The PROGRESS Trial - Continued Access Study Number: 2021-01-CAP
The purpose of this study is to evaluate an investigational treatment for people with moderate, calcific aortic stenosis: The SAPIEN 3, SAPIEN 3 Ultra, and SAPIEN 3 Ultra RESILIA system includes an artificial heart valve and accessories used to implant the valve. The selection of the study device will be made at the discretion of your doctor. The artificial valve is made of two parts, a stent and valve leaflets. The Stent (a metal mesh tube) holds the valve in position and valve leaflets (made of cow tissue) open and close fully to direct the flow of blood through your heart.
The PROP-RD Study: A Prospective Registry for the Study of Outcomes and Predictors in Pouchitis and Pouch-Related Disorders
Purpose: Create a prospective registry of patients with pouch-related conditions to allow for longitudinal assessment of outcomes.Participants: 320 patients from 8 centers who are diagnosed with acute pouchitis, CADP, CARP, or CD of the pouch.Procedures (methods): Following the enrollment data collection during a standard of care clinic visit, patients will complete online questionnaires at 3, 6, and 12 months following enrollment.These questionnaires will include standardized follow-up assessments of disease activity and detailed questions regarding patient-reported response to initial therapies.We will prospectively collect serial stool samples from 90 patients with acute pouchitis (a subset of the 320 patients enrolled in the registry). Samples will be collected at the time of diagnosis (day 0 in clinic) and the end of antibiotic therapy (day 14).
The Prospective Natural History of Congenital Insensitivity to Pain with the Anhidrosis (CIPA)
The purpose of this study is to create a database of clinical information collected from you/your child’s medical chart on a yearly basis from patients with Congenital Insensitivity to Pain with Anhidrosis (CIPA). The study will document the clinical features of patients with CIPA overtime by storing their routine clinical test results in a central database. The study will involve collaborators at other specialist clinics around the world who follow/evaluate patients annually. Providing blood for future use is optional.
THE PROSPECTIVE NATURAL HISTORY OF FAMILIAL DYSAUTONOMIA
Our ultimate goal is to develop new treatments for patients with familial dysautonomia (FD, OMIM 223900). We also want to learn which specific nerve populations are affected by the disease-causing mutation, whether these features are progressive, and how best to measure them in clinical trials. FD is caused by a founder mutation in the IKBKAP gene that is carried by 1:30 people of European Jewish ancestry. Over 99% of affected patients have two copies of the identical founder mutation. This affects the development of the sensory nervous system, which relays information to the brain. FD is both developmental and progressive. Current drug treatments are supportive and none specifically target the on-going neurological decline. Partnership between academic centers, advocacy groups and federal agencies has allowed the creation of a pipeline for drug development and an infrastructure for translational research. The first aim of this project will be to enroll patients with FD from around the world in a non-interventional natural history study. We will score the severity of their clinical features and follow how they evolve over time. The natural history will focus on establishing disease-specific milestones to use as outcome measures in future clinical trials. We will find ways to measure the progressive neurological aspects of the disease including blindness and gait ataxia, which are intrinsically related and most devastating to the patient’s quality of life overtime. We will also explore other potential biomarkers that quantify renal, cardiovascular, respiratory, orthopedic and cognitive aspects of the disease, which will help us monitor adverse events. The second aim of this project will address one of the most intriguing questions about the disease; why some patients are more severely affected than others. The study will include genomic sequencing from patients with FD, to find specific modifier genes that influence the severity of traits as possible targets for future drug development. Some of these genes may be important in the general population, but discovering them could be easier in FD patients due to >99% being homozygous for the founder mutation.
The Psorcast Study: A Validation Study of a Smartphone Sensor-Based Patient-Driven Detection of Psoriatic Disease
Smartphone-based and physician-assessed measures will be collected at baseline and 12 weeks after initiating a new therapy for psoriatic disease. Weekly digital assessments will also be performed.
The RESTORATIVE303 Study: A Randomized Double-Blind Placebo-Controlled Phase 3 Study of VE303 for Prevention of Recurrent Clostridioides Difficile Infection
The purpose of this research study is to see if the study drug, VE303, compared with placebo is safe and effective in preventing patients from having another episode of Clostridioides difficile infection (CDI)following completion of a course of standard-of-care (SoC) antibiotics in participants with recurrent CDI (rCDI) and those with primary CDI who are at high risk for recurrence (pCDI-hr).
The Rhythm Evaluation for AntiCoagulaTion with Continuous Monitoring of Atrial Fibrillation
REACT-AF is a research study for people withoccasional episodes of atrial fibrillation (AFib).We are studying if it is safe and effective tostop blood thinning medicine during timeswhen your heart rhythm is normal.
The role of exercise in the consolidation of fear extinction learning in adults with high anxiety sensitivity
This is an experimental study (not a treatment study) aiming to examine the effects of acute exercise (vs. seated control) on fear extinction learning in a 3-day paradigm. 50 eligible (after screening) men and women ages 18-60 with high anxiety sensitivity (AS) and an anxiety disorder (generalized anxiety disorder, panic disorder, social anxiety disorder) will participate in a 3-day paradigm. Day 1 includes habituation and conditioning procedures. Day 2 includes extinction procedures. Additionally, participants will be randomized to 1 of 2 conditions immediately following extinction: 1) moderate intensity exercise (n=25) or 2) seated control (n=25), occurring immediately after extinction for 20 minutes. Day 3 includes extinction recall, renewal, and reinstatement procedures. Primary outcomes are physiological arousal (skin conductance, heart rate) during Day 3 procedures. Mechanistic factors, including expected negative consequences of exercise (e.g., fainting), affect during exercise, threat/shock expectancy, and changes pre-post exercise in stress related neuroendocrine markers (cortisol and alpha-amylase) and their effects on extinction recall will be measured.
The Role of Residual Hearing in Cochlear Implant Outcomes
Cochlear implants (CIs) have the potential to restore high frequency hearing electrically while maintaining residual low frequency hearing, allowing for simultaneous electric and acoustic listening in the same ear. Residual hearing has been thought to be beneficial for all patients, prompting extensive effort into designing electrodes and surgical techniques to preserve it. However, the benefits of this combined electro-acoustic stimulation (EAS) are highly variable across patients, as is acceptance of EAS. Currently, the audiologist decides whether to fit EAS based on the patient’s audiometric thresholds and their personal predictions about its potential efficacy for the individual patient. Unfortunately, patients with similar audiograms show outcomes ranging from interference to benefit with EAS, making the audiogram a poor predictor of performance.. This is because pure-tone audiometry measures only the detection of a sound and not the ability to functionally interpret the stimuli. Determining the factors that relate to EAS benefit and acceptance is important for understanding residual hearing as well as counseling prospective patients. Therefore, a need exists for a clinically feasible measure (i.e., brief and self-administered) to evaluate the usefulness of residual hearing in a given patient. The goal of this study is to develop such a tool.
THE ROLE OF THE MICROBIOME IN MONOZYGOTIC TWINS WITH PSORIASIS AND PSORIATIC ARTHRITIS
We propose a longitudinal study of monozygotic twins discordant for psoriatic disease. Subjects will be characterized with detailed clinical questionnaires in addition to blood, fecal, skin swab, skin biopsy (optional) and urine (optional) specimen collections. These will be used to perform microbiome analysis and explore markers of inflammation as well as immunologic parameters. Healthy and psoriasis (not psoriatic arthritis) twins will be followed for up to five years. Data and biospecimen collections will be repeated on an annual basis or earlier in the case of disease conversion.