ClinicalTrials.gov record NCT06847607
A Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA)
A Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA)
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This is a non-randomized, open-label study giving iptacopan to adults with relapsed or refractory warm or cold AIHA. Participants receive iptacopan and are followed for blood-count responses and safety. Main outcomes are overall response at 3 months and complete response at 6 months.
What it is studying. The study tests iptacopan as a drug treatment for people with relapsed or refractory warm or cold autoimmune hemolytic anemia and monitors blood-count responses and safety over time.
What the researchers hope to learn. To measure overall response rate at 3 months and complete response rate at 6 months in this patient group. To collect safety data on iptacopan in people with relapsed or refractory warm and cold AIHA.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
To evaluate the efficacy and safety of ipecopam in the treatment of refractory/relapsed AIHA.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Iptacopan
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 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;
- 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.
Exclusion criteria
- 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;
- 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.
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants receive the study drug Iptacopan and attend regular clinic visits for physical exams and blood tests to monitor hemoglobin, direct antiglobulin test (DAT), and safety. They must meet lab and health thresholds and may need meningococcal and pneumococcal vaccination (or antibiotic prophylaxis) per study rules; certain background medicines must be stable before entry. Participants should avoid disallowed treatments (for example recent herbal treatments that interfere with assessment) and will be monitored over time with response assessments noted at 3 and 6 months.
How the study is structured. Interventional (treatment) study with a single treatment group. Participants receive iptacopan (no placebo or comparison arm), the study is not randomized and not masked, and outcomes are assessed at defined time points including 3 and 6 months.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on autoimmune hemolytic anemia
This trial is also listed on this site under cold agglutinin disease.
Important dates
- Study start
- Mar 1, 2025 (estimated)
- Primary completion
- Oct 30, 2025 (estimated)
- Study completion
- Jun 30, 2026 (estimated)
- Target enrollment
- 20 participants (estimated)
- Last updated on registry
- Feb 26, 2025
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Bing Han
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Bing Han Han 📞 13601059938 ✉ Hanbing_li@sina.com.cn
Participating sites (1)
- Bing Han
Beijing · China
Terms used on this page · explanations by MedicalUpdates.ai
- refractory
- A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
- relapsed
- A condition that improved with treatment and has since returned or worsened.
- informed consent form
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- ECOG
- A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
- glucocorticoid
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- contraindicated
- A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
- immunosuppressants
- A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- Neutrophil count
- The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
- × ULN
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- creatinine clearance
- A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
- inclusion criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- QTcF
- A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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