A Phase 2, multicenter, randomized, placebo-controlled, doubleblind study in patients with acquired thrombotic thrombocytopenic purpura (aTTP) to evaluate the pharmacokinetics, safety, and efficacy of rADAMTS-13 (SHP655) administered in addition to standard of care (SoC) treatment

Date Added
September 8th, 2020
PRO Number
Pro00098276
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

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.

Institution
MUSC
Recruitment Contact
Karen Hawkins
843-792-0560
hawkink@musc.edu

A Phase 1/2 randomized, double-blind, placebo-controlled, multicenter, ascending dose, safety and Pharmacokinetic/Pharmacodynamic PK/PD study of SHP655 (rADAMTS13) in sickle cell disease at baseline health and during acute vaso-occlusive crisis.

Date Added
August 25th, 2020
PRO Number
Pro00101087
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

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.

Institution
MUSC
Recruitment Contact
Karen Hawkins
843-792-0560
hawkink@musc.edu

A Prospective Phase II, Open-Label, Single-arm, Multicenter, Study to Assess Efficacy and Safety of SEG101 (crizanlizumab), in Sickle Cell Disease Patients with Priapism (SPARTAN)

Date Added
February 11th, 2020
PRO Number
Pro00091529
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

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.

Institution
MUSC
Recruitment Contact
Karen Hawkins
843-792-0560
hawkink@musc.edu

A phase 2, Multicenter, Open-Label Study to Assess Appropriate Dosing and to Evaluate Safety of Crizanlizumab, with or without Hydroxyurea/Hydroxycarbamide, in Sequential, Descending Age Groups of Pediatric Sickle Cell Disease Patients with Vaso-Occlusive Crisis

Date Added
August 14th, 2018
PRO Number
Pro00079784
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

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.

Institution
MUSC
Recruitment Contact
Karen Hawkins
843-792-0560
hawkink@musc.edu

A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF with or without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed with Severe von Willebrand Disease

Date Added
February 28th, 2017
PRO Number
Pro00062418
Researcher
Shayla Bergmann

List of Studies


Keywords
Drug Studies, Pediatrics
Summary

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.

Institution
MUSC
Recruitment Contact
Lauren Card
843-792-5935
cardl@musc.edu

A Study to Compare Bone Marrow Transplantation to Standard Care in Adolescents and Young Adults with Severe Sickle Cell Disease

Date Added
December 6th, 2016
PRO Number
Pro00056206
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders, Transplant
Summary

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.

Institution
MUSC
Recruitment Contact
Brandi Day
843-792-3379
dayb@musc.edu

A RANDOMIZED, OPEN-LABEL, ACTIVE CONTROLLED, SAFETY AND DESCRIPTIVE EFFICACY STUDY IN PEDIATRIC SUBJECTS REQUIRING ANTICOAGULATION FOR THE TREATMENT OF A VENOUS THROMBOEMBOLIC EVENT

Date Added
November 10th, 2015
PRO Number
Pro00048857
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

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.

Institution
MUSC
Recruitment Contact
Karen Hawkins
843-792-0560
hawkink@musc.edu



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