AIHA - Warm Autoimmune Hemolytic AnemiaNCT06847607
A Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA)
A study testing Iptacopan in people with AIHA - Warm Autoimmune Hemolytic Anemia and AIHA - Cold Autoimmune Hemolytic Anemia, run by Bing Han at 1 site in China.
Trial summary
To evaluate the efficacy and safety of ipecopam in the treatment of refractory/relapsed AIHA. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- AIHA - Warm Autoimmune Hemolytic Anemia, AIHA - Cold Autoimmune Hemolytic Anemia
- What is being tested?
- Iptacopan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 1 Mar 2025to 30 Jun 2026
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Voluntarily signed an informed consent form (ICF);
- Males or females aged 18 or older;
- Physical status score [Eastern Cooperative Oncology Group (ECOG) score] ≤2;
- Confirmed diagnosis of primary AIHA or secondary autoimmune disease (except rheumatoid arthritis and systemic lupus erythematosus) with underlying disease in a stable state;
- Poor response to at least previous glucocorticoid therapy, including ineffective (defined as failure to achieve stabilization of Hb levels at 100 g/L or erythrocyte hematocrit <30% despite at least 4 weeks of treatment with previously recommended doses), or glucocorticoid-dependent (defined as maintenance of equal doses of prednisone exceeding 15 mg/d), or relapsed (defined as treatment that is effective and then again has an Hb of <100 g/L or an erythrocyte hematocrit of <30%), or otherwise contraindicated. 30%), or otherwise contraindicated or intolerant to glucocorticoid therapy;
- Hemoglobin (Hb) <100 g/L before drug administration;
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- Positive direct anti-human globulin test (DAT) (IgA, IgM or IgG+, with or without C3+).
- Combination of one anti-AIHA therapy [glucocorticoids only (≤15 mg prednisone equivalent), immunosuppressants (azathioprine, cyclosporine, and merti-macrolide only)] is permitted in this study, provided that the dose has been stable for at least 28 days prior to enrollment;
- Laboratory tests meet the following criteria (no treatment for the abnormality of the index within 2 weeks prior to blood collection, or no long-acting G-CSF treatment within 2 weeks)
- Neutrophil count >1.5×109/L and platelet >30×109/L;
- ALT and AST ≤ 2 × ULN;
- Serum creatinine concentration ≤ 2 × ULN and creatinine clearance ≥ 50mL/min;
- No active infection; no pregnancy or lactation;
- cAIHA patients presenting with skin cyanosis and thrombosis;
- Written evidence of Neisseria meningitidis and Streptococcus pneumoniae vaccinations within 2 years or, if none, antibiotic prophylaxis until 2 weeks after completion of vaccination.
Who cannot join
- Presence of secondary AIHA outside the inclusion criteria;
- Hb <100 g/L due to non-AIHA factors; and
- Infections requiring systemic therapy;
- Those with a past history of malignancy (except cured basal cell carcinoma of the skin or cervical carcinoma in situ);
- With history of vital organ transplantation or hematopoietic stem cell/bone marrow transplantation;
- Those who have undergone splenectomy within 24 weeks prior to enrollment;
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- Those who have had major surgery within four weeks prior to enrollment or who require major elective surgery during the study period;
- History of severe cardiovascular disease [e.g., class III/IV congestive heart failure, arrhythmia or angina requiring medication, unstable angina, coronary stenting, angioplasty or coronary artery bypass grafting, or corrected Q-T interval (QTcF) ≥ 90 mmHg]
- Patients with medically uncontrolled hypertension (systolic blood pressure ≥140 mmHg or diastolic blood pressure ≥90 mmHg); or comorbid portal hypertension;
- Patients with severe gastrointestinal disorders such as dysphagia, active gastric ulcers, etc., who are unable to take drugs orally or have impaired absorption of oral drugs;
- Human immunodeficiency virus (HIV) infection
- Uncontrolled or active HBV infection [Hepatitis B surface antigen (HBsAg) or Hepatitis B core antibody (HBcAb) positive patients, need to confirm Hepatitis B Virus Deoxyribonucleic Acid (HBV DNA) positive]; or Hepatitis C [patients with Hepatitis C Virus Ribonucleic Acid (HCV RNA) positive patients]; or cirrhosis of the liver;
- Those who had received herbal treatment within one week prior to enrollment that interfered with the assessment of efficacy;
- Patients with severe psychological or psychiatric abnormalities;
- Alcohol or drug abusers;
- Female patients who are pregnant or breastfeeding;
- Patients who, in the opinion of the investigator, are not suitable for participation in this study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
To evaluate the efficacy and safety of ipecopam in the treatment of refractory/relapsed AIHA.
- Being tested
- Iptacopan
- 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.
IptacopanDrug
Iptacopan
| Study group | Role | What they receive |
|---|---|---|
| Iptacopan-AIHA | Experimental | Iptacopan-AIHA Receives: Drug: Iptacopan |
What is being measured
The primary outcome is the main question the study is designed to answer.
OR
Overall response rate
Measured: 3 months
CR
complete response rate
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Feb 2025
- Study start1 Mar 2025 (estimated)
- Primary completion30 Oct 2025 (estimated)
- Study completion30 Jun 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AIHA - Warm Autoimmune Hemolytic AnemiaStudies of the same condition, open and under way.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Bing Han
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Bing Han, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06847607
- Sponsor's protocol ID
- AIHA-Iptacopan
- Status last verified
- Feb 2025
- First submitted
- 21 Feb 2025
- Last updated
- 26 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA)
- ClinicalTrials.gov ID
- NCT06847607
- Acronym / sponsor ID
- AIHA-Iptacopan
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Bing HanUniversity, hospital or other
- Study start
- 1 Mar 2025 (estimated)
- Primary completion
- 30 Oct 2025 (estimated)
- Study completion
- 30 Jun 2026 (estimated)
- First posted
- 26 Feb 2025 (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.
To evaluate the efficacy and safety of ipecopam in the treatment of refractory/relapsed AIHA.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Iptacopan-AIHA | Experimental | Drug: IptacopanIptacopan-AIHA |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Voluntarily signed an informed consent form (ICF);
- Males or females aged 18 or older;
- Physical status score [Eastern Cooperative Oncology Group (ECOG) score] ≤2;
- Confirmed diagnosis of primary AIHA or secondary autoimmune disease (except rheumatoid arthritis and systemic lupus erythematosus) with underlying disease in a stable state;
- Poor response to at least previous glucocorticoid therapy, including ineffective (defined as failure to achieve stabilization of Hb levels at 100 g/L or erythrocyte hematocrit <30% despite at least 4 weeks of treatment with previously recommended doses), or glucocorticoid-dependent (defined as maintenance of equal doses of prednisone exceeding 15 mg/d), or relapsed (defined as treatment that is effective and then again has an Hb of <100 g/L or an erythrocyte hematocrit of <30%), or otherwise contraindicated. 30%), or otherwise contraindicated or intolerant to glucocorticoid therapy;
Key exclusion criteria (17 listed)
- Presence of secondary AIHA outside the inclusion criteria;
- Hb <100 g/L due to non-AIHA factors; and
- Infections requiring systemic therapy;
- Those with a past history of malignancy (except cured basal cell carcinoma of the skin or cervical carcinoma in situ);
- With history of vital organ transplantation or hematopoietic stem cell/bone marrow transplantation;
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 |
|---|---|---|
| IptacopanIptacopan | Drug | Iptacopan-AIHA |
Primary outcome
OR
Time frame: 3 months
Overall response rate
CR
Time frame: 6 months
complete response rate
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Feb 2025.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Iptacopan in people with AIHA - Warm Autoimmune Hemolytic Anemia and AIHA - Cold Autoimmune Hemolytic Anemia, run by Bing Han 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 15 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: opening soon, as of the sponsor's last update on 26 Feb 2025. 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.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Bing Han Han (13601059938, Hanbing_li@sina.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
OR (time frame: 3 months). The study lists 2 primary outcomes in all. 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 26 Feb 2025.