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

April 17, 2026 updated by: Alexion Pharmaceuticals, Inc.

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

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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
20

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Japan
      • Bunkyō City, Japan, 113-8655
        • Not yet recruiting
        • Research Site
      • Hirakata-shi, Japan, 573-1191
        • Not yet recruiting
        • Research Site
      • Iruma-Gun, Japan, 350-0495
        • Recruiting
        • Research Site
      • Kyoto, Japan, 602-8566
        • Not yet recruiting
        • Research Site
      • Matsumoto-shi, Japan, 390-8621
        • Not yet recruiting
        • Research Site
      • Miyazaki, Japan, 889-1692
        • Not yet recruiting
        • Research Site
      • Nagoya, Japan, 466-8650
        • Recruiting
        • Research Site
      • Nara, Japan, 630-8581
        • Not yet recruiting
        • Research Site
      • Nerima-ku, Japan, 177-8521
        • Not yet recruiting
        • Research Site
      • Sapporo, Japan, 060-8638
        • Not yet recruiting
        • Research Site
      • Shinjuku-ku, Japan, 162-8666
        • Not yet recruiting
        • Research Site
      • Tsu, Japan, 514-8507
        • Recruiting
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Ravulizumab
Participants will receive a weight-based loading dose of ravulizumab, followed by a weight-based dose 2 weeks after loading dose administration, then weight-based maintenance doses every 8 weeks via intravenous (IV) infusion.
Drug: Ravulizumab
Participants will receive ravulizumab via IV infusion.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Percentage of Participants Showing Improvement in Platelet Count During the 26-week Ravulizumab Treatment
Time Frame: Baseline up to Week 26
Baseline up to Week 26

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Percentage of Participants Showing Improvement in Renal Function During the 26-week Ravulizumab Treatment
Time Frame: Baseline up to Week 26
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
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
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
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
Baseline (Day 1) up to Week 26
Change from Baseline in Platelet Count
Time Frame: Baseline (Day 1), Week 26
Baseline (Day 1), Week 26
Change From Baseline in Hemoglobin
Time Frame: Baseline (Day 1), Week 26
Baseline (Day 1), Week 26
Change From Baseline in Lactate Dehydrogenase
Time Frame: Baseline (Day 1), Week 26
Baseline (Day 1), Week 26
Change From Baseline in Estimated Glomerular Filtration Rate
Time Frame: Baseline (Day 1), Week 26
Baseline (Day 1), Week 26

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Alexion Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 19, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 25, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 25, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 17, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 17, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 29, 2025

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 20, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 17, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • D928BL00001
  • NEPH-ULT-501 (Registry Identifier: Alexion)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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