Ripertamab Plus Eltrombopag vs. Eltrombopag in ITP Patients Post-Steroid Failure (RPE-ITP)

A Multicenter, Randomized Controlled, Open-Label Study of Ripertamab Combined With Eltrombopag Versus Eltrombopag for ITP Patients After First-Line Steroid Therapy

This study aims to evaluate the efficacy and safety of Ripertamab in combination with Eltrombopag compared to Eltrombopag alone for patients with Primary Immune Thrombocytopenia (ITP) who have not responded to or have relapsed after first-line steroid therapy. Participants will be randomly assigned to one of two treatment groups and followed for 52 weeks to assess response rates and safety.

Study Overview

• Status: Not yet recruiting
• Conditions: ITP - Immune Thrombocytopenia
• Intervention / Treatment: Combination product: Ripertamab/Eltrombopag; Drug: Eltrombopag

Detailed Description

This is a multicenter, open-label, randomized controlled clinical trial designed to compare the effects of Ripertamab plus Eltrombopag versus Eltrombopag monotherapy in adults with ITP. The study will enroll approximately 78 participants, aged 18 to 80 years, who have had an inadequate response to or relapsed after first-line steroid therapy. The primary outcome measure is the sustained response rate at 12 weeks post-treatment initiation. Secondary outcomes include overall response rate, complete response rate, median time to response, median sustained response time, and safety. Participants will undergo regular blood tests and clinical assessments throughout the study period.

• Study Type: Interventional
• Enrollment (Estimated): 78
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Heng Mei, Ph.D&M.D; Phone Number: 027-8572600 027-8572600; Email: hmei@hust.edu.cn
• Study Contact Backup: Name: QZ Wei, Ph.D.; Phone Number: 15271897896; Email: 576346468@qq.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Diagnosis: Confirmed Primary Immune Thrombocytopenia (ITP) with an age range of 18 to 80 years, inclusive, and no gender restrictions.
2. Bone Marrow Cytology: Diagnosis via bone marrow cytology excluding secondary thrombocytopenia caused by other diseases, with primary ITP patients who are unresponsive to or have relapsed after first-line steroid therapy (platelet count drops below 30×10^9/L).
3. Coagulation Status: Laboratory tests show prothrombin time (PT/INR) and activated partial thromboplastin time (APTT) not exceeding 20% above the upper limit of normal values, with no history of coagulation disorders other than ITP.
4. Bone Marrow Function: Normal bone marrow function indicated by an absolute neutrophil count (ANC) ≥1.5×10^9/L and hemoglobin (Hb) ≥90 g/L.

Liver and Kidney Function: Normal liver and kidney function with serum direct bilirubin and indirect bilirubin ≤1.5 times the upper limit of normal (ULN); 5.alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤3 times the upper limit of normal (ULN); serum creatinine ≤1.5 times the upper limit of normal (ULN).

6.Psychiatric History: No history of psychiatric disorders. 7.Informed Consent: Voluntary signing of an informed consent form.

Exclusion Criteria:

1. Life-Threatening Bleeding: Presence of bleeding that poses an immediate threat to life, such as severe anemia or central nervous system bleeding.
2. Recent Treatment: Use of intravenous immunoglobulin, thrombopoietin receptor agonists, recombinant human thrombopoietin (rhTPO), immunosuppressants, anti-CD20 monoclonal antibodies, or systemic corticosteroids within 28 days prior to enrollment.
3. Malignancy History: History of malignancy.
4. Cardiac Conditions: Presence of severe heart failure or other diseases significantly impacting cardiac function (e.g., unstable angina, congestive heart failure, uncontrolled hypertension, arrhythmias, or prolonged QTc interval within the last 3 months).
5. Coagulation Disorders: History of coagulation disorders other than ITP, such as disseminated intravascular coagulation (DIC), hemolytic uremic syndrome (HUS), or thrombotic thrombocytopenic purpura (TTPP).
6. Viral Markers: Hepatitis C virus (HCV) antibody positive with HCV RNA quantitative test ≥10^4 copies/mL; or Hepatitis B virus (HBV) markers positive for HBsAg and/or HBcAb with HBV DNA positivity.
7. Immunocompromised: History of immunodeficiency, including HIV positivity or other acquired or congenital immunodeficiency diseases, autoimmune diseases, or a history of organ transplantation.
8. Tuberculosis: Suspected active or latent tuberculosis.
9. Active Infections: Presence of active infections at enrollment, or any significant infectious events within 4 weeks prior to enrollment or major surgery within 4 weeks.
10. Pregnancy and Lactation: Pregnant or nursing women, or women of childbearing potential or considering pregnancy during the study period.
11. Other Conditions: Any other conditions deemed by the investigator as contraindications for study participation.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Eltrombopag/Ripertamab; Eltrombopag (starting at 2.5mg once daily, 28 days, adjusted based on platelet count),Ripertamab (375 mg/m² BSA on Day 1)，If the participant remains in remission at the 3-month, an additional intravenous dose of 375 mg/m² BSA Ripertamab is administered	Combination product: Ripertamab/Eltrombopag; Ripertamab, a novel anti-CD20 monoclonal antibody, in combination with Eltrombopag . Ripertamab is administered intravenously at a dose of 375 mg/m² BSA on Day 1 . If the participant remains in remission at the 3-month mark, an additional intravenous dose of 375 mg/m² Ripertamab is administered. Eltrombopag are given orally at an initial dose of 2.5 mg once daily, with dosage adjustments made every two weeks based on platelet count response, for a total treatment duration of up to Day 28.; Other Names: Anti-CD20/TPO-RA
Active Comparator: Eltrombopag; Eltrombopag (starting at 2.5mg once daily, 28 days, adjusted based on platelet count)	Drug: Eltrombopag; Eltrombopag as a single therapy. Eltrombopag is a thrombopoietin receptor agonist given orally at an initial dose of 2.5 mg once daily, with dosage adjustments made every two weeks based on platelet count response, for a total treatment duration of up to Day 28.; Other Names: TPO-RA

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Sustained Response Rate at 12 Weeks	The primary outcome measure is the proportion of patients who achieve a sustained response at 12 weeks after the initiation of treatment. A sustained response is defined as a platelet count of ≥30×10^9/L without bleeding and without the need for rescue therapy.	up to 12weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Response Rate (ORR)	The percentage of patients who achieve any response (complete or partial) to the treatment as measured by an increase in platelet count from baseline.	through study completion, an average of 1 year
Complete Response Rate (CR%)	The proportion of patients who achieve a complete response, defined as a platelet count of ≥100×10^9/L without bleeding and without the need for rescue therapy.	through study completion, an average of 1 year
Median Time to Response (mTTR)	The time at which 50% of the patients achieve a response, measured from the start of treatment until the first evidence of therapeutic effect.	1 year
Median Sustained Response Time (mSRT)	The time at which 50% of the patients maintain a response without relapse, measured from the start of treatment until the response is no longer sustained.	1 year
24-week Sustained Response Rate (24-week SR)	The proportion of patients who maintain a sustained response at 24 weeks post-treatment initiation, defined by a platelet count of ≥100×10^9/L without bleeding and without rescue therapy.	up to 24 weeks
52-week Sustained Response Rate (52-week SR)	The percentage of patients who continue to show a sustained response at 52 weeks after starting treatment, with platelet counts meeting predefined criteria.	up to 52 weeks

Collaborators and Investigators

• Sponsor: Wuhan Union Hospital, China
• Collaborators: Sinocelltech Ltd.
• Investigators: Study Chair: Heng MEI, Ph.D&M.D, Wuhan Union Hospital, China

Study record dates

Study Major Dates

• Study Start (Estimated): February 1, 2025
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027

Study Registration Dates

• First Submitted: December 15, 2024
• First Submitted That Met QC Criteria: January 19, 2025
• First Posted (Actual): March 25, 2025

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 19, 2025
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Keywords: Ripertamab
• Additional Relevant MeSH Terms: Cytopenia; Pathologic Processes; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Skin Manifestations; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Thrombocytopenia; Purpura, Thrombocytopenic, Idiopathic
• Other Study ID Numbers: UHCT240527

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No