SELECT-SLE: A Phase 3 Program to Evaluate the Safety and Efficacy of Upadacitinib in Subjects with Moderately to Severely Active SLE
The purpose of this program is to assess the safety and efficacy of upadacitinib compared with placebo (fake drug) for the treatment of signs and symptoms in adults with moderately to severely active systemic lupus erythematosus (SLE). Upadacitinib is an experimental drug that blocks the actions of proteins known as Janus kinases (Jaks). Jaks are involved in the immune response and cell growth including blood cells. The study drug has been approved by regulatory authorities to treat other medical conditions such as rheumatoid arthritis, psoriatic arthritis, and axial spondylarthritis but is not approved to treat lupus. Therefore, the use of the study drug is investigational (experimental) for the purposes of this program.
The Hirschsprung Disease Research Collaborative
Hirschsprung disease (HSCR) is a neurodevelopmental defect resulting from the absence of nerve (ganglion) cells in the gastrointestinal tract. The disorder has a population incidence of 1/5,000 live births and most often occurs as an isolated phenotype. However, approximately 30% of HSCR cases are associated with other birth defects such as Down syndrome, deafness, hypopigmentation, and Congenital Central Hypoventilation syndrome (CCHS, aka Ondine’s curse). Hirschsprung disease is a genetic condition sometimes recognized with autosomal dominant and autosomal recessive inheritance, but is generally multifactorial. While several genes associated with HSCR have been identified, it is expected that additional genes play important roles in the disorder. Furthermore, much remains to be understood about the mechanisms of genetic variants involved in the disease and how variants in multiple genes interact to lead to the diverse forms of HSCR. The objective of the Hirschsprung Disease Research Collaborative (HDRC) is to build a large collection of data and biological samples of individuals with HSCR by which genetic data can be linked to detailed and accurate phenotypic information. HDRC members (surgeon champions at diverse medical centers) will collect samples and data through a multi-site study where Dr. Aravinda Chakravarti’s laboratory at NYU School of Medicine serves as the coordinating center. The data and samples collected by HDRC members will not only be available for genetic studies, but will form a biobank from which HDRC members can request access to de-identified samples and data for use in their own IRB-approved studies. The goal of the genetic studies carried out with HDRC samples is to complete the identification of HSCR susceptibility genes and to better understand the complex inheritance of HSCR in families by whole genome mapping and sequencing studies. We also intend to ascertain the frequency with which HSCR gene variants, individually and together, lead to the diverse forms of HSCR. Finally, we use these results together with the clinical information we collect to investigate possible genotype-phenotype correlations and their relationship with medical, surgical and pathological data on participants.
Gaucher Disease Outcome Survey (GOS)
The Gaucher Outcome Survey (GOS) is a long term observational survey. This survey aims to collect as much information as possible from as many Gaucher disease patients about the course of their disease, their medical management & the use of current or past medications or treatments, if any. Patient participation in GOS will be voluntary & GOS will be open to patients with Gaucher disease of any phenotype. Patients who are naïve to treatment, patients who are currently or have been previously treated with VPRIV, as well as patients who have been exposed to or are currently receiving other treatments for Gaucher disease may be included. This study is designed to gain a better understanding of the clinical course of the disease and its response to VPRIV therapy, thereby improving the clinical management of patients affected by Gaucher disease.
Tracking Sexual Dysfunction Over Time in Patients with Inflammatory Bowel Disease
This study will track sexual dysfunction over time in patients with inflammatory bowel disease (IBD) and correlate sexual dysfunction with disease activity and different treatment modalities. Researchers will also correlate sexual dysfunction with illness perception, anxiety and depression which have been known to affect sexual dysfunction. Researchers will use verified sexual dysfunction and disease activity scales in survey format in order to obtain this data. Scales for depression, quality of life and illness perception will also be used. Medical record data will also be searched in order to obtain biopsy, colonoscopy and treatment data. The overall aim is to examine the persistence of sexual dysfunction over time and how it varies with treatment in IBD. Researchers also hope to correlate sexual dysfunction with both disease activity and psychosocial aspects of illness.
Our Locations | NYU Langone Health
NYU Langone Heart has locations in New York City and on Long Island, as well as in Westchester County and Florida.
School Health Program—Family Health Centers at NYU Langone | NYU Langone Health
The School Health Program—Family Health Centers at NYU Langone brings healthcare into schools where it is convenient for children to access.
Characterization and Investigation of Hematologic Disorders
The purpose of this study is to maintain a research registry and biorepository for patients with disorders of the hematologic system and enable the use of peripheral blood, bone marrow, and tissue samples by basic and clinical investigators for research into the biology, causes, prevention, and treatment of these disorders. Research will also at times pair specimens and clinical information about patient outcomes to obtain clinically relevant findings. We wish to prospectively collect clinical data and biospecimens from pediatric patients to enable investigators in the future to use this material. We wish to obtain the relevant clinical information about the patients and continue to follow-up the patients through their medical records. The overarching goal is to facilitate clinical-translational research evaluating biology, diagnosis, treatments, and ouctomes in patients with disorders of the hematologic system.
Cochlear implants and listening effort: the interaction of cognitive and sensory constraints
Hearing impairment is the third most prevalent chronic medical condition among older adults in the United States. Untreated, hearing loss can interfere with effective communication, quality of life, and lead to accelerated cognitive decline. Cochlear implants (CI) have seen increasing use for older adults who can no longer gain significant benefit from a conventional hearing aid, and it is estimated that over 150,000 adults over 70 years of age in the United States would likely be CI candidates today. Older adult CI users can show impressive adaptation to the sharply degraded signal produced by CIs, sometimes in moderate levels of noise. Both patients and clinicians, however, frequently complain that these standard measures of speech perception are not informative with respect to real-world performance, and clinicians are often puzzled by patients who seem to do well in clinical tests but report major difficulties in everyday interactions.
Gut Microbiome in Food Protein-Induced Enterocolitis Syndrome: An Observational Study
Food protein-induced enterocolitis syndrome (FPIES) is a non IgE-mediated food allergy that manifests with predominantly gastrointestinal symptoms and usually starts in the first year of life. There is no diagnostic biomarker and no treatment for FPIES beyond food avoidance, reflecting poorly understood pathophysiology. This is a prospective observational study, which will compare fecal microbiota, fecal short chain fatty acids and metabolomics between infants with FPIES and control infants and infants without food allergy, 0-12 months old. It will also compare mucosal immunity via epigenetic markers in blood in infants with FPIES, and infants with IgE-mediated food allergy. Stool samples will be collected from 50 infants with FPIES and 50 sex and age matched control infants birth through 12 months. Optional blood samples will be collected only from infants with FPIES and infants with IgE-mediated food allergies (0-12 months old). A questionnaire will be used to obtain information detailing the infant’s feeding, medical, birth, and family history.
Integrated multi-omics approaches to precision medicine in kidney disease (iMAP-Kidney)
The primary objective of the study is to establish a biorepository (tissue bank) for the storage of plasma, serum, DNA, RNA, urine, and fresh tissue from kidney and kidney allograft biopsies, from patients undergoing kidney biopsy. This objective will be accomplished through the following: 1) establishing a clinical registry containing information linked from the patient’s medical record; 2) digitizing kidney histology slides from kidney tissue biopsies; 3) obtaining leftover archival tissue if extra fresh tissue collection is deemed unsafe by the physician, if the patient is pregnant, or if the patient refuses extra tissue collection and 4) allowing for accessibility to newly obtained samples for related studies.