NCT07400250

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

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

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 28 (estimated)
• Sponsor: Peking Union Medical College Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

This is a prospective, single-arm, open-label Phase II study, enrolling adult patients with chronic primary immune thrombocytopenia (ITP) who failed first-line therapy. The study evaluates the efficacy and safety of orelabrutinib combined with romiplostim N01, focusing on the treatment regimen as follows: * Core Treatment Phase (Weeks 1-24) Patients receive orelabrutinib 50mg orally once daily (fixed dose) and romiplostim N01 subcutaneously. Romiplostim N01 starts at 250ug/week (≈3ug/Kg) and is titrated within 1-10ug/Kg/week to maintain platelet count (PLT) at 50-200×10⁹/L. Dose adjustments: increase by 1-3ug/Kg/week if PLT \<50×10⁹/L; decrease by 1-3ug/Kg/week if PLT 200-400×10⁹/L; suspend if PLT \>400×10⁹/L. Patients with PLT \<50×10⁹/L after 28 days of maximum-dose romiplostim N01 withdraw. * Romiplostim N01 Tapering Phase (Weeks 25-32) Patients with PLT ≥50×10⁹/L in the last two core phase visits discontinue orelabrutinib. Romiplostim N01 is tapered: doses \>3ug/Kg/week are reduced to 2-3ug/Kg/week, then dosing intervals extended (weekly→every 10 days→every 2 weeks). Patients with two consecutive PLT \<30×10⁹/L withdraw. * Follow-up Phase (Weeks 33-56) Successfully tapered patients are followed up every 4 weeks to monitor PLT and adverse events (graded per NCI-CTC AE 5.0). Relevant events are reported to the sponsor's Pharmacovigilance Department.

Conditions

• Primary Immune Thrombocytopenia (ITP)

Interventions

Type	Name	Description
DRUG	Orelabrutinib	Orelabrutinib will be given as 50mg per day orally, week 1-24
DRUG	Romiplostim N01	Core Treatment Phase Recommended starting dose: 3 μg/kg subcutaneously once weekly. Monitor platelet count (PLT) and symptoms weekly for dose adjustment, max 10 μg/kg/week. Therapeutic target: Maintain PLT within the range of 50-200×10⁹/L. 1. PLT \< 50×10⁹/L: Increase by 1-3 μg/kg/week to max dose. Discontinue study if PLT remains \< 50×10⁹/L after 28d of max dose. 2. 50-200×10⁹/L: Maintain the minimum effective dose to reduce bleeding risk. 3. 200-400×10⁹/L: Decrease by 1-3 μg/kg/week. Discontinue if PLT ≥200×10⁹/L at 250 μg/week; resume when PLT \<50×10⁹/L (extend interval if needed). 4. PLT \>400×10⁹/L: Suspend; resume at 1-3 μg/kg lower dose when PLT \<200×10⁹/L (weekly monitoring). Tapering Phase For maintenance dose \>3 μg/kg/week: Taper by 1-3 μg/kg/week to ≤3 μg/kg (250 μg/week), then extend intervals (weekly→every 10 days→every 2 weeks). 1. PLT \<30×10⁹/L: Maintain current dose. 2. Two consecutive PLT \<30×10⁹/L: Discontinue study.

Timeline

• Start date: 2026-04-01
• Primary completion: 2026-10-01
• Completion: 2028-10-01
• First posted: 2026-02-10
• Last updated: 2026-02-10

Locations

1 site across 1 country: China