This is a Phase 3, randomized, double-blind, placebo-controlled, efficacy and safety study of subjects with IPF treated with inhaled treprostinil over a 52-week period. This study is investigating whether a drug called inhaled treprostinil (brand name Tyvaso®) works to help people with IPF improve their lung function tests. The purpose of this research study is to see how well inhaled treprostinil works in participants with IPF and to gather information on how safe it is. This study will look at changes in your breathing tests, also called lung function tests. About 396 people will participate in this study from about 100 medical centers. Your participation in this study is voluntary and will last approximately 58 weeks. This time includes a Screening Period that could last up to approximately 6 weeks plus a 52-week Treatment Period.
The purpose of this study is to see if taking mepolizumab is safe and effective in treating Hypereosinophilic syndrome (HES) in children and adolescent patients ages 6 to 17 years who are receiving standard of care (SoC) therapy. Mepolizumab is administered through a subcutaneous injection (shot). Participation in the study will last for about 64 weeks approximately (1 year and 3 months).
The purpose of this study is to examine the relationship between common clinical assessments and measurements of the function of brain-spinal cord-muscle connections. For examining brain-to-muscle pathways, we use a transcranial magnetic stimulator. This stimulator produces a magnetic field for a very short period of time and indirectly stimulates brain cells with little or no discomfort. We hope that the results of this training study will help us in developing therapy strategies for individuals, better understanding clinical assessments, and understanding treatments that aim to improve function recovery in people with SCI.
There are 2 aims for this study. The purpose of the first is to examine the relationship between assessments commonly used in therapy and doctor's offices (clinical assessments) and measurements of the function of brain-spinal cord- muscle connections. This will require 2 visits, and each visit will last approximately 2 hours.
The purpose of the second aim is to examine the effects of training on brain-spinal cord-muscle response. This will require 30 visits, and each visit will last approximately 1.5 hours.
Constraint-induced movement therapy (CIMT) is the most efficacious treatment for children with hemiparesis from a perinatal arterial stroke but instead, weekly low-dose OT and/or PT is typical. The aims of this study are to compare 2 high doses of treatment to usual care in helping infants improve skills on the hemiplegic hand/arm and to improve bimanual activities. In addition, the association with gross motor, language and cognition will be explored.
This study is examining the use, safety and performance of an investigational, meaning not approved for commercial use or sale by the US Food and Drug Administration (FDA) device called Transcatheter Mitral Valve Replacement (TMVR). The TMVR is a device used to treat mitral valve disease in patients with heart failure (weakened heart muscle). The device is placed by a non surgical approach using a delivery catheter (hollow tube) placed in your blood vessel at the top of your leg and directed up to your heart. This five year study will include a screening process to determine you meet eligibility criteria. If you qualify you will then be seen for a baseline visit, undergo the procedure to place the device, day after procedure, at hospital discharge, 1,3,6, 12 months, then yearly for up to 5 years. Study related testing includes physical exams, medication review, 6 minute hall walk test, questionnaires, blood work, CT scans, electrocardiogram or ECG (recording of your heart's electrical activity) and echocardiogram (ultrasound test of your heart).
The study will include 9 key informant interviews discussing how patients developed their impression of the benefit of an implant and how patients prefer expectation data be relayed. No video recordings will be performed during interviews. Interview notes or audio recordings will be taken and uploaded into a password protected network server. The results of interviews will mainly be descriptive. To test the plasticity of preoperative patient expectations and the impact of the CI evaluation the CIQOL expectations form will be administered prior to CI evaluation, directly after the CI evaluation, and prior to surgery for all patients who are undergoing cochlear implantation and meet inclusion/exclusion criteria and agree to participate. The decisional conflict scale will also be administered directly after the CI evaluation and prior to surgery for the above patient group. The 9 patients who undergo key informant interviews will also be part of the larger study group of 200 patients and have the same surveys administered and be subject to the same inclusion/exclusion criteria. The group of patients will also be asked about how they prefer expectation and QOL data to be relayed. All survey data will be collected in a secure RedCap database. Patients expectations at the three preoperative timepoints will be compared using paired T-tests. Data regarding how patients develop expectations and how they prefer expectation data to be presented to them will be mainly qualitative and discussed in terms of the most common themes from patient surveys and interviews. When exploring how patients prefer expectations and QOL data to be relayed we intend to include numbered clinical vignettes relaying differing levels of hearing capability. We will compare relaying information through vignettes to more conventional outcome measures such as speech recognition and QOL scores to determine which is the more effective means of communication.
We plan to obtain skin biopsies from patients with systemic sclerosis and healthy donors to grow cells from biopsies. With those cells, we will use them in experiments to see why the cells from systemic sclerosis patients have more fibrosis compared to patients without systemic sclerosis.
The International Intestinal Failure Registry (IFR) is an initiative of the Intestinal Rehabilitation and Transplant Association (IRTA) and The Transplantation Society (TTS) and will be managed by these organizations. The primary objective of this project is to create a large international database of children with intestinal failure to characterize their management and outcome and guide the development of best practices and evidence-based management.
The primary objective of this project is to create a large international database of children with intestinal failure to characterize their management and outcome and guide the development of best practices and evidence-based management.
This open-label, LTFU study will evaluate the safety, tolerability, and efficacy of sotatercept in participants with PAH previously treated with sotatercept or placebo. Participants eligible to enroll in this study will have participated in and completed the relevant study participants of the parent PAH sotatercept clinical trials. The estimated duration of the A011-12 study is up to 7 years; however, the estimated duration of enrollment for each participants is approximately 4 years. There is no formal sample size calculation for the study. The number of participants in this LTFU study is dependent upon the enrollment in the parent protocols. Approximately 700 participants are anticipated to enroll in the study.
This research study evaluates the effects of anFDA-approved medication Gabapentin in individuals with Bipolar Disorder who smoke marijuana. Participants in the study will will be assigned to take either Gabapentin or a matched placebo. Study medication will be taken for 17 days. There will be 5 study visits, with 2 MRI brain imaging scans completed. Questionnaires and clinical interview measures will be completed at study visits along with consistent assessment of potential side effects from study medication.