The purpose of this study is to find out whether a web-based intervention using a mobile app is helpful for teens and young adults with sickle cell disease (SCD) in learning how to care for and manage their symptoms. 272 teens and adults with SCD will be enrolled in this study which is being conducted at the Medical University of South Carolina in Charleston SC., East Carolina University in Greenville NC., University of Miami in Miami FL., and the University of Alabama in Birmingham AL.
This study will assess the appropriate dosing and evaluate the safety of NDec in patients with sickle cell disease. Patients may or may not be currently treated with Hydroxyurea to participate in this study. This study is for male and female patients above the age of 18 years who weight between 110 to 308 pounds. Patients must experience at least 2 but no more than 10 Vaso-occlusive crises within a 12-month period. This is an oral medication that is take twice a week and they will participate in this study for approximately 52 weeks.
Researchers want to find out more about how to manage and treat suspected infections and sepsis. Sepsis is a life-threatening condition in which your body is fighting a severe infection.
This study is a collection of health information, blood samples and possibly a nose swab. The samples collected will depend on your doctor's recommendation.
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 purpose of this study is to compare the safety and efficacy of the PMX cartridge ( Toramyxin) (in Addition to Standard Medical Care for Patients with Endotoxemic Septic Shock:
Eligible and consented subjects will be randomized to receive either the PMX cartridge (administered twice for 1½ to 2 hours per treatment session approximately 24 hours apart) plus standard medical care or standard medical care alone. For all subjects in whom treatment has been initiated, a follow-up visit (if they are still in the hospital) or a telephone call will be completed at Day 28 (or later) to determine their mortality status. In surviving subjects, a follow-up visit or telephone call to determine their mortality status will also take place at approximately three months (i.e. Day 90) and 12 months after the subject was randomized.
Over 2400 people who have sickle cell disease and are between the ages of 15 and 45 have been enrolled into the National Registry (SCDIC-I) of patients with Sickle Cell Disease (SCD). A rich resource of natural history data, the SCDIC-I Registry has longitudinal data collected yearly since 2016 from patient surveys (e.g. self reported pain incidences, sleep, barriers to care, experiences during and after pregnancy), medical record abstraction (e.g. medications, transfusion history, co-morbidities) and laboratory results. The 150 patients (or 1200 among the 8 sites) will be selected from both MUSC adult and pediatric SCD clinics starting at 12 years of age; those not previously enrolled in the SCDIC National Registry will be offered the possibility to enroll in SCDIC-II.
We will look at the following:
1- Compare the effect of new SCD medications – crizanlizumab, voxelotor, and L-glutamine – on clinical outcomes in individuals with SCD.
2 - Identify genetic and genomic predictors of response to crizanlizumab, voxelotor, and L-glutamine
3 - Integrate study data into the CureSCi metadata catalog (MDC) to enhance future cross-study analyses.
This study is for participants who have been diagnosed with a pulmonary embolism or PE. A PE is a blood clot in the lung. This study is comparing the FlowTriever Retrieval/Aspiration System to any commercially available Catheter Directed Thrombolysis system in participants who have a PE and undergo a procedure to remove the clot. Participants will be randomized which means they will be randomly assigned like the flip of a coin to have the procedure done wit the the FlowTriever system or a CDT system. All of the devices being used in this study are FDA approved for this indication. This study involves collecting data from the time of diagnosis through 30 days post procedure. Study participation will last about 30 days.
Over 2400 people who have sickle cell disease and are between the ages of 15 and 45 have been enrolled into the National Registry (SCDIC-I) of patients with Sickle Cell Disease (SCD). A rich resource of natural history data, the SCDIC-I Registry has longitudinal data collected yearly since 2016 from patient surveys (e.g. self reported pain incidences, sleep, barriers to care, experiences during and after pregnancy), medical record abstraction (e.g. medications, transfusion history, comorbidities) and laboratory results. The SCDIC-II Registry will continue follow-up of this patient cohort and enroll new patients of all ages to enrich the data resource as a natural history study.
This registry is an observational study designed to evaluate the effect of Oxbryta in individuals with Sickle Cell Disease (SCD) in a real-world setting.
Participants who are currently taking or have been prescribed and initiating standard of care treatment with Oxbryta are eligible to participate. This registry will collect data that is recorded in participants' medical records and other off site sources. Study data will be collected a regular intervals and entered in case reports (CRFs) by electronic capture system (EDC) by the study staff. Participants will be on the study for 5 years after their first dose of Oxbryta treatment or until they choose to withdraw from the study. Participants who stop Oxbryta treatment prior to the 5 years will continue to be monitored at regular intervals unless they request to withdraw from study. Participants quality of life (QoL) questionnaires will be collected throughout the 5 years of the study.
Participants safety and tolerability will be assessed throughout the study data collection period by the study doctor and reported to the Sponsor.
Vaso-occulsive crisis is a complication of Sickle cell disease in which the red blood cells (RBC) change shape, causing congestion within the blood vessels that leads to pain and tissue damage.
The study medication FT-4202, an oral tablet, is believed to reduce the rate of sickle cell polymerization and improve RBC membrane function, thereby reduction sickling of RBCs and their hemolysis (breakdown of red cells) that causes vascular obstruction and anemia.
This study will consist of a 52-week, randomized (volunteers are selected by chance to receive study either study medication or placebo) , placebo controlled (a placebo is a look-alike pill that contains no active medication). There will be 17 study visits.
The study is followed by a 52-week open label extension study in which all participants will receive study medication. There will be 11 study visits.