This study is intended to provide open-label, early access to voxelotor for pediatric patients with Sickle Cell Disease between the ages of 4 to 11 years old who have no alternative treatment options. Voxelotor is approved by the Food and Drug Administration in patients ages 12 and up with Sickle Cell Disease. Voxelotor has not been approved by the Food and Drug Administration for patients less than 12 years of age. Participants will follow their standard of care schedule and receive supply of medication every 3 months during study period.
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.
For patients with thrombotic thrombocytopenic purpura (aTTP), research involving SHP655 added to current standard of care treatment for aTTP may be effective by reducing the severity of TTP episodes, length time on therapy and reducing clinical complications of the disease. rADAMTS-13 is an essential protein that is inactivated by antibodies. The study drug, SHP655, is given by intravenous injection (IV). It is an essential synthetic protein used to replace decreased volumes of the rADAMTS-13 protein which will prevent or lessen the symptoms of aTTP. The study will last approximately 1.5 years with the Treatment phase occurring during hospitalization for the treatment of aTTP illness and the follow-up phase that will consist of study visits occurring every week for 4 visits, then biweekly for the 2 visits with a final completion visit 1 month after the last follow-up visit lasting for a 3-month period. The length of time for each visit should last approximately 2 hours. If a recurrence or relapse of aTTP occurs during the follow-up phase, subjects will not receive additional study drug, but subjects will be followed on a bi-weekly visit schedule until remission is achieved or 4 months after the initial remission, whichever is sooner.
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 is to evaluate the effectiveness of crizanlizumab-an monoclonal antibody on male patients between 16 to 65 years of age with Sickle cell disease experiencing vaso-occlusive crisis (VOC) priapism. The study will review the number of VOC-priapism events, their duration of the episodes and requirement of opioid treatment. Male patients may also take Hydroxyruea (HU) during study but must be receiving HU for at least 14 weeks before screening and continue HU during study.
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.
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.
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.