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.

Researchers are conducting the GENESIS clinical study to map the HLA genomic region in the Greek population and explore its possible links with various underlying diseases. This non-interventional, multicenter study aims to provide a pilot map of genetic variation in HLA that may be useful in medical research and clinical applications related to selected diseases. The study plans to include 12,000 participants over a total duration of 36 months. Each participant will attend one visit at a participating site during which they will provide demographic data, lifestyle information such as smoking and alcohol use, blood pressure measurements, details on diagnosed diseases and treatments, and recent laboratory test results if available. Buccal swab samples will be collected from each participant to extract DNA for HLA genotyping analysis. Selected samples will undergo further whole genome sequencing to investigate associations with autoimmune diseases. Participants will receive a personalized ancestry report after analysis completion. During the study visit, data collection includes demographic and health information, as well as laboratory and clinical test results from the past year. The genetic material from buccal swabs will be stored and processed for genetic analysis. Researchers will measure allele frequency of HLA alleles in the Greek population and assess the prevalence and risk associations of selected HLA-related diseases. The study's total duration is 36 months with results available at the end of this period.

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 1/2 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 platelet count and increased risk of bruising or bleeding. This research aims to evaluate the long-term safety, tolerability, effectiveness, and how the body processes mezagitamab in adults with chronic primary ITP. Participants who previously took part in related mezagitamab studies (TAK-079-3002 and TAK-079-1004) are invited to join this continuation study. Eligible participants will receive mezagitamab injections under pre-specified criteria and as needed, based on their clinical condition and the investigator's judgment. This on-demand treatment may be repeated multiple times during the study. The study is an open-label, phase 3 trial that monitors participants over an extended period with repeated dosing as required. Participants will visit the study clinic multiple times for assessments throughout the study, which may last up to approximately 108 weeks. Researchers will monitor treatment-emergent adverse events, platelet response duration, use of other ITP medications, and the presence of antibodies against the drug. Blood samples will be collected to measure drug levels and immune responses. Safety and effectiveness will be closely followed, including any need for rescue therapies.

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 1b/2a open-label study aims to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary clinical effects of budoprutug in patients with platelet counts below 30,000/µL 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 evaluating the safety, tolerability, pharmacokinetic and pharmacodynamic profiles, and immune response of multiple doses of STSA-1301 subcutaneous injection in healthy adults and patients with primary immune thrombocytopenia (ITP). This Phase Ib/II randomized, double-blind, placebo-controlled trial also aims to explore the initial effects of STSA-1301 on platelet counts in patients with ITP. The study is sponsored by Jiangsu BioJeTay Biotechnology Co., Ltd and includes participants aged 18 to 75 years. Participants receive varying doses of STSA-1301 or placebo through subcutaneous injections according to the study protocol. The trial includes three cohorts receiving low, median, or high doses of the study drug or placebo. Treatments are given multiple times over the study period, with careful monitoring of drug levels, immune response, and platelet counts to assess pharmacokinetics, pharmacodynamics, and potential benefits. During the 78-day study, participants undergo regular assessments including blood tests for platelet counts, immune markers, and drug concentration levels. Safety is closely monitored by tracking adverse events and immunogenicity. Additional measures include evaluating bleeding scores and cytokine levels. The trial requires informed consent and adherence to contraception requirements, with follow-up to assess outcomes and side effects throughout the study period.

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 treatment's 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 drug's impact and safety profile.

Researchers are evaluating the effectiveness and safety of Kedrion Intravenous Human Normal Immunoglobulin (IVIg) 10% in adults with chronic primary Immune Thrombocytopenia (ITP), a condition characterized by a low platelet count lasting more than 12 months. This Phase III, open-label, single-arm study focuses on adult patients aged 18 to 70 years who have chronic primary ITP with platelet counts below 30 × 10^9/L. The study is sponsored by Kedrion S.p.A. and aims to assess how well the treatment increases platelet counts and controls bleeding symptoms. Participants will receive a single treatment course of Kedrion IVIg 10% at a dose of 2 g/kg, administered intravenously over two days. The treatment period is followed by monitoring to evaluate the platelet count response and bleeding regression. The study does not include a control group or placebo, and all subjects receive the same investigational treatment. The treatment is administered at the start of the study, with follow-up visits extending up to 30 days after treatment. During the study, participants will undergo platelet count assessments and evaluation of bleeding symptoms from the start of treatment (Day 1) through to the end of the study (Day 30). Researchers will monitor the rate and duration of platelet response, complete response rates, and safety and tolerability of the treatment. The primary outcome is the rate of subjects who respond by Day 14. Additional assessments include time to response, duration of response, regression of hemorrhages, and safety monitoring throughout the 30-day period. Participants' health information will be accessed with authorization, and adherence to the protocol is required throughout the study.