It has been established that individual's with Sickle cell disease (SCD) have an increased risk of mental changes (cognitive impairment) related to neurological damage such as a stroke (cerebral vascular accident) or decreased vascular blood flow in the brain decreases oxygenation (hypoxia) in the blood stream related to decreased blood counts and severe anemia.
Voxelotor is an oral medication taken once a day, that was approved by the Food and Drug administration (FDA) in 2019 for patients with SCD ages 12 years and older to improve patients' hemoglobin and the delivery of oxygen to the brain.
This study is designed to assess patient neurocognitive (ability to concentrate, remember things, process information, learn, speak, and understand) functions by having subjects answer questionnaires at the week 1 and end of treatment visits. The study will have 5 visits and last approximately 4 1/2 months,
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.
This study is testing an IV infusion medication to treat painful pain episodes, called vaso-occulsive crises, in participants with Sickle Cell Disease. Participants will have a screening visit followed by a 24-hour inpatient hospitalization visit to receive the IV medication and then 8 follow up visits. The purpose of this study is to learn more about how a new potential drug for treating SCD symptoms affects the body and how the body processes it.
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.
This study will evaluate if Food and Drug Administration (FDA) approved recombinant von Willebrand Factor (rVWF) is safe and effective with long term use in adult and pediatric/adolescent patients with von Willebrand Disease (VWD). Dosing is an optional prophylactic (preventative) dosing on a weekly basis, or on demand (OD) for bleeding episodes and in the management of surgical bleeding. This study may last up to 3 years. You will have clinic visits in 3 month intervals during this time.
Sickle cell disease (SCD) is a inherited disease that can cause sudden, severe pain. The management of this pain is accomplished through analgesic medications. This study for for male and female subjects between the ages of 18 and 65 years. This study will assess the appropriate dose and the evaluate the safety of SHP 655 in SCD patients at a baseline health state. The study medication is given by infusion as a single dose. SHP 655 is believed to increase blood flow and decrease blood cells from being trapped in low blood flow areas such as joints which can lead to tissue death.
This study will assess the appropriate dosing and evaluate the safety of crizanlizumab in pediatric sickle cell disease patients. The study is for male and female subjects between the ages of 6 months to 17 years old 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 has the potential to reduce the amount of sickle cell pain crisis a participant may experience. Participants can expected to participant in this study for up to 2 years.
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 a study to determine the use of recombinant Von Willebrand Factor (rVWF) in the treatment and control of nonsurgical bleeding episodes and bleeding during elective and emergency surgery in children with severe Von Willebrand Disease. The study will last approximately 14 months and will involve regular visits to a research clinic.
Blood clots in children are rare when compared to the adult population. However, in the past ten years the increased survival of children with serious illnesses and improved diagnostic techniques have led to an increasing awareness of the occurence and consequences of blood clots in the pediatric population.
Children and adults are thought to share a common physiology of blood clots. In adults several risk factors are known to start one or more of the clotting cascade. The physiology in children is similar but the contribution of each factor differs among age groups. Once a blood clot occurs the progression of hte disease and the aim of antithrombotic (anti-clotting) therapy is the same for both adults and children. These aims are to 1)reduce the risk of death due to blood clots ; 2)reduce the occurence of recurrent blood clots; 3)reduce the occurrence of post clot syndrome by limiting the vascular damage; and 4) maintain vessel patency and vascular access.
Anticoagulation therapy in children can be administered prophylactically to prevent blood clots or in therapeutic doses om those with confirmed blood clots. There is no standard of care for all children for the treatment of blood clots, recommendations include the use of unfractionated heparin, low molecular weight heparin, and/or a vitamin K antagonist.
Apixaban is an orally active factor Xa inhibitor that is being developed for the treatment of venous blood clots in children. The safety and effectiveness profile of Apixaban in other trials indicates a possible benefit to oral apixaban in children over standard of care for the treatment of blood clots. This trial will enroll pediatric patients who require anticoagulation therapy for newly diagnosed blood clots. Subjects will be randomized to receive either apixaban or standard of care. The primary oal of the study is to assess whether apixaban is safe and effective in children for the treatment of blood clots over 12 weeks or over 6 to 12 weeks of therapy in neonates.