We are conducting a research study on the effect of high insulin levels on the buildup of excessive liver fat ("fatty liver disease") in people at risk for type 2 diabetes. We are seeking adult volunteers, ages 18-65, who have obesity with a body mass index of 30-45, and have been diagnosed with, or are at risk for, prediabetes and metabolic…
The purpose of this study is to find out if the study medicine called EDIT-301 will help in the treatment of Severe Sickle Cell Disease (SCD) and how safe it is to be used in people. EDIT-301 (study medicine) is a new investigational therapy which uses the patient's own stem cells, modifies them by CRISPR technology, and infuses them back to the…
We are conducting an academic study to understand how people solve problems related to health. In the study, participants will be presented with a hypothetical problem related to diabetes self-management and asked to use an online health application to explore information and propose a solution. We invite all participants between 18 and 65 years old, who…
Seeking participants for a study about how individuals prefer to receive nutrition recommendations to support health management and preferences for visualizing health data. Participants will be invited to complete a 60-minute session to answer questionnaires and evaluate different styles of nutrition recommendations. Alternatively, they will complete one 60…
Seeking participants for a study about how individuals prefer to receive nutrition recommendations to support health management and preferences for visualizing health data. Participants will be invited to complete a 60-minute session to answer questionnaires and evaluate different styles of nutrition recommendations. Alternatively, they will complete one 60…
Although supraventricular tachycardia (SVT), including atrial flutter (AF), are the most common causes of intended in-utero fetal therapy, none of the medication used to date has been evaluated for their effects on the mother and her baby in a randomized controlled clinical trial (RCT). In the absence of such evidence, there is no consensus for the optimal…
The purpose of this study is to learn about whether the study drug (PF-07209326) is safe and effective in people with sickle cell disease and learn about what the right dose is. We will look at drug levels in the blood, see how fast drug levels rise and fall, and what effects the drug has, all of which are known as the pharmacokinetics (PK) and…
We will enroll and randomly assign 30 sedentary, healthy overweight men and women to two groups. Participants will either start by consuming peanuts for 4 weeks, and then go on to exercise at high intensity intervals (HIIT) for 4 weeks, or the reverse order. We will test and compare the effect of peanuts and exercise on inflammation and heart rate…
Over time, children and young adults who have had a Fontan operation may find they are more tired, have difficulty breathing and cannot exercise as well as before. Currently, there are no approved medicines to prevent this decline. The purpose of this research study is to learn whether children who have had the Fontan operation can improve their ability to…
The purpose of this research is to learn how to help parent youth pairs work together to help youth with sickle cell disease take hydroxyurea every day as a daily habit. This study is for children and teens ages 10-18 with Sickle Cell Anemia taking hydroxyurea, and their parents (or guardians). Many children and teens do not use hydroxyurea every day. Daily…
We are currently looking for overweight (body mass index 25 kg/m2) men and women, age 18-65 y, to participate in a sleep study. To participate you must have been diagnosed with Obstructive Sleep Apnea within the past year, and have been prescribed continuous positive airway pressure (CPAP) but not have not yet started treatment. This is a 2-phase,…
This study is being done to see if giving your child a lower dose of chemotherapy (drug therapy), than has been used historically in bone marrow transplant, to kill his/her own diseased cells before he/she receives a bone marrow transplant, will possibly result in the permanent control of your child's sickle cell disease. Patients will receive busulfan…
This is a Phase 3 multi-center open label randomized clinical trial. The purpose of this study is to learnmore about which anti-rejection (or immunosuppressive) medications best protect infants, children,adolescents, and young adults after they have had a heart transplant. Two different drug regimens will bewill be compared. All of these drugs are FDA-…