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 which is Part A of this study. 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 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.
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.