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

Last updated: January 31, 2025
Sponsor: Novartis Pharmaceuticals
Overall Status: Active - Recruiting

Phase

3

Condition

Kidney Failure (Pediatric)

Platelet Disorders

Renal Failure

Treatment

Iptacopan

Clinical Study ID

NCT04889430
CLNP023F12301
2020-005186-13
  • Ages 18-100
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Main Inclusion Criteria:

  • Adult patients with evidence of active thrombotic microangiopathy (TMA), includingthrombocytopenia, evidence of hemolysis, and acute kidney injury

  • Vaccinations against Neisseria meningitidis, Streptococcus pneumoniae andHaemophilus influenzae infections are required prior to the start of studytreatment. If the patient has not been previously vaccinated, or if a booster isrequired, vaccine should be given according to local regulations, at least 2 weeksprior to first study drug administration. If study treatment has to start earlierthan 2 weeks post vaccination or before vaccination is given, prophylacticantibiotic treatment must be administered at the start of study treatment and for atleast 2 weeks after vaccination

Exclusion

Main Exclusion Criteria:

  • Treatment with complement inhibitors, including anti-C5 antibody

  • ADAMTS13 deficiency (<10% activity or <0.1U/ml), and/or Shiga toxin-relatedhemolytic uremic syndrome (STx-HUS), and/or Positive direct Coombs test

  • Identified drug exposure-related HUS or HUS related to known genetic defects ofcobalamin 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 thecurrent 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 ofaHUS or impedes the ability to manage the aHUS disease, active infection (or historyof recurrent invasive infections) caused by encapsulated bacteria

  • Kidney disease suggestive of other disease than aHUS or of chronic kidney failure orfamily history of non-complement mediated genetic kidney disease

  • Liver disease or liver injury at screening

  • Systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), orantiphospholipid antibody positivity or syndrome

  • Chronic hemo- or peritoneal dialysis

Other protocol-defined inclusion/exclusion criteria may apply

Study Design

Total Participants: 50
Treatment Group(s): 1
Primary Treatment: Iptacopan
Phase: 3
Study Start date:
January 17, 2022
Estimated Completion Date:
March 29, 2029

Study Description

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.

Connect with a study center

  • Novartis Investigative Site

    Innsbruck, Tyrol 6020
    Austria

    Active - Recruiting

  • Novartis Investigative Site

    Wien, 1090
    Austria

    Active - Recruiting

  • Novartis Investigative Site

    Fortaleza, CE 60430 370
    Brazil

    Active - Recruiting

  • Novartis Investigative Site

    Brasilia, DF 71635-580
    Brazil

    Active - Recruiting

  • Novartis Investigative Site

    Belo Horizonte, MG 30150-221
    Brazil

    Active - Recruiting

  • Novartis Investigative Site

    Porto Alegre, RS 90035-074
    Brazil

    Active - Recruiting

  • Novartis Investigative Site

    Pernambuco, Recife 50740-900
    Brazil

    Active - Recruiting

  • Novartis Investigative Site

    Botucatu, SP 18618-970
    Brazil

    Active - Recruiting

  • Novartis Investigative Site

    Sao Paulo, SP 05403 000
    Brazil

    Active - Recruiting

  • Novartis Investigative Site

    São Paulo, SP 04038-002
    Brazil

    Site Not Available

  • Novartis Investigative Site

    Rio de Janeiro, 22270-60
    Brazil

    Active - Recruiting

  • Novartis Investigative Site

    Salvador, 40301-155
    Brazil

    Active - Recruiting

  • Novartis Investigative Site

    Guangzhou, Guangdong 510080
    China

    Active - Recruiting

  • Novartis Investigative Site

    Nanjing, Jiangsu 210009
    China

    Active - Recruiting

  • Novartis Investigative Site

    Yantai, Shandong 264000
    China

    Active - Recruiting

  • Novartis Investigative Site

    Beijing, 100730
    China

    Active - Recruiting

  • Novartis Investigative Site

    Shanghai, 200025
    China

    Active - Recruiting

  • Novartis Investigative Site

    Shanxi, 710063
    China

    Active - Recruiting

  • Novartis Investigative Site

    Ostrava Poruba, Czech Republic 708 52
    Czechia

    Site Not Available

  • Novartis Investigative Site

    Ostrava, Poruba 708 52
    Czechia

    Active - Recruiting

  • Novartis Investigative Site

    Prague 4, 146 24
    Czechia

    Active - Recruiting

  • Novartis Investigative Site

    Praha, 12808
    Czechia

    Active - Recruiting

  • Novartis Investigative Site

    Praha 4, 140 00
    Czechia

    Site Not Available

  • Novartis Investigative Site

    Thessaloniki, GR 570 10
    Greece

    Active - Recruiting

  • Novartis Investigative Site

    Athens, 115 27
    Greece

    Active - Recruiting

  • Novartis Investigative Site

    Heraklion, 711 10
    Greece

    Site Not Available

  • Novartis Investigative Site

    Heraklion Crete, 71110
    Greece

    Active - Recruiting

  • Novartis Investigative Site

    Hyderabad, Andhra Pradesh 500012
    India

    Active - Recruiting

  • Novartis Investigative Site

    Thiruvananthapuram, Kerala 695011
    India

    Active - Recruiting

  • Novartis Investigative Site

    Nagpur, Maharashtra 440015
    India

    Active - Recruiting

  • Novartis Investigative Site

    Pune, Maharashtra 411011
    India

    Active - Recruiting

  • Novartis Investigative Site

    Chandigarh, Punjab 160012
    India

    Active - Recruiting

  • Novartis Investigative Site

    Chennai, Tamil Nadu 600006
    India

    Active - Recruiting

  • Novartis Investigative Site

    Vellore, Tamil Nadu 632 004
    India

    Active - Recruiting

  • Novartis Investigative Site

    Lucknow, Uttar Pradesh 226014
    India

    Active - Recruiting

  • Novartis Investigative Site

    Iruma-gun, Saitama 350-0495
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Izumo-city, Shimane 693 8501
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Bunkyo ku, Tokyo 113 8655
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Seoul, 06591
    Korea, Republic of

    Active - Recruiting

  • Novartis Investigative Site

    Bratislava, 83305
    Slovakia

    Active - Recruiting

  • Novartis Investigative Site

    Martin, 03601
    Slovakia

    Active - Recruiting

  • Novartis Investigative Site

    Ljubljana, 1000
    Slovenia

    Active - Recruiting

  • Novartis Investigative Site

    Taichung, 40447
    Taiwan

    Active - Recruiting

  • Novartis Investigative Site

    Taoyuan, 33305
    Taiwan

    Active - Recruiting

  • Novartis Investigative Site

    London, NW1 2BU
    United Kingdom

    Active - Recruiting

  • Novartis Investigative Site

    Newcastle Upon Tyne, NE7 7DN
    United Kingdom

    Active - Recruiting

  • Novartis Investigative Site

    Newcastle upon Tyne, NE7 7DN
    United Kingdom

    Active - Recruiting

  • Uni Of Alabama At Birmingham

    Birmingham, Alabama 35233
    United States

    Active - Recruiting

  • Uni Of Alabama At Birmingham Division of Nephrology

    Birmingham, Alabama 35233
    United States

    Active - Recruiting

  • Novartis Investigative Site

    Los Angeles, California 90033
    United States

    Active - Recruiting

  • USC Norris Cancer Center

    Los Angeles, California 90033
    United States

    Active - Recruiting

  • Univ of California at Los Angeles

    Los Angeles, California 90095
    United States

    Active - Recruiting

  • Univ Cali Irvine ALS Neuromuscular

    Orange, California 92868
    United States

    Active - Recruiting

  • Univ Cali Irvine ALS Neuromuscular Nephrology Department

    Orange, California 92868
    United States

    Active - Recruiting

  • Harbor-UCLA Medical Center .

    Torrance, California 90502
    United States

    Active - Recruiting

  • Novartis Investigative Site

    Torrance, California 90502
    United States

    Active - Recruiting

  • Georgetown University Lombardi Cancer Center

    Washington, District of Columbia 20007 2197
    United States

    Active - Recruiting

  • Novartis Investigative Site

    Washington, District of Columbia 20007 2197
    United States

    Active - Recruiting

  • University Of Miami

    Miami, Florida 33136
    United States

    Active - Recruiting

  • Brigham and Womens Hospital

    Boston, Massachusetts 02115
    United States

    Active - Recruiting

  • University of Minnesota

    Minneapolis, Minnesota 55455
    United States

    Active - Recruiting

  • WA Uni School Of Med

    Saint Louis, Missouri 63110
    United States

    Active - Recruiting

  • WA Uni School Of Med .

    Saint Louis, Missouri 63110
    United States

    Active - Recruiting

  • Rut Univ for Translational Med Scie

    New Brunswick, New Jersey 08901
    United States

    Active - Recruiting

  • Novartis Investigative Site

    Albuquerque, New Mexico 87131-0001
    United States

    Active - Recruiting

  • University of New Mexico

    Albuquerque, New Mexico 87131-0001
    United States

    Active - Recruiting

  • University of New Mexico Clin and Transl Science Ctr

    Albuquerque, New Mexico 87131-0001
    United States

    Active - Recruiting

  • Montefiore Medical Center .

    Bronx, New York 10461
    United States

    Active - Recruiting

  • Novartis Investigative Site

    Bronx, New York 10461
    United States

    Active - Recruiting

  • Duke University Medical Center

    Durham, North Carolina 27710
    United States

    Active - Recruiting

  • Duke University Medical Center .

    Durham, North Carolina 27710
    United States

    Active - Recruiting

  • Novartis Investigative Site

    Durham, North Carolina 27710
    United States

    Active - Recruiting

  • University of Cincinnati

    Cincinnati, Ohio 45267-0585
    United States

    Active - Recruiting

  • University of Cincinnati Div of Transplantation

    Cincinnati, Ohio 45267-0585
    United States

    Active - Recruiting

  • Cleveland Clinic Foundation

    Cleveland, Ohio 44195
    United States

    Active - Recruiting

  • Cleveland Clinic Foundation Nephrology and Hypertension

    Cleveland, Ohio 44195
    United States

    Active - Recruiting

  • Novartis Investigative Site

    Cleveland, Ohio 44195
    United States

    Active - Recruiting

  • Comprehensive Transplant Ctr at OSU

    Columbus, Ohio 43210
    United States

    Active - Recruiting

  • Comprehensive Transplant Ctr at OSU Division of Transplant Surgery

    Columbus, Ohio 43210
    United States

    Active - Recruiting

  • UT Southwestern Medical Center

    Dallas, Texas 75390
    United States

    Active - Recruiting

  • UT Southwestern Medical Center .

    Dallas, Texas 75390
    United States

    Active - Recruiting

  • Baylor Scott and White Research

    Temple, Texas 76502
    United States

    Active - Recruiting

  • Baylor Scott and White Research .

    Temple, Texas 76502
    United States

    Active - Recruiting

  • Novartis Investigative Site

    Temple, Texas 76502
    United States

    Active - Recruiting

  • Virginia Commonwealth University

    Richmond, Virginia 23298
    United States

    Active - Recruiting

  • Virginia Commonwealth University .

    Richmond, Virginia 23298
    United States

    Active - Recruiting

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.