ADVANCE NEXT global phase 3 confirmatory study is open for certain people living with ITP to join. The study, or clinical trial, is looking at a drug called efgartigimod IV for treating primary immune thrombocytopenia (ITP). The study lasts about 2.5 years and includes a screening period, a treatment period, and a follow-up period. During the first 24 weeks, participants will get either the new drug or a placebo. After that, everyone can get the new drug. The study is looking for 69 adults with primary ITP. You may join if you were diagnosed at least a year ago and have tried other treatments that may no longer be working well for you. There is no cost to join, and you may get travel expenses covered. For more details, please contact your hematologist, visit clinicaltrials.gov and search for NCT06544499 or email argenx Patient Advocacy Lead at sgerson@argenx.com.
USA and International
https://clinicaltrials.gov/study/NCT06544499?term=NCT06544499&rank=1
Actively Recruiting
Massachusetts General Hospital in Boston is conducting a longitudinal observational study to evaluate for the prevalence and extent of cognitive dysfunction in adults with persistent and chronic ITP. Following enrollment, subjects will complete neurocognitive testing and will complete surveys for patient-reported outcomes of quality of life, fatigue, self-reported cognitive function, and depression. Background characteristics and relevant clinical history will be obtained through an enrollment survey and chart review. The primary aim of the study is to describe the prevalence and extent of cognitive impairment in ITP and explore associated risk factors. This study will also assess the feasibility of longitudinal cognitive testing in patients with ITP and describe any observed changes in cognition over time.
This study is open to individuals with confirmed persistent or chronic ITP who are over the age of 18 years, defined as having a platelet count less than 50 x 109/L on two occasions in the preceding 3 to 12 months or greater with a documented response to at least 1 prior ITP-directed therapy. Participants must also be able to follow instructions in English.
USA
Actively Recruiting
Sanofi Is sponsoring a multi-center, hospital based, cross-sectional study based on data from patient medical records, including laboratory results. The study will include adult patients with chronic (> 1 year duration) primary immune thrombocytopenia (ITP) only. Patients will be identified based on a laboratory confirmed diagnosis of ITP from medical records, where there is also a physician-confirmed diagnosis of primary ITP. If patients consent to take part in the study, a routine clinical visit will also serve as the study visit. All study participants will have a routine blood test as part of their standard clinical care at the study visit, and this same procedure will be used to collect supplementary blood samples to assess a variety of biomarkers.
Participants will be excluding from participating if they have secondary ITP, or an inherited platelet disorder. ITP patients in remission for one year will also be excluded, in addition to those who have received a recent vaccination or recently received a new ITP treatment called rilzabrutinib before the index date of the study.
USA and UK
Actively Recruiting
Chugai Pharmaceutical is conducting a phase one interventional ‘basket’ study to investigate the safety, tolerability, and efficacy of RAY121, an inhibitor of the classical complement pathway in the immune system. Participants are eligible for this study if they have at least one of the following autoimmune conditions: antiphospholipid syndrome (APS), bullous pemphigoid (BP), Behçet's Syndrome (BS), dermatomyositis (DM), immune-mediated necrotizing myopathy (IMNM), or immune thrombocytopenia (ITP).
Participants must be between the ages of 18-75 years. For those with ITP, having a platelet count less than 30,000 is another requirement for study participation.
USA, Australia, Austria, Bulgaria, Canada, Croatia, Czechia, France, Germany, Hungary, Italy, Japan, Netherlands, Norway, Poland, Portugal, Romania, Spain, Taiwan, and Turkey
Actively Recruiting
Pfizer is conducting a phase 2 multicenter study to look at the safety and efficacy of using a drug called PF-06835375 for treatment of adults (between 18-70 years) with persistent or chronic primary ITP. This drug is administered either intravenously or subcutaneously, however in this trial subcutaneous injection would be the method of drug delivery. The drug acts by targeting a C-X-C chemokine receptor type 5 (CXCR5) inhibitor. Eligible participants have on-going ITP with a platelet count less than 50,000.
Canada, USA, Australia, Czechia, Hungary, and United Kingdom
https://classic.clinicaltrials.gov/ct2/show/NCT05070845
Actively Recruiting
Alpine Immune Sciences is conducting a Phase 1b Open-Label Study to Assess the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of a new treatment for immune thrombocytopenia (and other autoimmune cytopenias) called Povetacicept (also known as APLN-303). This study is open to adults with persistent or chronic ITP. Povetacicept is a is a dual B cell cytokine antagonist engineered to inhibit BAFF (BLyS) and APRIL which are clinically validated cytokine pathways that play a key role in B cell development, differentiation, and survival. The drug will be provided to participants every 4 weeks for 6 months, with the possibility of participating in a 6-month study treatment extension period. Adult participants must have shown failure to at least two previous treatments for ITP, including exposure to a TPO-RA, unless otherwise contraindicated or unavailable to them. Participants must also have a documented platelet count of less than 30,000 µL.
USA, Australia, Austria, Canada, Germany, Italy, Norway, Spain, Turkey, and the United Kingdom
https://www.clinicaltrials.gov/study/NCT05757570?term=%20Povetacicept%20&rank=2
Actively Recruiting
Novartis Pharmaceuticals is conducting a Phase 3 study involving multiple centers designed to assess the efficacy and safety of using two different doses of ianalumab, a monoclonal antibody. Eligibility is restricted to adults with primary ITP who are over the age of 18 years, and who had a platelet count less than 30,000 µL before starting first line therapy options and meet criteria for treatment.
After completion of the screening period to ensure eligibility, participants will be given (randomly) either ianalumab or placebo as a healthy control to compare responses. All participants will be monitored for efficacy and/or safety.
Participants with current serious bleeding will be excluded, in addition to those who have had a previous splenectomy or ITP treatment within 28 days prior to the start of the study post-screening. Participants will also be excluded if they have used rituximab in past and have an absolute neutrophil count below 1.0 G/L at randomization. Anyone with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication (with an exemption of low dose of acetylsalicylic acid) will also be excluded from participating in this study.
USA, Argentina, Australia, Austria, Belgium, Bulgaria, China, Czechia, France, Germany, Hong Kong, Hungary, India, Italy, Japan, Malaysia, Mexico, Norway, Romania, Singapore, Spain, Thailand, Turkey, United Kingdom, and Vietnam
https://www.clinicaltrials.gov/ct2/show/NCT05653349?recrs=a&cond=immune+thrombocytopenia&draw=2&rank=15
Actively recruiting
This global ITP registry study is FDA approved and developed in partnership with the National Organization for Rare Disorders (NORD) to follow ITP patients of all ages, in all phases of disease to learn more about the natural history of ITP. The goal of the registry is to learn more about the clinical characteristics of ITP, the incidence of co-morbidities, treatment patterns, adverse outcomes, how quality of life is impacted, how family and socioeconomics is affected, and how ITP patients respond to COVID-19 and vaccinations. Note: The registry is in English only, currently.
USA, Canada, International (all places).
https://www.pdsa.org/registry.html
Recruiting
This study is conducted by the New York Medical College and is open for newly diagnosed pediatric ITP patients between 1 year – 21 years of age who have bleeding symptoms and show no evidence of a recent infection. Platelet counts less than 20, 000 µL are required. A bone marrow biopsy is preferred but not required. The aim of the study is to determine the effect of early administration of Rituximab on relapse rates following treatment. To determine impact on quality of life, the kids ITP tools (KIT) will be used according to protocol.
USA
Recruiting
This is a prospective, open-label, single-arm, multicenter, Phase 4 study that is evaluating the efficacy and safety of PANZYGA, an IVIg product designed to be used as an antibody replacement therapy in ITP chronic ITP patients between the ages of 1 year – 18 years of age in participants who have a platelet count less than 30K microliters.
USA
https://www.clinicaltrials.gov/ct2/show/NCT03866798?cond=Immune+Thrombocytopenia&draw=2&rank=33
Active, no longer recruiting
This Is a cross sectional multi-site tissue repository for approved investigator-directed biomedical research. Using blood samples and other specimens, researchers from Sanguine Biosciences hope to bridge the gap between researchers and a cure to various diseases, including immune thrombocytopenia. Using DNA collected from various samples, between health and unhealthy controls, the study aims to gain a greater understanding of the etiology of common diseases affecting adults living in the USA.
USA
Recruiting