Last Update: Dec 26, 2023
A Multicenter, Single-arm, Open Label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily LNP023 in Adult aHUS Patients Who Are Naive to Complement Inhibitor Therapy
ClinicalTrials.gov Identifier:
Novartis Reference Number:CLNP023F12301
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 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.

Atypical Hemolytic Uremic Syndrome
Phase 3
Recruiting
50
Jan 17, 2022
Jan 06, 2026
All
18 Years - (Adult, Older Adult)

Interventions

Drug

Iptacopan

Iptacopan 200mg twice daily oral

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

Study Location

Novartis Investigative Site

Recruiting

Wien,1090,Austria

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Innsbruck,Tyrol,6020,Austria

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Fortaleza,CE,60430 370,Brazil

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Sao Paulo,SP,05403 000,Brazil

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Porto Alegre,RS,90020-090,Brazil

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Belo Horizonte,MG,30150-221,Brazil

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Brasilia,DF,71635-580,Brazil

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Salvador,40301-155,Brazil

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São Paulo,SP,04038-002,Brazil

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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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Nanjing,Jiangsu,210009,China

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Shanghai,200025,China

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Shanxi,710063,China

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Ostrava,Poruba,708 52,Czech Republic

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Praha 4,140 00,Czech Republic

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Praha,12808,Czech Republic

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Heraklion Crete,711 10,Greece

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Thessaloniki,GR,570 10,Greece

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Athens,115 27,Greece

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Vellore,Tamil Nadu,632517,India

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Hyderabad,Andhra Pradesh,500012,India

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Chandigarh,Punjab,160012,India

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Lucknow,Uttar Pradesh,226014,India

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Chennai,Tamil Nadu,600 006,India

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Nagpur,Maharashtra,440015,India

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Pune,Maharashtra,411011,India

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Thiruvananthapuram,Kerala,695011,India

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Bunkyo ku,Tokyo,113 8655,Japan

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Iruma-gun,Saitama,350-0495,Japan

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Izumo-city,Shimane,693 8501,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,833 05,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

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Bronx,New York,10461,United States

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Washington,District of Columbia,20007 2197,United States

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Los Angeles,California,90033,United States

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Temple,Texas,76502,United States

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Cleveland,Ohio,44195,United States

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Albuquerque,New Mexico,87131-0001,United States

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Torrance,California,90502,United States

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Durham,North Carolina,27710,United States

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