Active clinical trials for "Purpura, Thrombotic Thrombocytopenic"

This is a single group, treatment, Phase 3, open-label, single-arm study to evaluate the efficacy and safety of caplacizumab and immunosuppressive therapy (IST) without firstline therapeutic plasma exchange (TPE) with primary endpoint of remission in male and female participants aged 18 to 80 years with immune-mediated thrombotic thrombocytopenic purpura (iTTP). The anticipated study duration per participant without a recurrence while on therapy is maximum 24 weeks (ie, approximately 1 day for screening + maximum 12 weeks of treatment for the presenting episode + 12 weeks of follow-up). Participants will have daily assessments during hospitalization and weekly visits for assessments during ongoing treatment with caplacizumab and IST. There will be 3 outpatient visits for assessments during the follow-up period. There will be two additional follow-up visits for participants who do not have ADAMTS13 activity levels of ≥50% at the time of caplacizumab discontinuation.

This is a study of TAK-755 in adults with immune-mediated thrombotic thrombocytopenic purpura (iTTP). The main aim of this study is to determine the percentage of participants with a clinical response without plasma exchange during the study. Participants who have an acute attack of iTTP will receive TAK-755 and immunosuppressive therapy during their stay at the hospital until they achieve a clinical response. Participants will also be treated with TAK-755 for an additional time of up to 6 weeks after the acute phase. In total, participants will stay in the study for approximately 3 months.

Thrombotic thrombocytopenic purpura (or TTP for short) is a condition where blood clots form in small blood vessels throughout the body. The clots can limit or block the flow of oxygen-rich blood to the body's organs, such as the brain, kidneys, and heart. As a result, serious health problems can develop. The increased clotting that occurs in TTP uses up the cells that help the blood to clot, called platelets. With fewer platelets available in the blood, bleeding problems can also occur. People who have TTP may bleed underneath the skin forming purple bruises, or purpura. TTP also can cause anemia, a condition in which red blood cells break apart faster than the body can replace them, leading to fewer red blood cells than in normal. TTP is caused by a lack of activity in the ADAMTS13 enzyme, a protein in the blood involved in controlling clotting of the blood. The ADAMTS13 enzyme breaks up another blood protein called von Willebrand factor that forms blood clots by clumping together with platelets. Some people are born with this condition, while others develop the condition during their life. Many people who are born with TTP experience frequent flare-ups that need to be treated right away. TAK-755 is a medicine that replaces ADAMTS13 and may prevent or control TTP flare-ups, called acute TTP events. The main aim of the study is to check for side effects of long-term treatment with TAK-755. Treatment will be given in 2 ways: TAK-755 treatment given either every week or every other week to prevent acute TTP events from happening (the "prophylactic" cohort). TAK-755 treatment given to control an acute TTP event when it happens (the "on-demand" cohort). Participants in the prophylactic cohort will receive treatment in the clinic or at home for up to approximately 3 years. They will visit the clinic at least every 12 weeks. Participants in the on-demand cohort will receive daily treatment for the acute TTP event until the flare-up has gotten better. They will have a follow-up visit at the clinic 4 weeks later.

Thrombotic thrombocytopenic purpura (or TTP for short) is a condition where blood clots form in small blood vessels throughout the body. The clots can limit or block the flow of oxygen-rich blood to the body's organs, such as the brain, kidneys, and heart. As a result, serious health problems can develop. The increased clotting that occurs in TTP uses up the cells that help the blood to clot, called platelets. With fewer platelets available in the blood, bleeding problems can occur. People who have TTP may bleed underneath the skin forming purple bruises or purpura, or from the surface of the skin. TTP also can cause anemia, a condition in which red blood cells break apart faster than the body can replace them leading to lower than normal number of red blood cells. A lack of activity in the ADAMTS13 enzyme, a protein in the blood involved in blood clotting, causes TTP. The enzyme breaks up another blood protein called von Willebrand factor that clumps together with platelets to form blood clots. Some people are born with this condition, others get the condition during their life. Many people who born with TTP experience frequent flareups that need to be treated right away. If not treated It can be fatal or cause lasting damage, such as brain damage or a stroke. BAX 930 is a medicine that replaces ADAMTS13 and can prevent or control TTP flareups, called TTP events. The main aim of this study is to compare the number of TTP events in people born with severe TTP when they treated with BAX 930 versus when they are treated with the standard treatment. Treatment will be given in 2 ways: BAX 930 or standard treatment given to prevent TTP events from happening. BAX 930 or standard treatment given to control an acute TTP event when it happens, according to the clinic's standard practice. Both BAX 930 and standard treatment are given slowly through a vein (infusion). At the first visit, the study doctor will check if you can participate in the study. If you are eligible and enter the study, you will follow an assigned schedule and either start with BAX 930 (Period 1) and then switch to standard treatment (Period 2) or start with standard treatment (Period 1) and then switch to BAX 930 (Period 2). Everyone will be treated with BAX 930 again for Period 3. Each Period will last approximately 6 months. If you enter the study to control an acute TTP event, you will follow a schedule receiving either BAX 930 or standard care to treat your acute TTP event. Once the acute TTP event has gotten better, you can decide to continue in the study and be given treatment to prevent TTP events from happening, following the schedule above. Another study's aim is to assess side effects from treatment with BAX 930 and standard treatment. To do that, the study doctor will ask you questions about your health at each study visit. The study doctors will also check how long BAX 930 stays in the blood of the participants, over time. They will do this from blood samples taken after participants receive their specific infusions of BAX 930. This will happen at different times during the study. 1 month after all treatment has been completed, participants will visit the clinic for a final check-up.

To evaluate the efficacy and safety of bortezomib in the first-line treatment of patients with acquired TTP，we design this prospective, multi-center, single-arm interventional study.All enrolled TTP patients were given bortezomib 1.3 mg/m2 intravenous injection d1, 4, 8, on the basis of standard single membrane plasma exchange (2L/d) and hormone therapy (1mg/kg prednisone or equivalent methylprednisolone). 11 (4 doses in total). Bortezomib should be administered immediately after each plasma exchange, and the interval between the next plasma exchange is> 24h. Plasma exchange continued until the patient's platelet count was >100×109/L for 2 consecutive days, and then changed to once every other day for a total of two times and then stopped.

Hereditary thrombotic thrombocytopenic purpura (Upshaw-Schulman syndrome) is a rare disorder characterized by thrombocytopenia as a result of platelet consumption, microangiopathic hemolytic anemia, occlusion of the microvasculature with von Willebrand factor-platelet-thrombic and ischemic end organ damage. The underlying patho-mechanism is a severe congenital ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin type 1 motif, 13) deficiency which is the result of compound heterozygous or homozygous ADAMTS13 gene mutations. Although considered a monogenic disorder the clinical presentation in Upshaw-Schulman syndrome patients varies considerably without an apparent genotype-phenotype correlation. In 2006 we have initiated a registry for patients with Upshaw-Schulman syndrome and their family members to identify possible triggers of acute bouts of TTP, to document individual clinical courses and treatment requirements as well as possible side effects of long standing plasma substitution, e.g. alloantibody formation or viral infections.

The lack of ADAMTS13 is the only biological marker that is specific for aTTP diagnosis8 and the assessment of ADAMTS13 is of clinical importance because it is essential for the rapid differential diagnosis between aTTP and other TMA. Furthermore, monitoring of ADAMTS13 activity is useful to ensure biological remission (ADAMTS13 levels > 10%) as well as predicting relapses. Due to the high mortality rate of aTTP, treatment should be started as soon as the disease is suspected, sometimes even before confirmation with the ADAMTS13 test results. This situation may lead to misdiagnose some patients and leave them without the appropriate treatment. In conclusion, ADAMTS13 activity assay is crucial for an early diagnosis and optimal management of acute aTTP and any delay in ADAMTS13 results will have a negative impact on the diagnosis, treatment and prognosis of the patient. There are currently 2 techniques available for the ADAMTS13 activity determination, the fluorescence resonance energy transfer (FRET) and the Technozym chromogenic enzyme-linked immunosorbent assay (ELISA). Both are considered reference methods but they require considerable skill because they are highly manual and this increases the risk of error. Furthermore, these methods are time-consuming, not widely available and, in case of the ELISA method, it requires a new calibration at each run. The inter-laboratory variability is also a challenge and therefore a validation and/or interpretation method could be needed. Recently, a new and first fully automated HemosIL AcuStar ADAMTS13 Activity assay (Instrumentation Laboratory, Bedford, Massachusetts, United States) has been developed. HemosIL AcuStar ADAMTS13 Activity assay is a two steps chemiluminescent immunoassay (CLIA) with an analytical time of 33 minutes for the quantitative measurement of ADAMTS13 activity in human-citrated plasma on the ACL AcuStar analyser. The immunoassay uses the GST-VWF73 substrate in combination with magnetic particles for rapid separation and chemiluminescence technology detection. The ADAMTS13 present in the plasma sample cleavages the GST-VWF73 substrate and the detection of the generated fragments is based upon an isoluminol-labelled monoclonal antibody that specifically reacts with the cleaved peptide. The emitted light is proportional to the ADAMTS13 activity in the sample. This new ADAMTS13 assay method has been compared with the other two available techniques in two different studies. First, Favresse et al. published the results of the comparison between Technozym activity ELISA assay and the new HemosIL AcuStar chemiluminescent assay. On the other hand, Valsecchi et al. have recently published the results of validation of this new technique in comparison with ELISA and FRETS in 176 samples. Both studies conclude that the new chemiluminescent ADAMTS13 activity assay showed a good correlation and excellent clinical performance for the diagnosis of severe ADAMTS13 deficiency with the FRETS-VWF73 assay and a commercial ELISA when considering only ADAMTS13 activity values below 10% (the internationally accepted cut-off for a diagnosis of severe ADAMTS13 deficiency typical of aTTP). Finally, Stratmann et al. have just published another study comparing the HemosIL AcuStar chemiluminescent assay with two commercially available ADAMTS13 assay kits using 24 paired test samples derived from 10 consecutively recruited patients13 and their results corroborate the previously published data suggesting that the AcuStar assay could be a valuable and accurate tool for ADAMTS13 activity testing and aTTP diagnostic. In this context, a unique opportunity to validate this new technique is generated, both retrospectively with our already available data from frozen samples and also in the context of a large prospective study. This will be the first study worldwide testing HemosIL AcuStar method in real clinical practice aTTP population (Spanish and Portuguese aTTP populations) with the aim to standardize the diagnosis and follow-up methodology for the disease.

Thrombotic thrombocytopenic purpura (TTP) is a rare disease with a mortality rate of over 90% if left untreated [1]. TTP is a prototype of the thrombotic microangiopathies (TMAs), and it is characterized by disseminated formation of platelet-rich thrombi in arterioles and capillaries resulting in microangiopathic hemolytic anemia (MAHA), thrombocytopenia, and potential end-organ injury mainly involving the brain, heart, and kidneys leading to significant morbidity/mortality

The objective of this national, prospective, multi-centre observational study is to describe the prescription rational and practice in Germany, confirm the efficacy of caplacizumab in a real-world setting, and identify predicting factors in iTTP-patients with regard to persistent autoimmune activity, therapy guidance and risk of complications. The rational is to develop new treatment algorithms that optimize overall patient outcome and reduce treatment cost.

Thrombotic thrombocytopenic purpura (TTP) is a rare and life-threatening thrombotic microangiopathy characterized by thrombocytopenia, microangiopathic hemolytic anemia, and microvascular thrombosis causing neurological and renal abnormalities; it is associated with massive depletion of platelets in the microvasculature to form microthrombi1 . Long-term follow-up of patients with congenital TTP (cTTP) revealed frequent strokes and renal injury. Of 217 surviving patients, 62 (29%) had a stroke; the median age was 21 years. iTTP patients also require long-term follow-up. iTTP patients with low ADAMTS13 activity (<70%) in remission have a 28% risk of stroke. Survival rates of iTTP patients in remission were lower than those of age-, race-, and sex-matched populations. In terms of stable treatment, maintenance therapy is not recommended for patients with iTTP. Previous studies have shown that aspirin may be able to prevent stroke complications in patients with cTTP and iTTP. In addition to its potential efficacy, the risks of aspirin are small and inexpensive. Aspirin is very effective in secondary prevention of stroke 6. However, the therapeutic value of aspirin in TTP has not been studied previously. To improve the prognosis and survival of patients with cTTP and iTTP, we propose to conduct a prospective study to observe the efficacy and safety of aspirin in patients with cTTP and iTTP in remission.