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

NCT07400250 | Not Yet Recruiting Phase 2

• 2 other identifiers: PUMCH-ITP-IIInnoCare-ITP-001
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Primary Immune Thrombocytopenia (ITP)

Keywords

primary immune thrombocytopenia; orelabrutinib; romiplostim N01

Outcome Measures

Primary Outcomes (1)

• 24-Week Sustained Platelet Response Rate

  The proportion of subjects with a platelet count (PLT) ≥ 50×10⁹/L in at least 4 out of the last 6 visits during the 24-week treatment period, without rescue therapy administered in the previous 4 weeks

  Up to 24 weeks

Secondary Outcomes (11)

• The sustained remission off-treatment (SROT)

  Week 56

• The cumulative number of weeks of platelet response

  Up to 24 weeks

• The time to first achievement of a platelet count ≥ 50×10⁹/L

  Up to 24 weeks

• Cumulative response time

  Up to 24 weeks

• Complete response rate

  Up to 24 weeks

Study Arms (1)

Orelabrutinib combined with Romiplostim N01

EXPERIMENTAL

The experimental arm will be treated orelabrutinib plus romiplostim N01. The study consists of three phases: 1) Core Treatment Phase (Weeks 1-24): Orelabrutinib 50mg orally once daily (fixed dose) + romiplostim N01 subcutaneously (starting dose 3ug/kg/week, administered once weekly). Platelet count and clinical symptoms are assessed weekly to adjust the dose of romiplostim N01 as appropriate, with a maximum dose of 10ug/kg/week. Patients with platelet count (PLT) \<50×10⁹/L after 28 days of maximum-dose romiplostim N01 withdraw. 2) Tapering Phase (Weeks 25-32): Eligible patients (PLT ≥50×10⁹/L in the last two core phase visits) discontinue orelabrutinib, then taper romiplostim N01 (dose reduction + extended intervals); patients with two consecutive PLT \<30×10⁹/L withdraw. 3) 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).

Drug: Orelabrutinib; Drug: Romiplostim N01

Interventions

Orelabrutinib DRUG

Orelabrutinib will be given as 50mg per day orally, week 1-24

Orelabrutinib combined with Romiplostim N01

Romiplostim N01 DRUG

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.

Orelabrutinib combined with Romiplostim N01

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects voluntarily participate in this study and provide written informed consent；
• Age 18-80 years, inclusive, regardless of gender；
• ECOG score 0 to 2；
• Documented diagnosis of chronic primary Immune Thrombocytopenia (ITP) with a disease duration \>12 months；
• Patients with an inadequate sustained response, relapse, intolerance, or insufficient response to first-line ITP therapy (corticosteroids and/or intravenous immunoglobulin). Prior receipt of other ITP treatments is allowed, with no limit on the number of prior lines；
• A history of response to prior standard ITP therapy (defined as achieving a platelet count ≥50×10⁹/L)；
• During or following the most recent ITP treatment, patients must have experienced either: treatment failure (platelet count \<30×10⁹/L after treatment, or failure to double the baseline count, or occurrence of bleeding), relapse after initial response (platelet count decreased to \<30×10⁹/L, or fell below twice the baseline, or bleeding symptoms recurred), treatment intolerance, or an inability to maintain response after treatment discontinuation；
• Subjects demonstrate adequate comprehension of and are able to comply with the study protocol requirements, and are willing to complete the study according to the schedule.

You may not qualify if:

• Subjects suffer from severe ITP at screening;
• Subjects have other diseases which mention in protocol;
• Subjects develop intracranial hemorrhage within 6 months prior to screening;
• Active and uncontrollable infection;
• \. Subjects have a history of coagulopathy other than ITP; 7. Subjects with a history of malignancies; 8. History of major organ transplantation or hematopoietic stem cell/bone marrow transplantation; 9. Subjects with a known history of hypersensitivity to the investigational drug as described in the Protocol, or any ingredients; 10. Subjects with a Medication history and surgical history which mention in protocol; 11. Subjects do not meet the criterion of the laboratory test in protocol.
• Withdrawal Criteria:
• If, after 4 consecutive weeks of Romiplostim N01 administration at the maximum dose (10 µg/kg once weekly), the platelet count remains \<50×10⁹/L and the investigator judges the investigational product to be ineffective for the subject, such that continued use is not in the subject's best interest；
• Subjects who are unable to successfully undergo treatment tapering or discontinuation；
• Subjects who, during the treatment period, require rescue therapy based on clinical assessment；
• Subjects who withdraw their informed consent.
• Occurrence of pregnancy during the trial period
• Poor subject compliance or a significant protocol violation；
• Loss to follow-up；
• The investigator decides that withdrawal is necessary for the subject's safety；
• Presence of other conditions, as determined by the investigator, that may affect the study results or lead to premature termination of the study；

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100010, China

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

orelabrutinib

Condition Hierarchy (Ancestors)

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

Study Officials

• Tienan Zhu, M.D.

  Peking Union Medical College Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Tienan Zhu, M.D.

CONTACT

010-69155027; zhutn@pumch.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 3, 2026
• First Posted: February 10, 2026
• Study Start: April 1, 2026
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): October 1, 2028
• Last Updated: February 10, 2026

Record last verified: 2026-01

Locations

CN (1)