20 Clinical Trials for Platelet Disorders
To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD.
To learn about the safety and effects of low-dose sirolimus in participants with RUNX1-FPD.
This is an open-label, multicenter study to evaluate the safety, tolerability, and efficacy of HMPL-523 in adult subjects with ITP.
Background: Runt-related transcription factor 1 (RUNX1) gene regulates the formation of blood cells. People with mutations of this gene may bleed or bruise easily; they are also at higher risk of getting cancers of the blood, bone marrow, and lymph nodes. Objective: To test a drug (imatinib) in people with RUNX1 mutations that cause symptoms. Eligibility: Adults aged 18 and older with RUNX1 mutations. Healthy people without this mutation, including family members of affected participants, are also needed. Design: Participants with the RUNX1 mutation will be screened. They will have a physical exam with blood and urine tests. They will have a test of their heart function. They may need a new bone marrow biopsy: A sample of soft tissue will be removed from inside a bone. Imatinib is a tablet taken by mouth once a day, every day, at home. Affected participants in different parts of the study will take imatinib for either 28 days or up to 84 days. Participants will visit the clinic once a week for the first 28 days that they are taking the imatinib. Then they will come once every 2 weeks if they are taking the drug for 84 days. Blood, urine, and tests of heart function will be repeated. They may opt to have the bone marrow biopsy repeated after they finish their course of imatinib. Participants will have a follow-up visit 30 days after they stop taking imatinib. Participants who do not have the RUNX1 mutation will have 1 clinic visit. They will have blood tests. They will fill out questionnaires. They may opt to have a bone marrow biopsy....
In parallel with the growth of American Thrombosis and Hemostasis Network's (ATHN) clinical studies, the number of new therapies for all congenital and acquired hematologic conditions, not just those for bleeding and clotting disorders, is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have yet to demonstrate long-term safety and effectiveness beyond the pivotal trials that led to their approval. In addition, results from well-controlled, pivotal studies often cannot be replicated once a therapy has been approved for general use.(1,2,3,4) In 2019 alone, the United States Food and Drug Administration (FDA) has issued approvals for twenty-four new therapies for congenital and acquired hematologic conditions.(5) In addition, almost 10,000 new studies for hematologic diseases are currently registered on www.clinicaltrials.gov.(6) With this increase in potential new therapies on the horizon, it is imperative that clinicians and clinical researchers in the field of non-neoplastic hematology have a uniform, secure, unbiased, and enduring method to collect long-term safety and efficacy data. ATHN Transcends is a cohort study to determine the safety, effectiveness, and practice of therapies used in the treatment of participants with congenital or acquired non-neoplastic blood disorders and connective tissue disorders with bleeding tendency. The study consists of 7 cohorts with additional study "arms" and "modules" branching off from the cohorts. The overarching objective of this longitudinal, observational study is to characterize the safety, effectiveness and practice of treatments for all people with congenital and acquired hematologic disorders in the US. As emphasized in a recently published review, accurate, uniform and quality national data collection is critical in clinical research, particularly for longitudinal cohort studies covering a lifetime of biologic risk.(7)
Primary immune thrombocytopenia (ITP) is a condition where the immune system mistakenly destroys platelets, which are cells that help stop bleeding. This leads to a low number of platelets, making it easier to bruise or bleed. The main aim of this study is to learn whether mezagitamab, when given just under the skin (subcutaneously \[SC\]), is effective in keeping the platelet count of adults with ITP stable when compared to a placebo. A placebo looks like medicine but doesn't have any active ingredients in it. The participants will be treated with mezagitamab for up to 6 months. During the study, participants will visit their study clinic several times. Participants who complete the TAK-079-3002 study or do not have any response to study treatment by week 16 (according to study criteria) will be given the opportunity to participate in a continuation study to receive open label mezagitamab (if they are eligible and the site is able to open the continuation study).
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) Condition or disease: Primary Myelofibrosis/Post-Polycythemia Vera Myelofibrosis/ Post-essential Thrombocythemia Myelofibrosis Intervention/treatment: Drug-Pacritinib
The purpose of the SENTRY (Stability Enhanced Transcriptional Analytics) Study is to test whether combining a unique analytical approach with changes in platelet RNA expression accurately diagnoses ovarian cancer. Using retrospective data, the investigators have developed an approach that appears to accurately classify ovarian cancer with relatively high sensitivity and specificity. The SENTRY Study will build upon these retrospective analyses to prospectively recruit women with ovarian cancer or an ovarian mass (and healthy control women), obtain platelet RNA samples from whole blood, and perform validation analyses to test our hypothesis.
This study looks at how lipoproteins, which are particles in the blood that transport cholesterol, influence heart and blood vessel health. Beyond just their levels, the way these particles function plays a key role in preventing or contributing to disease. In some conditions, like high cholesterol or diabetes, lipoproteins may not work properly, increasing the risk of clogged arteries and other complications. The investigators aim to study these changes in people with lipid disorders to better understand their impact on blood health and to find new ways to prevent and treat heart disease.
The objective of the NeoPlaTT trial is to test whether, among extremely preterm infants born at 23 0/7 to 26 6/7 weeks' gestation, a lower platelet transfusion threshold, compared to a higher threshold, improves survival without major or severe bleeding up to 40 0/7 weeks' postmenstrual age (PMA).
The researchers are testing a medication named ravulizumab for the treatment of severe preeclampsia and Hemolysis, Elevated Liver enzymes, Low Platelets (HELLP) syndrome.
The purpose of this study is to understand how the drug rivaroxaban improves symptoms associated with peripheral artery disease.
Sagittal split osteotomy (SSO) is a common operation done to move the mandible to correct dentofacial deformities and obstructive sleep apnea (OSA). Inferior alveolar nerve (IAN) injury and associated paresthesia is a well-known negative outcome following SSO, causing temporary or sometimes, permanent numbness in the chin and/or lip. There are limited methods to decrease the occurrence and duration of neurosensory dysfunction. Recent research has shown that platelet-rich fibrin (PRF) aids neurosensory recovery after SSO. Another method to minimize nerve injury is proximal segment grooving (PSG) to create space for the nerve to rest. This grooving method has never been formerly reported. The purpose of this study is to answer the following question: Among patients undergoing bilateral sagittal split osteotomy (BSSO) for dentofacial deformity or OSA, do those who receive PRF with or without PSG, compared to those who do not, have shorter times to functional sensory recovery (FSR) of the IAN? The null hypothesis is that there is no difference among 4 treatment groups and neurosensory outcomes. The specific aims of this proposal are to 1) enroll and randomize subjects who will undergo BSSO for correction of dentofacial deformity or OSA into 4 different treatment groups (PSG with PRF, PSG alone, PRF alone, neither PSG or PRF), 2) measure objective and subjective post-operative nerve function at fixed intervals post-operatively for up to 1 year, 3) compare differences in neurosensory outcomes among treatment groups, and 4) identify other variables that might be associated with differences in neurosensory outcomes.
MitoQ is commercially available as a dietary supplement and it has been tested as a potential drug in other diseases, but it has never been tested in patients with sickle cell disease. The goal of this research is to study if MitoQ, a molecule that works as an antioxidant by removing potentially damaging agents in a living organism, improves platelet function in patients with sickle cell disease (SCD).
Blood contains red blood cells, white blood cells, and platelets, as well as a fluid portion termed plasma. We primarily study blood platelets, but sometimes we also analyze the blood of patients with red blood cell disorders (such as sickle cell disease), white blood cell disorders, and disorders of the blood clotting factors found in plasma. Blood platelets are small cell fragments that help people stop bleeding after blood vessels are damaged. Some individuals have abnormalities in their blood platelets that result in them not functioning properly. One such disorder is Glanzmann thrombasthenia. Most such patients have a bleeding disorder characterized by nosebleeds, gum bleeding, easy bruising (black and blue marks), heavy menstrual periods in women, and excessive bleeding after surgery or trauma. Our laboratory performs advanced tests of platelet function and platelet biochemistry. If we find evidence that a genetic disorder may be responsible, we analyze the genetic material (DNA and RNA) from the volunteer, and when possible, close family members to identify the precise defect.
Ticagrelor currently represents the most tested and commonly used P2Y12 inhibitor monotherapy following percutaneous coronary intervention. The purpose of this study is to conduct a head-to-head comparison on the pharmacodynamic efficacy of ticagrelor-based and prasugrel-based single antiplatelet therapy. To determine if the PD profiles of ticagrelor- and prasugrel-based SAPT are comparable, we aim to conduct a non-inferiority study between the two strategies.
The proposed study aims to assess the antiplatelet effects of more potent oral P2Y12 inhibition with low-dose ticagrelor (60 mg bid) compared with standard of care clopidogrel in patients with a high ABCD-GENE score (≥10). We hypothesize that ticagrelor is associated with better pharmacodynamic effects (i.e., lower platelet reactivity and high platelet reactivity rates) compared with clopidogrel in stable coronary artery disease patients undergoing percutaneous coronary intervention with a high ABCD-GENE score.
Despite the relative safety of PCI with new generation stents, peri-PCI thrombotic complications, including myocardial infarction and myocardial injury, are common in elective PCI, occurring in up to 30% of patients. Importantly, these events are associated with poor prognosis. The risk of peri-PCI myocardial infarction/myocardial injury has been in part attributed to HPR. The aim of this study is to prospectively validate the accuracy of the ABCD-GENE score in identifying stable CAD patients undergoing elective PCI treated with standard of care clopidogrel who are at risk of peri-PCI myocardial infarction/myocardial injury. This investigation will be a prospective cohort study conducted in a population of patients (n=500) with stable CAD undergoing elective PCI treated with standard of care clopidogrel. By integrating genetic data with clinical variables, patients will be stratified into 2 cohorts based on their ABCD-GENE score (using a cut-off of 10). Assessments to define HPR status and myocardial infarction/myocardial injury will be performed post-PCI.
This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.
Hermansky-Pudlak Syndrome (HPS) is an inherited disease which results in decreased pigmentation (oculocutaneous albinism), bleeding problems due to a platelet abnormality (platelet storage pool defect), and storage of an abnormal fat-protein compound (lysosomal accumulation of ceroid lipofuscin). The disease can cause poor functioning of the lungs, intestine, kidneys, or heart. The major complication of the disease is pulmonary fibrosis and typically causes death in patients ages 40 - 50 years old. The disorder is common in Puerto Rico, where many of the clinical research studies on the disease have been conducted. Neither the full extent of the disease nor the basic cause of the disease is known. There is no known treatment for HPS. The purpose of this study is to perform research into the medical complications of HPS and begin to understand what causes these complications. Researchers will clinically evaluate patients with HPS of all ethnic backgrounds. They will obtain cells, blood components (plasma), and urine for future studies. Genetic tests (mutation analysis) to detect HPS-causing genes will also be conducted.\<TAB\>