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

NCT07308574 | Recruiting Phase 4

• 2 other identifiers: D928BL00001NEPH-ULT-501
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 12

Brief Summary

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).

Conditions

aHUS; Atypical Hemolytic Uremic Syndrome

Keywords

aHUS; atypical hemolytic uremic syndrome; ravulizumab

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants Showing Improvement in Platelet Count During the 26-week Ravulizumab Treatment

  Baseline up to Week 26

Secondary Outcomes (9)

• Percentage of Participants Showing Improvement in Renal Function During the 26-week Ravulizumab Treatment

  Baseline up to Week 26

• Percentage of Participants Showing Improvement in Platelet Count

  Day 4 and on Weeks 1, 2, 10, 18, and 26

• Percentage of Participants Showing Improvement in Renal Function

  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

  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

  Baseline (Day 1) up to Week 26

Study Arms (1)

Ravulizumab

EXPERIMENTAL

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

Interventions

Ravulizumab DRUG

Participants will receive ravulizumab via IV infusion.

Ravulizumab

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (12)

Research Site

Bunkyō City, 113-8655, Japan

NOT YET RECRUITING

Research Site

Hirakata-shi, 573-1191, Japan

NOT YET RECRUITING

Research Site

Iruma-Gun, 350-0495, Japan

RECRUITING

Research Site

Kyoto, 602-8566, Japan

NOT YET RECRUITING

Research Site

Matsumoto-shi, 390-8621, Japan

NOT YET RECRUITING

Research Site

Miyazaki, 889-1692, Japan

NOT YET RECRUITING

Research Site

Nagoya, 466-8650, Japan

RECRUITING

Research Site

Nara, 630-8581, Japan

NOT YET RECRUITING

Research Site

Nerima-ku, 177-8521, Japan

NOT YET RECRUITING

Research Site

Sapporo, 060-8638, Japan

NOT YET RECRUITING

Research Site

Shinjuku-ku, 162-8666, Japan

NOT YET RECRUITING

Research Site

Tsu, 514-8507, Japan

RECRUITING

MeSH Terms

Conditions

Atypical Hemolytic Uremic Syndrome

Interventions

ravulizumab

Condition Hierarchy (Ancestors)

Hemolytic-Uremic Syndrome; Uremia; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia

Central Study Contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

1-855-752-2356; clinicaltrials@alexion.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 17, 2025
• First Posted: December 29, 2025
• Study Start: December 19, 2025
• Primary Completion (Estimated): June 25, 2027
• Study Completion (Estimated): June 25, 2027
• Last Updated: April 20, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR

Locations

JP (12)