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 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 is a first-in-humans gene therapy study for subjects diagnosed with severe hemophilia A. A one time infusion of the gene therapy product (BAX888) will be given with the hope of eliminating or reducing the need for prophylactic and/or on-demand use of FVIII concentrate therapy. Up to 10 subjects will be enrolled study-wide with up to two subjects enrolled at MUSC. This study will require weekly visits to the study clinic after initial infusion for the first 15 weeks followed by monthly visits for the first year. Compensation will be provided.
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.
This study is meant to compare transplant to standard care (regular care) for sickle cell patients. By comparing the health outcomes for patients who receive bone marrow transplant to those patients who receive standard of care, this study will be able to determine whether the two treatments are the same, or if one is better than the other.
The main symptom of sickle cell disease is pain-and it is the main reason for which patients are admitted. This study introduces a new drug, Rivaipansel, which may shorten the duration of this pain (called a "crisis"). This is a phase 3 open label extension research study using the drug Rivipansel in sickle cell patients in the inpatient setting. Patients who consented and participated in the first part of the study (B5201002) may be consented to receive the drug Rivipansel during a subsequent pain crisis to test the efficacy and safety of repeated treatments for pain crises.
Study participants in this study will be recruited because of development of a clot. Clots in pediatric patients are typically treated with blood thinner medicine for 3 months (sometimes longer, depending on confounding factors). It is possible that treatment for 3 months may be longer than needed in children whose clots do not block blood flow through the vein after 6 weeks of treatment.
Over a period of five years, up to 750 children will be enrolled in this study internationally. Each participant will be randomized to a total of 6 weeks or 3 months of venous thrombosis treatment. Doppler ultrasounds of the blood clot will be taken at approximately 6 weeks after diagnosis of the blood clot (the time of the first picture). If the picture at 6 weeks shows that the blood clot is completely blocking blood flow through the vein, the patient will still be followed in the study, but will not be eligible to get the 6 week length of blood thinner treatment. Instead, the patient will receive the usual length of treatment (3 months).
Patients initially positive with an antiphospholipid antibody test that was still positive at approximately 6 weeks after diagnosis of the blood clot, will still be followed in the study, but will not be eligible to get the 6 week length of blood thinner treatment. Instead the primary doctor will decide length of treatment as part of standard care, which will be at least 3 months in total, and possibly much longer.
At the 6 week and 3 month visits, the patient will have an additional 10 mL (2 teaspoons) [for infants: 6 ml, or just over 1 teaspoon] of blood drawn for clotting research tests designed to identify new risk factors for blood clots and their long-term effects.
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.