Menu
Recruiting NCT07308574

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

Phase IV Interventional aHUS Atypical Hemolytic Uremic Syndrome

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: Ravulizumab.
Who it may be relevant to
Registry conditions: aHUS, Atypical Hemolytic Uremic Syndrome. 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
Japan
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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).

Interventions

  • Drug Ravulizumab
    Participants will receive ravulizumab via IV infusion.

Primary outcome measures

  • Percentage of Participants Showing Improvement in Platelet Count During the 26-week Ravulizumab Treatment [Time frame: Baseline up to Week 26]
Secondary outcome measures (9)
  • Percentage of Participants Showing Improvement in Renal Function During the 26-week Ravulizumab Treatment [Time frame: Baseline up to Week 26]
  • Percentage of Participants Showing Improvement in Platelet Count [Time frame: Day 4 and on Weeks 1, 2, 10, 18, and 26]
  • Percentage of Participants Showing Improvement in Renal Function [Time frame: 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 [Time frame: 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 [Time frame: Baseline (Day 1) up to Week 26]
  • Change from Baseline in Platelet Count [Time frame: Baseline (Day 1), Week 26]
  • Change From Baseline in Hemoglobin [Time frame: Baseline (Day 1), Week 26]
  • Change From Baseline in Lactate Dehydrogenase [Time frame: Baseline (Day 1), Week 26]
  • Change From Baseline in Estimated Glomerular Filtration Rate [Time frame: Baseline (Day 1), Week 26]

Eligibility criteria

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

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

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Japan · 12 centers
  • 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
  • … and 4 more centers

Identifiers

NCT: NCT07308574 · D928BL00001 · NEPH-ULT-501

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗