Ravulizumab for people with clinical aHUS

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

This is a post-marketing, phase 4 interventional study enrolling participants clinically diagnosed with aHUS to receive ravulizumab. The primary objective is to measure platelet count response after treatment; eligible participants must weigh at least 20 kg and meet laboratory signs of thrombotic microangiopathy such as platelet count <150,000/µL, hemoglobin <10 g/dL, and acute kidney injury by specified creatinine changes. The protocol includes people with complicating conditions (infections except pneumococcal and STEC, pregnancy or postpartum, post-renal transplant, malignant hypertension, or connective tissue diseases) and those whose TMA has not improved after treatment for secondary TMA. Platelet counts and clinical markers of hemolysis and renal function will be followed over time to document response to ravulizumab.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Body weight ≥20 kilograms (kg)
* Participants clinically diagnosed as aHUS who have any of diseases/conditions listed below (including participants in whom Thrombotic microangiopathy (TMA) has not been improved even after treatment for the pathogenesis of diagnosed secondary TMA and therefore, diagnosis of aHUS was made).
* Infection (except for pneumococcal infection and Siga toxin-producing Escherichia coli infection)
* During pregnancy or postpartum
* Post-renal transplantation
* Hypertensive crisis/malignant hypertension
* Systemic lupus erythematosus and related diseases (e.g. dermatomyositis, mixed connective tissue disease, etc.)
* Participants with the following three signs:
* Thrombocytopenia: Platelet count \<150,000/microliter (μL)
* Microangiopathic haemolytic anaemia: Hb \< 10 grams per deciliter (g/dL) (\*)
* Acute kidney injury: one of the following is fulfilled; 1. ΔsCr ≥ 0.3 milligrams per deciliter (mg/dL) (within 48 hours), 2. 1.5-fold increase from baseline sCr (within 7 days), 3. urinary output ≤ 0.5 mL/kg/hour for ≥ 6 hours.
* No prior treatment with complement inhibitors.
* The investigator plans to provide the participant with 26-week treatment with ravulizumab in accordance with the treatment policy in clinical practice.
* Ravulizumab treatment is planned to be initiated within 14 days after onset of the latest TMA episode.
* Participants consenting to meningococcal vaccine administration and appropriate antibiotic prophylaxis (if required).

Exclusion Criteria:

* Participants with TTP, STEC-HUS, secondary TMA that is obviously unrelated to complement abnormality.
* Participants with TMA caused by malignant tumors, abnormal Cobalamin C metabolism, Streptococcus pneumoniae, drugs, autoimmune diseases other than systemic lupus erythematosus and related diseases (e.g. scleroderma etc.), or hematopoietic stem cell transplantation
* Participants with pathological complement gene variants (CFH, CFI , CD46 (MCP), C3, CFB, THBD, DGKE) associated with the development of aHUS at enrolment
* Participants with positive anti-factor H antibodies
* More than 14 day from onset of TMA to the planned start of ravulizumab treatment
* Chronic kidney disease or irreversible renal impairment that requires chronic dialysis
* Presence of unresolved meningococcal disease
* Judgement by the investigator that the participant is not eligible for the study

Where it's running

• Research Site
  Iruma-Gun, Japan Recruiting
• Research Site
  Nagoya, Japan Recruiting
• Research Site
  Tsu, Japan Recruiting
• Research Site
  Bunkyō City, Japan Not yet recruiting
• Research Site
  Hirakata-shi, Japan Not yet recruiting
• Research Site
  Kyoto, Japan Not yet recruiting

Study contacts

• Study coordinator: Alexion Pharmaceuticals, Inc. (Sponsor)
• Email: clinicaltrials@alexion.com
• Phone: 1-855-752-2356

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Ravulizumab for children with IgA nephropathy or IgA vasculitis nephritis

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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.