Sickle cell disease is the most common single-gene disease in the world.
1 France site
20 to 40 Years
This is a phase 1, first-in-human, multi-center, open-label, single ascending dose (SAD) cohort study to evaluate the safety and tolerability, pharmacokinetics (PK), exploratory pharmacodynamics (PD), and biomarkers of target engagement of CSL889 following single intravenous (IV) doses in subjects with stable sickle cell disease (SCD).
18 to 60 Years
Children with sickle cell disease systematically receive a transfusion 2 to 5 days before scheduled surgery (with the exception of minor surgeries) in order to avoid post-operative complications of which the vaso-occlusive crisis and acute thoracic syndrome are the most frequent.
1 Belgium site
< 18 Years
The purpose of this study is to find out whether a web-based intervention using a mobile device is helpful for teens learning to care for and manage symptoms of sickle cell disease.
11 to 17 Years
The aim of the present study is comparing the effectiveness of different treatment regimens for investigating the therapeutic potential for each one in management of Vaso-occlusive pain in pediatric sickle cell disease.
2 Egypt sites
1 Saudi Arabia site
5 to 15 Years
Phase 2/Phase 3
The purpose of this study is to compare the effect of ticagrelor vs placebo for the reduction of Vaso-Occlusive crises in paediatric patients with Sickle Cell Disease .
6 to 17 Years
This study is a single center, prospective exploratory pilot study of Sickle Cell Anemia (SCA) participants.
> 18 Years
Phase 1/Phase 2
This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects ≥2 and ≤50 years of age with sickle cell disease (SCD).
2 United States sites
2 to 50 Years
This study measures the effect of exercise on a variety of biomarkers in blood and urine selected to evaluate the physiological pathways of hemolysis, myolysis, thrombosis, inflammation, and renal function in subjects with sickle cell trait.
18 to 70 Years
This is a dose escalation study to evaluate the safety and tolerability of voxelotor at daily doses of 1500 mg to 3000 mg in participants with sickle cell disease (SCD).
6 United Kingdom sites
18 to 60 Years