ClinicalTrials.gov record NCT05795140

Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Aug 22, 2026

A Multi-center, Single Arm, Open-label Extension Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Iptacopan in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Who Have Completed a Preceding Iptacopan Phase 3 Study in aHUS

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
Age18 years old to 100 years old
SexAll
LocationBrazil, China, Czechia, India, Japan, Turkey
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This open-label, single-arm extension study follows adults with aHUS who completed a preceding Novartis Phase 3 iptacopan trial and remain on iptacopan (investigator must judge they derive benefit). Participants continue iptacopan 200 mg taken twice daily and are seen on-site every 4 months; a safety phone visit occurs 7 days after the last dose. The study collects safety data and measures of thrombotic microangiopathy and kidney function for up to 4 years.

What it is studying. The study is collecting long-term data on iptacopan given twice daily in people with aHUS. It focuses on safety and tolerability (adverse events, labs, vital signs, ECGs) and on measures of disease control and kidney health: presence or absence of thrombotic microangiopathy (TMA) signs, complete TMA response, estimated glomerular filtration rate (eGFR) and chronic kidney disease stage, and need for dialysis over time.

What the researchers hope to learn. To evaluate the long-term safety and tolerability of iptacopan in people with aHUS and to collect longer-term data on disease control (TMA outcomes), kidney function (eGFR and CKD stage), and dialysis requirements over up to 4 years.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

This is a multicenter, single arm, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of iptacopan in participants with aHUS.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Iptcaopan 200 mg

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Signed informed consent must be obtained prior to participation in the open label extension study
  • Willing and able to comply with the study Schedule of Activities
  • 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
  • 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

  • Concomitant treatment with any complement inhibitor as well as concomitant treatment with any of the prohibited drugs
  • 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
  • Active infection or history of recurrent invasive infections caused by encapsulated bacteria such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
  • History of hypersensitivity to iptacopan or its excipients or to drugs of similar chemical classes
  • Pregnant or nursing (lactating) women
  • 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.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

The extension study Baseline/Day 1 visit is equivalent to the End of Treatment visit of the parent study. The study will begin on Day 1 followed by on-site visits every 4 months during the study treatment period. A Safety Follow Up tele-visit must be conducted 7 days after last study treatment to collect information on Adverse Events.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants continue taking study-supplied iptacopan capsules (200 mg, hard gelatin capsules, taken twice daily). The extension begins with a Baseline/Day 1 visit (equivalent to the parent study's end-of-treatment visit), then on-site visits every 4 months during treatment with regular safety monitoring (blood tests, vital signs, ECGs) and assessments of kidney and blood measures. A safety follow-up telephone visit is done 7 days after the last study treatment to collect adverse event information. The study collects safety and efficacy data throughout the study period (up to 4 years as specified in the record).

How the study is structured. Interventional, open-label, single-group extension study. Enrolled participants receive iptacopan 200 mg capsules taken twice a day. Study visits occur approximately every 4 months during treatment, with a safety follow-up phone visit 7 days after the final dose. Participants are followed for safety and disease measures for up to about 4 years.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in Brazil, China, Czechia, India, Japan, Turkey. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on atypical hemolytic uremic syndrome

Clinical trials13 trials listed for atypical hemolytic uremic syndromeEvery trial the registry lists for atypical hemolytic uremic syndrome, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in atypical hemolytic uremic syndrome researchLatest monthly update: September 2026. Includes: Ravulizumab and labs. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
May 8, 2024
Primary completion
Jul 16, 2032 (estimated)
Study completion
Jul 27, 2032 (estimated)
Target enrollment
65 participants (estimated)
Last updated on registry
May 11, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Novartis Pharmaceuticals

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (8)

  • Novartis Investigative SiteActive Not Recruiting

    São Paulo, São Paulo, 05403 000 · Brazil

  • Novartis Investigative SiteRecruiting

    Rio de Janeiro, 22270-060 · Brazil

  • Novartis Investigative SiteRecruiting

    Beijing, 100034 · China

  • Novartis Investigative SiteRecruiting

    Ostrava, Poruba, 708 52 · Czechia

  • Novartis Investigative SiteActive Not Recruiting

    Nagpur, Maharashtra, 440015 · India

  • Novartis Investigative SiteRecruiting

    Pune, Maharashtra, 411011 · India

  • Novartis Investigative SiteRecruiting

    Iruma-gun, Saitama, 3500495 · Japan

  • Novartis Investigative SiteRecruiting

    Mersin, Yenisehir, 33110 · Turkey (Türkiye)

Terms used on this page · explanations by MedicalUpdates.ai

single arm
All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
extension study
A continuation study in which participants who completed an earlier study can keep receiving the treatment, usually with everyone knowing what they are given.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
Concomitant treatment
Other medicines a participant takes during the study. Some must be reported; some are restricted.
investigational drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
exclusion criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
Follow Up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
Adverse Events
Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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