This study is to better understand barriers to and resources for transcranial Doppler (TCD) screening in children with sickle cell anemia (SCA) ages 2 to 16 years. Surveys and interviews are being conducted with children with SCA and their parents/caregivers, healthcare providers, and other stakeholders to learn about their thoughts and experiences.
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 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.
GBT440 is an study drug that has been shown to increase oxygen in the blood. This study is recruiting patients with sickle cell disease to establish efficacy and safety of GBT440 as compared with placebo.
The purpose of this research protocol is to collect information about individuals living with sickle cell disease to help improve the care of those patients. We hope to understand more about the disease itself, the best ways to treat the disease, and the best ways to help patients with sickle cell disease get care. The goal is to have clinical information about every person diagnosed with sickle cell disease in South Carolina so that we can better treat the disease and help develop new ways to improve outcomes.
Ferriprox is an iron chelator that is approved by the FDA of the USA in October of 2011 for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate. LA38-EXT is an open-labeled extension trial of LA38-0411 where patients who completed LA38-0411 will be invited to enroll in the extension trial to determine the long-term safety and tolerability of Ferriprox in iron-overloaded patients with sickle cell disease or other anemias.