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Could a simple pill replace infusions for aHUS patients?

NCT ID NCT05935215

Disease control Sponsor: Novartis Pharmaceuticals 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study is for adults with a rare blood disorder called aHUS who are currently receiving antibody infusions. Researchers want to see if switching to a daily pill called iptacopan is safe and works just as well to prevent dangerous blood clots. About 50 participants will take iptacopan for 12 months and be monitored closely.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 50 people

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

Started

Feb 2024

Expected to finish

Jul 2029

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 to 100 years

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: * Male and female adult participants ≥ 18 years of age with diagnosis of aHUS for whom etiologies of other types of TMA and non-aHUS kidney disease have been excluded. •. Currently on the recommended (as per label) dosage regimen of anti-C5 antibody treatment, for at least 3 months prior to entering the screening period. * In the opinion of the investigator the participant has responded to anti-C5 antibodytreatment prior to screening and has clinical evidence of response (in absence of PE/PI) during the Screening period. Clinical evidence of response to anti-C5 antibody treatment (in absence of PE/PI) confirmed during the Screening period by central laboratory at two visits 12 weeks apart. Clinical evidence of response is defined as: 1. Hematological normalization in platelet count ≥150 x 10\^9/L and LDH below upper limit of normal \[ULN\], and 2. Stable kidney function as defined by serum creatinine values within ±15% during the Screening period * Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections is required prior to the start of treatment with iptacopan. * If not received previously or if a booster is required, vaccination against Haemophilus influenzae infection, should be given, if available and according to local regulations. Exclusion Criteria: * History of aHUS disease relapse while on anti-C5 antibody treatment. * eGFR \< 30 ml/min/1.73m\^2 * Active infection or history of recurrent invasive infections caused by encapsulated bacteria, i.e., meningococcus, pneumococcus (eg., N. meningitidis, S. pneumoniae) or H. influenzae. * Participants with sepsis or active systemic bacterial, viral (including COVID-19) or fungal infection within 14 days prior to study treatment administration. * Kidney, bone marrow transplant (BMT)/hematopoietic stem cell transplant (HSCT), heart, lung, small bowel, pancreas, liver transplantation or any other cell or solid organ transplantation * Female patients who are pregnant or breastfeeding, or intending to conceive during the course of the study * Any medical condition deemed likely to interfere with the patient's participation in the study

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Conditions

The condition(s) this trial relates to.

atypical hemolytic-uremic syndrome thrombotic microangiopathy

As listed by the trial registrant

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

Atypical hemolytic uremic syndrome

How to take part

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

  1. The places running it

    26 sites in 7 countries. The list below names each one and where it is.

  2. 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 ↗

  3. 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

Locations

  • Novartis Investigative Site

    WITHDRAWN

    Nanjing, Jiangsu, 210009, China

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Beijing, 100034, China

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Beijing, 100730, China

  • Novartis Investigative Site

    WITHDRAWN

    Shanghai, 200025, China

  • Novartis Investigative Site

    RECRUITING

    Bordeaux, 33076, France

  • Novartis Investigative Site

    RECRUITING

    Paris, 75015, France

  • Novartis Investigative Site

    RECRUITING

    Paris, 75970, France

  • Novartis Investigative Site

    RECRUITING

    Rouen, 76031, France

  • Novartis Investigative Site

    RECRUITING

    Toulouse, 31054, France

  • Novartis Investigative Site

    RECRUITING

    Tours, 37044, France

  • Novartis Investigative Site

    RECRUITING

    Essen, 45147, Germany

  • Novartis Investigative Site

    RECRUITING

    Kiel, 24105, Germany

  • Novartis Investigative Site

    RECRUITING

    Ranica, BG, 24020, Italy

  • Novartis Investigative Site

    RECRUITING

    Milan, MI, 20122, Italy

  • Novartis Investigative Site

    RECRUITING

    Roma, RM, 00168, Italy

  • Novartis Investigative Site

    RECRUITING

    Matsumoto-shi, Nagano, 3908510, Japan

  • Novartis Investigative Site

    RECRUITING

    Iruma-gun, Saitama, 3500495, Japan

  • Novartis Investigative Site

    RECRUITING

    Bunkyo Ku, Tokyo, 113-8655, Japan

  • Novartis Investigative Site

    RECRUITING

    Santiago Compostela, A Coruna, 15706, Spain

  • Novartis Investigative Site

    RECRUITING

    Barcelona, 08036, Spain

  • Novartis Investigative Site

    RECRUITING

    Córdoba, 14004, Spain

  • Novartis Investigative Site

    RECRUITING

    Málaga, 29010, Spain

  • Novartis Investigative Site

    RECRUITING

    Seville, 41013, Spain

  • Novartis Investigative Site

    RECRUITING

    Valencia, 46026, Spain

  • Novartis Investigative Site

    WITHDRAWN

    Izmir, Balcova, 35340, Turkey (Türkiye)

  • Novartis Investigative Site

    RECRUITING

    Köseköy, Kocaeli, 41380, Turkey (Türkiye)

  • Novartis Investigative Site

    RECRUITING

    Ankara, Yenimahalle, 06500, Turkey (Türkiye)

  • Novartis Investigative Site

    RECRUITING

    Mersin, Yenisehir, 33110, Turkey (Türkiye)

  • Novartis Investigative Site

    RECRUITING

    Glasgow, Scotland, G51 4TF, United Kingdom

  • Novartis Investigative Site

    RECRUITING

    Newcastle upon Tyne, Tyne and Wear, NE7 7DN, United Kingdom

  • Novartis Investigative Site

    RECRUITING

    London, NW1 2BU, United Kingdom

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