REHAB-HFpEF is a multicenter, randomized, attention-controlled, single-blind trial to examine the hypothesis that, a novel, tailored, progressive, multi-domain physical rehabilitation intervention administered to older patients with acute decompensated heart failure (ADHF) with preserved ejection fraction (HFpEF) beginning early during hospitalization and continuing for 12 weeks in a structured outpatient setting, and continuing as a maintenance program will reduce the rate of combined all-cause rehospitalization and death at 6 months (the primary outcome), and reduce major mobility disability (MMD) prevalence at 6 months (the secondary outcome). This trial, REHAB-HFpEF, builds upon preliminary studies, including the phase 2 REHAB-HF trial, which suggests this intervention may yield significant benefits for this population which are largely older, frail, and with few evidence-based treatment options.
The study will recruit a total of 880 consenting patients >=60 years old hospitalized with ADHF with HFpEF. Following informed consent and baseline testing, the participants will be randomized in a 1:1 fashion to receive a novel, progressive, multi-domain rehabilitation and exercise training intervention or attention control. The intervention will include strength, balance, endurance, and mobility training and the specific training exercises will be tailored based on participant performance in each of these domains. The intervention will begin as soon as possible after randomization during the hospitalization and will continue 3 times per week in an outpatient setting for 12 weeks. Those randomized to the attention control will be contacted bi-weekly by study staff to maintain contact, collect information regarding health status, clinical events, and physical activity/exercise, and ensure retention; they do not receive any specific exercise recommendations. Both arms will receive all services ordered by their primary physician (usual care) and undergo measures of physical function, cognitive function, and quality of life (QOL).
This study's objective is to determine if electrical stimulation can safely reduced atrial fibrillation, which is an abnormal heart rhythm that begins in the top chambers of the heart. Electrical stimulation will be delivered to the wrists or in the ear. Participation will last about 6 weeks and require 3 clinic visits. Participants will be asked to wear a heart monitor and patch to monitor the heart rate and rhythm, keep a diary to collect information on atrial fibrillation, undergo randomization (being randomly assigned, like drawing straws) and use the study device per randomization assignment. Participants are randomized in a 1:1:1 fashion to a wrist worn study device, an ear piece study device or a sham wrist worn study device. A sham device looks like the real wrist worn device but does not deliver any electrical stimulation.
This study will evaluate the safety and effectiveness of fazirsiran (investigational drug) compared with placebo (an inactive substance) in patients with alpha-1 antitrypsin deficiency associated liver disease (AATD-LD). If eligible, subjects will be randomized (assigned to a group by chance) to receive either fazirsiran or placebo to be administered subcutaneously (an injection under the skin). Subjects will be treated on Day 1, at Week 4, and then every 12 weeks for 196 weeks. Subjects will be followed for 6 months after their last dose of study drug or placebo for a total study duration of approximately 230 weeks (including 10 weeks of the screening period which is the time needed to assess if a subject is eligible for the study).
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 Advanced Systemic Mastocytosis. The investigational drug is known as CGT9486 and will be taken daily orally. Participation in the study is expected to be approximately 6 years.
The objective of this research study is to identify brain biomarkers using MRI scans that can predict an individual's response to Deep Brain Stimulation (DBS). A total of 55 participants with Parkinson's Disease planning to undergo DBS will be recruited from MUSC's Clinical DBS Program. Participants will undergo two visits which include a 2.5-hour pre-DBS questionnaire and MRI scanning visit, and a 3.5-hour post-DBS cognitive assessment visit. In addition control participants without Parkinson's Disease will be recruited to undergo MRI scanning and cognitive assessments.
The study will be an open-label, randomized, parallel arm study that will include a
treatment arm and control arm. Participants will have clinic visits at screening, randomization (day 1) and weeks 4, 12, 18, and 24. After week 24, participants will have clinic visits at weeks 32, 40, and 48. Participants will also have a telehealth visit on day 2 and phone calls to assess adverse events (AEs), serious adverse events (SAEs), and review patient education will occur during weeks 5, 8, 36, and 44. The phone calls may be converted to telehealth visits or clinic visits and / or followed by clinic visits, if the study team deems it necessary. Pulmonary function testing, quality of life survey (St. George's Respiratory Questionnaire (SGRQ)), and blood draw will occur at each clinic visit.
This study wants to understand how we can better identify children who have experienced or are at risk to experience child maltreatment. We will be interviewing caregivers of children and primary care providers (nurses, pediatricians, social workers) to understand their perspectives on screening for child safety risks in primary care settings. Interviews with providers will also focus on their feedback on a new tool to designed to detect child safety risks within health care settings. Findings will help inform best practice in integrated health care settings.
Ritlecitinib (PF 06651600) is an investigational drug (referred to as "study drug" from here on) and is being developed as possible study treatment for participants with non segmental vitiligo (both active and stable vitiligo). The study drug will be compared with a placebo to find out if the study drug is better than the placebo for the study treatment of vitiligo. The duration of this study is a maximum of 60 weeks. There will be an initial screening period of 30 days where the study doctor will determine eligibility. If eligible, participants will be randomly assigned to receive the study drug or a placebo during a 52 week study treatment period. At the end of the treatment period, participants will enter a 4 week Follow Up Period.
The purpose of this research study is to determine if an already FDA approved drug called atorvastatin can help adults, age 75 or older, live well for longer by preventing dementia, disability, or heart disease. Participants will be assigned by chance to receive either atorvastatin (study drug) or a matching placebo (a tablet that looks like atorvastatin but does not contain any medicine). Study engagement may last up to five years and involves in clinic and/or remote visits, survey questions, potential blood draws, in addition to memory and physical tests. After the first 1-2 visits performed in clinic or remote, the study team will follow-up via phone on a yearly basis to see how participants are doing. Participants will be compensated for their time and participation in this research study.
The purpose of this research study is to determine if an already FDA approved drug called atorvastatin can help adults, age 75 or older, live well for longer by preventing dementia, disability, or heart disease. Participants will be assigned by chance to receive either atorvastatin (study drug) or a matching placebo (a tablet that looks like atorvastatin but does not contain any medicine). Study engagement may last up to five years and involves in clinic and/or remote visits, survey questions, potential blood draws, in addition to memory and physical tests. After the first 1-2 visits performed in clinic or remote, the study team will follow-up via phone on a yearly basis to see how participants are doing. Participants will be compensated for their time and participation in this research study.