The New York City Study of Fertility and Environmental Exposures (NYC FREE): A single-center observational study of environmental and lifestyle influences on health and reproduction
NYC FREE is a reproductive health study that will enroll people of reproductive age who have the potential to become pregnant in the near future and partners of these individuals. The overall aim of this project is to create a biobank that will facilitate future studies on the relation of environmental and lifestyle factors with reproductive health and human fecundity.
THE NEW YORK MOVEMENT DISORDER STUDY
Movement disorders represent a major public health crisis. Large longitudinal clinicopathological studies form the basis for studying the symptomatology, epidemiology, progression and pathogenesis of these diseases. In addition, by studying blood and brain tissue for biomarker and genetic studies has the potential to pave the way towards new diagnostics and uncover underlying pathogenic mechanisms that can be targeted for therapeutic development. The goal of this project is to perform a clinicopathological study of a large cohort of patients with movement and related disorders drawn from the patients at the Robert and John M. Bendheim (RJB) Parkinson and Movement Disorders Center and at The NYU Langone Health The Marlene and Paolo Fresco Institute for Parkinson’s and Movement Disorders (Fresco Institute). Movement disorders represent a large group of related diseases that include Parkinson’s disease, dystonia, essential tremor, myoclonus, chorea, and others. Here, volunteers will be assessed for clinical and neuroradiological features in an ongoing way during their disease course. Blood will be collected for genetic and biomarker studies. In addition, we will recruit healthy adult volunteers who will serve as controls to test the specificity of our findings. Finally, patients will be offered the opportunity to serve as brain tissue donors. These clinical and neuropathological variables will stored using the secure Research Electronic Data Capture (REDCap) web-based software and correlative studies will be performed.
The Parkinson s Progression Markers Initiative (PPMI) 2.0 Clinical - Establishing a Deeply Phenotyped PD Cohort
The Parkinson Progression Marker Initiative 2.0 (PPMI 2.0) is a longitudinal, observational, multi-center natural history study to assess progression of clinical features, digital outcomes, and imaging, biologic and genetic markers of Parkinson’s disease (PD) progression in study participants with manifest PD, prodromal PD, and healthy controls. The overall goal of PPMI 2.0 is to identify markers of disease progression for use in clinical trials of therapies to reduce progression of PD disability.
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.