Orelabrutinib in the Treatment of Relapsed/Refractory AIHA

Recruiting now · Phase 2

Conditions studied: Autoimmune Hemolytic Anemia (AIHA)

In brief

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.

Key facts

Study ID

NCT07453368

Run by

Peking Union Medical College Hospital

People needed

50

Starts

2026-03-01

Expected to finish

2028-03-01

Last updated by the study team

2026-06-16

Who can join

Age: 18 and older, up to 85. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Peking Union Medical College Hospital — Beijing, Beijing Municipality, China (enrolling)

Full record on ClinicalTrials.gov

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