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 effect of ticagrelor versus placebo in reducing the rate of pain crisis in sickle cell disease patients. The study is for male and female subjects between the ages of 2 to 17 who have experienced at least two pain crisis within a 12 month period. The drug is given by mouth in an outpatient setting and consists of 13 visits over an 18 month period.
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 16 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 is a multi-center, longterm safety and efficacy follow-up study for subjects who have been treated with the LentiGlobin BB305 drug product on HGB-206 sickle cell gene therapy parent study. This study is observational only, and no investigational treatment will be given on this follow-up study. Participants for this study are requested to follow-up every six months for the first three years and then once every year for the remaining Year 4 through Year 13, totaling thirteen years in this longterm study. Study assessments will include exams, laboratory studies, imaging, ECG, surveys, and record review.