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Last Updated: 09/18/2022

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Phase 1/2 Randomized, Double-Blind, Placebo-Controlled Study of Safety and Efficacy of Abatacept for Treating Chronic Cytopenia in Cytotoxic T-lymphocyte Antigen 4 (CTLA4) Haploinsufficiency

Summary: Background:~CTLA4 stands for cytotoxic T-lymphocyte antigen-4. It is a protein the body makes naturally to check its immune system from attacking itself. Some people don t produce enough CTLA4 protein, causing problems due to overactive immune system such as big spleens, repeated lung infections, breathing problems, stomach and intestine symptoms as well as inflamed brain and nerve problems. Many ...

IMMUNE THROMBOCYTOPENIA (ITP) AND COVID-19: National Retrospective and Prospective Observational Investigation on the Incidence and Course of COVID-19 in Patients With Prior, Ongoing or de Novo ITP. Evaluation of the Impact of COVID-19 Pandemic on the Management of ITP

Summary: Multicenter retrospective and prospective observational study based on the collection of sequential anonymized data from principal Italian ITP centers, to describe the clinical course of ITP patients becoming infected by SARS-CoV-2 and of COVID-19 patients developing de novo ITP.

A Phase III, Multicenter, Single-Arm Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Adult and Adolescent Patients With Atypical Hemolytic Uremic Syndrome (aHUS)

Summary: This study aims to evaluate the efficacy and safety of crovalimab in adult and adolescent participants with aHUS.


Summary: This is a Phase 2, open-label, single arm, multicenter, multiple subcutaneous injection, safety and efficacy study of PF-06835375 in adult participants with primary immune thrombocytopenia (ITP). This study will focus on participants with persistent (>3 months and ≤12 months), or chronic (>12 months) ITP

Optimization of Glucocorticoid Taper Strategies for Maintenance Therapy of Systemic Lupus Erythematosus Associated Immune Thrombocytopenia (SLE-ITP)

Summary: SLE associated immune thrombocytopenia (SLE-ITP) is one of the main clinical manifestations of SLE. Approximately 70% of SLE patients follow a relapsing-remitting course. Similarly, SLE-ITP often relapses during GCs tapering. At the same time, patients with SLE-ITP may suffer from thrombocytopenia and damage to vital organs when they relapse, seriously affecting their lives. Therefore, maintenance...

A Randomized, Controlled Phase 3 Study of Pacritinib Versus Physician's Choice in Patients With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis With Severe Thrombocytopenia (Platelet Count <50,000/μL)(PACIFICA)

Summary: This study (study ID PAC203 North America; PAC303 ex-North America) is evaluating 200 mg BID of pacritinib compared to physician's choice (P/C) therapy in patients with MF and severe thrombocytopenia (platelet count <50,000/μL). Approximately 399 patients in total will be enrolled, randomized 2:1 to either pacritinib (approximately 266 patients) or to P/C therapy (approximately 133 patients)~Condi...

An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® (Avatrombopag) in Adult Patients With Immune Thrombocytopenia (ITP)

Summary: This is a multi-center, observational, Phase 4 study in patients with Immune Thrombocytopenia (ITP) designed to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base regarding the use of Doptelet in routine medical practice. Patients eligible for participation will, as part of their routine medical care, be receiving Doptelet for...

A Phase III, Multicenter, Single-Arm Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Pediatric Patients With Atypical Hemolytic Uremic Syndrome (aHUS)

Summary: This study aims to evaluate the efficacy and safety of crovalimab in pediatric participants with aHUS.

Intraoperative Application of Nimodipine to the Facial and Cochlear Nerves During Vestibular Schwannoma Resection to Avoid Spasm-related Postoperative Facial Paralysis and Deafness - a Prospective Randomized Pilot Study

Summary: Title:~Intraoperative application of nimodipine to the facial and cochlear nerves during vestibular schwannoma resection to avoid spasm-related postoperative facial paralysis and deafness - a prospective randomized study~Background:~In patients undergoing microsurgical resection of a vestibular schwannoma, the facial and vestibulocochlear nerves are at risk. Prior studies suggested positive effect...

Investigation of the Natural Progression of Clonal Hematopoiesis of Indeterminate Potential and Clonal Cytopenia of Undetermined Significance.

Summary: Background:~Clonal Hematopoiesis of Indeterminate Potential (CHIP) is a change in a person s DNA that can increase a person s risk of developing blood cancers or cardiovascular disease. CHIP occurs mostly occurs in older people. Clonal cytopenia of undetermined significance (CCUS) occurs when one or more blood cell types is lower than it should be and is associated with a change in their DNA. Rese...

Prospective, Multi-center, Open-label Study Measuring Safety and Treatment Satisfaction in Adult Subjects With Chronic Immune Thrombocytopenia (ITP) After Switching to Avatrombopag From Eltrombopag or Romiplostim

Summary: Evaluate the safety and tolerability of avatrombopag given for 90 days after stopping treatment with eltrombopag or romiplostim.

A Prospective, Natural History Study to Assess the Occurrence of HPA-1a Alloimmunization in Women Identified at Higher Risk for Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)

Summary: A prospective, non-interventional, natural history study to assess the occurrence of higher FNAIT risk across a broad population of different racial and ethnic characteristics and the occurrence of HPA-1a alloimmunization in these women.
Showing 1-12 of 206

Last Updated: 09/18/2022