Active clinical trials for "Purpura, Thrombotic Thrombocytopenic"

The SABRE study is a single-arm prospective study measuring safety, tolerability and pharmacokinetics of two SARS-CoV-2 neutralising antibodies (BMS-986414 and BMS-986413) amongst high-risk special populations of vaccine non-responders. The aim is to test the hypothesis that for individuals who fail to mount a measurable immune response to a routinely offered SARS-CoV-2 prophylactic vaccine or for those who are not able to receive such a vaccine (for example those receiving a bone marrow transplant or starting chemotherapy treatment), the receipt of subcutaneous injection of two long-acting neutralising antibodies BMS-986414 and BMS-986413 will confer durable high titres and subsequent immunological protection against SARS-CoV-2 infection.120 eligible participants will be enrolled and followed up for 48 weeks after the one-time dosing visit. Primary inclusion criteria are patients age 18 years and older and either 1) have received two doses of a routine NHS standard of care SARS-Cov-2 vaccine and do not have detectable serum SARS-CoV-2 anti-spike antibodies in routine NHS assays more than two weeks post-vaccination, or do not have protective levels of antibody or 2) be ineligible to receive a SARS-CoV-2 prophylactic vaccine. This could be because they need to commence immediate systemic chemotherapy or receive bone marrow and therefore the requirement to initiate profound immune suppression. Primary objectives are to determine the safety, tolerability and detectable SARS-CoV-2 antibody by specific PPD assay in serum at 12 weeks after enrolment.

The general objective of this study is to assess the efficacy and safety of Rituximab in the management of patients with refractory or relapsed thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS). There have been several case reports and case series describing the use of Rituximab in patients with TTP-HUS; however its use has not been studied in a large trial. It is hypothesized that Rituximab may ameliorate the severity of certain cases of TTP-HUS by decreasing the number of activity of B-cells which may result in decreased production of the ADAMTS13 protease inhibitor. Patients with TTP-HUS not responding to standard therapy or patients with relapsed disease may have particular benefit. Treatments that decrease the frequency of relapse or shorten the time to remission of TTP-HUS will be of benefit by decreasing the need for blood product support.

Thrombotic Thrombocytopenic Purpura (TTP) is a potentially life-threatening thrombotic microangiopathy caused by a severe deficiency of ADAMTS13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif member 13). Decreased ADAMTS13 activity leads to an accumulation of ultralarge von Willebrand factor (vWF) multimers which induce aggregation of platelets and microthrombi. These microthrombi may involve the brain, heart, kidneys and lead to life-threatening organ failures. In experimental models, magnesium sulfate increases cleavage of newly released vWF by ADAMTS13, decreases the endothelial secretion of ultralarge vWF and inhibits the interaction of vWF with platelets. In another thrombotic microangiopathy, magnesium sulfate has been shown to reduce the risk of seizures in women with severe pre-eclampsia. In analogy with its evidence-based therapeutic application in pre-eclampsia and based on a strong rationale for magnesium supplementation in TTP, we propose a phase 3, double blind, placebo controlled, and randomized study to evaluate the efficacy of magnesium sulfate in more rapidly restoring normal platelet counts as measure of prevention of further microvascular thrombosis in patients with Thrombotic Thrombocytopenic Purpura.

This is a multicenter, randomized, double-blind, parallel, placebo-controlled phase II clinical study. It is planned to recruit 74 patients with acquired thrombotic thrombocytopenic purpura (TTP). To evaluate the efficacy and safety of Anfibatide as an adjuvant therapy for plasma exchange in patients with acquired TTP.

The aim of the study is to evaluate the efficacy of a personalized caplacizumab regimen based on ADAMTS13 activity monitoring in adult acquired thrombotic thrombocytopenic purpura (aTTP): This study is a phase II, prospective, multicenter non-inferiority single-arm study.

The aim of this study is to determine the frequency of cardiac and cerebral involvements in patients with idiopathic thrombotic microangiopathies on diagnosis. Patients will be assessed for cardiac involvement (troponin Ic level and cardiac ultrasonography) and cerebral involvement (cerebral MRI). The investigators will assess whether serum troponin Ic on diagnosis can predict morbidity and mortality of patients with a thrombotic microangiopathy at the acute phase. The primary outcome measurement is the event free survival at day 30, as defined by death, myocardial ischemia, arrhythmia, severe cerebral injury and disease exacerbation. An increase in troponin Ic on diagnosis is defined as at least one result above 0.2 ng/ml among the three daily analyses performed after TMA diagnosis.

This observational study enrolls patients who have received at least one infusion of octaplasLG®. OctaplasLG® will be administered standard of care and observation occurs during the treatment and 24 hours after the end of treatment. Characteristics of the use of octaplasLG®, tolerance criteria, and efficacy criteria will be collected.

Thrombotic thrombocytopenic purpura (TTP) is a thrombotic microangiopathy defined by the spontaneous formation of platelet thrombi in the microvessels. These platelet microthrombi are responsible for a mechanical hemolytic anemia, a thrombocytopenia and a multivisceral ischemia. TTP is a rare but life-threatening disease in the absence of appropriate treatment (PLASMATHERAPY). The onset of the disease usually occurs in adulthood (MOSCHCOVITZ syndrome) and rarely in childhood (UPSHAW-SCHULMAN syndrome). TTP is either sporadic or recurrent with multiple unpredictable relapses. TTP pathophysiology has remained obscure until a new metalloprotease, ADAMTS13, has been demonstrated to be involved in about 90% of all cases. Physiologically, ADAMTS13 function consists in limiting the size of von Willebrand factor (VWF) multimers and consequently, their hemostatic capacity. A large majority of TTP is associated with a severe deficiency of ADAMTS13. In most cases, ADAMTS13 severe deficiency is acquired via auto-antibodies to ADAMTS13; more rarely, ADAMTS13 deficiency is hereditary via ADAMTS13 gene mutations. ADAMTS13 auto-antibodies are either inhibitory of the catalytic activity or non inhibitory. ADAMTS13 mutations are spread all over the gene. TTP prognosis is quite heterogeneous. Indeed, in about one third of the patients, TTP is refractory to PLASMATHERAPY and/or chronic relapsing. Until now, TTP prognosis factors are not known. Their identification is however crucial both to adapt the curative treatment of an acute episode (addition of first intention immunosuppressive agents to PLASMATHERAPY) and to prevent relapses. In this context, the aim of the current project is to identify some ADAMTS13 related prognosis factors in TTP. A national prospective multicenter study including both adult and pediatric patients with TTP related to a severe ADAMTS13 deficiency will be designed over a three-year period. This study will involve our group as the French reference center for ADAMTS13 and 10 clinical departments from various French hospitals. Patients will be tested for ADAMTS13 activity and antigen, ADAMTS13 antibodies and ADAMTS13 gene sequencing. Our main hypothesis is that the inactivation of the ADAMTS13 domains crucial for its catalytic activity, either by inhibitory auto-antibodies (acquired TTP) or by genetic mutations (hereditary TTP) is a major bad prognosis factor.

Congenital thrombotic thrombocytopenic purpura (TTP), also called Upshaw-Schulman Syndrome or hereditary or familial TTP is a rare, but severe disease. The purpose of this study is to determine how infusions of plasma to patients with congenital TTP correlate with symptoms and signs of activity of the disease, and to determine why some patients need more frequent infusions of plasma than others to prevent acute attacks of the disease.

This registry will collect clinical data and store biosamples (seru, plasma, urine, and DNA) annually from pediatric patients with thrombotic mcroangiopathy