Romiplostim for Oral TPO-RA Resistant ITP

NCT07612319 | Recruiting DMC

• 1 other identifier: 20251200
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

This study is a prospective, multicenter, open-label, single-arm cohort study to evaluate the effectiveness and safety of romiplostim in patients with immune thrombocytopenia (ITP) who have not responded to oral thrombopoietin receptor agonists (TPO-RAs) such as eltrombopag, hetrombopag, or avatrombopag. ITP is a blood disorder in which the immune system attacks and destroys the body's own platelets, leading to low platelet counts and an increased risk of bleeding. While oral TPO-RAs are effective for many patients, approximately 20-30% of patients do not respond adequately or lose response over time. For these patients, alternative treatments are urgently needed. Participants in this study will discontinue their current oral TPO-RA and receive romiplostim as a weekly subcutaneous injection. The starting dose is 3 µg/kg, and the dose may be adjusted weekly based on platelet counts (maximum 10 µg/kg). The total treatment and follow-up period is 24 weeks. The primary outcome measure is the durable platelet response rate at week 24, defined as maintaining platelet counts at ≥50×10⁹/L for at least two scheduled visits during weeks 22-24 without rescue therapy. Secondary outcomes include the sustained response rate at week 12, complete response rate at week 24, time to first response, improvement in bleeding events, and quality of life assessment. Safety outcomes include monitoring for thromboembolic events, bone marrow fibrosis, hepatotoxicity, injection site reactions, and other adverse events according to CTCAE v5.0. Approximately 60 participants will be enrolled across multiple centers in China. This study aims to provide evidence for romiplostim as a treatment option for ITP patients who have failed oral TPO-RAs.

Conditions

Immune (Idiopathic) Thrombocytopenic Purpura (ITP)

Keywords

Immune Thrombocytopenia; Romiplostim; Thrombopoietin Receptor Agonists; Drug Resistance

Outcome Measures

Primary Outcomes (1)

• Durable Platelet Response Rate at Week 24

  Defined as the proportion of participants achieving platelet count ≥50×10⁹/L for at least two scheduled visits during weeks 22-24, without receiving rescue therapy (IVIG, high-dose corticosteroids, or platelet transfusion) during weeks 20-24.

  24 weeks

Study Arms (1)

Romiplostim Treatment Arm

EXPERIMENTAL

Drug: Romiplostim

Interventions

Romiplostim DRUG

Romiplostim is administered as a subcutaneous injection once weekly for 24 weeks. The starting dose is 3 µg/kg. Platelet count is assessed weekly, and the dose is adjusted based on platelet count and bleeding symptoms as follows: If platelet count \<50×10⁹/L: increase dose by 1 µg/kg (maximum dose: 10 µg/kg per week) If platelet count 50-200×10⁹/L: maintain the lowest dose that reduces bleeding risk If platelet count 200-400×10⁹/L: decrease dose by 1 µg/kg If platelet count \>400×10⁹/L: withhold dosing. After platelet count decreases to \<200×10⁹/L, resume at a dose 1 µg/kg lower than the dose before withholding.

Romiplostim Treatment Arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥18 years Diagnosed with primary immune thrombocytopenia (ITP) according to current guidelines
• Failed prior oral TPO-RA therapy (eltrombopag, hetrombopag, or avatrombopag), defined as:
• Platelet count persistently \<30×10⁹/L after ≥4 weeks of treatment at the maximum recommended or tolerated dose, OR Prior achieved response (platelet ≥50×10⁹/L) then lost response with platelet count \<30×10⁹/L on two consecutive visits (≥1 week apart), OR Intolerance to oral TPO-RA leading to discontinuation (with documented reason) Baseline platelet count \<30×10⁹/L (at least two measurements, ≥5 days apart) ECOG performance status 0-2 Willing and able to receive weekly subcutaneous injections and comply with study procedures For women of childbearing potential: negative pregnancy test at screening and agreement to use effective contraception during the study and for 3 months after the last dose Able to provide written informed consent

You may not qualify if:

• Secondary ITP (e.g., associated with autoimmune diseases, lymphoproliferative disorders, drug-induced, or infections such as HIV/HCV/HBV) Known hypersensitivity to romiplostim or any of its excipients Prior treatment with romiplostim Received rituximab or other B-cell depleting therapy within 6 months prior to screening Received immunosuppressants (e.g., cyclosporine, mycophenolate mofetil, azathioprine, danazol) within 4 weeks prior to screening Splenectomy within 8 weeks prior to screening Use of corticosteroids (prednisone \>10 mg/day or equivalent) or IVIG within 2 weeks prior to screening History of bone marrow fibrosis (MF grade ≥2) or known reticulin fibrosis History of arterial or venous thromboembolic event (e.g., myocardial infarction, stroke, deep vein thrombosis, pulmonary embolism) within 6 months prior to screening Known thrombophilic conditions (e.g., antiphospholipid syndrome, protein C/S deficiency, ATIII deficiency) Severe hepatic impairment (ALT or AST \>3×ULN, or total bilirubin \>1.5×ULN) Severe renal impairment (creatinine clearance \<30 mL/min) New York Heart Association (NYHA) functional class III or IV heart failure Malignancy within 5 years prior to screening (except non-melanoma skin cancer, cervical carcinoma in situ, or ductal carcinoma in situ of the breast) Pregnancy or breastfeeding Participation in another interventional clinical trial within 30 days prior to screening Any other condition that, in the investigator's judgment, would make the participant unsuitable for the study

Sponsors & Collaborators

• The First Affiliated Hospital of Soochow University: lead
• Affiliated Hospital of Nantong University: collaborator
• Changshu Affiliated Hospital of Soochow University: collaborator
• The First People's Hospital of Changzhou: collaborator
• Shanghai Tong Ren Hospital: collaborator
• The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School: collaborator
• Zhangjiagang First People's Hospital: collaborator
• Soochow Hopes Hematonosis Hospital: collaborator

Study Sites (1)

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215000, China

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic; Purpura, Thrombocytopenic, Idiopathic

Interventions

romiplostim

Condition Hierarchy (Ancestors)

Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms; Hemorrhagic Disorders; Autoimmune Diseases

Central Study Contacts

The First Affiliated Hospital of Soochow University

CONTACT

86-0512-67781521; tianhong0718@163.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 21, 2026
• First Posted: May 28, 2026
• Study Start: March 15, 2026
• Primary Completion (Estimated): September 1, 2027
• Study Completion (Estimated): June 1, 2028
• Last Updated: May 28, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)