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Discover 19,675 clinical trials near Pennsylvania. Find research studies in your area.
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NCT05467891
This is an open label, multicenter, single arm phase II study to evaluate the efficacy and safety of ribociclib and ET in patients with locoregional recurrence of HR-positive, HER2-negative breast cancer.
NCT06958211
The purpose of this study is to evaluate the efficacy and safety of ruxolitinib cream in participants with hidradenitis suppurativa.
NCT07223762
The Hyperangulated versus Standard Geometry Laryngoscope Blade (ANGLE) Trial is a multi-center, non-blinded, parallel-group, randomized clinical trial evaluating the effect of use of a hyperangulated video laryngoscope blade versus use of a standard geometry video laryngoscope blade. Critically ill adults undergoing tracheal intubation in participating EDs and ICUs who meet eligibility criteria will be enrolled and randomly assigned in a 1:1 ratio to either intubation using a hyperangulated video laryngoscope blade or a standard geometry video laryngoscope blade. The primary outcome is the incidence of successful intubation on the first attempt. The secondary outcome is incidence of hypoxemia during the interval between induction and 2 minutes after tracheal intubation.
NCT05321342
The investigators' overall objective is to demonstrate the efficacy of the Bullying Classroom Check-Up (BCCU) on elementary aged students' aggressive and bullying behaviors, teacher practices, and student and teacher relationships. The investigators' principal hypothesis is that the BCCU will improve students' aggression and bullying behaviors and their perceptions of the school climate and relationships in the building as well as teacher practices.
NCT05776134
The goal of this study is to provide access to brexucabtagene autoleucel for patients diagnosed with a disease approved for treatment with brexucabtagene autoleucel, that is otherwise out of specification for commercial release.
NCT07215624
This study evaluates the use of extended venous thromboembolism prophylaxis (ePPx) following abdominopelvic cancer surgery within the NCI Community Oncology Research Program (NCORP) network, targeting surgeons and surgical advanced practice providers (APPs).
NCT06794996
The purpose of the study is to evaluate the efficacy, safety, and tolerability of Inaxaplin (IXP) in participants with proteinuric APOL1- mediated kidney disease (AMKD).
NCT02156102
Background: \- People with sickle cell disease and other blood disorders sometimes get chronic leg ulcers. These are wounds that develop on the skin and don t go away. Current treatments do not work very well, so researchers want to learn more about why the ulcers happen. They want to find out which bacteria may cause it, and if external factors play a role. Objective: \- To study social and environmental factors of sickle cell disease and the causes of sickle cell disease leg ulcers. Eligibility: \- People age 18 and older who have sickle cell disease or another red cell disorder, with or without an active leg ulcer. Design: * Participants will have a medical history and clinical evaluation. They will also have blood drawn. * Participants will complete questionnaires about their life, health, environment, stress, and other topics. * Participants may provide a small sample of hair. * Participants will be asked to collect a small amount of saliva. * Participants with leg ulcers will have their skin microbiome sampled. The microbiome is all of the microbes (bacteria and and/or fungi) and their genes in and on the body. Researchers will use swabs to collect skin samples. Photographs will be taken of the skin sample area. * Some participants without leg ulcers also will have their skin microbiome sampled. * Some participants who have their skin microbiome sampled will return for a second visit. At this visit, their microbiome will be resampled. It will take place more than 30 days after the first visit.
NCT00345930
The purpose of this study is to identify individuals who have suffered a liver injury arising as an idiosyncratic reaction to a prescription drug or a complementary and alternative medicine. Recently added acute cases enrollment that meets criteria to the protocol. Also added Fibroscans to the protocol that will be completed at baseline and follow-up on chronic subjects.
NCT05077709
A Phase II Multi-Arm (basket) Trial Investigating the Safety and Efficacy of IO102-IO103 in Combination with pembrolizumab, as First-line Treatment for Patients with Metastatic Non-Small Cell Lung Cancer (NSCLC), Squamous Cell Carcinoma of Head or Neck (SCCHN), or Metastatic Urothelial Bladder Cancer (mUBC)
NCT06150820
Pompe disease is a genetic condition which causes muscle weakness over time. People with Pompe disease have a faulty gene that makes an enzyme called acid alpha-glucosidase (or GAA). This enzyme breaks down a type of sugar called glycogen. Without this enzyme, there is a build-up of glycogen in the cells of the body. This causes muscle weakness and other symptoms. Pompe disease can happen at any age, but in late-onset Pompe disease, symptoms generally start from 12 months old onwards. The standard treatment for people with Pompe disease is to receive regular infusions of the GAA enzyme. This is known as enzyme replacement therapy. However, people can build up antibodies against the GAA enzyme over time. Gene therapy is used to treat conditions caused by a faulty gene. It works by replacing the faulty gene with a working gene inside the cells of the body. The working gene is delivered into the cells using certain viruses as carriers (vectors). Viruses are often used as carriers as they can easily get inside cells. The genetic material of the original virus is replaced with the working gene, so only the working gene gets inside the cells. A common virus used as a carrier in gene therapy is the adeno-associated virus (or AAV). This is like an adenovirus, which causes the common cold. The original type of AAV does not cause any harm to humans. However, people that have previously been infected with the original type of AAV may have built up antibodies against AAV. These antibodies may stop the AAV carrier with the working gene getting inside the cells. Researchers want to learn more about antibody levels against AAV and the GAA enzyme in people with late-onset Pompe disease. They also want to learn about other substances in the blood that provide more information about late-onset Pompe disease. These are known as biomarkers. In this study, older teenagers and adults with late-onset Pompe disease will take part. They will not have had gene therapy using AAV. There will be 2 groups - those who have never had enzyme replacement therapy, and those who have had enzyme replacement therapy for 6 months or more. No study treatment will be given during the study, but blood and urine samples will be taken for testing. The main aims of the study are to check antibody levels against AAV8 (a type of AAV) in people with late-onset Pompe disease who had not received any treatment using AAV, to check antibody levels against the GAA enzyme in people previously treated with GAA as part of enzyme replacement therapy, to check levels of biomarkers for Pompe disease, and to check for medical problems. In the study, people will visit the study clinic several times. Some visits may be in the person's home. The first visit is to check if they can take part. Those who can take part will have a medical examination, and have their vital signs checked. Vital signs include blood pressure, heart rate, breathing rate and temperature. Blood samples will be taken to check antibody levels against the GAA enzyme and against AAV8. Blood and urine samples will also be taken to check for biomarkers for Pompe disease. Blood and urine samples will be taken about every 4 months for up to 2 years.
NCT04892017
This is a multicenter, open label, first in human (FIH) study of inlexisertib as monotherapy, and in combination with trametinib, binimetinib, or sotorasib in participants with advanced or metastatic solid tumors with RAS/MAPK pathway mutation. The study consists of 2 parts, a dose-escalation phase, and an expansion phase.
NCT05061004
The objective of this study is to evaluate the preliminary safety and effectiveness of the Cephea Mitral Valve System for the treatment of symptomatic patients with mitral valve disease (including mitral regurgitation, mitral stenosis and mixed mitral valve disease) in whom transcatheter therapy is deemed more appropriate than open heart surgery.
NCT06971731
The goal of this Phase 3, randomized study is to assess the safety, efficacy, tolerability, and pharmacokinetics (PK) of oral JNT-517 in adults (18 years of age or older) with PKU. Participants will receive either JNT-517 or placebo and will be blinded to their treatment assignment. Participants will have a 2 in 3 (or approximately 67%) chance of receiving JNT-517 during the first part of the study which will last approximately six weeks. During the second part of the study every participant who continues in the study will receive one of two doses of JNT-517 for an additional 46 weeks. The study requires a screening period of up to 35 days to ensure dietary stabilization and amino acid levels required to meet study eligibility. In total, participation in the study could last for up to 400 days. Participants will: Take 75 mg JNT-517 or 150 mg JNT-517, or a placebo BID (2x per day) for approximately 365 days; Visit the clinic or have a mobile health nurse visit your home for checkups and tests; Collect urine sample at home and bring to clinic on specified days; Keep a food diary 3 days before each study visit
NCT03255174
The objective of this study is to evaluate the safety and hemostatic effectiveness of EVARREST as an adjunct to controlling mild to moderate soft hepatic parenchyma or soft tissue bleeding during open hepatic, abdominal, pelvic, retroperitoneal, and thoracic (non-cardiac) surgery in pediatric population.
NCT05952934
This is a Phase II randomized, double-blind, placebo controlled, multi-site study of Candin. It is designed to show the efficacy and safety of a 7-dose regimen of Candin over a two-year period in terms of reducing cancer recurrence rate by comparing the recurrence rates between the Candin and the placebo arm. The ratio of the number of subjects who will receive Candin versus placebo will be 3:1. Up to 100 subjects will be screened until 80 subjects are eligible for injection.
NCT07250737
The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping. The patient's Administering Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.
NCT05362721
Coarctation of the aorta accounts for 4-7% of all congenital heart disease. While stent therapy, when feasible, is the standard of care for coarctation, it may not completely improve the work (and afterload) of the heart due to its effects on the elasticity of the aorta. This study will provide the information needed to understand the effects of current management on the cardiac mechanics and work.
NCT06079398
This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg CNP/kg of Navepegritide (TransCon CNP) administered SC once-weekly for 52 weeks in infants with genetically verified heterozygous ACH, aged 0 to \< 2 years at the time of randomization.
NCT07516964
Nephrotic syndrome is a kidney condition that mainly affects children and is characterized by high levels of protein in the urine, low levels of protein in the blood, and swelling. While many children respond well to steroid treatment, a large proportion experience relapses or become dependent on therapy. In some cases, the disease does not respond to standard treatments and may progress to chronic kidney disease. Recent research suggests that, in addition to genetic factors, immune system mechanisms may play a key role in the development and progression of nephrotic syndrome. In particular, some patients produce autoantibodies against nephrin, an essential protein of the kidney filtration barrier. These autoantibodies may be associated with disease activity and treatment response. The aim of this study is to investigate the presence of anti-nephrin autoantibodies in children with nephrotic syndrome and to better understand their role in disease mechanisms and clinical outcomes.The study will also explore the presence of other autoantibodies targeting components of the glomerular filtration barrier. The study will use advanced laboratory techniques, including blood tests and detailed analysis of kidney biopsy samples, to identify these antibodies and their relationship with kidney structure and function. By integrating laboratory findings with clinical data, this study aims to improve the understanding of nephrotic syndrome and support the development of more personalized diagnostic and therapeutic strategies, with the goal of improving patient outcomes and reducing unnecessary or ineffective treatments.
