Orelabrutinib in the Treatment of Relapsed/Refractory AIHA

March 5, 2026 updated by: Chen Miao, Peking Union Medical College Hospital

Orelabrutinib in the Treatment of Relapsed/Refractory Autoimmune Hemolytic Anemia: A Prospective, Dose-Escalation Cohort Study

  1. wAIHA Treatment Regimen: Group A (50mg group): Orelabrutinib 50 mg, orally, once daily. After 4 weeks of treatment, if still transfusion-dependent or hemoglobin increase is < 20 g/L, the dose may be increased to 100 mg qd. Treatment can be discontinued if ineffective at 12 weeks.

    Group B (100mg group): Orelabrutinib 100 mg, orally, once daily. The treatment course is at least 12 weeks. Treatment can be discontinued if ineffective at 12 weeks. Patients who respond and tolerate the drug well may continue treatment for up to 52 weeks or longer to observe long-term efficacy and safety.

  2. cAIHA Treatment Regimen: Group C (150mg group): Orelabrutinib 150 mg, orally, once daily. The treatment course is at least 12 weeks. Patients who respond and tolerate the drug well may continue treatment for up to 52 weeks or longer to observe long-term efficacy and safety.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Detailed Description

  1. wAIHA Treatment Regimen: Group A (50mg group): Orelabrutinib 50 mg, orally, once daily. After 4 weeks of treatment, if still transfusion-dependent or hemoglobin increase is < 20 g/L, the dose may be increased to 100 mg qd. Treatment can be discontinued if ineffective at 12 weeks.

    Stable doses of glucocorticoids (e.g., prednisone ≤ 15 mg/day) are permitted, and their tapering process should be recorded.

    Group B (100mg group): Orelabrutinib 100 mg, orally, once daily. The treatment course is at least 12 weeks. Treatment can be discontinued if ineffective at 12 weeks. Patients who respond and tolerate the drug well may continue treatment for up to 52 weeks or longer to observe long-term efficacy and safety.

    Stable doses of glucocorticoids (e.g., prednisone ≤ 15 mg/day) are permitted, and their tapering process should be recorded.

  2. cAIHA Treatment Regimen: Group C (150mg group): Orelabrutinib 150 mg, orally, once daily. The treatment course is at least 12 weeks. Patients who respond and tolerate the drug well may continue treatment for up to 52 weeks or longer to observe long-term efficacy and safety.
  3. Red blood cell transfusion is permitted when hemoglobin (HGB) is below 60 g/L, or under necessary conditions. Platelet transfusion is permitted when platelet count is below 10×10^9^/L or with significant bleeding tendency. To ensure patient safety, in the above situations, intravenous immunoglobulin (IVIG) infusion for no more than 5 days or glucocorticoids at 1-2 mg/kg/day for no more than 2 weeks are allowed. Efficacy will not be assessed within 8 weeks of receiving IVIG or glucocorticoids. Recombinant human erythropoietin at 10,000-20,000 units/week is allowed.
  4. If the neutrophil count falls below 1.0×10^9^/L, G-CSF may be used until recovery to above 1.0×10^9^/L.
  5. Follow-up schedule: Every 1-2 weeks during the first month; at least monthly from the second month onwards; at least every 2 months from the fourth month onwards. Record patient symptoms, treatment-related adverse events, signs, transfusion requirements, direct antiglobulin test (DAT), complete blood count (including reticulocytes), biochemistry (liver and kidney function) to monitor efficacy and safety. At 12 weeks, 24 weeks, and 52 weeks, re-assess immunoglobulins, lymphocyte subset analysis, and cytokines to evaluate immune function status.

Study Type

Interventional

Enrollment (Estimated)

50

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • China
    • Beijing Municipality
      • Beijing, Beijing Municipality, China, 100730
        • Peking Union Medical College Hospital
        • Principal Investigator:
          • Miao Chen
        • Contact:
        • Contact:
        • Principal Investigator:
          • Chuanhuan Liu

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Age ≥ 18 years.
  • Patients with a definitive diagnosis of AIHA. wAIHA or Evans syndrome, primary or secondary to connective tissue disease (CTD) or lymphoproliferative disorder (LPD). If secondary, CTD should have no indication for treatment for other system involvement, and LPD should be in an asymptomatic, observation phase without treatment indication.

cAIHA, primary or secondary to LPD. If secondary, LPD should be in an asymptomatic, observation phase without treatment indication.

  • For wAIHA: No response (no partial response after 3 weeks) to full-dose glucocorticoid therapy (1-2 mg/kg/day), intolerance, or relapse (after initial response, hemoglobin drop ≥20 g/L or requiring re-initiation of immunosuppressive therapy). And hemoglobin ≤ 100 g/L.
  • For cAIHA: No response or relapse after anti-CD20 monoclonal antibody therapy (after initial response, hemoglobin drop ≥20 g/L or recurrence of vascular symptoms, or requiring re-initiation of immunosuppressive therapy), or unsuitable for anti-CD20 therapy. And hemoglobin ≤ 100 g/L or presence of vascular symptoms.
  • Baseline liver and kidney function (ALT, Cr) within 2 times the upper limit of normal (elevated AST, LDH, Bil due to hemolysis are not used as indicators for liver function assessment).
  • Agreement to sign the informed consent form.

Exclusion Criteria:

  • Active involvement of other major organs due to connective tissue disease.
  • Uncontrolled infection or bleeding per standard treatment.
  • Uncontrolled active HIV, HCV, or HBV infection per standard treatment.
  • Concurrent advanced uncontrolled malignancy, lymphoma.
  • Presence of other uncorrected types of anemia at screening, such as nutritional anemia, thalassemia, etc.
  • Currently receiving glucocorticoid therapy at screening and unable to discontinue or taper to ≤ 15 mg/day (prednisone equivalent) within 1 week before enrollment.
  • Currently receiving treatment with medications such as cyclosporine, tacrolimus, sirolimus, cyclophosphamide, azathioprine, stanozolol, testosterone undecanoate, danazol, etc., at screening, and the treatment duration at a stable dose is < 12 weeks, or unable to discontinue directly.
  • Last dose of rituximab < 3 months prior to enrollment.
  • Prior treatment with any BTK inhibitor.
  • Liver cirrhosis or portal hypertension.
  • Pregnant or breastfeeding women.
  • Participation in another clinical trial within the past 3 months.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: wAIHA-A

Orelabrutinib 50 mg, orally, once daily.After 4 weeks of treatment, if still transfusion-dependent or hemoglobin increase is < 20 g/L, the dose may be increased to 100 mg qd. Treatment can be discontinued if ineffective at 12 weeks.

Stable doses of glucocorticoids (e.g., prednisone ≤ 15 mg/day) are permitted, and their tapering process should be recorded.
Drug: Orelabrutinib-50

Orelabrutinib 50 mg, orally, once daily. After 4 weeks of treatment, if still transfusion-dependent or hemoglobin increase is < 20 g/L, the dose may be increased to 100 mg qd. Treatment can be discontinued if ineffective at 12 weeks.

Stable doses of glucocorticoids (e.g., prednisone ≤ 15 mg/day) are permitted, and their tapering process should be recorded.

Other Names:
  • wAIHA-A
Experimental: wAIHA-B

Orelabrutinib 100 mg, orally, once daily. The treatment course is at least 12 weeks. Treatment can be discontinued if ineffective at 12 weeks. Patients who respond and tolerate the drug well may continue treatment for up to 52 weeks or longer to observe long-term efficacy and safety.

Stable doses of glucocorticoids (e.g., prednisone ≤ 15 mg/day) are permitted, and their tapering process should be recorded.
Drug: Orelabrutinib-100

Orelabrutinib 100 mg, orally, once daily. The treatment course is at least 12 weeks. Treatment can be discontinued if ineffective at 12 weeks. Patients who respond and tolerate the drug well may continue treatment for up to 52 weeks or longer to observe long-term efficacy and safety.

Stable doses of glucocorticoids (e.g., prednisone ≤ 15 mg/day) are permitted, and their tapering process should be recorded.

Other Names:
  • wAIHA-B
Experimental: cAIHA-C
Orelabrutinib 150 mg, orally, once daily. The treatment course is at least 12 weeks. Patients who respond and tolerate the drug well may continue treatment for up to 52 weeks or longer to observe long-term efficacy and safety.
Drug: Orelabrutinib-150
Orelabrutinib 150 mg, orally, once daily. The treatment course is at least 12 weeks. Patients who respond and tolerate the drug well may continue treatment for up to 52 weeks or longer to observe long-term efficacy and safety.
Other Names:
  • wAIHA-C

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
ORR
Time Frame: 12weeks
Overall Response Rate (ORR) at 12 weeks. Overall Response Rate (ORR) is defined as the proportion of patients achieving Complete Response (CR) + Partial Response (PR).
12weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The incidence of adverse events
Time Frame: 12weeks
Safety analysis includes assessing the incidence and severity of adverse events; all adverse events occurring or worsening during treatment, as well as later events considered by the investigator to be related to the study drug, will be reported.
12weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Peking Union Medical College Hospital

Investigators

  • Principal Investigator: Miao Chen, Peking Union Medical College

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

March 1, 2026

Primary Completion (Estimated)

March 1, 2027

Study Completion (Estimated)

March 1, 2027

Study Registration Dates

First Submitted

March 1, 2026

First Submitted That Met QC Criteria

March 1, 2026

First Posted (Actual)

March 6, 2026

Study Record Updates

Last Update Posted (Actual)

March 9, 2026

Last Update Submitted That Met QC Criteria

March 5, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Orelabrutinib-AIHA

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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