This study is enrolling participants with heart failure, a condition where the heart muscle does not pump blood efficiently, with preserved ejection fraction, meaning the heart muscle contracts (squeezes) normally but is unable to relax appropriately. The study involves implanting a pacemaker, a small device that is placed in the upper left chest, and then programming it to either standard settings or personalized settings for you based on your height and heart function. The programming is randomized, meaning assigned by chance, like flipping a coin so you do not get to choose which group you are in nor does your doctor. The study is trying to determine if using the pacemaker to control your heart rate can help you heart failure.
Your participation will last at least 18 months and may be as long as 4.5 years depending on when you join the study. The study will include about 8 visits and include the pacemaker implant procedure as well as testing such as physical exams, 6 minute walk test, echocardiograms (ultrasound test of your heart), blood work, questionnaires and pacemaker checks.
There are risks associated with the pacemaker implant procedure, risks related to study related procedures and the risk of loss of confidentiality. There may be benefit to you and to others with this condition in the future.
BTX-302-001 is a research study investigating the safety (how many side effects participants may have) and tolerability (how tolerable the side effects are) of BEAM-302 for individuals with Alpha-1 Antitrypsin Deficiency (AATD)-associated lung and/or liver disease. This study also aims to gather additional information regarding how BEAM-302 moves through the participant's body, how long it stays, and how long it takes to eliminate it - which is defined as the study drug's pharmacokinetics or "PK". Researchers would like to determine through this research study how BEAM-302 impacts the disease course (progression) of AATD in terms of AATD blood biomarkers, which are substances in blood that the body normally makes and will help show if an individual's AATD is improving, staying the same, or getting worse, along with lung and liver function testing results and the quality of life of participants.
This research study will be split into two main parts, Part A (which is for individuals with AATD-associated lung disease with no clear evidence of AATD-associated liver disease) and Part B (which is for individuals with AATD-associated liver disease). Additionally, each Part will be split into two separate cohorts, where one cohort will receive a single intravenous (IV) infusion of BEAM-302 (single-dose cohort) and the other will receive two IV infusions of BEAM-302 approximately 8 weeks apart (multi-dose cohort). Within these cohorts (single-dose and multi-dose), there are also separate smaller cohorts that will vary by the dose of BEAM-302 administered to participants, so a participant in this study could receive any of the following dosages - 15mg, 30mg, 60mg, 75mg, or 90mg. Overall, the research study will last up to around 29 months for each participant, depending on which cohort they are in, and their participation will be split into three main study periods - Screening, Dose and Dose-limiting toxicity (DLT), and Follow-up. It is also important to note that when a participant is receives their infusion(s) of BEAM-302 during the Dose and DLT period, the administration of the study drug will be done as a part of an in-patient hospital stay that will last up to 48 hours so that they can be closely monitored by the study team.
The key eligibility criteria for this study are that individuals (male or female) must be 18 to 70 years old, possess the PiZZ type of AATD, and have either AATD-associated lung disease with no clear evidence of AATD-associated liver disease or AATD-associated liver disease. There are additional eligibility criteria that must be met in order to be able to participate in the study, which will be assessed across up to 2 study visits that will occur during the Screening period.
Children with amblyopia (lazy eye) are often treated with patch therapy. Patch therapy often results in leftover amblyopia and has many challenges associated with its use. This study will determine how traditional patch therapy results compare to those achieved with the Luminopia digital therapeutic system.
Preterm and term infants with brain injury frequently have difficulty learning to feed by mouth. Transcutaneous vagus nerve stimulation (taVNS) may be paired with the motor activity of feeding to boost brain circuits involved with feeding. This study will test a taVNS-paired bottle system in a blinded, randomized, controlled trial in infants who have reached term age and failed to learn to feed. Our preliminary data indicates that most infants improve their volume of oral feeds with the BabySTrong system, and 50-70% achieve full oral feeds and avoid placement of a gastrostomy tube (G-tube) or home nasogastric tube. The BabySTrong feeding system may improve oral feeding volumes and help infants and their families avoid a long hospital stay while trying to learn to feed.
Children with amblyopia (lazy eye) are often treated with patch therapy. Patch therapy often results in leftover amblyopia and has many challenges associated with its use. This study will determine how traditional patch therapy results compare to those achieved with the Luminopia digital therapeutic system.
The purpose of this research study is to find out if giving radiation therapy (RT) early to high-risk bone metastases that are not causing pain can reduce the chance of skeletal-related events (SREs) such as fractures, spinal cord compression, or surgery to bone. This will be compared to the current standard of care (SOC), which usually treats bone metastases only when symptoms like pain occur. The study will enroll 16 participants locally over 25 months, and each subject will remain in the study for at least 40 months. You are being asked to join because you have metastatic cancer that has spread to your bones and is considered "high-risk," though it is not causing pain at this time. High-risk bone metastases are typically located in the spine, hip, shoulder, or long bones, and larger tumors in these areas are more likely to cause complications. If you join this study, you will receive radiation therapy to high-risk bone sites in addition to standard care. The main risk is that radiation therapy may not work better than the usual approach at preventing bone complications. Radiation can also cause side effects, including skin changes, tiredness, and inflammation of the esophagus, bowel, or lungs. There may also be risks that are not yet known to the study doctors.
The aim of this supplement study is to learn more about the emotional health and symptom needs of long-term cancer survivors (over 5 years since diagnosis) living with likely incurable disease. Participants in this qualitative substudy will be individuals who are long-term cancer survivors who participated in another study of depression in individuals with likely incurable cancer. We will recruit long-term cancer survivors with likely incurable cancer to participate in one-time, 45- to 60-minute in-depth, semi-structured interviews. We will ask patients about emotional health and symptom burden concerns and elicit attitudes toward digital mental health interventions (DMHI) and preferences for intervention type and delivery. Findings will inform the development of digital mental health interventions tailored to this patient population.
The aim of this supplement study is to learn more about the emotional health and symptom needs of long-term cancer survivors (over 5 years since diagnosis) living with likely incurable disease. Participants in this qualitative substudy will be individuals who are long-term cancer survivors who participated in another study of depression in individuals with likely incurable cancer. We will recruit long-term cancer survivors with likely incurable cancer to participate in one-time, 45- to 60-minute in-depth, semi-structured interviews. We will ask patients about emotional health and symptom burden concerns and elicit attitudes toward digital mental health interventions (DMHI) and preferences for intervention type and delivery. Findings will inform the development of digital mental health interventions tailored to this patient population.
The aim of this supplement study is to learn more about the emotional health and symptom needs of long-term cancer survivors (over 5 years since diagnosis) living with likely incurable disease. Participants in this qualitative substudy will be individuals who are long-term cancer survivors who participated in another study of depression in individuals with likely incurable cancer. We will recruit long-term cancer survivors with likely incurable cancer to participate in one-time, 45- to 60-minute in-depth, semi-structured interviews. We will ask patients about emotional health and symptom burden concerns and elicit attitudes toward digital mental health interventions (DMHI) and preferences for intervention type and delivery. Findings will inform the development of digital mental health interventions tailored to this patient population.
The aim of this supplement study is to learn more about the emotional health and symptom needs of long-term cancer survivors (over 5 years since diagnosis) living with likely incurable disease. Participants in this qualitative substudy will be individuals who are long-term cancer survivors who participated in another study of depression in individuals with likely incurable cancer. We will recruit long-term cancer survivors with likely incurable cancer to participate in one-time, 45- to 60-minute in-depth, semi-structured interviews. We will ask patients about emotional health and symptom burden concerns and elicit attitudes toward digital mental health interventions (DMHI) and preferences for intervention type and delivery. Findings will inform the development of digital mental health interventions tailored to this patient population.