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New hope for aHUS patients: Long-Term iptacopan study launches

NCT ID NCT05795140

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 looks at the long-term safety and effectiveness of the drug iptacopan in people with a rare blood disorder called atypical hemolytic uremic syndrome (aHUS). Participants must have completed a previous iptacopan study and still benefit from the drug. The goal is to monitor side effects and see if the drug continues to control the disease over time.

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 65 people

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

Started

May 2024

Expected to finish

Jul 2032

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: 1. Signed informed consent must be obtained prior to participation in the open label extension study 2. Willing and able to comply with the study Schedule of Activities 3. Participants who have completed the full study treatment period of any prior "Novartis sponsored" iptacopan Phase 3 clinical trial in aHUS (e.g. CLNP023F12301 , CLNP023F12302), are still on iptacopan study treatment and derive benefit from it as per Investigator's judgement 4. Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections should be up to date (i.e., any boosters required should be administered according to local guidelines) Exclusion Criteria: 1. Concomitant treatment with any complement inhibitor as well as concomitant treatment with any of the prohibited drugs 2. Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the Investigator could put the participant at risk 3. Active infection or history of recurrent invasive infections caused by encapsulated bacteria such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae 4. History of hypersensitivity to iptacopan or its excipients or to drugs of similar chemical classes 5. Pregnant or nursing (lactating) women 6. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of investigational drug and for 1 week after stopping of investigational drug. Other protocol-defined inclusion/exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

atypical hemolytic-uremic syndrome membranoproliferative glomerulonephritis 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

    7 sites in 6 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

    ACTIVE_NOT_RECRUITING

    São Paulo, São Paulo, 05403 000, Brazil

  • Novartis Investigative Site

    RECRUITING

    Rio de Janeiro, 22270-060, Brazil

  • Novartis Investigative Site

    RECRUITING

    Beijing, 100034, China

  • Novartis Investigative Site

    RECRUITING

    Ostrava, Poruba, 708 52, Czechia

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Nagpur, Maharashtra, 440015, India

  • Novartis Investigative Site

    RECRUITING

    Pune, Maharashtra, 411011, India

  • Novartis Investigative Site

    RECRUITING

    Iruma-gun, Saitama, 3500495, Japan

  • Novartis Investigative Site

    RECRUITING

    Ankara, Yenimahalle, 06500, Turkey (Türkiye)

  • Novartis Investigative Site

    RECRUITING

    Mersin, Yenisehir, 33110, Turkey (Türkiye)

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