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Not yet recruiting NCT06847607

A Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA)

No phase Interventional AIHA - Warm Autoimmune Hemolytic Anemia AIHA - Cold Autoimmune Hemolytic Anemia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Iptacopan.
Who it may be relevant to
Registry conditions: AIHA - Warm Autoimmune Hemolytic Anemia, AIHA - Cold Autoimmune Hemolytic Anemia. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Overview

To evaluate the efficacy and safety of ipecopam in the treatment of refractory/relapsed AIHA.

Interventions

  • Drug Iptacopan
    Iptacopan

Primary outcome measures

  • OR [Time frame: 3 months]
  • CR [Time frame: 6 months]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

China · 1 center
  • Bing Han — Beijing

Identifiers

NCT: NCT06847607 · AIHA-Iptacopan

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗