Philadelphia, PA Paid Clinical Trials

Minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), and Membranous nephropathy (MN), generate an enormous individual and societal financial burden, accounting for approximately 12% of prevalent end stage renal disease (ESRD) cases (2005) at an annual cost in the US of more than $3 billion. However, the clinical classification of these diseases is widely believed to be inadequate by the scientific community. Given the poor understanding of MCD/FSGS and MN biology, it is not surprising that the available therapies are imperfect. The therapies lack a clear biological basis, and as many families have experienced, they are often not beneficial, and in fact may be significantly toxic. Given these observations, it is essential that research be conducted that address these serious obstacles to effectively caring for patients. In response to a request for applications by the National Institutes of Health, Office of Rare Diseases (NIH, ORD) for the creation of Rare Disease Clinical Research Consortia, a number of affiliated universities joined together with The NephCure Foundation the NIDDK, the ORDR, and the University of Michigan in collaboration towards the establishment of a Nephrotic Syndrome (NS) Rare Diseases Clinical Research Consortium. Through this consortium the investigators hope to understand the fundamental biology of these rare diseases and aim to bank long-term observational data and corresponding biological specimens for researchers to access and further enrich. Read Less

The primary objective of the study is to investigate the safety and efficacy of KPI-012 compared to vehicle in participants who have a documented clinical diagnosis of PCED. Read Less

The aim of this study is establish the reliability and clinical utility of microneuromas as identified via in vivo confocal microscopy as the diagnostic biomarker for NCP. Read Less

The purpose of this study is to assess whether oxygen supplementation during sleep improves working memory and other clinical and patient-reported outcomes among children who have Down Syndrome (DS) with moderate to severe Obstructive Sleep Apnea (OSA). Read Less

The objective of this study protocol is to test whether intermittent theta-burst transcranial magnetic stimulation (iTBS-TMS) to the inferior parietal lobule (IPL) can strengthen functional connectivity with a key region in emotion regulation (ER) neurocircuitry (anterior insula, AI) and improve performance on ER-related tasks in patients with bipolar disorder. Individual IPL sites for stimulation will be identified through baseline, pre-TMS functional magnetic resonance imaging (fMRI) scans. Patient-specific IPL subregions showing positive functional connectivity with the anterior insula and falling within the patient-specific frontoparietal control network will be used as individualized target sites for TMS stimulation. Patients will be randomized to receive 24 sessions of active versus sham iTBS to patient-specific IPL targets (6 sessions/day, 4 days, 43,200 pulses total). Post-iTBS resting-state and task-based fMRI scans will be acquired 3 days after the final day of iTBS administration (Day 4) following identical procedures as baseline. Effects of iTBS-TMS on ER will be evaluated by comparing pre-TMS versus post-TMS functional connectivity and behavior during performance on ER tasks. Read Less

To leverage access to patients across the primary care network, EPIC tools for identifying eligible patients, and the Way to Health platform to launch and enroll a program that will be evaluated in a clinical trial that is focused on changing patient behavior and powered to detect differences in improving blood pressure and cholesterol over 6 months for Penn Medicine patients in West/Southwest Philadelphia and Lancaster. Read Less

The Goal of this study is to investigate if individuals ages 12 years and older, carrying the IL-4RαR576 gene variant, will have a greater response to therapy acting directly on the anti-IL-4R. This will be conducted by examining the effect of a 48 week therapy with dupilumab on the rate of asthma exacerbations. Read Less

The purpose of the study is to assess the efficacy of durvalumab plus tremelimumab in combination with chemotherapy compared with pembrolizumab in combination with chemotherapy in metastatic NSCLC patients with non-squamous histology who have mutations and/or co-mutations in STK11, KEAP1, or KRAS. Read Less

This HTX treatment study evaluates the effects of chest tube size and the benefits of daily irrigations on acute HTX. 20 acutely injured but stable trauma patients requiring a chest tube for HTX will be enrolled. Patients will be assigned a 28Fr or 14 Fr chest tube with serial lavage and drainage. The endpoints will be HTX volume (by CT scan), complications, additional interventions, hospital length of stay, chest tube duration, provider feedback, and patient-reported outcomes. Read Less

The Subharmonic Aided Pressure Estimation (SHAPE) technique is a noninvasive ultrasound-based imaging technique that can estimate ambient pressure using subharmonic emissions from ultrasound contrast agents. Ultrasound contrast agents are encapsulated microbubbles (mean diameter \< 8 µm) with a lipid, protein, or polymer shell that traverse the entire vasculature. When the contrast microbubbles are insonated with relatively high acoustic pressures (\>100-150 kPa), these microbubbles act as nonlinear oscillators yielding energy components in the received echo signals at frequencies ranging from the subharmonic (half of transmit frequency) to higher harmonics and even ultraharmonics. Based on empirical evidence, the subharmonic signal exhibits a sigmoidal relationship with incident acoustic pressure i.e., subharmonic signal can be divided into occurrence, growth and saturation stages. In the growth stage, the subharmonic signal has shown sensitivity to ambient pressure characterized by an inverse linear relationship between subharmonic signal and ambient pressure changes. This inverse linear relationship forms the basis for the SHAPE technique. Several pre-clinical and clinical studies have been conducted to utilize the SHAPE technique for in vivo pressure estimation e.g., to diagnose portal hypertension, to estimate intra-cardiac pressures and to determine interstitial fluid pressures. The core imaging mode underpinning the SHAPE technology (subharmonic imaging) is now available on some commercial scanners. In the proposed pilot study, the SHAPE technique will be leveraged for monitoring intracranial pressure (ICP). The use of SHAPE technique for the ophthalmic artery could potentially provide an accurate noninvasive method of measuring ICP in patients with idiopathic intracranial hypertension (IIH) and other conditions of raised ICP, which would revolutionize the field. Read Less

The current protocol is to determine the biodistribution, metabolism, excretion and brain uptake of 11C-M503. The goal of this radiotracer is to quantify alpha-synuclein that is abnormally deposited in the brain of people with Parkinson's disease (PD). Investigators will compare uptake in participants with PD versus participants with multiple system atrophy (MSA) and progressive supranuclear palsy (PSP), as well as non-Parkinsonism volunteers. This multicenter project funded by an NIH U19 grant, is centered at U Pennsylvania (Penn, Grant PI: Robert Mach) in collaboration with U Pittsburgh (Pitt) (non-clinical site) Yale U, U of California at San Francisco (UCSF) and Washington University in St. Louis (WU). The University of Pennsylvania will act as the sIRB for this multi-center human subjects project and participants will be recruited from all sites. Read Less

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. Read Less