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

Novartis Pharmaceuticals is conducting this open-label, multicenter, phase 2 basket study to assess the effectiveness and safety of Iptacopan (LNP023) in adults with persistent or chronic ITP, who are at least 18 years of age. Iptacopan is an orally administered therapy that targets the alternative complement pathway of the immune system. The drug will also be given to participants who have cold agglutinin disease, a different condition than ITP, to compare how well the drug does in both patient groups. Participants must be vaccinated against Neisseria meningitidis (meningitis) and Streptococcus pneumoniae (pneumonia) infections in addition to Haemophilus influenzae (the flu) infection. Participants will be excluded if they have secondary ITP or have used any therapies for their ITP apart from low-dose corticosteroids or a thrombopoetin receptor agonist (TPO-RA) at least four weeks before enrolling into the study.

United States, Germany, Italy, Republic of Korea, Spain, United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05086744?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=50

Status: Recruiting

Pfizer is conducting an interventional phase two, open-label, one-arm, multicenter study to determine the safety and efficacy of a drug, PF-06835375, in adults with moderate-severe bleeding due to ITP. Specifically, the study will determine if the drug can increase platelet counts to reduce bleeding risks, without a significant side effect profile. PF-06835375 is a chemokine (CXCR5) receptor antagonist that works as an immune system regulator and has been reported as a critical chemokine regulator in several autoimmune diseases. Its mechanism of action is not fully understood yet. The drug would be provided through a subcutaneous injection every month for a total of three months. Eligible participants include adults (between the ages of 18-70 years) with primary persistent or chronic ITP (over 3 months since diagnosis) who have a platelet count less than 50,000/μL and have not experienced severe bleeding within 1 month. Individuals who have had a splenectomy within three months of the study initiation period will be excluded from participating.

Australia, Canada, Czechia, Hungary, Poland, USA.

https://www.clinicaltrials.gov/ct2/show/NCT05070845?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=15

Status: 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

https://www.clinicaltrials.gov/ct2/show/NCT04323748?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=40

Recruiting

Swedish Orphan Biovitrum AB International (SOBI) pharmaceuticals is conducting a phase 4, prospective, multi-center, open-label study to evaluate the safety, platelet count response, and subject reported satisfaction of treatment in adult subjects (18 years of age or older) with chronic ITP after switching to avatrombopag from eltrombopag or romiplostim. This study is for participants who have been undergoing treatment for primary ITP with eltrombopag or romiplostim for at least 90 days. Participants must have had a previous response (at any time) to either eltrombopag or romiplostim, defined as at least 2 platelet counts above 50,000uL. Patients will be treated with avatrombopag for up to 90 days.

Recruiting

USA

https://clinicaltrials.gov/ct2/show/NCT04638829

Swedish Orphan Biovitrum AB International (SOBI) pharmaceuticals is conducting a randomized, double-blind, placebo-controlled study with an open-label extension phase to evaluate the efficacy and safety of avatrombopag for the treatment of thrombocytopenia in pediatric subjects. The study is for participants between 1-17 years of age with a diagnosis of primary ITP for at least 6 months duration who have had an insufficient response to a previous treatment. Participants must have an average of two platelet counts less than 30×109/L with no single count greater than 35×109/L during the screening period. Avatrombopag is a thrombopoietin receptor agonist currently available for use in adults only with ITP.

France, Germany, Hungary, Poland, Russia, Turkey Ukraine, UK, and USA

https://clinicaltrials.gov/ct2/show/NCT04516967

Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Greece, Ireland, Isreal, Italy, Japan, Jordan, Republic of Korea, Mexico, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Serbia, South Africa, Spain, Taiwan, Thailand, Tunisia, Turkey, and USA.

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Isreal, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

Takeda pharmaceuticals is conducting a multi-centered phase two placebo-controlled double-blinded study to look at the safety, tolerability, and efficacy of a drug called TAK-079, which is a monoclonal antibody (anti-CD38 antibody). The trial is for adult ITP patients whose ITP is persistent or chronic, with a platelet count less than 30,000/µL for four consecutive weeks prior to participating in the trial. TAK-079 would be administered as a subcutaneous injection.

Bulgaria, Croatia, Germany, Greece, Italy, Slovenia, Spain, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04278924?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=11

Status: Recruiting

Baylor College of Medicine is conducting a prospective, open label, randomized, two-arm, multi-center Phase 3 trial using eltrombopag, a thrombopoietin receptor agonist, in newly diagnosed children, between the ages of 1-18 years. Participants must have a platelet count below 30 X 109/L. Children will be randomized (2:1) into one of two groups, either the treatment group (where they will receive eltrombopag), or the standard group (where they will receive standard first line therapies). The goal of the study is to see which group elicits a greater overall platelet response.

USA

https://www.clinicaltrials.gov/ct2/show/NCT03939637?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=1

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

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

https://www.clinicaltrials.gov/ct2/show/NCT01931644?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=57

Recruiting

Through Baylor College of Medicine, this prospective, open-label, randomized two-arm multi-center study focuses on pediatric patients between the ages of 1 years of age – less than 18 years of age, who are newly diagnosed with ITP. Participants will be randomized into groups to receive either eltrombopag or one of three typical first-line ITP therapies. For those in the eltrombopag group, participants can expect to receive this drug for twelve weeks with the option to continue up to one year, depending on their response to the drug. The goal of this study is to determine if platelet counts in pediatric patients with ITP respond better to eltrombopag versus standard first-line therapies.

USA

https://www.clinicaltrials.gov/ct2/show/NCT03939637?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=25

Recruiting

Boston Children’s Hospital is conducting an observational cross-sectional study in hopes to replicate larger scale findings from smaller studies that suggest a positive correlation between platelet parameters (such as immature platelet function, absolute immature platelet count, and P-selection and glycoprotein 11b-11a) with bleeding severity in severe immune thrombocytopenia, regardless of platelet counts. This study center hopes to confirm previous findings in a multi-center patient population to investigate the use of these parameters and better predict disease severity and bleeding events.

USA

https://www.clinicaltrials.gov/ct2/show/NCT03810352?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=3

Recruiting

This is a phase two double-blind, randomized, multicenter study through Oscotec Inc. designed to evaluate the efficacy and safety of SKI-0-703, an oral SYK inhibitor. This study is targeting adult ITP patients who have persistent or chronic ITP and have failed to respond adequately to other approved therapies, or who have relapsed following exposure to other approved ITP treatments. SYK inhibitors play important roles in signalling within the immune system, particularly in the adaptive immune system. Inhibition of SYK leads to reduced antibody mediated destruction of platelets. Eligibility includes individuals diagnosed with ITP for at least three months who have a platelet count less than 30,000 µL and are at least 18 years of age.

USA, Greece, Republic of Korea, Poland, Spain

https://www.clinicaltrials.gov/ct2/show/NCT04056195?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=2

Status: Unknown