Eltrombopag Plus Telitacicept vs. Eltrombopag Alone for Steroid-refractory/Relapsed ITP

Official title

Eltrombopag Combined With Telitacicept Versus Eltrombopag Monotherapy for the Treatment of Immune Thrombocytopenia Refractory or Relapsed to Corticosteroid Therapy: A Randomized Exploratory, Controlled, Open-label, Phase II Clinical Study

Overview

Background: Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder. Corticosteroids are first-line therapy, but about one-third of patients relapse or are refractory. Eltrombopag (a TPO-RA) promotes platelet production, yet some patients show no response or relapse upon discontinuation. Telitacicept, a TACI-Fc fusion protein, dual-targets BAFF and APRIL, inhibiting B cell and plasma cell function and reducing autoantibody production, potentially providing synergistic immunomodulatory benefit. Objective: To evaluate the sustained response rate of eltrombopag plus telitacicept vs. eltrombopag alone in patients with steroid-refractory/relapsed ITP. Design: Randomized, open-label, controlled, exploratory Phase II study. 40 patients planned (20 combination, 20 monotherapy). Combination group: eltrombopag + telitacicept . Monotherapy group: eltrombopag alone for 12 weeks. Monotherapy patients with no response after 4 weeks may cross over to the combination group. After 12 weeks, treatment is stopped and patients are followed until Week 24. Primary endpoint: Proportion of patients maintaining platelet count ≥30×10⁹/L with no bleeding at 24 weeks post-treatment. Secondary endpoints include platelet response rates during 12 weeks, safety, bleeding events, etc. Expected results: The combination group is expected to have a significantly higher proportion of patients achieving the primary endpoint without increased adverse events. Population: Age ≥18, diagnosed ITP ≥3 months, baseline platelets \<30×10⁹/L, prior corticosteroid failure. Safety: Monitoring for bleeding, infection, thrombosis, cytopenia, etc., with dose adjustment/cessation as per protocol. Conclusion: This study explores whether dual-targeting (platelet production + autoimmune suppression) with eltrombopag and telitacicept can provide more durable remission for steroid-refractory/relapsed ITP patients.

Interventions

• Drug Eltrombopag
  eltrombopag plus telitacicept vs. eltrombopag alone

Primary outcome measures

• Sustained Response Comparison [Time frame: Within 24weeks of first dose]

Secondary outcome measures (9)

• Platelet Response Within 12 Weeks [Time frame: Within 12 weeks of first dose]
• Platelet ≥50×10⁹/L at Week 12 [Time frame: Within 12 weeks of first dose]
• Platelet ≥100×10⁹/L at Week 12 [Time frame: Within 12 weeks of first dose]
• Time to First Platelet Response [Time frame: Within 12 weeks of first dose]
• Proportion of Days with Platelet ≥30×10⁹/L [Time frame: Within 12 weeks of first dose]
• Platelet Response After Crossover (Monotherapy Group) [Time frame: With in 24 weeks of first dose]
• Safety and Tolerability [Time frame: Within 24 weeks of first dose]
• Time to Rescue Therapy or Platelet Decline [Time frame: Within 24 weeks of first dose]
• Proportion Requiring Rescue Therapy [Time frame: Within 24 weeks of first dose]

Eligibility criteria

Inclusion criteria

• Age ≥18 years, regardless of sex.
• Clinically diagnosed with immune thrombocytopenia for at least 3 months prior to enrollment. Platelet count <30×10⁹/L within 48 hours before the first dose of study drug.
• Previous failure (ineffective, unable to maintain response, or relapse) to first-line standard corticosteroid therapy for ITP as recommended by guidelines.
• Any prior emergency treatment for ITP (e.g., corticosteroids, platelet transfusion, intravenous immunoglobulin) must have been completed at least 2 weeks before the first dose.
• Patients receiving maintenance corticosteroid therapy must be on a stable dose for at least 2 weeks prior to the first dose; patients receiving immunosuppressants (e.g., azathioprine, danazol, cyclosporine A, tacrolimus, sirolimus, etc.) must be on a stable dose for at least 4 weeks prior to the first dose; anti-CD20 antibody therapy must have been completed >3 months prior.

Understand the study procedures and voluntarily provide written informed consent.

Exclusion criteria

• Received anti-CD20 antibody therapy within 3 months.
• Uncontrolled primary disease of vital organs, such as malignant tumors, liver failure, heart failure, renal failure, etc.
• Positive for HIV.
• Uncontrolled active viral or bacterial infections, including positive for hepatitis B, hepatitis C, cytomegalovirus, Epstein-Barr virus, or syphilis.
• Extensive and severe bleeding, such as hemoptysis, upper gastrointestinal hemorrhage, intracranial hemorrhage, etc.
• Currently have cardiac disease requiring treatment, arrhythmia, or hypertension poorly controlled as judged by the investigator.
• Patients with thrombotic diseases such as pulmonary embolism, thrombosis, atherosclerosis, etc.
• Patients with mental disorders who are unable to give informed consent or undergo study procedures and follow-up normally.
• Patients whose toxic symptoms from prior treatment before enrollment have not yet resolved.
• Other serious diseases that may limit the patient's participation in this study (e.g., poorly controlled diabetes; severe cardiac insufficiency; myocardial infarction, unstable arrhythmia, or unstable angina within the past 6 months; gastric ulcer; active autoimmune disease, etc.).
• Pregnant women, suspected pregnancy (positive urinary human chorionic gonadotropin pregnancy test at screening), or lactating patients.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Open label

Primary purpose

Treatment

Study locations

China · 1 center

• Ethics Committee of Blood disease hospital, Chinese Academy of Medical Sciences — Tianjin

Identifiers

NCT: NCT07687134 · TACI-ITP-2026

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗