aHUSPhase 4NCT07308574
Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS
A Phase 4 study testing Ravulizumab in people with aHUS and Atypical Hemolytic Uremic Syndrome, run by Alexion Pharmaceuticals, Inc. at 12 sites in Japan.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- aHUS, Atypical Hemolytic Uremic Syndrome
- What is being tested?
- Ravulizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 12 sitesJapan
- Study dates
- 19 Dec 2025to 25 Jun 2027
- Phase
- Phase 4
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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).
Who cannot join
- 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
Trial locations and contacts
12 sites in 1 country. Contact the study team or a site near you to ask about taking part.
12 sites in 1 country
Japan12 sites, 12 recruiting
- Not yet recruiting
Research Site
Bunkyō City, 113-8655
- Not yet recruiting
Research Site
Hirakata-shi, 573-1191
- Recruiting
Research Site
Iruma-Gun, 350-0495
- Not yet recruiting
Research Site
Kyoto, 602-8566
- Not yet recruiting
Research Site
Matsumoto-shi, 390-8621
- Not yet recruiting
Research Site
Miyazaki, 889-1692
- Recruiting
Research Site
Nagoya, 466-8650
- Not yet recruiting
Research Site
Nara, 630-8581
- Not yet recruiting
Research Site
Nerima-ku, 177-8521
- Not yet recruiting
Research Site
Sapporo, 060-8638
- Not yet recruiting
Research Site
Shinjuku-ku, 162-8666
- Recruiting
Research Site
Tsu, 514-8507
About this study
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
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
- Being tested
- Ravulizumab
- Keywords
- aHUS, atypical hemolytic uremic syndrome, ravulizumab
- Medical subject headings
- Atypical Hemolytic Uremic Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RavulizumabDrug
Participants will receive ravulizumab via IV infusion.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Ravulizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Showing Improvement in Platelet Count During the 26-week Ravulizumab Treatment
Measured: Baseline up to Week 26
Secondary outcomes (9)
Percentage of Participants Showing Improvement in Renal Function During the 26-week Ravulizumab Treatment
Measured: Baseline up to Week 26
Percentage of Participants Showing Improvement in Platelet Count
Measured: Day 4 and on Weeks 1, 2, 10, 18, and 26
Percentage of Participants Showing Improvement in Renal Function
Measured: 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
Measured: 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
Measured: Baseline (Day 1) up to Week 26
Change from Baseline in Platelet Count
Measured: Baseline (Day 1), Week 26
Change From Baseline in Hemoglobin
Measured: Baseline (Day 1), Week 26
Change From Baseline in Lactate Dehydrogenase
Measured: Baseline (Day 1), Week 26
Change From Baseline in Estimated Glomerular Filtration Rate
Measured: Baseline (Day 1), Week 26
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Dec 2025
- Study start19 Dec 2025
- Primary completion25 Jun 2027 (estimated)
- Study completion25 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for aHUSStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07308574
- Sponsor's protocol ID
- D928BL00001
- Other IDs
- NEPH-ULT-501
- Status last verified
- Apr 2026
- First submitted
- 17 Dec 2025
- Last updated
- 20 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.