Eculizumab for high-risk TA-TMA in adult HSCT recipients

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

In adults who recently had an allogeneic HSCT and develop high-risk transplant-associated thrombotic microangiopathy, this study tests early treatment with the C5 inhibitor eculizumab. It is a multicenter, prospective study using eculizumab with a defined loading, induction, and maintenance schedule for up to 24 weeks. The primary goal is to see if survival at 6 months after TA-TMA diagnosis improves compared with historical controls, with secondary aims including organ dysfunction at 6 months and 1-year post-transplant survival. Safety and efficacy will be monitored in treated patients. Eligible participants are adults with diagnosed TA-TMA within 14 days before enrollment and meeting high-risk criteria such as significant proteinuria, MODS, or elevated IL-10.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Age ≥18 years.
* Patients who have undergone hematopoietic stem cell transplantation for any indication within 12 months prior to enrollment.
* Meet the diagnostic criteria for TA-TMA within ≤14 days prior to enrollment (at least 4 of the following 7 criteria present simultaneously):① Lactate dehydrogenase (LDH) above the age-adjusted upper limit of normal;② Presence of schistocytes on peripheral blood smear;③ New-onset thrombocytopenia or requirement for platelet transfusions;④ New-onset anemia or requirement for red blood cell transfusions;⑤ Hypertension;⑥ Random urine protein-to-creatinine ratio (rUPCR) ≥1 mg/mg;⑦ Elevated plasma soluble C5b-9 (sC5b-9) level (≥244 ng/mL).
* Meet the criteria for high-risk TA-TMA (presence of any of the following):① Proteinuria (rUPCR ≥2 mg/mg);②Multiple organ dysfunction syndrome (MODS);③ Elevated IL-10 (≥2× upper limit of normal \[ULN\]).
* Meet the following condition: TA-TMA has not resolved after ≥72 hours of management of triggering factors/conditions, including: ① Discontinuation or dose reduction of inciting medications (e.g., calcineurin inhibitors, CNI); ② Treatment of any underlying infection; ③ Treatment of underlying acute graft-versus-host disease (aGVHD).
* Provide written informed consent.

Exclusion Criteria:

* Known hypersensitivity to eculizumab.
* Uncontrolled severe infection (including meningococcal infection).
* Prior treatment with complement inhibitors.
* Known hereditary or acquired ADAMTS13 deficiency (activity \<10%).
* Disseminated intravascular coagulation (DIC).
* Respiratory failure (any cause) requiring mechanical ventilation, occurring within 72 hours prior to enrollment.
* Acute and/or chronic heart failure with ejection fraction ≤40%.
* Expected survival \<48 hours.
* Any subject who, in the investigator's opinion, is not suitable for participation in this study.

Where it's running

• Xiangya Hospital of Central South University
  Changsha, Hunan, China Recruiting
• Sir Run Run Shaw Hospital, Zhejiang University School of Medicine
  Hangzhou, Zhejiang, China Recruiting
• The First Affiliated Hospital, Zhejiang University School of Medicine.
  Hangzhou, Zhejiang, China Recruiting
• The Second Affiliated Hospital of Zhejiang University School of Medicine
  Hangzhou, Zhejiang, China Recruiting
• Jinhua Central Hospital
  Jinhua, Zhejiang, China Recruiting
• The Affiliated People's Hospital of Ningbo University
  Ningbo, Zhejiang, China Recruiting

Study contacts

• Principal investigator: Yi Luo — First Affiliated Hospital of Zhejiang University
• Study coordinator: Yibo Wu
• Email: wuyibo7@126.com
• Phone: +8657187233801

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.