Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS

Официальное название

Multicenter, Open-label, Single-arm, Post-Marketing Clinical Study to Evaluate the Efficacy and Safety of Ravulizumab in Participants Clinically Diagnosed as Atypical Hemolytic Uremic Syndrome

Обзор

The primary objective of this study is to assess the platelet count response to ravulizumab in participants clinically diagnosed as atypical hemolytic uremic syndrome (aHUS).

Вмешательства

• Препарат Ravulizumab
  Participants will receive ravulizumab via IV infusion.

Первичные конечные точки

• Percentage of Participants Showing Improvement in Platelet Count During the 26-week Ravulizumab Treatment [Срок оценки: Baseline up to Week 26]

Вторичные конечные точки (9)

• Percentage of Participants Showing Improvement in Renal Function During the 26-week Ravulizumab Treatment [Срок оценки: Baseline up to Week 26]
• Percentage of Participants Showing Improvement in Platelet Count [Срок оценки: Day 4 and on Weeks 1, 2, 10, 18, and 26]
• Percentage of Participants Showing Improvement in Renal Function [Срок оценки: Day 4 and on Weeks 1, 2, 10, 18, and 26]
• Percentage of Participants Showing Improvement in Complete Thrombotic Microangiopathy (TMA) Response or Partial TMA Response [Срок оценки: Day 4 and on Weeks 1, 2, 10, 18, and 26]
• Percentage of Participants who are on Dialysis on Day 1 and are Able to Withdraw From Dialysis by Week 26 [Срок оценки: Baseline (Day 1) up to Week 26]
• Change from Baseline in Platelet Count [Срок оценки: Baseline (Day 1), Week 26]
• Change From Baseline in Hemoglobin [Срок оценки: Baseline (Day 1), Week 26]
• Change From Baseline in Lactate Dehydrogenase [Срок оценки: Baseline (Day 1), Week 26]
• Change From Baseline in Estimated Glomerular Filtration Rate [Срок оценки: Baseline (Day 1), Week 26]

Критерии участия

Критерии включения

• 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).

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Япония · 12 центров

• Research Site — Bunkyō City
• Research Site — Hirakata-shi
• Research Site — Iruma-Gun
• Research Site — Kyoto
• Research Site — Matsumoto-shi
• Research Site — Miyazaki
• Research Site — Nagoya
• Research Site — Nara
• … и ещё 4 центра

Идентификаторы

NCT: NCT07308574 · D928BL00001 · NEPH-ULT-501

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗