A Study of Rilzabrutinib in Adult Patients With Immune Thrombocytopenia (ITP)
Immune ThrombocytopeniaImmune Thrombocytopenic PurpuraThis is a 2 part (Part A and B) adaptive, open-label, dose-finding study of PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options, with a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. The dose-finding portion of the study has been completed. Part B treatment dose is 400 mg twice daily.
Evaluation of Avatrombopag for the Treatment of Thrombocytopenia in Japanese Adults With Chronic...
Immune ThrombocytopeniaEvaluate the efficacy, safety, and PK of avatrombopag given for 26 weeks in Japanese adults with chronic immune thrombocytopenia (ITP).
Phase III Study on HMPL-523 for Treatment of ITP
Primary Immune Thrombocytopenia (ITP)The purpose of this study is to determine whether HMPL-523 (sovleplenib) is safe and effective in the treatment of chronic Immune Thrombocytopenic Purpura (ITP).
Safety and Treatment Satisfaction in Adults With Chronic ITP After Switching to Avatrombopag From...
Immune ThrombocytopeniaEvaluate the safety and tolerability of avatrombopag given for 90 days after stopping treatment with eltrombopag or romiplostim.
Post IVIG Medication in Children With Immune Thrombocytopenia
Immune ThrombocytopeniaThis study is a single hospital system, single-arm year-long pilot to evaluate the feasibility of enrolling children with ITP who are receiving IVIG for treatment of disease to a scheduled post-infusion medication for 72 hours following IVIG infusion. This year-long feasibility pilot will test the (1) feasibility of enrollment and the willingness of families to participate in a scheduled medication regimen and (2) adherence of patients and families to the scheduled medication regimen. Clinical outcomes, as defined by rates of headache or nausea/vomiting or other adverse event following IVIG, return to medical care, and need for further laboratory or imaging studies, will be collected. These rates will be compared to retrospective, historical data from Texas Children's Hematology Center from 2010 to 2019. However, due to the rate at which these events occur following IVIG, this feasibility pilot is not fully powered to detect differences in clinical outcomes.
Study of Human Umbilical Cord-derived Mesenchymal Stem Cells for Treatment of Refractory Immune...
ThrombocytopeniaMesenchymal Stem CellsPrimary Objective: To evaluate the safety and efficacy of human umbilical cord-derived mesenchymal stem cells(hUC-MSCs) to treat refractory immune thrombocytopenia(ITP). Secondary Objective: To observe the changes of immune function in refractory ITP patients with human umbilical cord-derived mesenchymal stem cells(hUC-MSCs) after infusion, and to explore and reveal the mechanism of hUC-MSCs in treating ITP.
Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological...
Immune Thrombocytopenia (ITP)Cold Agglutinin Disease (CAD)The main purpose of this study is to evaluate the efficacy and safety of iptacopan in participants with autoimmune benign hematological disorders such as primary immune thrombocytopenia and primary cold agglutinin disease.
A Long-term Study to Assess the Safety and Efficacy of Efgartigimod in Adult Patients With Primary...
Primary Immune ThrombocytopeniaThis is an open-label long-term multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in adult patients with primary ITP.
A Study to Evaluate the Efficacy and Safety of Efgartigimod PH20 Subcutaneous in Adult Patients...
Primary Immune ThrombocytopeniaThis is a phase 3, multicenter, randomized, double-blinded, placebo-controlled, parallel-group trial to evaluate the efficacy, safety, and effect on QoL/PRO of efgartigimod PH20 SC treatment in adult patients with primary ITP.
ASPirin in Immune thRombocytopenia Patients With Cardiovascular disEase
PurpuraThrombocytopenicThe incidence of immune thrombocytopenia increases with older age. This population is at risk for arterial thrombosis. Due to an increased turn-over of platelets, low-dose aspirin once daily may be insufficient in this population to protect against arterial thrombosis. This study is aimed at assessing the pharmacodynamics of aspirin once daily on platelet function in these patients.