ClinicalTrials.gov record NCT07453368

Orelabrutinib in the Treatment of Relapsed/Refractory AIHA

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Aug 22, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
Age18 years old to 85 years old
SexAll
LocationChina
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 2 interventional study evaluates oral orelabrutinib in people with relapsed or refractory autoimmune hemolytic anemia (AIHA), including warm and cold types. Participants are assigned to one of three daily dose groups (50 mg with optional increase to 100 mg, 100 mg, or 150 mg) for at least 12 weeks; responders may continue treatment up to about 52 weeks or longer. The study measures response at 12 weeks and monitors safety and immune markers over time. The trial is randomized, parallel-group, and open-label (no masking).

What it is studying. Whether oral orelabrutinib given at different daily doses leads to hematologic responses and what adverse events and immune changes occur in people with relapsed/refractory AIHA.

What the researchers hope to learn. Primary scientific goal: measure overall response rate (complete + partial responses) at 12 weeks. Secondary goals: assess the incidence and severity of adverse events and monitor immune function (immunoglobulins, lymphocyte subsets, cytokines) and longer-term efficacy/safety.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

  • 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.

  • 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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Orelabrutinib-50
  • Orelabrutinib-100
  • Orelabrutinib-150

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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 description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Show the detailed description from the registry
  • 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.

  • 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.
  • 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.
  • 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.
  • 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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants take daily oral orelabrutinib (assigned to 50 mg, 100 mg, or 150 mg groups) for at least 12 weeks; some wAIHA patients may have a dose increase after 4 weeks, and responders may continue treatment up to 52 weeks or longer. They attend frequent clinic visits (every 1–2 weeks in month 1, at least monthly in month 2, then at least every 2 months from month 4) with blood tests to monitor hemoglobin, reticulocytes, DAT, liver and kidney function, and adverse events. Immune tests (immunoglobulins, lymphocyte subsets, cytokines) are reassessed at weeks 12, 24, and 52. Supportive care allowed includes red blood cell or platelet transfusion when needed, short courses of IVIG (≤5 days) or glucocorticoids at 1–2 mg/kg/day for ≤2 weeks in emergencies (efficacy is not assessed within 8 weeks after receiving IVIG or glucocorticoids); recombinant erythropoietin and G‑CSF use are also permitted per the protocol.

How the study is structured. Phase 2 interventional drug trial. Participants are randomly assigned to one of three parallel dose groups and receive daily oral orelabrutinib for at least 12 weeks; the trial is open-label (no masking). Blood tests and safety monitoring are frequent early on and continue at scheduled visits; some immune tests are repeated at 12, 24, and 52 weeks. Responders who tolerate the drug may stay on treatment longer to observe longer-term effects.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on autoimmune hemolytic anemia

Clinical trials49 trials listed for autoimmune hemolytic anemiaEvery trial the registry lists for autoimmune hemolytic anemia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in autoimmune hemolytic anemia researchLatest monthly update: September 2026. Includes: Negative DAT cases. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Mar 1, 2026
Primary completion
Mar 1, 2027 (estimated)
Study completion
Mar 1, 2028 (estimated)
Target enrollment
50 participants (estimated)
Last updated on registry
Jun 16, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Peking Union Medical College Hospital
Principal investigator
Chen Miao, Director
Peking Union Medical College Hospital

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

glucocorticoid
Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
relapse
A condition that improved with treatment and has since returned or worsened.
immunosuppressive
A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
Baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
upper limit of normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
informed consent form
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
standard treatment
Treatment generally considered appropriate for a condition in a particular clinical setting. It can vary by patient, place and over time. In many studies the new treatment is added to, or compared with, standard of care.
at screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
stable dose
A dose that has not changed for a defined period before the study starts. Studies may require this so that background treatment is relatively stable when study measurements begin.
Prior treatment
Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
neutrophil count
The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
Follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
adverse events
Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

← Back to clinical trials