The purpose of this study is determine the optimal dose, efficacy and safety of an investigational drug (a new drug not yet approved by the U.S. Food and Drug Administration) in adults with Nonadvanced Systemic Mastocytosis. The investigational drug is known as bezuclastinib and will be taken daily orally. Participation in the study is expected to be approximately 3 years.
Individuals with obesity and a history of heart or kidney-related disease will be eligible for participation. Study participants will have a 50:50 chance to be randomly assigned to either the treatment (retatrutide) or control (placebo) group. Retatrutide is currently considered an investigational medication as it has not been approved by the FDA for the treatment of obesity or any other medical condition at this time. Study participation will last approximately 5 years and begin with a screening period to ensure correct patient selection. Research clinic visits will occur every 4 weeks initially and then every 12 weeks. Some visits may be completed virtually through telehealth or by phone. Study procedures include but are not limited to: blood draws, questionnaires, self injection of study medication, medical history review, vital signs, and electrocardiogram. The medication will be injected subcutaneously once a week. The medication's purpose is to facilitate weight loss and decrease a subject's risk for heart or kidney related disease progression or events.
The purpose of this study is to evaluate how certain procedures work for individuals with chronic neck pain.
Individuals who are 18 years of age or older and have chronic neck pain will participate in this study. The study involves randomly assigning individuals into two treatment groups (i.e. manual therapy and resistance exercise). Both treatments are recommended treatments and are part of clinical practice guidelines and are well supported in the literature.
Participants will be asked to complete a 4-week training program which includes physical examination, education, treatment, and an exercise plan. Participants will also complete a brief set of questionnaires at their baseline visit, 4 weeks, and 6 months after their initial visit
This is a research study involving an investigational drug called RT234. RT234 is being developed to treat pulmonary arterial hypertension (PAH). Eligible subjects must have a confirmed diagnosis of PAH and be on stable disease-specific PAH background therapy. The purpose of this study is to collect more information about the ability of RT234 to improve exercise capacity and safety of RT234 for the treatment of pulmonary arterial hypertension. The total duration of participation in the study, including screening, will be up to 4 study visits and a follow-up telephone call, over a period of up to 14 weeks. Up to 60 subjects will take part in this study.
Study LTI-401 is an open-label, multicenter study which will evaluate the safety and tolerability of LIQ861, the study drug, in subjects who have World Health Organization (WHO) Group 1 & 3 Pulmonary Hypertension. The purpose of this research study is to evaluate the long-term safety and tolerability of LIQ861 in patients with WHO Group 3 Pulmonary Hypertension associated with interstitial lung disease (PH-ILD). The investigational form of Treprostinil in this study is called LIQ861, it is delivered to your lungs using a hand-held device called a dry powder inhaler (DPI). Dose levels may be adjusted by the Study Doctor between 26.5 micrograms to 318 micrograms based on your PH-ILD symptoms. The study will include approximately 60 subjects and participation will last 52 weeks.
This is an inpatient clinical trial of a new form of transcranial magnetic stimulation with 10 treatments/day for 5 days, called SAINT. It is jointly funded by the NIH and a TMS company, MAGNUS. It is being done at 5 enrolling sites in the US, with total sampe of 100. Depressed and suicidal inpatients will have an MRI scan, then 5 days of treatment, and then a followup MRI scan. Outcome measures are depression and suicide ratings, as well as MRI changes. It is randomized and double blind, so some patients will not be getting active TMS. Importantly this is an adjunctive treatment study on top of all routine clinical treatments.
The purpose of this medical research study is to evaluate the safety and effectiveness of a new medication called imatinib mesylate in the treatment of Lymphangioleiomyomatosis (LAM). LAM is a rare disease in which abnormal cells (called LAM cells) grow out of control. Over time, LAM cells destroy healthy lung tissue and cause respiratory disease or failure.
Many patients with LAM are currently treated with a medication called sirolimus (rapamycin). Sirolimus slows the growth of LAM cells.
Imatinib mesylate (hereafter called imatinib) is approved by the Food and Drug Administration (FDA) for the treatment of some cancers that share common pathways with LAM cells. Laboratory studies suggest that imatinib could completely block the growth of LAM cells through initiation of targeted cell death.
An important purpose of this research is to determine the safety of imatinib in people with LAM. This study will also evaluate the short-term effectiveness of imatinib. Participants will be randomized to receiving imatinib (study medication) or placebo (no treatment) for the 180 day duration of participation. The study is being conducted at the Medical University of South Carolina and at Columbia University in New York (CUMC). Each site will enroll 10 participants.
As people get older, understanding speech in a noisy environment can become more difficult. The MUSC Hearing Research Program is seeking healthy adults between 25 and 89 years of age to participate in a research study to investigate the connection between listening difficulties and the brain. Payment is provided for participation and scheduling is flexible. The study involves two visits. Participants must be able to complete an MRI. Please contact us if you would like to participate in this research or learn more about our study.
The aim of this study is to use magnetic resonance imaging (MRI) to capture images of participants' brains and apply an image processing method called INSCAPE to assess brain states. This study will recruit participants who are scheduled to receive accelerated Transcranial Magnetic Stimulation (TMS) therapy for Major Depressive Disorder (MDD). Participation in this study involves two visits, each including a 30-minute MRI scan. The interval between each visit is about one week. Depressed participants will also complete mood assessments at various intervals while they are receiving accelerated TMS for depression.
Up to 67% of patients undergoing heart surgery may experience complications after surgery. These complications may include longer stay in the intensive care unit (ICU), more time on a ventilator (also known as a breathing machine), longer overall stay in the hospital, having to return to the hospital soon after being discharged, and even death.
This research study is being performed to see whether RBT-1, an investigational new drug, can reduce complications after heart surgery
In this research study, you will have a 50% chance of being assigned to receive RBT-1 and a 50% chance of being assigned to receive placebo.
If you decide to participate, you will receive a single intravenous (IV) infusion (an infusion into your vein) of either: 1) RBT-1 at a dose level of 45 mg SnPP/240 mg FeS or 2) placebo in the form of normal saline (salt water solution). You cannot choose whether you are given RBT-1 or placebo.