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Switching from anti-C5 antibody treatment to iptacopan for aHUS patients

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

Phase 3 Interventional Novartis · NCT05935215

This study is testing if switching to a new treatment called iptacopan can help people with atypical hemolytic uremic syndrome (aHUS) feel better after using a different medication.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment50 (estimated)
Ages18 Years to 100 Years
SexAll
SponsorNovartis Industry-sponsored
Locations31 sites (Nanjing, Jiangsu and 30 other locations)
Trial IDNCT05935215 on ClinicalTrials.gov

What this trial studies

This Phase 3 study evaluates the efficacy and safety of iptacopan in patients with atypical hemolytic uremic syndrome (aHUS) who are switching from anti-C5 antibody treatment. It is a multicenter, single-arm, open-label study that includes a screening period of up to 8 weeks, followed by a 12-month core treatment period and an additional 12-month extension treatment period. The study will assess various efficacy outcomes relevant to aHUS to determine the benefits of iptacopan compared to the previous treatment.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with a confirmed diagnosis of aHUS who have been on anti-C5 antibody treatment for at least 3 months and have shown a clinical response.

Not a fit: Patients who have not responded to anti-C5 antibody treatment or have other types of thrombotic microangiopathy may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a more effective management option for patients with aHUS.

How similar studies have performed: Other studies have shown promising results with similar treatment approaches, indicating potential for success.

Eligibility criteria

Show full inclusion / exclusion criteria 
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

Where this trial is running

Nanjing, Jiangsu and 30 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Atypical Hemolytic Uremic SyndromeaHUS,thrombotic microangiopathyTMA
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.