UnknownPhase 2

The Safety and Efficacy of Zanubrutinib in Refractory/Relapsed Autoimmune Hemolytic Anemia

China33 participantsStarted 2023-09-01

Plain-language summary

The sample size of this study is calculated based on Simon's two-stage design. The first stage of the study enrolled a cohort of 12 patients. If after 12 weeks at least 6 patients achieved a response, then enrollment was expanded to a total of 26 patients. The null hypothesis was unaccepted if more than 14 of 26 patients achieved the response. Accounting for a 20% dropout rate, the estimated final sample size was 33 patients.

Who can participate

Age range

6 Years – 70 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Age from 6 to 70 * Diagnosis of Coombs-negative AIHA * Diagnosis of warm AIHA, mixed AIHA or Evans syndrome. * Meets the criteria of relapsed / refractory AIHA * ECOG ≤ 3 * Willing and able to comply with the requirements for this study and written informed consent. Exclusion Criteria: * Diagnosis of any of the following diseases: Cold agglutinin disease, cold agglutinin syndrome, mixed AIHA, paroxysmal cold hemoglobinuria (PCH). * Diagnosis of active stage of connective tissue disease. * History of lymphoproliferative tumors or any other malignant. * Diagnosis of other inherited or acquired hemolytic diseases. * Secondary AIHA caused by drugs or infection. * Previously received organ or stem cell transplantation. * History of thrombosis or organ infarction. * Received rituximab within 8 weeks before enrollment. * Previously treated with BTK inhibitor ≥ 2 weeks. * Received low-molecular-weight heparin or warfarin within 1 week before enrollment and need to continue the drug treatment. * Received CYP3A4 Enzyme Inhibitors or Inducers within 1 week before enrollment and need to continue the drug treatment. * Uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment), known human immunodeficiency virus (HIV), known evidence of active infectious hepatitis B, and/or known evidence of active hepatitis C.…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Overall response rate

Timeframe: within 12 weeks

Trial details

NCT IDNCT06021977

SponsorInstitute of Hematology & Blood Diseases Hospital, China

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2025-09-30

Contact for this trial

Weiwang Li, PhD

15332132036 liweiwang@ihcams.ac.cn

View on ClinicalTrials.gov More Refractory/Relapsed Autoimmune Hemolytic Anemia trials