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ACTIVE NOT RECRUITING

NCT04889430

PHASE3

Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy

Sponsor: Novartis Pharmaceuticals

View on ClinicalTrials.gov

Summary

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.

Official title: 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

Key Details

Gender

All

Age Range

18 Years - 100 Years

Study Type

INTERVENTIONAL

Enrollment

Start Date

2022-01-17

Completion Date

2026-08-13

Last Updated

2026-04-01

Healthy Volunteers

Conditions

Atypical Hemolytic Uremic Syndrome

Interventions

DRUG

Iptacopan

Iptacopan 200mg twice daily oral

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

Locations (48)

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

University of Minnesota

Minneapolis, Minnesota, 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

Fortaleza, Ceará, Brazil

Novartis Investigative Site

Brasília, Federal District, Brazil

Novartis Investigative Site

Belo Horizonte, Minas Gerais, Brazil

Novartis Investigative Site

Recife, Pernambuco, Brazil

Novartis Investigative Site

Porto Alegre, Rio Grande do Sul, Brazil

Novartis Investigative Site

Botucatu, São Paulo, Brazil

Novartis Investigative Site

São Paulo, São Paulo, Brazil

Novartis Investigative Site

São Paulo, São Paulo, Brazil

Novartis Investigative Site

São Paulo, São Paulo, Brazil

Novartis Investigative Site

Rio de Janeiro, Brazil

Novartis Investigative Site

Salvador, Brazil

Novartis Investigative Site

Nanjing, Jiangsu, China

Novartis Investigative Site

Yantai, Shandong, China

Novartis Investigative Site

Beijing, China

Novartis Investigative Site

Beijing, China

Novartis Investigative Site

Beijing, China

Novartis Investigative Site

Shanghai, China

Novartis Investigative Site

Ostrava, Poruba, Czechia

Novartis Investigative Site

Thiruvananthapuram, Kerala, India

Novartis Investigative Site

Nagpur, Maharashtra, India

Novartis Investigative Site

Pune, Maharashtra, India

Novartis Investigative Site

Chandigarh, Punjab, India

Novartis Investigative Site

Chennai, Tamil Nadu, India

Novartis Investigative Site

Vellore, Tamil Nadu, India

Novartis Investigative Site

Hyderabad, Telangana, India

Novartis Investigative Site

Lucknow, Uttar Pradesh, India

Novartis Investigative Site

Iruma-gun, Saitama, Japan

Novartis Investigative Site

Izumo, Shimane, Japan

Novartis Investigative Site

Bunkyo Ku, Tokyo, Japan

Novartis Investigative Site

Seoul, Korea, South Korea

Novartis Investigative Site

Taichung, Taiwan

Novartis Investigative Site

Taoyuan District, Taiwan

Clinical Research Directory

Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (48)