Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy
NCT ID: NCT04889430
Last Updated: 2025-12-11
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
34 participants
INTERVENTIONAL
2022-01-17
2029-03-29
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Iptacopan 200 mg b.i.d
Single arm open-label with 50 adult patients receiving 200mg oral twice daily doses of iptacopan
Iptacopan
Iptacopan 200mg twice daily oral
Interventions
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Iptacopan
Iptacopan 200mg twice daily oral
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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
18 Years
100 Years
ALL
No
Sponsors
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Novartis Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Novartis Pharmaceuticals
Role: STUDY_DIRECTOR
Novartis Pharmaceuticals
Locations
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Uni Of Alabama At Birmingham
Birmingham, Alabama, United States
USC Norris Cancer Center
Los Angeles, California, United States
Univ of California at Los Angeles
Los Angeles, California, United States
Univ Cali Irvine ALS Neuromuscular
Orange, California, United States
Univ of California at Sacramento
Sacramento, California, United States
Harbor-UCLA Medical Center .
Torrance, California, United States
Georgetown University Lombardi Cancer Center
Washington D.C., District of Columbia, United States
University Of Miami
Miami, Florida, United States
Brigham and Womens Hospital
Boston, Massachusetts, United States
University of Minnesota
Minneapolis, Minnesota, United States
St Barnabas Hospital
Livingston, New Jersey, United States
Rut Univ for Translational Med Scie
New Brunswick, New Jersey, United States
Montefiore Medical Center .
The Bronx, New York, United States
Montefiore Medical Center
The Bronx, New York, United States
Duke University Medical Center
Durham, North Carolina, United States
University of Cincinnati
Cincinnati, Ohio, United States
Cleveland Clinic Foundation
Cleveland, Ohio, United States
Comprehensive Transplant Ctr at OSU
Columbus, Ohio, United States
UT Southwestern Medical Center
Dallas, Texas, United States
Virginia Commonwealth University
Richmond, Virginia, United States
Novartis Investigative Site
Vienna, , Austria
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Fortaleza, Ceará, Brazil
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Brasília, Federal District, Brazil
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Belo Horizonte, Minas Gerais, Brazil
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Recife, Pernambuco, Brazil
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Porto Alegre, Rio Grande do Sul, Brazil
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Botucatu, São Paulo, Brazil
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São Paulo, São Paulo, Brazil
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São Paulo, São Paulo, Brazil
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São Paulo, São Paulo, Brazil
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Rio de Janeiro, , Brazil
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Salvador, , Brazil
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Guangzhou, Guangdong, China
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Nanjing, Jiangsu, China
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Yantai, Shandong, China
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Xian, Shanxi, China
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Beijing, , China
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Beijing, , China
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Beijing, , China
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Shanghai, , China
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Ostrava, Poruba, Czechia
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Thiruvananthapuram, Kerala, India
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Nagpur, Maharashtra, India
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Pune, Maharashtra, India
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Chandigarh, Punjab, India
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Chennai, Tamil Nadu, India
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Vellore, Tamil Nadu, India
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Hyderabad, Telangana, India
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Lucknow, Uttar Pradesh, India
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Iruma-gun, Saitama, Japan
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Izumo, Shimane, Japan
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Bunkyo Ku, Tokyo, Japan
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Bratislava, Slovakia, Slovakia
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Martin, Slovakia, Slovakia
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Seoul, Korea, South Korea
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Seoul, , South Korea
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Taichung, , Taiwan
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Taoyuan District, , Taiwan
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Newcastle upon Tyne, Tyne and Wear, United Kingdom
Countries
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Other Identifiers
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2020-005186-13
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
CLNP023F12301
Identifier Type: -
Identifier Source: org_study_id
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