Immune 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.

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 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 1/2 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 drug's 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 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.