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New drug ravulizumab aims to control rare blood disorder aHUS

NCT ID NCT07308574

Disease control Sponsor: Alexion Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study is testing the drug ravulizumab in 20 people with atypical hemolytic uremic syndrome (aHUS), a rare condition that causes blood clots and kidney damage. The goal is to see if the drug improves platelet counts and kidney function over 26 weeks. All participants receive the drug, and researchers will monitor safety and effectiveness.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ravulizumab
What this could lead to
If successful, this study could confirm ravulizumab as a reliable treatment to control aHUS and prevent kidney damage.
What could go wrong
This is a small, single-arm study with no placebo group, so results may not be definitive. Ravulizumab also carries risks like increased susceptibility to infections.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 4

Runs after approval, following long-term safety and how well the treatment works in everyday use.

Participants

About 20 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2025

Expected to finish

Jun 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

atypical hemolytic-uremic syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

AHUS Atypical hemolytic uremic syndrome

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    12 sites. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Research Site

    NOT_YET_RECRUITING

    Bunkyō City, 113-8655, Japan

  • Research Site

    NOT_YET_RECRUITING

    Hirakata-shi, 573-1191, Japan

  • Research Site

    RECRUITING

    Iruma-Gun, 350-0495, Japan

  • Research Site

    NOT_YET_RECRUITING

    Kyoto, 602-8566, Japan

  • Research Site

    NOT_YET_RECRUITING

    Matsumoto-shi, 390-8621, Japan

  • Research Site

    NOT_YET_RECRUITING

    Miyazaki, 889-1692, Japan

  • Research Site

    RECRUITING

    Nagoya, 466-8650, Japan

  • Research Site

    NOT_YET_RECRUITING

    Nara, 630-8581, Japan

  • Research Site

    NOT_YET_RECRUITING

    Nerima-ku, 177-8521, Japan

  • Research Site

    NOT_YET_RECRUITING

    Sapporo, 060-8638, Japan

  • Research Site

    NOT_YET_RECRUITING

    Shinjuku-ku, 162-8666, Japan

  • Research Site

    RECRUITING

    Tsu, 514-8507, Japan

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