ClinicalTrials.gov record NCT05935215

Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

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

A Multicenter, Single Arm, Open-label Study to Evaluate Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With 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
LocationChina, France, Germany, Italy, Japan, Spain, Turkey, United Kingdom
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 single-arm, open-label Phase 3 study gives adults with aHUS who have been treated with an anti‑C5 antibody a chance to switch to iptacopan (200 mg twice daily). After screening (up to 14 weeks), participants enter a 12-month core treatment period and a 12-month extension. The study will track blood and kidney measures and whether thrombotic microangiopathy (TMA) recurs after the switch.

What it is studying. The study is testing the efficacy and safety of switching from an anti‑C5 antibody to iptacopan in people with aHUS, by tracking TMA manifestations, blood counts, and kidney function over 12 and 24 months.

What the researchers hope to learn. To measure whether participants remain free of TMA after switching to iptacopan and to document safety. Outcomes include TMA recurrence at 12 and 24 months, time to TMA, and changes in platelets, LDH, hemoglobin, serum creatinine, urine protein-to-creatinine ratio, eGFR, CKD stage, and dialysis requirement.

What this study is about

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

The purpose of this Phase 3 study is to evaluate the efficacy and safety of iptacopan upon switching from anti-C5 antibody to iptacopan treatment in study 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
  • Iptacopan

Eligibility

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

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:
  • Hematological normalization in platelet count ≥150 x 10\^9/L and LDH below upper limit of normal [ULN], and
  • 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

Study description and what participation involves

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

Detailed description from the registry

The study is designed as a multicenter, single-arm, open label study to evaluate the efficacy and safety of iptacopan upon switching from anti-C5 antibody to iptacopan treatment in participants with aHUS. It consists of a screening period of up to 14 weeks followed by a 12-Month Core Treatment period and 12-Month Extension Treatment period. The study will assess the effects of iptacopan on a range of efficacy assessments relevant to aHUS.

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.

After a screening period of up to 14 weeks, participants stop anti‑C5 antibody and begin open‑label iptacopan 200 mg twice daily for a 12‑month Core Treatment and a 12‑month Extension (24 months total). They will attend regular clinic visits for safety and disease monitoring, including blood and urine tests to check platelets, LDH, hemoglobin, serum creatinine/eGFR and urine protein, with key assessments at baseline, month 12 and month 24 and central‑lab confirmations during screening. The study team will monitor for thrombotic microangiopathy (TMA) events and infections. Vaccination against meningococcus and pneumococcus must be completed before starting iptacopan (H. influenzae vaccine per local guidance).

How the study is structured. Interventional, single-group, open-label drug study. Eligible adults who have been on anti‑C5 antibody treatment are switched to iptacopan 200 mg twice daily. There is no placebo or masking. The study includes screening (up to 14 weeks), a 12-month core treatment period and a 12-month extension, with assessments at baseline, month 12 and month 24.

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 China, France, Germany, Italy, Japan, Spain, Turkey, United Kingdom. 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
Feb 28, 2024
Primary completion
Jul 21, 2028 (estimated)
Study completion
Jul 19, 2029 (estimated)
Target enrollment
50 participants (estimated)
Last updated on registry
Apr 29, 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 (31)

  • Novartis Investigative SiteWithdrawn

    Nanjing, Jiangsu, 210009 · China

  • Novartis Investigative SiteActive Not Recruiting

    Beijing, 100034 · China

  • Novartis Investigative SiteActive Not Recruiting

    Beijing, 100730 · China

  • Novartis Investigative SiteWithdrawn

    Shanghai, 200025 · China

  • Novartis Investigative SiteRecruiting

    Bordeaux, 33076 · France

  • Novartis Investigative SiteRecruiting

    Paris, 75015 · France

  • Novartis Investigative SiteRecruiting

    Paris, 75970 · France

  • Novartis Investigative SiteRecruiting

    Rouen, 76031 · France

  • Novartis Investigative SiteRecruiting

    Toulouse, 31054 · France

  • Novartis Investigative SiteRecruiting

    Tours, 37044 · France

Terms used on this page · explanations by MedicalUpdates.ai

screening period
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
upper limit of normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
relapse
A condition that improved with treatment and has since returned or worsened.
eGFR
A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
bone marrow transplant
A procedure that replaces blood-forming cells after high-dose treatment. 'Autologous' uses the person's own cells collected beforehand; 'allogeneic' uses cells from a donor.
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.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
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.

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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