Thrombotic Thrombocytopenic Purpura
Conditions
Sponsors
Saitama Medical University ,
Department of General Internal Medicine, Yoshitaka Miyakawa
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Tentative registration 1)Definitive or suspected case of acquired TTP according to the Provisional Diagnostic Guideline of TTP of Study Group for Coagulation Disorders (MHLW). -Definitive diagnosis: ADAMTS13 activity <5% and ADAMTS13 inhibitor positive, or presence of all of the 5 clinical symptoms(*) regardless of ADAMTS13 activity. Suspected diagnosis: presence of thrombocytopenia and microangiopathic hemolytic anemia regardless of ADAMTS13 activity. * thrombocytopenia, microangiopathic hemolytic anemia, renal insufficiency, fever, fluctuating levels of neuropsychiatric symptoms. 2) 20 to 79 years old at the time of informed consent. 3) Written informed consent from the subject or legally acceptable representative Registration 1) Patients with refractory or relapsed TTP who fulfill the criteria below. -refractory TTP: fulfill one of the following conditions i) Platelet count <50,000/microL after 5 procedures of plasma exchange. ii) ADAMTS13 inhibitor level of 2 bethesda unit (BU)/mL or more at the time of tentative registration. -relapsed TTP: relapse after at least 30 days from the previous episode.
Exclusion criteria
Exclusion criteria: Tentative registration 1) Secondary TTP associated with drug, hematopoietic stem cell transplantation, solid organ transplantation, collagen disease, malignancy, or pregnancy. 2) Congenital TTP (Upshaw-Shulman Syndrome) due to ADMTS 13 deficiency. 3) Presence of malignancy. 4) Severe hematological, neuropsychiatric, hepatic, pulmonary, endocrinological, immunological, or gastrointestinal conditions not related to TTP. 5) Previous exposure to rituximab. 6) Male patient who is not able to consent contraception during study period. 7) Female patient who is pregnant, lactating, or suspected of pregnancy. Female patient who wish to pregnant during study period. 8) Pariticipation in another registration clinical trial and administration of investigational drug during 12 weeks before informed consent. 9) Patients inadequate as the subject of the study judged by investigators. Registration 1) Known HIV seropositive status. 2) Positive status of HBs antigen, HBs antibody, HBc antibody, or HCV antibody except for HBV seropositivity due to vaccination.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Response rate at 4 weeks. Response is defined by fulfilling all the conditions below. 1) Normalization of platelet count 2) cessation of plasma exchange 3) Improvement of clinical symptoms | — |
Secondary
| Measure | Time frame |
|---|---|
| 1) Peripheral blood B-cell count (CD20 positive cell, CD19 positive cell), peripheral blood T-cell count (CD3 positive cell) 2) Changes of platelet count from base line. 3) Normalization of platelet count: the rate of patients who achieved plate count of >=150,000/microL. 4) Cessation of plasma exchange: the rate of patients who achieved plate count of >=150,000/microL for 2 consecutive days and cessation of plasma exchange. 5) Improvement of clinical symptoms: improvement of anemia (Normalization of hemoglobin level or increase of hemoglobin level by 2 g/dL or more), Neuropsychiatric symptoms (Improvement of sum of GCS from base line). | — |
Countries
Japan
Contacts
Public ContactKazuo Watanabe
CTD Inc.
Outcome results
None listed