14 Clinical Trials for Various Conditions
Registry program for volunteers who are willing to serve as control subjects in future research studies.
Human induced pluripotent stem cells (hiPSCs) have driven a paradigm shift in the modeling of human disease; the ability to reprogram patient-specific cells holds the promise of an enhanced understanding of disease mechanisms and phenotypic variability, with applications in personalized predictive pharmacology/toxicology, cell therapy and regenerative medicine. This research will collect blood or skin biopsies from patients and healthy controls for the purpose of generating cell and tissue models of Mendelian heritable forms of heart disease focusing on cardiomyopathies, channelopathies and neuromuscular diseases. Cardiomyocytes derived from hiPSCs will provide a ready source of disease specific cells to study pathogenesis and therapeutics.
The purpose of this study is to look at the progression of cardiovascular disease (CVD) in normal control individuals and in patients with controlled diabetes.
The purpose of this study is to directly characterize the pharmacokinetic (PK) profiles of resmetirom and its major metabolite (MGL-3623) following oral administration of 100 mg resmetirom (QD x 6 days) in subjects with severe renal impairment (RI) compared to healthy matched control subjects with normal renal function.
This is a two-part study which will evaluate the PK, safety, and tolerability of a single 1500 milligram (mg) oral dose of gepotidacin in subjects with normal hepatic function and in subjects with mild, moderate, and severe hepatic impairment. In Part 1, subjects with moderate hepatic impairment and subjects with normal hepatic function will be enrolled. Matching subjects with normal hepatic function in Part 1 (Group D), will be enrolled following the completion of all Day 3 assessments of the respective matched, hepatically impaired subject. In Part 2, subjects with mild (optional) and severe hepatic impairment and subjects with normal hepatic function will be enrolled concurrently based on the PK, safety, and tolerability data of Part 1. Subjects with mild hepatic impairment, may be studied if there is a significant difference in PK between subjects with moderate hepatic impairment and subjects with normal hepatic function. Subjects with severe hepatic impairment, will be studied in Part 2, provided that, the PK objectives are achieved in Part 1. A totals of 48 subjects, are planned to be enrolled in the study. The study duration is approximately of 44 days from Screening to Follow-up visit. The results from this study will enable the development of appropriate dosing recommendations in subjects with impaired hepatic function.
The purpose of this research study is to understand whether there is any difference in the amount of tasimelteon (including its breakdown products) in the blood of individuals with severe renal impairment compared to individuals who have normal renal function. The safety and tolerability of tasimelteon will also be assessed throughout this study.
The investigators want to see if healthy people who are given 2 doses of a medication called anakinra, which is used to treat moderate to severe rheumatoid arthritis, (RA) and then exposed to a form of air pollution, called Endotoxin, have less inflammation with the medication. Endotoxin is believed to be one of the causes of asthma attacks.
Compare the objective tear film dynamic measurements in three different populations (MGD \[Meibomium Gland Dysfunction\], ADDE \[Aqueous Deficient Dry Eye\] and normal/control \[non-dry eye\]) and the subjective experience before and after instilling a single drop of saline in each eye. Currently available office-based tests for dry eye do not reliably correlate with patients' subjective symptoms. This study is interested in assessing how individuals with these two different ocular surface diseases compare with the normal population when measured objectively using two different instruments.
This study will examine and compare what happens in the brains of patients with Parkinson's disease with that of healthy normal subjects while they train to react as fast as possible to the appearance of a visual signal. Particularly, we will measure the amount of the chemical dopamine released in the brain as well as the electrical activity during training. Indeed, patients with Parkinson's disease frequently complain of slowness and early fatigue during movements. These symptoms are believed to be related to a decrease of dopamine in the brain which may be associated with abnormalities in cerebral electrical activity. Adult patients with Parkinson's disease who are right-handed, do not have dementia, and are not depressed may be eligible for this study. Healthy volunteers who match patients in age, gender, handedness, and level of education will also be studied. Candidates will be screened with a medical history, physical and neurological examinations, memory test, mood evaluation, and urine toxicology. All participants will be required to stop taking any medications that can influence the central nervous system and to abstain from alcohol consumption for 1 week before the screening examination and during the study training period. Patients with Parkinson's disease will also be required to stop using antiparkinsonian medications for at least 12 hours before the first visit and each training session. Participants will have several 1-hour training sessions. During these sessions, they will sit facing a computer screen at a distance of about 32 inches (80 centimeters) from their eyes. Six permanent position markers will be displayed. A keyboard with six spatially compatible response keys will be within reach of their right hand. Participants will respond as quickly and as accurately as possible to the appearance of a stimulus (e.g., white circle) below one of the markers by pressing the spatially corresponding key. About a second later, the next stimulus will be displayed below one of the other markers, and so on. Reaction times and accuracy will be recorded. After 3 to 10 minutes of practice (one block), there will be a rest period during which the computer will display information about the subject's accuracy of movements and reaction time. Then, a new block will start. There will be about 6 to 20 practice blocks per training session. The number of training session will vary between 3 and 6 depending on accuracy and reaction time during the task. During each training session, subjects will have encephalographic (EEG) recordings to measure the electrical activity of the brain. In addition, they will have one or two positron emission tomography (PET) scans during the first training session, and some will also have one or two PET scans during the last session. For the PET scan, the subject will be injected with a substance called raclopride, which is taken up by the brain. The raclopride is tagged with a radioactive substance so that it can be detected by the PET camera. The amount of raclopride detected in the brain will provide an indirect measure of the amount of dopamine released during training. Before or after one of the training sessions, participants will undergo magnetic resonance imaging (MRI) to study brain anatomy. MRI uses a strong magnetic field and radio waves to produce images of the brain. The subject lies on a table in a space enclosed by a metal cylinder (the scanner). The test takes about 45 to 60 minutes, during which the participant must lie very still for 10 to 15 minutes at a time.
The purpose of this study is to investigate how changes in the brain associated with Parkinson's disease influence the control of motor and thinking speed. There is disagreement over whether patients with Parkinson's disease are slow in thinking as well as movement. This study may provide a new framework to explain the relationship between motor and cognitive aspects of human behavior and help to clarify the pathophysiology of Parkinson's disease. There are two parts to the study: behavioral tests and functional magnetic resonance imaging (fMRI). All participants will be asked about their medical history, have a physical examination, and complete a questionnaire. They must not take regular medications, including levodopa and dopamine agonists, for 8 hours prior to the study. The behavioral study involves computer-generated neuropsychological tasks, including hand movements, imagination of movements, and mental calculations. Response will be recorded and evaluated. Electrodes may be placed on the participant's skin to measure surface electromyogram (EMG). The fMRI study involves MRI scanning in which motor and thinking tasks are performed. Electrodes may be placed on the skin to monitor muscle activity. Thirty patients, ages 40 and up, with early-stage Parkinson's disease will be recruited. Sixty normal volunteers ages 21-75 will be included as well for comparison.
To assess if the CMB305 vaccine regimen may help the body's immune system to slow or stop the growth of synovial sarcoma tumor and improve survival.
This study is to evaluate the safety, tolerability, and pharmacokinetics of ND-L02-s0201 in normal, healthy, subjects
This will be a single center, open label study comparing baseline characteristics of recovered sputum cells (collected on screening day) to those of cells recovered 6 hours after inhalational challenge with 20,000 EU Clinical Center Reference Endotoxin (CCRE, a component of air pollution)) within each group as well as cross group comparisons between individuals with allergic asthma (AA's)and normal volunteers (NV's). The primary objective of this study is to test the hypothesis that persons with allergic asthma will have an increased neutrophil response to challenge with 20,000 EU CCRE compared to normal volunteers. Secondary objectives include post CCRE comparison between AA's and NV's with regard to changes in airway cells and blood as well as changes in mucociliary clearance (MCC) in response to inhalation of 20,000 EU CCRE.
The present study is designed to confirm and extend the observation from previous studies that lixivaptan therapy corrects hyponatremia, in euvolemic subject, including subjects with SIADH.