Refractory/Relapsed Autoimmune Hemolytic AnemiaPhase 2NCT06021977
The Safety and Efficacy of Zanubrutinib in Refractory/Relapsed Autoimmune Hemolytic Anemia
A Phase 2 study testing Zanubrutinib in people with Refractory/Relapsed Autoimmune Hemolytic Anemia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 70 years
- Conditions the study is for
- Refractory/Relapsed Autoimmune Hemolytic Anemia
- What is being tested?
- Zanubrutinib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- 1 siteChina
- Study dates
- 1 Sep 2023to 31 Dec 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
- Any severe and/or uncontrolled medical conditions: refractory hypertension, clinically significant cardiac diseases, renal diseases, liver diseases and metabolic diseases, etc.
- History of mental illness.
- Participation in another clinical trial within 4 weeks before the start of this trial.
- Pregnant or breast-feeding patients.
- Patients considered ineligible for the study by the investigator for reasons other than the above.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Regenerative Medicine Center
Tianjin, Tianjin Municipality, 300131
Jingyu Zhao, 13752253515, zhaojingyu94@ihcams.ac.cn
About this study
Official title: A Phase II, Single Center, Open-label Trial to Determine the Safety and Efficacy of Zanubrutinib in Refractory/Relapsed Autoimmune Hemolytic
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.
- Being tested
- Zanubrutinib
- Medical subject headings
- Anemia, Hemolytic, Autoimmune
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ZanubrutinibDrug
Zanubrutinib was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity.
| Study group | Role | What they receive |
|---|---|---|
| Zanubrutinib | Experimental | Each recruited subject will accept Zanubrutinib treatment. Receives: Drug: Zanubrutinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate
The percentage of patients achieved complete response, complete response with incomplete hemolysis recovery, and partial response.
Measured: within 12 weeks
Secondary outcomes (2)
Incidence of adverse events and severe adverse events
Measured: within 24 weeks
Relapse free survival rate
Measured: within 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Sep 2023
- Study start1 Sep 2023
- Primary completion30 Sep 2025 (estimated)
- Study completion31 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory/Relapsed Autoimmune Hemolytic AnemiaStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 33 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Fang Liwei, Institute of Hematology & Blood Diseases Hospital, China
- Investigators
- Liwei Fang, MD, Principal investigator, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06021977
- Sponsor's protocol ID
- IHBDH-IIT20230711
- Status last verified
- Nov 2023
- First submitted
- 27 Aug 2023
- Last updated
- 29 Nov 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Single Center, Open-label Trial to Determine the Safety and Efficacy of Zanubrutinib in Refractory/Relapsed Autoimmune Hemolytic
- ClinicalTrials.gov ID
- NCT06021977
- Acronym / sponsor ID
- IHBDH-IIT20230711
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 33 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 1 Sep 2023 (actual)
- Primary completion
- 30 Sep 2025 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 1 Sep 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Zanubrutinib | Experimental | Drug: ZanubrutinibEach recruited subject will accept Zanubrutinib treatment. |
Who may qualify
- Ages
- 6 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (17 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ZanubrutinibZanubrutinib was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. | Drug | Zanubrutinib |
Primary outcome
Overall response rate
Time frame: within 12 weeks
The percentage of patients achieved complete response, complete response with incomplete hemolysis recovery, and partial response.
2 secondary outcomes
Incidence of adverse events and severe adverse events
Time frame: within 24 weeks
Relapse free survival rate
Time frame: within 48 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Nov 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Liwei Fang, MD principal investigator
Institute of Hematology & Blood Diseases Hospital, China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Zanubrutinib in people with Refractory/Relapsed Autoimmune Hemolytic Anemia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 29 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Weiwang Li, PhD (15332132036, liweiwang@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response rate (time frame: within 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Nov 2023.