Study Description
The purpose of this Phase 3 study is to determine whether iptacopan (LNP023) is efficacious and safe for the treatment of aHUS in adult patients who are treatment naive to complement inhibitor therapy. The study is designed as a multicenter, single-arm, open label study to demonstrate the efficacy and safety of LNP023 (iptacopan) at a dose of 200 mg b.i.d. in adult patients with aHUS who are treatment naive to complement inhibitor therapy (including anti-C5 antibody). The study will enroll approximately 50 participants and assess the effects of iptacopan on a range of efficacy assessments relevant to aHUS including hematological and kidney parameters, dialysis requirement, changes in chronic kidney disease (CKD) stage, as well as patient reported outcomes (PRO) for fatigue and quality of life.
Interventions
Iptacopan
Eligibility Criteria
Main Inclusion Criteria:
* Adult patients with evidence of active thrombotic microangiopathy (TMA), including thrombocytopenia, evidence of hemolysis, and acute kidney injury
* Vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections are required prior to the start of study treatment. If the patient has not been previously vaccinated, or if a booster is required, vaccine should be given according to local regulations, at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post vaccination or before vaccination is given, prophylactic antibiotic treatment must be administered at the start of study treatment and for at least 2 weeks after vaccination
Main Exclusion Criteria:
* Treatment with complement inhibitors, including anti-C5 antibody
* ADAMTS13 deficiency (\<10% activity or \<0.1U/ml), and/or Shiga toxin-related hemolytic uremic syndrome (STx-HUS), and/or Positive direct Coombs test
* Identified drug exposure-related HUS or HUS related to known genetic defects of cobalamin C metabolism or known diacylglycerol kinase ε (DGKE) mediated aHUS
* Receiving PE/PI, for 14 days or longer, prior to the start of screening for the current TMA
* Bone marrow transplantation (BMT)/hematopoietic stem cell transplantation (HSCT), heart, lung, small bowel, pancreas, or liver transplantation
* Patients with sepsis or active severe systemic bacterial, viral (including COVID-19) or fungal infection, systemic infection which confounds an accurate diagnosis of aHUS or impedes the ability to manage the aHUS disease, active infection (or history of recurrent invasive infections) caused by encapsulated bacteria
* Kidney disease suggestive of other disease than aHUS or of chronic kidney failure or family history of non-complement mediated genetic kidney disease
* Liver disease or liver injury at screening
* Systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), or antiphospholipid antibody positivity or syndrome
* Chronic hemo- or peritoneal dialysis
Other protocol-defined inclusion/exclusion criteria may apply
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Innsbruck,Tyrol,6020,Austria
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Wien,1090,Austria
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Belo Horizonte,MG,30150-221,Brazil
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Rio de Janeiro,22270-60,Brazil
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Pernambuco,Recife,50740-900,Brazil
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Salvador,40301-155,Brazil
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Porto Alegre,RS,90035-074,Brazil
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Botucatu,SP,18618-970,Brazil
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Sao Paulo,SP,01327 001,Brazil
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Fortaleza,CE,60430 370,Brazil
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Sao Paulo,SP,04038-002,Brazil
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Brasilia,DF,71635-580,Brazil
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Sao Paulo,SP,05403 000,Brazil
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Beijing,100191,China
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Beijing,100730,China
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Shanghai,200025,China
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Guangzhou,Guangdong,510080,China
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Shanxi,710063,China
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Nanjing,Jiangsu,210009,China
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Yantai,Shandong,264000,China
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Beijing,100034,China
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Ostrava,Poruba,708 52,Czechia
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Prague 4,146 24,Czechia
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Praha,12808,Czechia
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Thessaloniki,GR,570 10,Greece
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Athens,115 27,Greece
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Heraklion Crete,71110,Greece
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Pune,Maharashtra,411011,India
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Chandigarh,Punjab,160012,India
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Chennai,Tamil Nadu,600006,India
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Vellore,Tamil Nadu,632 004,India
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Hyderabad,Andhra Pradesh,500012,India
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Lucknow,Uttar Pradesh,226014,India
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Thiruvananthapuram,Kerala,695011,India
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Nagpur,Maharashtra,440015,India
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Izumo-city,Shimane,693 8501,Japan
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Bunkyo ku,Tokyo,113 8655,Japan
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Iruma-gun,Saitama,350-0495,Japan
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Seoul,03080,Korea, Republic of
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Seoul,03722,Korea, Republic of
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Seoul,06591,Korea, Republic of
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Bratislava,83305,Slovakia
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Martin,03601,Slovakia
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Ljubljana,1000,Slovenia
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Taichung,40447,Taiwan
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Taoyuan,33305,Taiwan
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London,Nw1 2bu,United Kingdom
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Newcastle upon Tyne,Ne7 7dn,United Kingdom
Comprehensive Transplant Ctr at OSU
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Columbus,Ohio,43210,United States
Uday Nori
Dahlia Najjar
Uni Of Alabama At Birmingham
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Birmingham,Alabama,35233,United States
Jennifer Newby
Song Ong
Duke University Medical Center
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Durham,North Carolina,27710,United States
Gowthami Arepally
Montefiore Medical Center .
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Bronx,New York,10461,United States
Gretchen Mackie
Irina Murakhovskaya
WA Uni School Of Med
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Saint Louis,Missouri,63110,United States
Anuja Java
Teresa Arb
Harbor-UCLA Medical Center .
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Torrance,California,90502,United States
Sarah Tomassetti
UT Southwestern Medical Center
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Dallas,Texas,75390,United States
David Wojciechowski
University of Cincinnati
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Cincinnati,Ohio,45267-0585,United States
Amit Govil
Kyra Parker
Georgetown University Lombardi Cancer Center
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Washington,District of Columbia,20007 2197,United States
Catherine Broome
Univ of California at Los Angeles
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Los Angeles,California,90095,United States
Ahad Qureshi
Phuong-Thu Pham
Rut Univ for Translational Med Scie
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New Brunswick,New Jersey,08901,United States
Ashwin Sridharan
Baylor Scott and White Research
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Temple,Texas,76502,United States
Mohanram Narayanan
USC Norris Cancer Center
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Los Angeles,California,90033,United States
Ilene Weitz
Cleveland Clinic Foundation
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Cleveland,Ohio,44195,United States
Ziad Zaky
University of New Mexico
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Albuquerque,New Mexico,87131-0001,United States
Namita Singh
University Of Miami
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Miami,Florida,33136,United States
Adela Mattiazzi
Univ Cali Irvine ALS Neuromuscular
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Orange,California,92868,United States
Ramy Hanna
University of Minnesota
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Minneapolis,Minnesota,55455,United States
Nattawat Klomjit
Virginia Commonwealth University
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Richmond,Virginia,23298,United States
Gaurav Gupta
Brigham and Womens Hospital
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Boston,Massachusetts,02115,United States
Rania El Fekih
Worldwide Contacts
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