Clinical Research Directory

An Open-label Study of Intravenous Immunoglobulin (5%) for the Treatment of Primary Immune Thrombocytopenia

It is a clinical trial of a medicinal product Human Immunoglobulin for Intravenous Injection (5%) manufactured by Grand Shuyang Life Sciences (Chengdu) Co., Ltd. (hereinafter referred to as 5% IVIG). This clinical study is conducted to evaluate the efficacy and safety of 5% IVIG in patients with primary Immune Thrombocytopenia (ITP). The study includes 4 periods and 9 visits (designated as V): * Screening period (V1: 14 days before the first administration, preliminary examination); * Baseline Period (V2: within 24 h before the first dose, Day 0); * Treatment Period (V3: Day 1 to Day 5, administration of the drug in a hospital setting); * Follow-up Period (V4-V9: visits to Study Site on Day 6, Day 7, Day 14 + 2, Day 21 + 3, Day 28 + 4, and Day 90 (+ 14) after the first dose). In this study, the investigational product 5% IVIG will be administered at a dose of 0.4 g/kg/day (direct intravenous drip) for 5 consecutive days. The total duration of your participation in the study will be approximately 104 days. The investigational product will only be provided during the study period. It will not be supplied after the study ends. The study will be conducted at clinical centers in Turkey. It is planned to obtain data from 36 patients included in the study (no more than 48 screened patients).

A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)

The purpose of this study is to investigate the tolerability of ianalumab (9 mg/kg) with investigator's choice thrombopoietin receptor agonist (IC TPO-RA) in participants diagnosed with primary immune thrombocytopenia (ITP) who have been treated with at least one but no more than four prior treatments, and with no change in IC TPO-RA dose in at least the last 14 days prior to the start of ianalumab.

rhTPO Dose Escalation vs Eltrombopag Switch in ITP

This study is a prospective, multicenter, randomized controlled study, planning to enroll 110 ITP patients who failed to respond to conventional-dose rhTPO (300 IU/kg/d) after 14 days of treatment (PLT \< 30×10⁹/L). After a 2-week washout period, they will be randomized to the rhTPO double-dose group (Group A) and EPAG-pfos group (Group B), with blood routine monitored weekly and doses adjusted according to platelet levels, comparing the response rates of the two groups at 6 weeks after switching treatment.

A Study to Assess the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia

The main purpose of this study is to look at the effect (efficacy) and safety of efgartigimod IV in participants with primary immune thrombocytopenia (ITP). After an up to 2 weeks screening period, eligible participants will be randomized in a 2:1 ratio to receive either efgartigimod IV or placebo IV, respectively during the double-blinded treatment period (DBTP). At the end of the treatment period (up to 24 weeks), all participants will receive efgartigimod IV during the first 52-week open-label treatment period (OLTP1). At the end of the first OLTP1, participants may begin a second 52-week 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/advancenext

Study of PN20 in Adult Patients With Primary Immune Thrombocytopenia (ITP)

The main aim of this clinical trial is to assess the safety and tolerability of PN20 in adult patients with primary immune thrombocytopenia (ITP). The main questions it aims to answer are: * Is PN20 safe in these patients? * Could these patients potentially benefit from PN20 treatment? Participants will * Receive one subcutaneous injection of PN20 according to weight; * Visit the clinic for assessment.

Study of Ianalumab Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (ITP)

The purpose of this study is to evaluate the effect of two different doses of ianalumab versus placebo in addition to first-line corticosteroids in maintaining platelet count ≥30 G/L in adult participants with primary ITP.

Phase II Study of Orelabrutinib in Combination With Romiplostim N01 in Patients With Primary Immune Thrombocytopenia (ITP) Who Have Received At Least One Prior Line of Therapy

To evaluate whether orelabrutinib combined with romiplostim N01 can improve the quality of remission, increase the probability of successful drug withdrawal, and prolong the time to treatment failure in patients with primary immune thrombocytopenia (ITP) who have received at least one line of prior therapy.

A Study of Ianalumab (VAY736) in Patients With Primary Immune Thrombocytopenia (ITP) Previously Treated With at Least Two Lines of Therapies

The purpose of this study is to evaluate the therapeutic efficacy, safety and tolerability of ianalumab in adult patients with primary ITP previously treated with at least one corticosteroid and one TPO-RA.

A Phase Ib/II Clinical Trial of Multiple Doses of STSA-1301 Subcutaneous Injection in Healthy Subjects and Patients With Immune Thrombocytopenia (ITP)

A randomized, double-blind, placebo-controlled design was designed to evaluate the safety and tolerability, pharmacokinetic/pharmacodynamic profile, and immunogenicity of multiple administration of STSA-1301 subcutaneous injection in healthy subjects and patients with ITP, and to further explore the initial efficacy of STSA-1301 subcutaneous injection in patients with ITP

Sequential rhTPO and Eltrombopag Following Glucocorticoids for Severe Adult ITP

To address the clinical need for improving early response rates, maintaining sustained responses, reducing relapse rates, and minimizing adverse events in the treatment of immune thrombocytopenia (ITP), the investigators developed a comprehensive in-hospital and post-discharge management strategy. In this study, hospitalized participants will receive a 14-day regimen of high-dose dexamethasone (HD-DXM) plus recombinant human thrombopoietin (rhTPO), followed by a 10-week course of oral eltrombopag olamine dry suspension after discharge. The investigators aim to evaluate the efficacy and safety of this sequential treatment strategy in adult ITP patients.

Safety Study of CC312 in Autoimmune Disease Patients

This study is an open-label, multiple-dose escalation, Investigator-Initiated Trial (IIT) clinical trial designed to evaluate the safety and tolerability of CC312 in adult patients with relapsed and refractory autoimmune diseases. The trial also assesses pharmacokinetics (PK) and preliminary efficacy. CC312 is a trispecific T cell engager (TriTE) that targets the B cell surface antigen CD19, the T cell antigen CD3, and the T cell co-stimulatory molecule CD28. Given its mechanism of action, which is similar to the "biopharmaceutical version" of CAR-T, there is a higher risk of cytokine release syndrome (CRS) at the onset of infusion administration. Therefore, a lower priming dose will be administered before the therapeutic dosing phase to mitigate this risk and ensure safety, followed by a therapeutic dose to achieve and maintain efficacy. The study is divided into three dose groups, with 3-6 subjects enrolled in each group, resulting in a total of 9-18 subjects in the study. A "3+3" dose escalation design is employed to systematically evaluate the safety and determine the optimal dose of CC312.

A PHASE II TRIAL AIMING TO INVESTIGATE THE SAFETY AND CLINICAL ACTIVITY OF BELANTAMAB MAFODOTIN IN ADULT PATIENTS WITH PRIMARY IMMUNE THROMBOCYTOPENIA PREVIOUSLY TREATED WITH A THROMBOPOIETIN RECEPTOR AGONIST AND/OR RITUXIMAB AFTER CORTICOSTEROID FIRST-LINE THERAPY

This is a Phase II open label, prospective, multicenter trial designed to assess the safety and clinical activity of belantamab mafodotin in adult patients with primary immune thrombocytopenia (ITP) previously treated with a thrombopoietin receptor agonist (TPO-RA) and/or rituximab after first-line treatment with corticosteroids. Overall, 14 participants will be enrolled in the trial. Participants' follow-up will continue for up to 12 months after the last participant is enrolled. The accrual period will be approximately 12 months. Trial treatment will be given in 28-day cycles for a total period of one year per patient or until treatment failure, physician decision, unacceptable toxicity, withdrawal of consent, or death (whichever occurs first). Participants without confirmed Response (R) or better after two infusions with belantamab mafodotin will be discontinued from trial treatment and will not be replaced.

Romiplostim N01 Combined With Glucocorticoids as the First-line Treatment for Newly Diagnosed Adult Primary Immune Thrombocytopenia: A Multicenter, Interventional Trial

This prospective, multicenter, randomized study aim to evaluate the efficacy and safety of romiplostim N01 combined with glucocorticoids as the first-line treatment for newly diagnosed adult primary immune thrombocytopenia (ITP).

RESTI Registry: Spanish Registry of Primary Immune Thrombocytopenia and Other Immune Thrombocytopenia

Primary immune thrombocytopenia (PIT), previously referred to as idiopathic thrombocytopenic purpura, is an acquired disease characterized by accelerated platelet destruction and deficient platelet production by megakaryocytes. Although the existence of PIT has been known for decades, there is no consensus on its triggering mechanisms. On the other hand, this disease presents a very heterogeneous patient profile, both in its characteristics and treatments. In this sense, the diagnosis is still made by excluding other causes of thrombocytopenia. There is insufficient epidemiological data on the disease, a fact that is especially evident in our country. We have no data on the incidence or prevalence of this pathology in Spain. For all these reasons, this epidemiological study has been designed to increase the available knowledge about this disease and the detailed characterization of the population of patients with ITP in Spain. The main objective of this study is to describe the clinical, analytical and treatment characteristics of primary immune thrombocytopenia and other immune thrombocytopenia. The study is an epidemiological, ambispective, multicenter study carried out in hospitals in Spain. Information will be collected on the disease under study, as well as on the treatments received, using the patient's clinical history as a source document.

Phase III Study on HMPL-523 for Treatment of ITP

The purpose of this study is to determine whether HMPL-523 (sovleplenib) is safe and effective in the treatment of chronic Immune Thrombocytopenic Purpura (ITP).

Efficacy and Safety of Subcutaneous Belimumab or Placebo in Addition of Rituximab in Persistent or Chronic Immune Thrombocytopenia

Primary immune thrombocytopenia (ITP) is an autoimmune disease mainly mediated by autoreactive B cells and the presence of pathogenic anti-platelet auto-antibodies that enhance platelet destruction and impair platelet production. There are approximately 4,000 newly diagnosed ITP cases each year in France. For patients with a platelet count of less than 30x109/L and/or bleeding symptoms, corticosteroids alone or in combination with intravenous immunoglobulin (IVIg) is the standard first-line treatment. However, approximately two-thirds of adult patients responding to this first-line treatment relapse within days or weeks after corticosteroids withdrawal and overall, the course of the disease is chronic in about 70% of the cases. The anti-CD20 monoclonal antibody rituximab is commonly used off-label as a second-line therapy in many European countries including France for adults with persistent (i.e., disease duration of more than 3 months) or chronic (disease duration of more than 12 months) ITP. Rituximab leads to an overall response rate of only 40 % at 1 year but 29.5% of lasting (5 years and more) response The investigators have shown that the absence of response to rituximab in ITP could be explained by the settlement and expansion of long-lived autoreactive plasma cells in the spleen made possible by the high amount of BAFF. Belimumab is a fully humanized anti-BAFF/Blys monoclonal Ab licensed for SLE. Based on the preliminary results of a phase 2 open prospective pilot study performed in our center combining rituximab with i.v belimumab seems highly promising We hypothesized that combining subcutaneous belimumab weekly over a 24 weeks period (Arm A) with rituximab is superior to rituximab and subcutaneous placebo weekly over 24 weeks period (Arm B) to achieve an overall response at W52. The study design will be a prospective randomized, double-blind, multicenter (international), superiority phase III clinical study