Sirolimus for Autoimmune Disease of Blood Cells
Autoimmune PancytopeniaAutoimmune Lymphoproliferative Syndrome (ALPS)10 moreTreatment for patients with autoimmune destruction of blood cells is poor. The part of the body that fights infections is called the immune system and white blood cells (WBCs) are part of the immune system. Normally, a person's body creates WBCs to fight infections and eliminates WBCs which have stopped helping the body function. Patients with autoimmune destruction of blood cells have difficulty eliminating old WBCs. The abnormal WBCs build up and can damage other healthy cells, which can lead to anemia, fatigue, jaundice, internal bleeding, infection, and cancer. Few effective medications exist for treatment for patients with autoimmune cytopenias and those commonly used are fraught with side effects. Nevertheless, as scientific understanding of autoimmune diseases has improved, more directed and less toxic therapies are becoming available. A number of groups have been studying the efficacy of a medication called sirolimus in patients with autoimmune diseases. This medicine has been FDA-approved for over 20 years. Sirolimus is a medicine used in children with other diseases. Sirolimus works, in part, by eliminating old and abnormal WBCs. Our group and others have shown that sirolimus is effective in mice with autoimmunity and in children with a rare condition called Autoimmune Lymphoproliferative Syndrome (ALPS). We believe sirolimus will help children with autoimmune cytopenias. We believe it will improve their symptoms and make them less sick. We propose to study sirolimus in children with chronic and/or refractory autoimmune cytopenias.
Study of AKR-501 Tablets Taken Orally Once Daily for 28 Days in Patients With Chronic Idiopathic...
Chronic Idiopathic Thrombocytopenic PurpuraPurpura2 moreThe purpose of this study is to determine the efficacy, safety and tolerability, of AKR-501 (avatrombopag) tablets, as compared to placebo, in the treatment of participants with chronic Idiopathic Thrombocytopenic Purpura (ITP).
High Dose Dexamethasone Vs. Conventional Dose Prednisolone in Adult ITP
Idiopathic Thrombocytopenic PurpuraTo determine the long term effects of pulse high dose dexamethasone and conventional dose prednisolone in treatment of adult patients with ITP.
Rituximab and Prednisone as First-Line Therapy in Treating Patients With Immune Thrombocytopenic...
Nonneoplastic ConditionRATIONALE: Rituximab and prednisone may increase the number of platelets in patients with immune thrombocytopenic purpura. PURPOSE: This phase II trial is studying the side effects and how well giving rituximab together with prednisone works as first-line therapy in treating patients with immune thrombocytopenic purpura.
Clinical Evaluation of Eltrombopag in Chronic Idiopathic Thrombocytopenic Purpura (ITP)
Idiopathic Thrombocytopenic PurpuraPurpura2 moreAn open-label, dose-adjustment extension study to evaluate the safety and efficacy of eltrombopag for treatment of subjects with ITP who have previously been enrolled in the eltrombopag trial TRA108109 (NCT00540423).
Safety and Efficacy Study of PG2 to Treat Idiopathic Thrombocytopenic Purpura (ITP) Patients
Idiopathic Thrombocytopenic Purpura (ITP)This was a phase II multi-center, randomized, open-label study with two parallel study groups to evaluate the efficacy and safety of PG2 in ITP patients.
Alemtuzumab and Rituximab in the Treatment of Refractory Autoimmune Cytopenias
Idiopathic Thrombocytopenic PurpuraAnemia2 moreThe purpose of this study is to determine whether the combination of low doses of alemtuzumab and rituximab are effective in the treatment of patients with autoimmune cytopenias who has failed on steroids, relapsed after steroids withdrawal or required continuous steroids treatment.
Clinical Evaluation of SB-497115-GR in Chronic Idiopathic Thrombocytopenic Purpura (ITP)
Chronic Idiopathic Thrombocytopenic PurpuraPurpura2 moreThis is a Phase II/III multicenter study comprising of the double-blind, followed by open-label phases to evaluate and compare the efficacy and tolerability of eltrombopag (SB-497115-GR) in chronic ITP patients
P3 Study to Evaluate Efficacy and Safety of AMG 531 in Thrombocytopenic Japanese Subjects With Immune...
Idiopathic Thrombocytopenic PurpuraThe purpose of this study is to evaluate the efficacy and safety of AMG 531 compared with placebo in thrombocytopenic Japanese subjects with immune (idiopathic) thrombocytopenic purpura (ITP) .
Rituximab as Second Line Treatment for ITP
Immune Thrombocytopenia (ITP)Immune thrombocytopenic purpura (ITP) is an autoimmune disorder characterized thrombocytopenia. Splenectomy is the standard treatment for patients who fails the first-line treatment: corticosteroid. Rituximab, has recently emerged as a promising treatment for ITP. The aim of the study is to determine whether early treatment with Rituximab can result in durable remissions, and consequently, lead to the avoidance of splenectomy in a significant number of patients.