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Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
RecruitingPhase 3
Sponsor: Novartis Pharmaceuticals
Conditions: Atypical Hemolytic Uremic Syndrome
Interventions: Iptacopan
Countries: China, France, Germany, Italy, Japan, Spain, Turkey (Türkiye), United Kingdom
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.
Eligibility overview
Sex: ALL
Age: 18 Years to 100 Years
Healthy volunteers: No
Study type: INTERVENTIONAL
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
Locations (31)
- Nanjing, Jiangsu, China
- Beijing, China
- Beijing, China
- Shanghai, China
- Bordeaux, France
- Paris, France
- Paris, France
- Rouen, France
- Toulouse, France
- Tours, France
- Essen, Germany
- Kiel, Germany
- Ranica, BG, Italy
- Milan, MI, Italy
- Roma, RM, Italy
- Matsumoto-shi, Nagano, Japan
- Iruma-gun, Saitama, Japan
- Bunkyo Ku, Tokyo, Japan
- Santiago Compostela, A Coruna, Spain
- Barcelona, Spain
- Córdoba, Spain
- Málaga, Spain
- Seville, Spain
- Valencia, Spain
- Izmir, Balcova, Turkey (Türkiye)
- Köseköy, Kocaeli, Turkey (Türkiye)
- Ankara, Yenimahalle, Turkey (Türkiye)
- Mersin, Yenisehir, Turkey (Türkiye)
- Glasgow, Scotland, United Kingdom
- Newcastle upon Tyne, Tyne and Wear, United Kingdom
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