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 8 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.
Interventions
Iptacopan
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 weight-based dosage regimen of anti-C5 antibody treatment for at least 3 months prior to the screening visit.
* Clinical evidence of response to anti-C5 antibody treatment (in absence of PE/PI) for at least 3 months prior to entering the screening period as defined by:
1. Hematological normalization in platelet count ≥150 x 109/L and LDH below upper limit of normal \[ULN\], and
2. Stable or improving kidney function as defined by ≤15% increase in serum creatinine.
* 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
Novartis Investigative Site
Recruiting
Nanjing,Jiangsu,210009,China
Novartis Investigative Site
Recruiting
Beijing,100034,China
Novartis Investigative Site
Recruiting
Beijing,100730,China
Novartis Investigative Site
Recruiting
Shanghai,200025,China
Novartis Investigative Site
Recruiting
Iruma-gun,Saitama,350-0495,Japan
Novartis Investigative Site
Recruiting
Bunkyo ku,Tokyo,113 8655,Japan
Novartis Investigative Site
Recruiting
Izmir,35340,Turkey
Novartis Investigative Site
Recruiting
Kocaeli,41380,Turkey
Novartis Investigative Site
Recruiting
Mersin,33110,Turkey
Novartis Investigative Site
Recruiting
Ankara,06500,Turkey
Novartis Investigative Site
Recruiting
London,Nw1 2bu,United Kingdom
Worldwide Contacts
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