We are doing this study to test the use of a drug called bromocriptine for women with a condition called Peripartum cardiomyopathy (PPCM). The study will look at how the heart muscle improves in women taking bromocriptine compared to a group of women given a placebo or inactive pill.
This research study will look at a newer treatment option for Barrett's Esophagus (BE) which uses nitrous oxide to freeze unwanted tissue. The most commonly used treatment method is Radiofrequency Ablation (RFA) which uses heat to destroy the pre-cancerous tissue. Nitrous Oxide ablation using the C2 CryoBalloon Focal Ablation System, an FDA-cleared…
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…
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…
The purpose of this study is to determine the safety and long-term effects of CK-3773274 and to learn how well it is tolerated at different, increasing dose levels in participants with HCM.
The purpose of the study is to compare two types of endoscopic treatments in patients with Barretts esophagus (BE) with abnormal cells (dysplasia); radiofrequency ablation (RFA) and hybrid argon plasma coagulation (H-APC). H-APC is a newer technique in the treatment of BE and has not been adequately studied and is not approved by the FDA for routine use.…
The purpose of this trial is to determine if people with impaired heart function, that may have been induced with chemotherapeutic drugs and/or radiation, will have improved heart function with a cardiac device that can pace the heart more efficiently using cardiac resynchronization therapy.
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…
The purpose of this study is to compare the effect of the investigational drug, Mavacamten, to a placebo (an inactive drug). This is being done to evaluate whether Mavacamten is safe in obstructive Hypertrophic Cardiomyopathy (HCM) patients and helps to improve symptoms or ability to exercise. Mavacamten is an investigational drug. This means that it has…
C2 Therapeutics is currently enrolling patients to participate in the Coldplay 3 clinical trial. Coldplay 3 is evaluating the effectiveness of the Coldplay CryoBalloon Focal Ablation System in patients with Barrett's esophagus who have not yet received treatment. Participants will undergo treatment with the Coldplay CryoBalloon Focal Ablation System…
The purpose of this outpatient study is to examine if oral Spironolactone (100mg/day) in addition to loop diuretics (water pills) can improve signs and symptoms of fluid retention in patients with heart failure and prevent hospitalization.
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…
The purpose of this study is to evaluate a new technique called Wide Area Transepithelial Sample 3-Dimensional Esophageal Biopsy (WATS) with computer assisted method of analysis of brush biopsy. This study will assess whether performing standard biopsies in addition to the WATS brush biopsies identifies more patients with dysplasia than does performing…
This research study will evaluate the effectiveness of CEP-417 (Mesenchymal Precursor Cells (MPCs)) when administered through an injection catheter to your heart muscle improving your symptoms, helping your heart function better, improving your quality of life, and in reducing the number of times you may need to be hospitalized due to your heart failure.…