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NCT04761822
Background: Allergic reactions have been reported to occur after vaccination with both the Pfizer-BioNTech COVID-19 Vaccine and Moderna COVID-19 Vaccine. Allergic reactions range from mild to severe and include life- threatening anaphylactic reactions, although no deaths have been reported with either vaccine. This study is designed with two principal aims: * To estimate the proportions of systemic allergic reactions to the Pfizer-BioNTech COVID-19 Vaccine and the Moderna COVID-19 Vaccine in a High-Allergy/Mast Cell Disorder (HA/MCD) population, and * If the risk in the HA/MCD is demonstrable, to determine whether the proportions are higher in the HA/MCD in comparison to a representative population without severe allergies or mast cell disorders
NCT04136002
The ECLIPSE (Evaluation of the ctDNA LUNAR test in an Average Patient Screening Episode) study is a prospective, observational multi-site study without randomization. The primary objective of the study is to evaluate the performance characteristics of a blood-based ctDNA LUNAR-2 test to detect colorectal cancer in a screen-relevant, average risk population.
NCT05880836
The objective of ILAN is to assess the safety, feasibility and bronchodilator efficacy of in-line bronchodilator nebulizer delivery with VMN via HFNC system in hypoxemic respiratory failure patients treated with bronchodilators and compare this method to standard-nebulization using a jet nebulizer with a facial mask. The investigators hypothesized that aerosol nebulization using HFNC/VMN represents safer and more convenient approach in hypoxemic respiratory failure patients in comparison to conventional therapy while providing similar bronchodilator efficacy.
NCT06176066
Current standard of care therapy and all FDA approved adjuvant therapy for glioblastoma continue to provide less than 12 months of progression free survival (PFS) and less than 24 months of overall survival (OS). There is an extreme need for any novel therapy against glioblastoma that increases progression free survival and overall survival in patients diagnosed with this invasive form of cancer. A significant reason for such a poor prognosis is the infiltrative nature of this tumor in non-enhancing regions (NE) beyond the central contrast-enhancing (CE) portion of tumor, which is difficult to visualize and treat with surgical, medical, or radiotherapeutic means. Since tumor cells exhibit abnormal metabolic behavior leading to extracellular acidification, we theorize a newly developed pH-sensitive MRI technique called amine chemical exchange saturation transfer echoplanar imaging (CEST-EPI) may identify infiltrating NE tumor beyond what is clear on standard MRI with gadolinium contrast. This phase I safety study will use use intraoperative CEST-EPI guided resections in glioblastoma at increasing distances from areas of CE tumor to test whether this technique is safe and can remove additional areas of infiltrative NE tumor. The primary objective of this study is to assess the safety of pH-sensitive amine CEST-EPI guided resections for glioblastoma.The secondary objectives of this study include: 1. A preliminary efficacy analysis of CEST-EPI guided resections in extending progression free and overall survival. 2. To confirm that resected tissue obtained from pH-sensitive amine CEST-EPI guided resections contain infiltrating NE tumor. The primary endpoint for this study will be safety of resecting "CEST positive", acidic regions within T2 hyperintense regions of glioblastoma thought to contain active NE tumor at increasing distances from contrast enhancing tumor with development of a recommended maximal tolerated resection. 1. At the maximal tolerated resection, a preliminary efficacy study with endpoints of progression free survival (as defined by RANO Resect 2.0) 1 and overall survival. 2. Quantitation of infilitrating tumor burden on CEST-EPI resected tissue using immunohistochemical staining. 12 patients up to 24 patients based on resection limiting toxicities with potential expansion of up to 16 patients at the maximum tolerated resection. Inclusion Criteria: 1. Must be able to provide written informed consent 2. Male or female \> 18 years of age 3. Karnofsky Performance Scale (KPS) \> 70 (indicating good performance status). 4. Individuals with suspected, newly diagnosed or recurrent IDH wild type WHO IV glioblastoma (intraxial, expansile contrast-enhancing mass without evidence of metastatic disease. This will be reviewed by UCLA neuroradiology to only include patients with high likelihood of GBM) Exclusion Criteria: 1. Pediatric patients 2. Diagnostic uncertainty (reviewed by UCLA neuroradiology history extracranial malignancy or autoimmune disease) 3. Medical conditions that make patients a poor candidate for anesthesia and/or surgery (decision for surgery will follow standard pre-operative clearance guidelines and will not differ for this specific study from standard of care treatment plan) 4. Involvement of eloquent areas (as defined by MRI signal clearly involving areas that would lead to a qualifying neurologic deficit as defined in surgical limiting toxicity - this will specifically include: 1) primary motor cortex, 2) primary sensory cortex, 3) sensorimotor fibers as defined on pre-operative diffusion tensor imaging, 4) primary language areas (Broca, Wernicke), 5) arcuate fasiculus as defined on pre-operative diffusion tensor imaging Pre-operative: Standard of care pre-operative MRI including perfusion and pH-weighted amine CEST-EPI (which will add up to 15 minutes of scan time) for a single pre-operative exam prior to surgery. Surgery: 1 day (subjects to be admitted to the hospital) Follow-up: inpatient stay (1-3 days), 2 week clinical assessment (outpatient post-op clinic visit). MRI and clinical assessment at 4 weeks (end of resection limited toxicity window). Following this, there will be standard of care follow up with MRI and clinical assessment starting at 8 weeks +/- 4 weeks (per RANO 2.0). 1 Total study duration for recruitment, enrollment, and study completion of all subjects is up to 2 years. Single-arm, surgical resection escalation safety trial with a preliminary efficacy study at the maximal tolerated resection This safety evaluation will mimic a phase 1 dose escalation safety study using a rule based approach on based on a i3+3 design.2 Using standard of care resection of contrast enhancement as the baseline, we will begin with 3 subjects with maximal resection + "CEST positive" areas 0.7 cm from the contrast enhancing boundary within areas of T2 hyperintensity. If there is not \> 1 pre-determined resection limiting toxicity (RLT, defined below) in this cohort, the r
NCT05856331
Phase 2 study to compare SAR447537 (INBRX-101) to plasma derived A1PI therapy in adults with AATD emphysema
NCT03827265
The purpose of this study is to determine if brain stimulation using repetitive transcranial magnetic stimulation (rTMS) directed at different parts of the brain can decrease feelings of cigarette craving and symptoms of cigarette withdrawal, and also if men and women have different responses to rTMS. Participants will visit the University of California, Los Angeles (UCLA) five times: First, for in-person screening, then for four rTMS sessions, four three different brain regions. Everyone in the study will be assigned to all four treatment arms and they will take place in a random order. Before and after each rTMS session, a brief MRI will be performed, and participants will be asked to fill out questionnaires that describe how they are feeling.
NCT03974113
Primary Objective: \- To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to \<12 years of age) with severe hemophilia A or B Secondary Objectives: * To characterize the safety and tolerability * To determine fitusiran plasma concentrations at selected time points
NCT01461837
This study is being done to determine the safety and outcome (long-term control) of a high-dose chemotherapy regimen followed by an infusion of CD34 selected (immune cells) stem cells from a partially matched adult family member donor, called haploidentical stem cell transplantation, in high-risk sickle cell disease patients. Funding Source - FDA OOPD
NCT02003209
This randomized phase III trial studies docetaxel, carboplatin, trastuzumab, and pertuzumab with estrogen deprivation to see how they work compared to docetaxel, carboplatin, trastuzumab, and pertuzumab without estrogen deprivation in treating patients with hormone receptor-positive, human epidermal growth factor receptor 2 (HER2)-positive breast cancer that is operable or has spread from where it started to nearby tissue or lymph nodes (locally advanced). Drugs used in chemotherapy, such as docetaxel, carboplatin, trastuzumab, and pertuzumab, 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. Giving more than one drug (combination chemotherapy) may kill more tumor cells. Estrogen can cause the growth of breast cancer cells. Hormone therapy using goserelin acetate and aromatase inhibition therapy may fight breast cancer by blocking the use of estrogen by the tumor cells. Radiation therapy uses high energy x rays to kill tumor cells. Giving combination chemotherapy and radiation therapy with or without hormone therapy may be an effective treatment for hormone receptor-positive, HER2-positive, operable or locally advanced breast cancer.
NCT03266653
Related donor Epstein-Barr Virus (EBV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered in children, adolescents and young adults with refractory EBV infection post Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT), with primary immunodeficiencies (PID) or post solid organ transplant. Funding Source: FDA OOPD
NCT04825834
The primary objective of this study, DELFI-L101, is to train and test classifiers for lung cancer detection using the DELFI assay and other biomarker and clinical features.
NCT05842967
The purpose of this study is to learn about the safety and immunogenicity of a study vaccine (called RSVpreF) in several adult groups. Respiratory Syncytial Virus (RSV) is a common type of virus (germ) that can cause severe illness, where medical help is needed. RSV can lead to airway diseases in all ages. Vaccines help your body make antibodies which help fight against diseases. This is called an immune response. This study will measure how much antibody participants make after receiving RSVpreF (immunogenicity). The study consists of 2 groups (Substudy A and Substudy B). Substudy A is seeking approximately 675 participants who are: * Between 18 and 60 years of age. * Considered having a high likelihood of severe RSV disease due to certain long-term medical conditions. Such medical conditions do not include immunocompromising conditions. Participants will need to come to the study clinic at least 2 times. At the first clinic visit, participants will receive 1 shot of RSVpreF or placebo in the arm by chance. A placebo looks like the study vaccine but contains no active ingredients. At each clinic visit, a blood sample will be taken. A third (final) visit can be either completed in clinic or via telephone contact. This study is about 6 months long for each participant. Substudy B is seeking approximately 200 participants who are: * At least 18 years of age. About half of the participants will be at least 60 years of age. * Considered having a weakened immune system (immunocompromised). Participants will need to come to the study clinic at least 3 times. All participants will receive a shot of RSVpreF at the first study clinic visit. The second study clinic visit will be 1 month later. All participants will receive a second shot of the study vaccine at this second study clinic visit. Blood samples will be taken at the 3 study clinic visits. A fourth (final) visit can be either completed in clinic or via telephone contact. This study is about 7 months long for each participant.
NCT04129125
The trial is designed to assess the safety and efficacy of using the Zoom Reperfusion System in subjects diagnosed with acute ischemic stroke and undergoing a thrombectomy procedure within 8 hours of last known well.
NCT02546986
The purpose of this study is to determine whether the combination therapy of CC-486 (oral azacitidine) and pembrolizumab provides improved patient outcomes compared to pembrolizumab alone in patients with previously treated locally advanced or metastatic non-small cell lung cancer.
NCT03266627
Related donor Adenovirus (ADV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered intravenously in in children, adolescents and young adults with refractory ADV infection post Allogeneic Hematopoietic Stem Cell Transplantation (AlloHSCT), with primary immunodeficiencies (PID) or post solid organ transplant. Funding Source: FDA OOPD
NCT02900651
The purpose of this Phase I/II study is to establish the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) and to evaluate the safety, antitumor activity and pharmacokinetic (PK) profile of MAK683 in patients with advanced malignancies such as Diffuse Large B cell Lymphoma (DLBCL), nasopharyngeal carcinoma (NPC) or other advanced solid tumors for whom no further effective standard treatment is available.
NCT04164979
This is a phase 2 single-arm, open-label clinical trial determining efficacy of cabozantinib in combination with pembrolizumab in subjects with advanced gastric and gastroesophageal adenocarcinoma. These are subjects who have progressed, or not tolerated, at least one prior line of chemotherapy with a fluoropyrimidine and platinum agent.
NCT05226507
The purpose of the dose escalation phase is to evaluate the safety profile of escalating doses and dose schedules of NXP800. In the expansion phase the preliminary efficacy in subjects with ARID1a mutated ovarian clear cell and ovarian endometrioid cancers will be estimated.
NCT04814693
In acute pancreatitis, approximately 20% of the cases result in severe necrotizing pancreatitis which is associated with significant morbidity and mortality. Necrotizing pancreatitis is characterized by the development of an acute necrotic collection and as this collection persists beyond 4 weeks, walled off necrosis (WON) encapsulates the collection. To date, this is treated by the step-up approach, which contains percutaneous drainage and minimally invasive video assisted retroperitoneal debridement (VARD) or endoscopic ultrasound (EUS) guided drainage followed by direct endoscopic necrosectomy (DEN). Different DEN techniques are available for the treatment of WON, however, there is a lack of effective endoscopic instruments to perform DEN. Recently, the first dedicated alternative to conventional DEN has been cleared for use, namely the EndoRotor® Resection System. This device is a powered mechanical debridement device intended for use in endoscopic procedures to resect and remove necrotic debris during DEN for WON. Previous (pilot and feasibility) studies showed promising results in terms of the amount of procedures, adverse events and length of hospital stay. Therefore, aim of this study is to assess the performance of the EndoRotor, as compared to conventional endoscopic techniques, for direct endoscopic necrosectomy (DEN) of walled off necrosis (WON) in a randomized controlled trial.
NCT05791201
The purpose of the study is to evaluate the safety, tolerability, and efficacy of VX-264 in participants with type 1 diabetes (T1D).