Last Update: Jun 04, 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 ClinicalTrials.gov Identifier: [NCT05795140](https://clinicaltrials.gov/ct2/show/NCT05795140) Novartis Reference Number:CLNP023F12001B [See if you Pre-qualify](#trial-eligibility "See if you Pre-qualify") All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. ## Study Description 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. 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. Condition Atypical Hemolytic Uremic Syndrome Phase Phase3 Overall Status Recruiting Number of Participants65 Start Date May 08, 2024 Completion Date Jul 27, 2032 Gender All Age(s) 18 Years - 100 Years (Adult, Older Adult) ## Interventions Drug ### Iptcaopan 200 mg Open label, participant specific kits, hard gelatin capsules to be taken twice a day ## Eligibility 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 (e.g. CLNP023F12301 , CLNP023F12302), 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. Brazil #### Novartis Investigative Site Recruiting Rio de Janeiro,22270-060,Brazil China #### Novartis Investigative Site Recruiting Beijing,100034,China Czechia #### Novartis Investigative Site Recruiting Ostrava,Poruba,708 52,Czechia India #### Novartis Investigative Site Recruiting Pune,Maharashtra,411011,India Japan #### Novartis Investigative Site Recruiting Iruma-gun,Saitama,3500495,Japan Turkey (Türkiye) #### Novartis Investigative Site Recruiting Mersin,Yenisehir,33110,Turkey (Türkiye) #### Novartis Investigative Site Recruiting Ankara,Yenimahalle,06500,Turkey (Türkiye) ## Worldwide Contacts If the location of your choosing does not feature any contact detail, please reach out using the information below. #### Novartis Pharmaceuticals Phone: [ +81337978748](tel:+81337978748) Email: [](mailto:) #### Novartis Pharmaceuticals Phone: [ +41613241111](tel:+41613241111) Email: