A Prospective, One-arm and Open Clinical Study of CM313 in the Treatment of Immune Thrombocytopenia...
Immune ThrombocytopeniaTreatmentTo evaluate the safety and efficacy of CM313 in the treatment of immune thrombocytopenia in patients who have not responded adequately or relapsed after first-line treatment and at least one second-line therapy including rituximab and/or TPO-RA.
Two Regimens of IVIG in the Treatment of Newly Diagnosed ITP in Children
Newly Diagnosed Immune Thrombocytopenia in ChildrenFirst Line TreatmentTo compare the efficacy of two different dosage regimens of intravenous immune globulin (IVIG) in the treatment of children with newly diagnosed immune thrombocytopenia, and to reduce related adverse reactions and economic burdens on the premise of ensuring the remission rate
Anti-CD38 Antibody Treating APS With Thrombocytopenia
Antiphospholipid SyndromeThrombocytopeniaTo evaluate the safety and efficacy of anti-CD38 antibody in the treatment of antiphospholipid syndrome with secondary thrombocytopenia in patients who have not responded adequately or relapsed after first-line treatment and at least one second-line therapy including rituximab and/or TPO-RA.
Baricitinib for Steroid-resistant/Relapse Immune Thrombocytopenia
ITPImmune ThrombocytopeniaSingle-arm, open-label, single-center study to evaluate the efficacy and safety of baricitinib for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP).
Avatrombopag for Chemotherapy-induced Thrombocytopenia
Chemotherapy-induced ThrombocytopeniaAvatrombopagTo evaluate the efficacy and safety of Avatrombopag to treat chemotherapy-induced thrombocytopenia in solid tumors
Efficacy and Safety of Subcutaneous Belimumab or Placebo in Addition of Rituximab in Persistent...
Primary Immune Thrombocytopenia (ITP)Primary immune thrombocytopenia (ITP) is an autoimmune disease mainly mediated by autoreactive B cells and the presence of pathogenic anti-platelet auto-antibodies that enhance platelet destruction and impair platelet production. There are approximately 4,000 newly diagnosed ITP cases each year in France. For patients with a platelet count of less than 30x109/L and/or bleeding symptoms, corticosteroids alone or in combination with intravenous immunoglobulin (IVIg) is the standard first-line treatment. However, approximately two-thirds of adult patients responding to this first-line treatment relapse within days or weeks after corticosteroids withdrawal and overall, the course of the disease is chronic in about 70% of the cases. The anti-CD20 monoclonal antibody rituximab is commonly used off-label as a second-line therapy in many European countries including France for adults with persistent (i.e., disease duration of more than 3 months) or chronic (disease duration of more than 12 months) ITP. Rituximab leads to an overall response rate of only 40 % at 1 year but 29.5% of lasting (5 years and more) response The investigators have shown that the absence of response to rituximab in ITP could be explained by the settlement and expansion of long-lived autoreactive plasma cells in the spleen made possible by the high amount of BAFF. Belimumab is a fully humanized anti-BAFF/Blys monoclonal Ab licensed for SLE. Based on the preliminary results of a phase 2 open prospective pilot study performed in our center combining rituximab with i.v belimumab seems highly promising We hypothesized that combining subcutaneous belimumab weekly over a 24 weeks period (Arm A) with rituximab is superior to rituximab and subcutaneous placebo weekly over 24 weeks period (Arm B) to achieve an overall response at W52. The study design will be a prospective randomized, double-blind, multicenter (international), superiority phase III clinical study
The Combination of Sitagliptin and Danazol as the Treatment of Steroid-resistant/Relapse Immune...
Immune ThrombocytopeniaBlood Coagulation Disorders2 moreRandomized, open-label, multicenter study to compare the efficacy and safety of combination of Sitagliptin and danazol versus danazol for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP).
The SOLID Platelet Study
Platelet Transfusion Refractoriness (PTR)ThrombocytopeniaBackground: Platelets are cell fragments in the blood that help it clot. Some people get very low platelet counts during a disease or treatment. Low platelet counts can cause severe bleeding. Some people are not helped by platelet transfusions at the standard transfusion rate. This is called platelet transfusion refractoriness (PTR). Researchers want to learn more about transfusing platelets so they can make transfusions more effective. Objectives: To study the effects of transfusing platelets more slowly than the standard rate. To obtain data to improve the effectiveness of platelet transfusions in people with PTR and decrease the risk of bleeding in some people. Eligibility: Adults ages 18-100 who have very low platelet counts requiring platelet transfusion, and have evidence of PTR Design: Participants will be screened with a review their recent NIH medical records. They will have blood drawn. Participants will have up to three 12-hour treatment blocks. They can have only one block per day. During each block, they will have 2 platelet transfusions in those 12 hours. One transfusion will take place over 1 hour (SHORT infusion). The other will take place over 4 hours (LONG infusion). Participants will be randomly put in 1 of 2 treatment groups. This will dictate whether they get the SHORT or LONG infusion first. Participants will have blood drawn: When they enroll Right before each transfusion 2, 4, and 6 hours after each transfusion Each blood draw will consist of a complete blood count. Smaller tubes that require only small amounts of blood will be used to minimize the amount of blood drawn.
Eltrombopag vs Standard Front Line Management for Newly Diagnosed Immune Thrombocytopenia (ITP)...
Immune ThrombocytopeniaThis is an investigator initiated, multicenter, open label, randomized phase 3 study for subjects with newly diagnosed ITP from ages 1 to less than 18 years old.
Hetrombopag for Pediatric Patients With Chronic Immune Thrombocytopenia
Immune ThrombocytopeniaThe purpose of this study is to investigate the efficacy, safety of Hetrombopag in children with previously treated chronic immune thrombocytopenia who are between 6 and 17 years of age. This is a 2 part study. In part A, patients will receive Hetrombopag for 8 weeks. In part B, all patients will receive Hetrombopag for 24 weeks.