Idiopathic Thrombocytopenic Purpura (ITP)

Immune thrombocytopenia ITP is an autoimmune disease that causes a low platelet count leading to bleeding in the skin and mucous membranes. This trial studies the safety and effectiveness of nicotinamide adenine dinucleotide NAD and nicotinamide mononucleotide NMN for treating ITP in patients who have not responded well or have relapsed after first-line and at least one second-line therapy such as Anti-CD20 antibody or TPO receptor agonists. The study addresses the need for better treatments for adults with recurrent or difficult-to-treat ITP, a condition that severely impacts quality of life. Participants receive intravenous NAD 100 mg daily for 1 week and oral NMN 450 mg twice daily for 2 weeks in an open, single-arm design. The study has two stages the first 2 weeks focus on treatment safety and efficacy, followed by a 6-week observation period to monitor ongoing safety and effects after stopping the treatment. During the trial, participants will have regular blood tests to measure platelet counts and bleeding scores, along with tracking any side effects for up to 8 weeks. Researchers will evaluate how well the treatment increases platelet levels and reduces bleeding symptoms. The study also monitors adverse events and treatment tolerability. Overall participation lasts about 8 weeks, including treatment and follow-up assessments.

The GENESIS clinical study aims to map HLA genetic variation in the Greek population and evaluate possible correlations with selected underlying diseases. It is a multicenter, prospective, non-interventional clinical study targeting 12,000 subjects over an anticipated duration of 36 months, with the goal of creating a pilot HLA map for medical research and possible clinical applications. Each subject will complete one visit at a participating site and provide demographic information, including date of birth, gender, race, ancestry, height, and weight, as well as information about smoking or vaping, alcohol consumption, arterial blood pressure, diagnosed diseases, and current treatments. Recent clinical laboratory results from up to 12 months before sample collection may also be collected when available, including blood count, metabolic, liver enzyme, and biochemical parameters. Two buccal swabs will be collected from each subject for DNA extraction and HLA genotyping analysis. Selected DNA samples will also undergo low-pass whole genome sequencing to further investigate associations between the HLA region and autoimmune diseases. After the analysis is completed, an individualized ancestry report will be securely available to study subjects if they elect to access it.

Researchers are evaluating CID-103, a new anti-CD38 monoclonal antibody, in adults with chronic immune thrombocytopenia ITP to assess its safety and effectiveness. This global Phase 12 trial aims to find a safe dose range and the optimal dose for further studies, particularly for patients who have not responded well to current treatments. The study is sponsored by CASI Pharmaceuticals, Inc. and involves adults aged 18 to 65 with chronic ITP diagnosed according to established guidelines. The study has two parts Part A uses an accelerated dose escalation design to test increasing doses of CID-103 delivered by intravenous infusion. If significant side effects occur, the study switches to a standard dose escalation design. Part B is a randomized, open-label phase comparing up to three selected doses low, intermediate, and high to evaluate safety, tolerability, and platelet response. Treatment is given weekly for 6 weeks, then every two weeks up to week 12, and monthly if extended, for up to six months total. Participants will undergo regular monitoring during the treatment and a post-treatment safety follow-up period. Researchers will assess safety, tolerability, platelet counts, and platelet response over 12 weeks and up to 10 months. Other evaluations include organ function and response to prior treatments. The study includes detailed safety reviews and dose adjustments based on participant reactions, aiming to identify a dose regimen suitable for future clinical trials.

Primary immune thrombocytopenia ITP is a condition where the immune system mistakenly destroys platelets, leading to a lower number of platelets and increased risk of bruising or bleeding. This Phase 3 study evaluates the long-term safety, tolerability, and effectiveness of mezagitamab in adults with chronic primary ITP. The study also investigates how the body processes mezagitamab over an extended period. Participants who completed previous mezagitamab studies TAK-079-3002 or TAK-079-1004 will be invited to join this continuation trial. Eligible participants may receive mezagitamab injections on demand, with treatment courses repeated as needed based on specific criteria and the investigators clinical judgment. The treatment is administered subcutaneously. During the study, participants will visit the clinic several times for assessments. Researchers will monitor safety by tracking treatment-emergent adverse events, and evaluate effectiveness through platelet response and remission rates. Measurements of drug levels and antibodies will also be taken. The study may last up to approximately 108 weeks, allowing detailed long-term follow-up.

Researchers are evaluating budoprutug, a humanized monoclonal antibody targeting CD19, in adults with immune thrombocytopenia ITP, a condition characterized by low platelet counts. This Phase 1b2a open-label study aims to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary clinical effects of budoprutug in patients with platelet counts below 30,000L despite prior treatment attempts. The study is sponsored by Climb Bio, Inc. Participants will receive budoprutug as two intravenous infusions administered 14 days apart. The study includes sequential cohorts with escalating doses, followed by a dose expansion group. Each participant receives a single IV dose on Day 1 and another on Day 15. The trial monitors the effects of budoprutug on platelet counts and CD20 B-cell levels, among other factors. Throughout the study, participants will be closely monitored up to 48 weeks for treatment-related side effects, blood levels of the drug, immune cell changes, platelet responses, and the development of anti-drug antibodies. Safety labs including coagulation tests and bilirubin levels will be assessed. The study does not include placebo groups and participation involves scheduled visits for infusions and follow-up evaluations to track both safety and preliminary clinical outcomes over nearly a year.

Researchers are studying the safety, tolerability, and pharmacological effects of multiple doses of STSA-1301 subcutaneous injection in both healthy individuals and patients with Primary Immune Thrombocytopenia ITP. This randomized, double-blind, placebo-controlled trial also explores the initial effectiveness of STSA-1301 in patients with ITP. The study is sponsored by Jiangsu BioJeTay Biotechnology Co., Ltd. and aims to better understand how this treatment influences platelet counts and immune markers. Participants receive different doses of STSA-1301 or a placebo as subcutaneous injections according to the study protocol. The trial includes multiple dose levels low, median, and high doses, administered in separate cohorts. The study design is parallel and randomized, ensuring participants are assigned to one of the treatment groups or placebo without knowing their group. Treatments are given on specified dates, with monitoring for safety and immune responses over the course of the study. During the trial, participants undergo regular assessments including blood tests to measure platelet counts, immune markers such as serum IgG, cytokines, and anti-drug antibodies, as well as pharmacokinetic parameters like drug concentration in the blood. Researchers monitor any adverse events and changes in bleeding scores over a 78-day period following treatment. The study includes healthy volunteers and patients aged 18 to 75 years and lasts until September 2026. Participants will be closely observed throughout for treatment effects and safety.

Researchers are evaluating the safety and effectiveness of Obinutuzumab in treating immune thrombocytopenia ITP, a condition where the immune system attacks platelets causing low platelet counts and bleeding. This study focuses on adults who did not respond well or relapsed after initial treatments like corticosteroids. ITP is a challenging disease with limited effective options, and this trial aims to address the urgent need for better treatments for patients with persistent or recurring ITP. Participants will receive a single intravenous infusion of 1000 mg Obinutuzumab, a type of anti-CD20 monoclonal antibody. The study has two stages the first 12 weeks involve the core treatment and monitoring of safety and response, followed by a withdrawal period from week 12 to week 48 to observe ongoing safety and lasting effects after treatment. In total, 110 participants will be enrolled in this open-label, single-arm trial. During the study, participants will undergo regular assessments to evaluate treatment response, bleeding severity, and safety over one year. Key measures include the overall response within 12 weeks, sustained response rates at 6 and 12 months, recurrence-free survival, and safety monitoring. The study involves blood tests, physical exams, and questionnaires to track platelet counts and bleeding symptoms, ensuring close observation of the treatments effects and participant health throughout the trial period.

Researchers are evaluating the safety and effectiveness of Obinutuzumab, an anti-CD20 monoclonal antibody, for treating pediatric primary immune thrombocytopenia ITP in patients aged 12 to 18 years who have not responded well or have relapsed after first-line glucocorticoid treatment. ITP is an autoimmune disease causing low platelet counts and bleeding, and current treatments for pediatric ITP have limited success, leading to ongoing challenges and reduced quality of life. This open, single-arm study will enroll 60 pediatric participants who will receive one intravenous infusion of 1000 mg Obinutuzumab. The study has two stages the first 12 weeks focus on treatment safety and efficacy after infusion, and the following weeks up to 48 weeks monitor ongoing safety and lasting treatment effects without additional infusions. Participants will undergo regular assessments including platelet counts, bleeding evaluations, safety monitoring, and quality of life surveys over 12 months. Researchers will measure overall treatment response, duration of response, bleeding events, recurrence rate, and health-related quality of life to understand how Obinutuzumab affects pediatric ITP. Safety will be closely observed throughout the study period.

This research aims to evaluate the safety and effectiveness of zanubrutinib in treating immune thrombocytopenia ITP, a condition where the immune system causes low platelet counts leading to bleeding issues. ITP treatments currently have limited success, especially for patients who do not respond to standard therapies. Zanubrutinib is a newer drug that targets B cells involved in autoimmune diseases and has shown promise with fewer side effects compared to earlier treatments. In this study, 30 patients will take zanubrutinib starting at 80 mg once daily. If after 4 weeks the treatment is not effective and it is safe, the dose may be increased up to 160 mg twice daily. The treatment period lasts for 24 weeks. If severe side effects occur, the dose may be reduced or the patient may stop the treatment. This trial is designed to provide a new treatment option for adults with ITP who have not responded well to other therapies. Participants will have their platelet counts checked at 12 weeks to assess response and again at 24 weeks to evaluate sustained effects. The study also monitors bleeding severity, adverse events, and immune cell changes during treatment. Physical exams and lab tests will help ensure safety. The total participation time includes treatment and follow-up visits over 24 weeks to assess the drugs impact and safety profile.

Researchers are studying treatment patterns and outcomes in adults with primary immune thrombocytopenia ITP who have been treated with corticosteroids and then received eltrombopag. The study collects data from healthcare professionals in the United States, United Kingdom, and Germany to better understand how eltrombopag is used and its clinical effects in real-world settings. This observational study aims to describe patient responses and treatment decisions in a routine care environment. Data will be gathered from health care professionals through surveys and patient chart reviews. Participants include adults diagnosed with primary ITP who started eltrombopag between January 2020 and December 2024 after corticosteroid treatment. The study focuses on patients who have at least six months of follow-up data. The healthcare professionals involved have relevant experience and manage multiple ITP patients monthly. Participants health records and survey responses will provide information on platelet response within 12 weeks, treatment duration, bleeding symptoms, corticosteroid use during eltrombopag treatment, and reasons for treatment initiation or discontinuation. The study also collects data on patient demographics, prior treatments, and treatment outcomes over time. This approach helps researchers understand real-world treatment patterns and effectiveness of eltrombopag in managing primary ITP.