility to deliver the combined nivolumab and XL184 (cabozantinib) for a minimum of 4 cycles in at least 75% of the subjects in the expanded cohort with Kaposi sarcoma (KS) or to ...

Natural History Study of Progressive Multifocal Leukoencephalopathy (PML)

Oct 14th, 2022 - The objective of this study is to examine the risk factors and natural course of JCV infection and progressive multifocal leukoencephalopathy (PML). PML is a devastating, demyelinating neurological disease affecting the brain of patients with a compromised immune system. It is caused by reactivation of JC virus (JCV), a small DNA virus that infects the majority of the population without clinica...

AAV2-GDNF for Advanced Parkinson s Disease

Oct 14th, 2022 - Objective: While medications can temporarily alleviate the symptoms of Parkinson s disease (PD), they do not influence the degenerative process. Progressive loss of nigral dopaminergic (DA) neurons (the pathological hallmark of PD) results in progressive neurologic dysfunction and death. Glial cell line-derived neurotrophic factor (GDNF) was first identified based on its ability to promote the ...

Minnesota COVID-19 Testing Project

Oct 14th, 2022 - In the United States, recent statistics show that African American and Latinx communities bear a disproportionate burden from COVID-19. Reaching vulnerable and underserved populations is therefore crucial to combating the disease. However, most public messaging campaigns are not targeted toward underserved communities and don't address fears of social stigma, mistrust in the healthcare system, ...

CD4 CAR+ ZFN-modified T Cells in HIV Therapy

Oct 14th, 2022 - Step 1: Subject is screened, undergoes leukaphereses, and optional rectal biopsy, and safety evaluations before dosing. The University of Pennsylvania manufactures the study product. Step 2: Subjects receive a single infusion of 0.5-1x10(10) CD4 CAR+CCR5 ZFN modified T cells. Cohort 2 participants undergo a mini-leukapheresis and optional rectal biopsy at the end of step 2. The duration of Step...

COVID-19 Persistence in Stool

Oct 14th, 2022 - Approaching participants will mainly be performed in two ways: the Norfolk and Norwich University Hospital (NNUH) online booking system for SARS-CoV-2 testing will have a link to the Quadram Institute Bioscience (QIB) study webpage, which will include the Participant Information Sheet (PIS). When the NHS testing service informs individuals of their test results by text message, the QIB study pa...

Trial to Determine the Efficacy/Safety of Plitidepsin vs Control in Patients With Moderate COVID-19 Infection

Oct 14th, 2022 - This is a multicentre, open-label, controlled Phase 3 study in which adults requiring hospital admission and O2 supplementation for management of moderate COVID-19 infection will be randomised in 1:1:1 to: Plitidepsin 1.5 mg arm, Plitidepsin 2.5 mg arm and Control arm

First-In-Human Study of ChAdOx1-HBV & MVA-HBV Vaccines (VTP-300) for Chronic HBV

Oct 14th, 2022 - This is a multi-centre study conducted in 52 participants, who will each be administered 2 vaccine injections (IM) on Day 0 and Day 28 as follows: Group 1: MVA-HBV + MVA-HBV (now closed) Group 2: ChAdOx1-HBV + MVA-HBV Group 3: ChAdOx1-HBV + MVA-HBV + nivolumab (IV infusion) Group 4: ChAdOx1-HBV + nivolumab + MVA-HBV + nivolumab (now closed) Participants are randomised to treatment as the groups...

Investigating Vector-Borne Determinants of Aedes Transmitted Arboviral Infections in Cambodia: An Observational Longitudinal Cohort Study in Children

Oct 14th, 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...

Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5

Oct 14th, 2022 - This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, single dose study to determine the safety of valoctocogene roxaparvovec (an Adenovirus-Associated Virus (AAV) based gene therapy vector) in severe Hemophilia A patients with pre-existing antibodies against AAV5.

High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease

Oct 14th, 2022 - Study Description: Alemtuzumab, targeted busulfan, and TBI, with a 10/10 related or MUD donor graft or a 9/10 single HLA mismatch graft followed by post-transplant cyclophosphamide. Primary Objectives: To determine engraftment rates with the use of high cell doses, without increasing the risk of GvHD by using post-transplant cyclophosphamide and sirolimus in conjunction with a busulfan based co...

Hepatitis B and Hepatitis C as Risk Factors for Hepatocellular Carcinoma in African and Asian Immigrants

Oct 14th, 2022 - Specific Aim 1: We will determine whether exposures to HBV and HCV infections in African (Somalis, Kenyans, Liberians, and Ethiopians) and Southeast Asian (Hmong, Vietnamese, Laotian, and Cambodian) are associated with a single HBV/HCV genotype or few specific subtypes. In the following Sub-Aims we will: confirm the viral status of study subjects using serological and DNA tests including HBsAg,...

Follow-Up Evaluation for Gene-Therapy-Related Delayed Adverse Events After Participation in Pediatric Oncology Branch Clinical Trials

Oct 14th, 2022 - Background: Study subjects exposed to gene therapy interventions may be at risk for delayed or longterm adverse consequences. The U.S. Food and Drug Administration (FDA) has issued Long Term Follow-up After Administration of Human Gene Therapy Products: Guidance for Industry and Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Ma...

Anti-CD22 Chimeric Receptor T Cells in Pediatric and Young Adults With Recurrent or Refractory CD22-expressing B Cell Malignancies

Oct 14th, 2022 - Background: Adoptive cellular therapy with T cells genetically modified using viral-based vectors to express chimeric antigen receptors targeting the CD19 molecule have demonstrated dramatic clinical responses in patients with acute lymphoblastic leukemia (ALL). However, not all patients respond and CD19-negative escape has been observed following CD19 CAR therapy, as well as anti-CD19/CD3 bisp...

Dosage-Escalation Study of the Safety and Immunogenicity of a Novel Rabies Vaccine ChAd155-RG vs. the Comparator RABAVERT Vaccine in Healthy Adult Subjects

Oct 14th, 2022 - This is a single-center, observer-blinded, Phase 1, dosage-escalation trial to evaluate the safety, tolerability, reactogenicity, and immunogenicity of ChAd155-RG compared with RABAVERT in rabies virus-naïve healthy male and non-pregnant female adult subjects ages 18-49. Subjects who have never received a licensed or investigational rabies virus vaccine, or an Ad-based investigational vaccine, ...

Withdrawal of Therapy After Long-Term Antiviral Treatment for Chronic Hepatitis B

Oct 14th, 2022 - Chronic hepatitis B affects at least 1.5 million Americans and is a major cause of cirrhosis, end-stage liver disease and hepatocellular carcinoma. Five oral antiviral agents have been licensed for use in chronic hepatitis B in the United States. These agents are effective at suppressing viral replication, improving liver disease and reversing cirrhosis. The standard indications for starting an...

Non-Viral TCR Gene Therapy

Oct 14th, 2022 - Background: The administration of autologous tumor infiltrating lymphocytes (TIL) can mediate complete, durable regressions in 20-25% of patients with metastatic melanoma. Recent studies have shown that these TIL predominantly recognize unique mutated neoantigens expressed by the cancer not shared by other melanomas. Administration of bulk autologous TIL to patients with a variety of other soli...