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Testing iptacopan for treating atypical hemolytic uremic syndrome in adults

A Multicenter, Single-arm, Open Label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily LNP023 in Adult aHUS Patients Who Are Naive to Complement Inhibitor Therapy

Phase 3 Interventional Novartis · NCT04889430

This study is testing if a new medication called iptacopan can help adults with atypical hemolytic uremic syndrome feel better and improve their kidney health.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment50 (estimated)
Ages18 Years to 100 Years
SexAll
SponsorNovartis Industry-sponsored
Locations68 sites (Birmingham, Alabama and 67 other locations)
Trial IDNCT04889430 on ClinicalTrials.gov

What this trial studies

This Phase 3 study evaluates the efficacy and safety of iptacopan (LNP023) in adult patients with atypical hemolytic uremic syndrome (aHUS) who have not previously received complement inhibitor therapy. It is a multicenter, single-arm, open-label study that will enroll approximately 50 participants. The treatment involves administering iptacopan at a dose of 200 mg twice daily, with assessments focusing on hematological and kidney parameters, dialysis requirements, chronic kidney disease stage changes, and patient-reported outcomes related to fatigue and quality of life.

Who should consider this trial

Good fit: Ideal candidates are adult patients with active thrombotic microangiopathy, including thrombocytopenia, hemolysis, and acute kidney injury, who have not been treated with complement inhibitors.

Not a fit: Patients who have previously received treatment with complement inhibitors or have specific conditions like ADAMTS13 deficiency or Shiga toxin-related hemolytic uremic syndrome may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with aHUS, potentially improving their kidney function and overall quality of life.

How similar studies have performed: While this approach is novel in the context of aHUS treatment, similar studies targeting thrombotic microangiopathy have shown promise, indicating potential for success.

Eligibility criteria

Show full inclusion / exclusion criteria 
Main Inclusion Criteria:

* Adult patients with evidence of active thrombotic microangiopathy (TMA), including thrombocytopenia, evidence of hemolysis, and acute kidney injury
* Vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections are required prior to the start of study treatment. If the patient has not been previously vaccinated, or if a booster is required, vaccine should be given according to local regulations, at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post vaccination or before vaccination is given, prophylactic antibiotic treatment must be administered at the start of study treatment and for at least 2 weeks after vaccination

Main Exclusion Criteria:

* Treatment with complement inhibitors, including anti-C5 antibody
* ADAMTS13 deficiency (\<10% activity or \<0.1U/ml), and/or Shiga toxin-related hemolytic uremic syndrome (STx-HUS), and/or Positive direct Coombs test
* Identified drug exposure-related HUS or HUS related to known genetic defects of cobalamin C metabolism or known diacylglycerol kinase ε (DGKE) mediated aHUS
* Receiving PE/PI, for 14 days or longer, prior to the start of screening for the current TMA
* Bone marrow transplantation (BMT)/hematopoietic stem cell transplantation (HSCT), heart, lung, small bowel, pancreas, or liver transplantation
* Patients with sepsis or active severe systemic bacterial, viral (including COVID-19) or fungal infection, systemic infection which confounds an accurate diagnosis of aHUS or impedes the ability to manage the aHUS disease, active infection (or history of recurrent invasive infections) caused by encapsulated bacteria
* Kidney disease suggestive of other disease than aHUS or of chronic kidney failure or family history of non-complement mediated genetic kidney disease
* Liver disease or liver injury at screening
* Systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), or antiphospholipid antibody positivity or syndrome
* Chronic hemo- or peritoneal dialysis

Other protocol-defined inclusion/exclusion criteria may apply

Where this trial is running

Birmingham, Alabama and 67 other locations

+18 more sites — see ClinicalTrials.gov for the full list.

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 SyndromeLNP023iptacopanaHUSatypical hemolytic uremic syndromethrombotic microangiopathy
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.