Clinical Research Directory

Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)

This Phase 1b basket trial will investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity and preliminary efficacy of RAY121, a inhibitor of classical complement pathway, after multiple dose administration in patients with immunological diseases such as antiphospholipid syndrome (APS), bullous pemphigoid (BP), Behçet's Syndrome (BS), dermatomyositis (DM), immune-mediated necrotizing myopathy (IMNM) and immune thrombocytopenia (ITP).

Phase 1b Long-term Extension Trial of RAY121 in Immunological Diseases (RAINBOW-LTE Trial)

This is a long-term extension trial of RAY121 in patients with immunological diseases such as antiphospholipid syndrome (APS), bullous pemphigoid (BP), Behçet's Syndrome (BS), dermatomyositis (DM), immune-mediated necrotizing myopathy (IMNM) and immune thrombocytopenia (ITP).

A Phase 1b/2a Study of Budoprutug in Subjects With Immune Thrombocytopenia (ITP)

The main objective is to assess the safety and tolerability of budoprutug in adults with ITP. Pharmacokinetics, pharmacodynamics, and preliminary clinical efficacy will also be assessed.

A Study of Efgartigimod IV in Participants From 12 Years to Less Than 18 Years of Age With Chronic Immune Thrombocytopenia (ITP)

The main purpose of this study is to confirm the correct dose of efgartigimod IV for treating patients aged 12 to younger than 18 years with chronic immune thrombocytopenia (ITP). The study consists of a double-blinded treatment period (DBTP) in which the participants will be randomized in a 2:1 ratio to receive either efgartigimod IV or placebo IV. At the end of the treatment period (up to 24 weeks), all participants will receive efgartigimod IV during the first year open-label treatment period (OLTP1). At the end of the first OLTP1, participants may begin a second year (OLTP2). After the OLTP2, the participants will enter a follow-up period (approximately 8 weeks) while off study drug. The participants will be in the study for up to 138 weeks. More information can be found here: https://clinicaltrials.argenx.com/advancejunior

A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia

The purpose of the phase 1 part of this study is to evaluate how well pirtobrutinib is tolerated and what side effects may occur. The phase 2 part of the study will further investigate efficacy and safety of multiple pirtobrutinib dosages versus placebo. The study drug will be administered orally in participants with Primary Immune Thrombocytopenia (ITP). Blood tests will be performed to check how much pirtobrutinib gets into the bloodstream and how long it takes the body to eliminate it. The study will last up to approximately 16 weeks for phase 1 dose-escalation and 28 weeks for phase 2 dose-optimization, excluding screening.

A Clinical Study Evaluating the Safety and Preliminary Efficacy of Universal Allogeneic CAR T-cell Therapy Targeting CD19 and BCMA(QT-019C)in Patients With Refractory Primary Immune Thrombocytopenia

This is an investigator-initiated trial to evaluate the safety and efficacy of universal allogeneic anti-CD19/BCMA CAR T-cells(QT-019C) in Patients With Refractory Primary Immune Thrombocytopenia.

A Study of DZD8586 in Adults With Primary Immune Thrombocytopenia (ITP) (TAI-SHAN11)

This is a Phase 2, open-label, randomized, multicenter study to assess the efficacy and safety of DZD8586 in patients with primary immune thrombocytopenia (ITP). The target population of this study is patients with primary ITP who had failed to respond or relapsed after receiving at least one standard therapy. Participants who meet the inclusion criteria and do not meet the exclusion criteria will be randomized to different dose groups.

V-IMMUNE® for Immune Thrombocytopenia

This is a multicenter, prospective clinical trial evaluating the efficacy and safety of V-IMMUNE®, a 5% human normal immunoglobulin formulation administered intravenously, for the treatment of immune thrombocytopenia (ITP) in patients aged ≥1 year. The primary objective is to assess the proportion of patients achieving a platelet count ≥50,000/mm³ on or before Day 9 following the first infusion. The trial employs a single-group design, comparing outcomes to historical controls derived from the literature. Eligible patients must have a confirmed diagnosis of ITP with a platelet count ≤20,000/mm³ and no concurrent conditions likely to cause thrombocytopenia. Key exclusions include non-immune thrombocytopenia, active sepsis, pregnancy or lactation, hypersensitivity to blood products or IgG preparations, and various significant comorbidities (e.g., uncontrolled hypertension, severe hepatic or renal impairment, recent rituximab use). The intervention consists of V-IMMUNE® at a dose of 1 g/kg, administered once daily for two consecutive days, with infusion rates titrated from 0.01 mL/kg/min to 0.06 mL/kg/min. Standard pre-medication protocols (IV normal saline and diphenhydramine) are administered to mitigate infusion-related reactions and reduce the risk of thromboembolic events. Patients will be monitored at multiple time points from baseline through Day 90, with primary efficacy evaluation at Day 9. Secondary endpoints include duration of platelet response, overall treatment response rate, bleeding events, and incidence of infusion-related adverse events.

A Study of CM336 in Patients With Relapsed or Refractory Autoimmune Cytopenia

To evaluate the efficacy and safety of CM336 (BCMA/CD3 Bispecific Antibody) in the treatment of patients with relapsed or refractory autoimmune cytopenia

National Multicenter Retrospective Real-World Study on the Treatment Status of Newly Diagnosed and Persistent Primary Immune Thrombocytopenia (ITP) Patients

This study is a nationwide, multicenter, retrospective, real-world, non-interventional (observational) investigation. It retrospectively collects data on treatment modalities and therapeutic responses in patients with newly diagnosed or persistent immune thrombocytopenia (ITP), documents disease progression in both treated and untreated patients, and records the treatment regimens selected by those receiving therapy. The study aims to observe and evaluate the current treatment landscape for newly diagnosed and persistent ITP patients, while also analyzing the efficacy and safety in the treated population. All patient treatments and disease management are solely based on routine clinical practice and are not influenced by this study.

An Exploratory Study of Golidocitinib in Adult Patients With ITP

This is a multicenter clinical study to evaluate the safety and efficacy of golidocitinib in patients with primary immune thrombocytopenia (ITP). The study consists of two parts: Part A dose escalation and Part B dose expansion. Part A is designed to obtain the safety profile of golidocitinib in patients with ITP and the recommended dose for the randomized cohort in Part B. Part B is a randomized, double-blind, placebo-controlled study, and the primary objective of this part is to evaluate the preliminary efficacy of golidocitinib in patients with ITP.

The Efficacy and Safety of Combined Teriflunomide and High-dose Dexamethasone in Newly Diagnosed Primary Immune Thrombocytopenia (TEMPO-2)

A multicenter, open-label, randomized study to report the efficacy and safety of teriflunomide plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with newly diagnosed primary immune thrombocytopenia (ITP).

Anti CD19/BCMA CAR Gene Therapy for Relapsed/Refractory Immune Thrombocytopenia

This is an open label, single-site, dose-escalation study in up to 18 participants with treatment of relapsed and refractory immune thrombocytopenia. This study aims to evaluate the safety and efficacy of the treatment with an Anti- CD19/BCMA CAR gene vector injection

Safety, Tolerability, and Efficacy of NVG-2089 in Participants With Immune Thrombocytopenia

The purpose of the study is to evaluate the safety of NVG-2089 and to evaluate how well patients respond to this investigational treatment. NVG-2089 is a new drug that is being developed for treating patients with ITP. NVG-2089 is designed to mimic the effects of a protein called IVIg. NVG-2089 is designed to help the immune system by attaching (binding) to certain receptors in the body and activating them, which helps reduce inflammation and supports how the immune system works.

Longitudinal Cohort Study on Immune Thrombocytopenia Complicated With Acute Ischemic Stroke

Immune thrombocytopenia (ITP) is an acquired bleeding disorder mediated by immune-related platelet destruction and impaired platelet production. Immune thrombocytopenia may increase the risk of cerebral infarction and represents a relatively uncommon etiology of acute ischemic stroke. This disease is associated with high disability and mortality rates, poses significant therapeutic challenges, and constitutes a serious threat to human health. Therefore, research on the diagnosis, treatment, and prognosis of ITP combined with acute ischemic stroke is of great significance for improving patients' quality of life and survival outcomes. However, most current hematologic cohort studies are based on single-center or limited multicenter sample sizes, lacking comprehensive and large-scale prospective cohort studies. Our center plans to conduct a large-sample, combined retrospective and prospective longitudinal cohort study. This study will register patients' basic information and diagnosis, follow up with patients through questionnaires, telephone calls, video consultations, online platforms, and in-person visits to record treatment and comorbidity data, collect prognostic information, and retrieve hospitalization and outpatient costs through medical record systems. The study aims to provide comprehensive data on the incidence, treatment, prognosis, and healthcare costs of ITP patients with acute ischemic stroke in China.

Longitudinal Observational Study on Immune Thrombocytopenia

Immune Thrombocytopenia (ITP) is a bleeding disorder characterized by immune-mediated destruction and/or impaired production of platelets, leading to clinical manifestations such as petechiae, purpura, mucosal bleeding, and in severe cases, life-threatening visceral or intracranial hemorrhage. Research on the diagnosis, treatment, and prognosis of ITP is critical for improving patient outcomes and quality of life. However, most existing hematologic cohort studies are limited to single-center or small multicenter samples, lacking comprehensive, large-scale prospective investigations. To address this gap, our center plans to conduct a large-sample, combined retrospective and prospective longitudinal observational cohort study of ITP patients. The study will enroll patients to collect baseline demographic and diagnostic data, followed by longitudinal follow-up via questionnaires, telephone interviews, video consultations, online platforms, and in-person visits. Key variables including treatment regimens, comorbidities, and prognostic outcomes will be systematically recorded. Additionally, hospitalization and outpatient expenditure data will be extracted from electronic medical records. This study aims to provide high-quality real-world evidence on the epidemiology, treatment patterns, clinical outcomes, and healthcare costs of ITP patients in China, ultimately informing clinical decision-making and health policy.

Iguratimod Plus Low-dose Rituximab vs Low-dose Rituximab in Corticosteroid-resistant or Relapsed ITP

Randomized, open-label, multicentre study to assess the efficacy and safety of the combination of low-dose rituximab and Iguratimod in patients with steroid-resistant/relapsed ITP.

Level of Serum Lactate Dehydrogenase in Immune Thrombocytopenia

Assessment of relation between serum lactate dehydrogenase and immune thrombocytopenia

A Phase 1/2 Study of ESG206 in Patients With Primary Immune Thrombocytopenia

This is a multicenter, open-label Phase1/2 study aimed at evaluating the safety, tolerability, pharmacokinetic (PK) profile, pharmacodynamics (PD), immunogenicity, and preliminary efficacy of ESG206. The study will be conducted in patients with primary immune thrombocytopenia.

A Clinical Study of YTS109 Cell in R/R Systemic Lupus Erythematosus

This study evaluates the safety and efficacy of YTS109 cells in adults with refractory Lupus Nephritis (LN) and Systemic Lupus Erythematosus-Immune Thrombocytopenia (SLE-ITP). Approximately 36 patients aged 18-65 will receive a single infusion of YTS109 cells (1×10⁶-2×10⁶ cells/kg). The primary endpoint is observations of types, severity, and frequency of dose-limiting toxicities (DLTs) and adverse events (AEs). Secondary endpoints include the complete renal response (CRR) rate at week 12 in LN, and proportion of subjects achieving complete response (CR) or partial response (PR) at week 12 post-treatment in SLE-ITP. This single-arm, open-label trial will enroll patients across Beijing GoBroad Hospital in China.

An Open, Exploratory Clinical Study of CM336 in the Treatment of Immune Thrombocytopenia

To evaluate the efficacy and safety of CM336 in the treatment of refractory adult primary immune thrombocytopenia

IgIV Plus Prednisone vs High-dose Dexamethasone for ITP

ITP patients with low platelet count and active bleeding symptoms are at risk of life-threatening bleeding and therefore require a treatment with a rapid effect, reliable, and sustained. The combination of intravenous immunoglobulin (IVIg) and prednisone (1 mg/kg per day), is more rapidly and more frequently effective than high dose methylprednisolone to increase the platelet count. This combination is therefore usually given in patients with platelets count \< 20 x 109/L and moderate to severe bleeding manifestations. Based on common practice in France and on French ITP guidelines, on average 50 % of patients with ITP and profound thrombocytopenia do actually receive IVIg (mostly during the initial phase of the disease) corresponding to approximately 1,500 ITP patients per year in France. Whereas IVIg is usually well tolerated, renal insufficiency and congestive heart failure may occur, moreover IVIg are costly and non-easily available with supply difficulties in many countries including France. High dose dexamethasone (DXM) (ie: 40 mg/d for 4 days) has recently emerged as a promising treatment for ITP. One recent meta-analysis as well as a controlled prospective trial suggest that the initial overall response was higher (\> 80 %) and the time to response was shorter with dexamethasone (DXM) 40 mg/d given for 4 days compared to standard prednisone. The investigators hypothesize that DXM could be a reasonable non-inferior alternative to IVIg, more convenient for patients with less adverse events and economically cost-effective for patients with moderate and severe bleeding manifestations.