Last Update: Apr 29, 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 ClinicalTrials.gov Identifier: [NCT05935215](https://clinicaltrials.gov/ct2/show/NCT05935215) Novartis Reference Number:CLNP023F12302 [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 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. 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. Condition Atypical Hemolytic Uremic Syndrome Phase Phase3 Overall Status Recruiting Number of Participants50 Start Date Feb 28, 2024 Completion Date Jul 19, 2029 Gender All Age(s) 18 Years - 100 Years (Adult, Older Adult) ## Interventions Drug ### Iptacopan Open Label ## Eligibility 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 France #### Novartis Investigative Site Recruiting Paris,75970,France #### Novartis Investigative Site Recruiting Rouen,76031,France #### Novartis Investigative Site Recruiting Toulouse,31054,France #### Novartis Investigative Site Recruiting Tours,37044,France #### Novartis Investigative Site Recruiting Bordeaux,33076,France #### Novartis Investigative Site Recruiting Paris,75015,France Germany #### Novartis Investigative Site Recruiting Essen,45147,Germany #### Novartis Investigative Site Recruiting Kiel,24105,Germany Italy #### Novartis Investigative Site Recruiting Ranica,BG,24020,Italy #### Novartis Investigative Site Recruiting Roma,RM,00168,Italy #### Novartis Investigative Site Recruiting Milan,MI,20122,Italy Japan #### Novartis Investigative Site Recruiting Bunkyo Ku,Tokyo,113-8655,Japan #### Novartis Investigative Site Recruiting Iruma-gun,Saitama,3500495,Japan #### Novartis Investigative Site Recruiting Matsumoto-shi,Nagano,3908510,Japan Spain #### Novartis Investigative Site Recruiting Málaga,29010,Spain #### Novartis Investigative Site Recruiting Seville,41013,Spain #### Novartis Investigative Site Recruiting Córdoba,14004,Spain #### Novartis Investigative Site Recruiting Barcelona,08036,Spain #### Novartis Investigative Site Recruiting Santiago Compostela,A Coruna,15706,Spain #### Novartis Investigative Site Recruiting Valencia,46026,Spain Turkey (Türkiye) #### Novartis Investigative Site Recruiting Ankara,Yenimahalle,06500,Turkey (Türkiye) #### Novartis Investigative Site Recruiting Mersin,Yenisehir,33110,Turkey (Türkiye) #### Novartis Investigative Site Recruiting Köseköy,Kocaeli,41380,Turkey (Türkiye) United Kingdom #### Novartis Investigative Site Recruiting Newcastle upon Tyne,Tyne and Wear,Ne7 7dn,United Kingdom #### Novartis Investigative Site Recruiting Glasgow,Scotland,G51 4tf,United Kingdom #### Novartis Investigative Site Recruiting London,Nw1 2bu,United Kingdom ## Worldwide Contacts If the location of your choosing does not feature any contact detail, please reach out using the information below. #### Novartis Pharmaceuticals Phone: [ +41613241111](tel:+41613241111) Email: [](mailto:) #### Novartis Pharmaceuticals Phone: [ 1-888-669-6682](tel:1-888-669-6682) Email: