This is a intervention longitudinal study for children with CF and caregivers. Children aged 2-5 who are diagnosed with CF and are eligible for TRIKAFTA will be invited to participate in this study. The treating physicians will manage children with CF in accordance to their medical judgement and data will be collected throughout the study. Children with CF and caregivers may be enrolled up to 3 months before their child initiates TRIKAFTA and site personnel will record date for the study in that time (such as pulmonary exacerbations, medical history, and medication history.) Caregivers will also report PROS (patient reported outcomes) and CGRO (caregiver-reported outcomes).
This study is for people with non-small cell lung cancer that has come back or grown after receiving immunotherapy treatment. This study is being done to see if a new combination of 2 non-chemotherapy drugs will help patients live longer than the usual approach.
To determine the effect of randomized, once daily, oral administration of 80 mg
resmetirom versus matching placebo on patients as measured by time to
experiencing a first adjudicated Composite Clinical Outcome event, defined
as any of the following: all-cause mortality, liver transplant, and significant
hepatic events including hepatic decompensation events (ascites, hepatic
encephalopathy, or gastroesophageal variceal hemorrhage) and confirmed
increase of Model for End-stage Liver Disease (MELD) score from <12 to
≥15
This study is exploring the safety and efficacy of the study drug LTP001 in subjects with pulmonary arterial hypertension. This is a randomized study and the subjects will be be given the study drug or placebo in a capsule form which they are to take once daily in the morning. Participants will be screened for up to 8 weeks followed by 24 weeks of daily dosing- study visits to the site will occur approximately every 4 weeks. During these study visits there will be study procedures that may include labs for blood and urine, ECGs, vital signs, 6MWD, echocardiogram, or right heart catherization. There is the opportunity for an open-label portion of the study as well.
The purpose of this global study is to determine the efficacy, safety, and tolerability of 3 dose levels of OG-6219 (50, 100, and 150 mg twice a day [BID]) in pre-menopausal women between 18 and 49 years of age (inclusive), who have moderate to severe endometriosis-related pain.
In this research study, we are using examining how brain activity (electrical and blood flow) changes during tests of emotional processing and attention and memory in the magnetic resonance imaging (MRI) scanner. We are also using non-invasive brain stimulation to probe different parts of the brain and measuring brain activity (electrical and blood flow). This study includes healthy participants and researchers hope this will help us develop improved brain-based treatments for emotional difficulties like depression.
Hypermobile EDS and hypermobile spectrum disorder (collectively referred to as hEDS) are estimated to affect 1 in 500 individuals worldwide. hEDS patients have limited treatment options for their numerous symptoms that impact the quality of life. This clinical trial tests a new ear stimulation method in hEDS patients to determine if it may improve quality of life.
This study will evaluate the effects of elafibranor (the study drug) in participants with Primary Sclerosing Cholangitis (PSC). PSC is a rare disease of the liver that leads to injury and destruction of bile ducts. Damage to bile ducts leads to buildup of bile in the liver, which then causes further damage, and leads to disease progression. This study will compare elafibranor to a placebo, a dummy treatment. The main objective of the trial will be to study the safety and side effects of the study drug. The trial will also study the study drug's effects on blood tests and other tests related to PSC disease activity.
This is an Investigational Device Exemption (IDE) clinical research trial (referred to also as a trial or a research trial) sponsored by AtriCure, Inc (the Sponsor) to learn whether the use of the AtriClip® LAA Exclusion System to close off your left atrial appendage (LAA) during your heart surgery will reduce the risk of stroke. The AtriClip has not been well studied or approved by the FDA for stroke prevention; therefore, this procedure is considered investigational.
The goal of this study is to understand the knowledge, views and perceptions from diverse communities, primarily from the Lowcountry, about the new field of personalized medicine (or precision health), and what factors might influence participation in precision health research studies. It is hoped that results from this study will help inform future strategies to increase the diversity of participants in precision health research.
Participation involves completion of a one-time survey that takes approximately 20 minutes to complete. Compensation is available.