NCT07453368

Orelabrutinib in the Treatment of Relapsed/Refractory AIHA

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

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Peking Union Medical College Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

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.

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.

Conditions

• Autoimmune Hemolytic Anemia (AIHA)

Interventions

Type	Name	Description
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.
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.
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.

Timeline

• Start date: 2026-03-01
• Primary completion: 2027-03-01
• Completion: 2027-03-01
• First posted: 2026-03-06
• Last updated: 2026-03-09

Locations

1 site across 1 country: China