For Healthcare Professionals

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


About the study

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.

Who can take part

You may be eligible to participate in the study if you meet the following criteria:


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, 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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Study Locations

Enter your ZIP code/Postal code/PIN code to locate study sites near you:

How to Apply

Contact the study center to learn if this study is a good match for you.

Study’s details


Atypical Hemolytic Uremic Syndrome

Age (in years)



Phase 3

Participants needed


Est. Completion Date

Aug 23, 2029

Treatment type



Novartis identifier


Study number


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