ClinicalTrials.gg
RecruitingNCT07308574Updated Apr 20, 2026

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

A Phase 4 interventional study of Ravulizumab in aHUS and Atypical Hemolytic Uremic Syndrome, sponsored by Alexion Pharmaceuticals, Inc.. Recruiting at 12 sites in Japan. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-04-20.

Sponsored by Alexion Pharmaceuticals, Inc. (part of AstraZeneca) · Phase 4, Interventional, and Treatment

From the registry’s dates

  • Started Dec 2025; still recruiting 10 months later.

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Phase
Phase 4
Study type
Interventional
Enrollment
20
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Where this study stands

Counted across the records on this site · 2026-11-14

Among 19 studies in Atypical Hemolytic Uremic Syndrome by AstraZeneca — 4 open to participants now, 5 under way, none of them phase 3. Atypical Hemolytic Uremic Syndrome · AstraZeneca.

Planned enrollment
5th of 10
20 · median 18
Started
2025
with 1 other
Phase 4
Only one
of the 19
Registered
18th of 19
by date

Among the 19 studies in Atypical Hemolytic Uremic Syndrome by AstraZeneca, this one is the 5th largest by planned enrollment of the 10 interventional ones that give a count, is one of 2 that started in 2025, is the only one in phase 4, and was the 18th registered.

Atypical Hemolytic Uremic Syndrome

45 studies on the registry are indexed under Atypical Hemolytic Uremic Syndrome; 14 are open to participants now.

Browse Atypical Hemolytic Uremic Syndrome studies →

Open to participants now

4 of the 19 studies in Atypical Hemolytic Uremic Syndrome by AstraZeneca are open now, in 12 countries. Recruiting first, the newest first:

By country, each count listing its studies:

All 4 open studies →

Who these studies take

Of the 12 Atypical Hemolytic Uremic Syndrome studies recruiting now, 10 (83%) set no upper age limit and none (0%) stop at 75 or younger; 6 take children under 18; 2 take healthy volunteers.

This study: 18 Years and older · all sexes · no healthy volunteers.

The full table on the hub →

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Watch this landscapeDownload CSVNarrow it in search →With an account: a weekly mail of what changed among 19 studies in Atypical Hemolytic Uremic Syndrome by AstraZeneca, or all of them as a file.
02

Study 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).

03

Conditions studied

  • aHUS
  • Atypical Hemolytic Uremic Syndrome

Keywords

  • aHUS
  • atypical hemolytic uremic syndrome
  • ravulizumab
04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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: count \<150,000/microliter (μL)
  • Microangiopathic haemolytic anaemia: \< 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

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, 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 , 6 (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
Terms in these criteria, in plain language(4)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Platelets

Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.

Hemoglobin (Hb)

The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).

Autoimmune disease

A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.

Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.

CD4 count

The number of CD4 cells, a type of white blood cell, in a given amount of blood. It is used to follow how well the immune system is doing in HIV.

05

Study design

Phase
Phase 4
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
20 participants (estimated)

Study arms

  • 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

Interventions

  • Participants will receive ravulizumab via IV infusion.

06

What researchers measure

Primary outcomes

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

    Time frame: Baseline up to Week 26

Secondary outcomes

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

    Time frame: Baseline up to Week 26

  2. Percentage of Participants Showing Improvement in Platelet Count

    Time frame: Day 4 and on Weeks 1, 2, 10, 18, and 26

  3. Percentage of Participants Showing Improvement in Renal Function

    Time frame: Day 4 and on Weeks 1, 2, 10, 18, and 26

  4. 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

  5. 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

  6. Change from Baseline in Platelet Count

    Time frame: Baseline (Day 1), Week 26

  7. Change From Baseline in Hemoglobin

    Time frame: Baseline (Day 1), Week 26

  8. Change From Baseline in Lactate Dehydrogenase

    Time frame: Baseline (Day 1), Week 26

  9. Change From Baseline in Estimated Glomerular Filtration Rate

    Time frame: Baseline (Day 1), Week 26

07

Study locations

3 of 12 sites recruiting
  • 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
08

References and documents

Individual participant data

Plan to share: 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.

Supporting information: Study protocol, Statistical analysis plan, Clinical study report

No publications or documents are linked to this record.

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Dec 2025 (actual). Primary completion is estimated for Jun 2027, 18 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered
Dec 17, 2025
First posted
Dec 29, 2025
StartActual
Dec 19, 2025
Primary completionEst.
Jun 25, 2027
CompletionEst.
Jun 25, 2027
Last update
Apr 20, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Apr 20, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT07308574
Responsible party
Sponsor
First posted
Dec 29, 2025
Start date
Dec 19, 2025
Primary completion
Jun 25, 2027 (estimated)
Completion
Jun 25, 2027 (estimated)
Last update
Apr 20, 2026

Study contacts

Alexion Pharmaceuticals, Inc. (Sponsor)
Contact
clinicaltrials@alexion.com
1-855-752-2356

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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Studies of the drug tested here: Ravulizumab

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