This study will assess the safety and efficacy of voxelotor with long term, daily oral dosing compared to placebo in pediatric participants (ages 9 months to 12 years old) with SCD as measured by improvement in anemia. Participants can expected to be in this study for about 52 weeks with at least 12 visits to the study center.
This open label extension study is being conducted to assess the long-term safety and efficacy of GBT440 by providing participants from GBT440-031 continued access to treatment with GBT440 after completion of their participation in GBT440-031 and prior to the product potentially being available commercially. All participants enrolled into this study will receive GBT440.
This study is for patient with Sickle Cell Disease ages 16 to 70. The purpose of this study is to find out if participants with sickle cell disease have a reduction in the symptoms of their disease. Participants can expected to be in this study for about 12 weeks.
This study is for patients 18 years and older who have Sickle Cell Disease. The study will be evaluating the safety and tolerability of treatment with riociguat to potentially improvement in blood pressure, blood circulation, and disease symptoms. Participants can expect to be in this study for 20 weeks.
This is for patients aged 18 and older who have Sickle Cell Disease. The main purpose of this study is to find out how safe IMR-687 is for treating people with Sickle Cell, and whether it is has any unwanted effects. Participation in this study will last about 8 months.
This study will assess the efficacy of crizanlizumab in sickle cell disease patients. The study is for male and female subjects between the ages of 18 to 70 who have experienced at least one pain crisis within a 12 month period. The drug is given via an IV infusion in an outpatient setting and consists of 52 visits.
This study tests an intervention to help children with sickle cell disease ages 0-7 years and their families. The intervention is available on a smartphone or tablet, and includes an app for keeping track of symptoms and messaging with a health care provider. For more information, please contact Shannon at 843-792-9379.
The purpose of this study is to find out whether a web-based intervention using a mobile device over a 12-week period is helpful in managing symptoms of Sickle Cell Disease in children, such as pain and fatigue and quality of life. For more information, please contact Shannon at 843-792-9379.
iSCENSC is a three-part study that will use new methods (called implementation science) to identify ways to enhance care for people with sickle cell disease (SCD) who are in the (SC)2 South Carolina Sickle Cell network. The (SC)2 network is designed to help more people with SCD find a doctor with training in sickle cell disease and to improve their medical care. Part one of the study is a needs assessment where we look at the current barriers for people with SCD. Part two is a registry for individuals with SCD; and part three will be a study or studies to try and help improve the care for individuals with SCD.