Thrombocytopenia

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.

Researchers are evaluating the safety and potential benefits of YTS109 cells in adults with refractory Lupus Nephritis LN and Systemic Lupus Erythematosus-Immune Thrombocytopenia SLE-ITP. These are serious and difficult-to-treat conditions associated with systemic lupus erythematosus SLE, which affects multiple organs and involves persistent immune system problems. This exploratory study aims to gather early data on safety, effectiveness, and how the therapy behaves in the body in patients who have not responded to standard treatments. Participants will receive a single infusion of YTS109 cells, a universal allogeneic STAR-T cell therapy targeting CD19 to reduce B cells involved in autoimmune responses. The dose ranges from 1 million to 2 million STAR-T cells per kilogram of body weight. This is a single-arm, open-label trial conducted at one center, where all subjects receive the investigational treatment. The study will monitor patients for 52 weeks after treatment to assess safety and response. During the study, participants will be closely monitored for adverse events and dose-limiting toxicities over the first 28 days, with continued observation for up to a year. Researchers will evaluate kidney response in LN patients and platelet response in SLE-ITP patients at 12 weeks post-treatment. Blood samples will be collected to analyze pharmacokinetics and pharmacodynamics of the YTS109 cells. Participants will provide informed consent and undergo regular assessments throughout the study period.

Researchers are studying the use of herombopag to help elderly patients with immune thrombocytopenia ITP, a condition that affects platelet levels in the blood. This phase 2 clinical trial focuses on patients aged 60 years and older to evaluate how well herombopag works and how safe it is for treating this condition in older adults. The study is designed as a single-arm, prospective trial involving 80 participants. Participants will take herombopag tablets starting at 5 mg once daily on an empty stomach, with the possibility to eat two hours afterward. The treatment lasts for 24 weeks, and the dosage may be adjusted weekly based on platelet counts, with a maximum of 7.5 mg per day. If there is no response after 8 weeks, patients may be advised to stop treatment but will continue safety monitoring for an additional 4 weeks. Treatment continues unless toxicity, disease progression, withdrawal of consent, or other criteria require stopping. Throughout the study, participants will have weekly blood tests to monitor platelet levels and overall safety, including vital signs and lab tests. Researchers will assess treatment effects at 8 weeks and 24 weeks, including platelet count improvements and bleeding symptoms. After treatment ends, a 4-week safety follow-up visit is conducted. The study aims to measure how many patients reach target platelet levels and track any adverse effects experienced during the study period.

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 studying the use of unlicensed cryopreserved cord blood units CBUs for transplantation in both children and adults with blood cancers and other related disorders. This observational study involves patients with hematologic malignancies and various inherited and acquired disorders affecting the blood and immune system. The main goal is to monitor how well neutrophil recovery occurs after transplantation using these unlicensed CBUs in multiple institutions. Participants receive unlicensed cryopreserved CBUs as part of their transplant treatment. The study includes patients of any age receiving these CBUs for approved indications. The protocol focuses on the access and distribution of these unlicensed units rather than a specific treatment intervention. The study gathers data from recipients who receive these CBUs, tracking outcomes after transplantation. Participants are monitored for neutrophil recovery at 60 and 100 days after transplant, defined by a neutrophil count of at least 500mm3. Researchers also collect information on infection transmission, infusion reactions, survival rates at one year, and incidence of acute and chronic graft versus host disease. Platelet recovery is also evaluated. Safety and efficacy outcomes are followed over time to better understand the effects of unlicensed CBUs in this patient population.

Researchers are evaluating ESG206, an anti-BAFF-R monoclonal antibody, in patients with primary immune thrombocytopenia ITP to study its safety, tolerability, how the body processes the drug, immune response, and early signs of effectiveness. This Phase 12 open-label study is designed to understand these aspects in patients who have experienced insufficient or no response to prior ITP treatments such as corticosteroids and intravenous immunoglobulin. The study includes a dose escalation phase and an extended cohort phase where participants receive ESG206 intravenously at one of four different dose levels. The trial consists of a screening period of up to 28 days, followed by a 14-week treatment phase, and an 8-week post-treatment follow-up. Participants who experience treatment failure will undergo an additional safety follow-up 28 days after their last dose. During the study, participants will have their safety closely monitored, including tracking any adverse events, and their response to treatment evaluated by measuring platelet counts and immune cell levels over 24 weeks. Pharmacokinetic and pharmacodynamic assessments, immunogenicity testing, and other laboratory evaluations will be performed to understand the drugs effects. The total participation time includes screening, treatment, and follow-up periods, with regular visits for assessments and monitoring.

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.