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
Novartis Investigative Site
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Wien,1090,Austria
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Innsbruck,Tyrol,6020,Austria
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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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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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Nanjing,Jiangsu,210009,China
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Yantai,Shandong,264000,China
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Beijing,100034,China
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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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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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Thiruvananthapuram,Kerala,695011,India
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Nagpur,Maharashtra,440015,India
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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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Iruma-gun,Saitama,350-0495,Japan
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Izumo-city,Shimane,693 8501,Japan
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Bunkyo ku,Tokyo,113 8655,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
USC Norris Cancer Center
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Los Angeles,California,90033,United States
Ilene Weitz
University of New Mexico Clin and Transl Science Ctr
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Albuquerque,New Mexico,87131-0001,United States
Namita Singh
UT Southwestern Medical Center .
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Dallas,Texas,75390,United States
David Wojciechowski
Georgetown University Lombardi Cancer Center
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Washington,District of Columbia,20007 2197,United States
Catherine Broome
University of Cincinnati Div of Transplantation
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Cincinnati,Ohio,45267-0585,United States
Kyra Parker
Amit Govil
Baylor Scott and White Research .
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Temple,Texas,76502,United States
Mohanram Narayanan
University Of Miami
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Miami,Florida,33136,United States
Adela Mattiazzi
Cleveland Clinic Foundation Nephrology and Hypertension
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Cleveland,Ohio,44195,United States
Ziad Zaky
Univ Cali Irvine ALS Neuromuscular Nephrology Department
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Orange,California,92868,United States
Ramy Hanna
Montefiore Medical Center .
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Bronx,New York,10461,United States
Gretchen Mackie
Irina Murakhovskaya
University of Minnesota
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Minneapolis,Minnesota,55455,United States
Nattawat Klomjit
Harbor-UCLA Medical Center .
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Torrance,California,90502,United States
Sarah Tomassetti
Virginia Commonwealth University .
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Richmond,Virginia,23298,United States
Gaurav Gupta
Comprehensive Transplant Ctr at OSU Division of Transplant Surgery
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Columbus,Ohio,43210,United States
Dahlia Najjar
Uday Nori
Duke University Medical Center .
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Durham,North Carolina,27710,United States
Gowthami Arepally
Uni Of Alabama At Birmingham Division of Nephrology
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Birmingham,Alabama,35233,United States
Jennifer Newby
Song Ong
WA Uni School Of Med .
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Saint Louis,Missouri,63110,United States
Anuja Java
Jennifer Bruns
Worldwide Contacts
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