Newfane, New York

A Study to Evaluate the Safety of CMTX-101 in People with Cystic Fibrosis

A Study to Determine if BHV-7000 is Effective and Safe in Adults With Refractory Focal Onset Epilepsy

A Study to Assess the Effect of Dexpramipexole in Adolescents and Adults With Severe Eosinophilic Asthma.

This is a multicenter, randomized, double-blind, placebo controlled, parallel group study designed to evaluate the efficacy and safety of dexpramipexole in adults and adolescents with severe, inadequately controlled asthma with eosinophilic phenotype on medium to high-dose inhaled corticosteroids (ICS )and at least one additional asthma controller medication with or without oral corticosteroids (OCS). Approximately 1400 participants will be randomized globally. Participants will receive dexpramipexole, or placebo, administered orally, over a 52-week treatment period. The study also includes a post-treatment follow-up period of 4 weeks.

Chemoreflex and Baroreflex Alterations Causing Postural Tachycardia Syndrome With Orthostatic Hyperpnea and Hypocapnia

The Investigators will compare results from female POTS patients, free of known pulmonary or sleep disorders aged 15 to 39 years with, and without orthostatic hyperpneic hypocapnia, to healthy female volunteers with the following two (2) specific aims: 1. To test poikilocapnic (allowing carbon dioxide (CO2) to vary) orthostatic cardiorespiratory responses to determine whether prolonged initial orthostatic hypotension (IOH) precedes upright hypocapnia in hyperpneic POTS but not in controls or non-hyperpneic POTS. Subjects are instrumented for cerebral blood flow, respiratory and hemodynamic measurements, investigating splanchnic blood flow by indocyanine green infusion, and measuring changes of CBV, regional blood volumes, and cardiac output (CO) by impedance plethysmography during a 10 min stand to quantify IOH, and a 10 min tilt test to 70⁰ to quantitate cardiorespiratory changes. These changes during tilt stratify hyperpneic and non-hyperpneic POTS. 2. To test if chemoreflex sensitization of ventilation and sympathetic activity (by microneurography) are abnormal when supine and tilted upright at 45o and how that interacts with Oxford measured cardiovagal and sympathetic baroreflexes under controlled gas conditions which are: isocapnic hypoxia and isocapnic hyperoxia to measure carotid body reflex; hyperoxic isocapnia and hyperoxic hypercapnia to measure central chemoreflexes. Hyperoxia silences peripheral chemoreceptors and will normalize baroreflex and tilt responses.

American Lung Association (ALA) Lung Health Cohort

The scientific goal of the study is to establish the relationship between a variety of factors (lifetime environmental exposures, fitness and physical activity, biomarkers, nasal respiratory epithelial transcriptome) and ideal versus impaired peak lung health (reserve and markers of susceptibility in investigators' model) in young adulthood. In addition to the initial baseline assessment, remote contacts (e.g., via text message, email, mail, or phone) at regular intervals will allow for both the retention of the participants as well as the collection of additional short-term follow-up information, i.e., within 4.5 years of enrollment. If additional funding becomes available, investigators will extend the study for an additional period of time to study factors related to long-term changes in lung health over time, i.e., at 5 years and beyond. Recruitment of participants will be conducted by the participating study sites along with national and regional efforts from the American Lung Association. Recruitment efforts will be supported by the study web site www.lung.org/lung-study

Evaluation of Dupilumab in Patients With Atopic Keratoconjunctivitis (AKC)

A Study of Azenosertib (ZN-c3) in Subjects With Platinum-Resistant High-Grade Serous Ovarian, Fallopian Tube or Primary Peritoneal Cancer

A Phase 2 study to evaluate the efficacy and safety of azenossertib (ZN-c3) in subjects with Platinum-Resistant, High-Grade Serous Ovarian, Fallopian Tube, or Primary Peritoneal Cancer. Azenosertib is a selective and orally bioavailable inhibitor of WEE1. By inhibiting WEE1, azenosertib enables cell cycle progression, despite high levels of DNA damage, thereby resulting in the accumulation of DNA damage leading to mitotic catastrophe and cancer cell death. The study consists of two parts: Part 1: All comers, no biomarker status required (completed enrollment) Part 2: Cyclin E1 positive protein expression required

realMIND: Observational Study on Safety and Effectiveness of Tafasitamab in Combination With Lenalidomide in Patients With Relapsed or Refractory DLBCL

This observational study is intended to further characterize the safety and effectiveness of tafasitamab, in combination with lenalidomide, in patients with R/R DLBCL in the US, with a focus on racial and ethnic minorities. This study also characterizes the overall treatment patterns (e.g., line of treatment, dose modification, combination partners, use as monotherapy) of US patients with R/R DLBCL who have been treated with tafasitamab with a focus on racial and ethnic minorities This multicenter real-world study will help to characterize the use of tafasitamab (e.g., line of treatment, dose modification, combination partners, use as monotherapy) among US patients with R/R DLBCL with a focus on racial and ethnic minorities This is an observational study; as such, no study visits or assessments, laboratory tests or procedures are mandated by the study. Patients will be evaluated and treated according to the physician's usual practice and discretion. Patient data for this observational study will be collected in one of two ways; either - by prospective follow-up of patients included at study sites, or - by retrospective collection of data from patient records, at study sites or from vendor databases.

Safety and Efficacy Study of Cenobamate in Pediatric Subjects 2-17 Years of Age With Partial-onset (Focal) Seizures

Secondary objectives: - To evaluate the efficacy of cenobamate tablets and suspension in pediatric subjects with partial onset (focal) seizures - To evaluate the pharmacokinetics of cenobamate tablets and suspension in pediatric subjects with partial onset seizures - To evaluate the PK/pharmacodynamics of cenobamate in pediatric subjects with partial onset (focal) seizures - Acceptability and palatability assessment (determined by a 5-point Hedonic Scale) of the oral formulation and the 12.5 mg tablets - Day 1, and Day 15

A Study of ZN-c3 in Women With Recurrent or Persistent Uterine Serous Carcinoma

This is a Phase 2 open-label, multicenter study to evaluate the clinical activity and safety of ZN-c3 (also known as azenosertib; KP-2638) in adult women with recurrent or persistent uterine serous carcinoma (USC).