NCT04578834

Brief Summary

The study is designed as a multicenter, randomized, double-blind, placebo controlled study to demonstrate the superiority of iptacopan (LNP023) at a dose of 200 mg b.i.d. compared to placebo on top of maximally tolerated ACEi or ARB on reduction of proteinuria and slowing renal disease progression in primary IgA Nephropathy patients.

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Strong global presence with extensive site network
Enrollment
518

participants targeted

Target at P75+ for phase_3

Timeline
Completed

Started Jan 2021

Longer than P75 for phase_3

Geographic Reach
34 countries

175 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 9, 2020

Completed
29 days until next milestone

First Posted

Study publicly available on registry

October 8, 2020

Completed
4 months until next milestone

Study Start

First participant enrolled

January 25, 2021

Completed
4.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 19, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 19, 2025

Completed
Last Updated

December 23, 2025

Status Verified

December 1, 2025

Enrollment Period

4.7 years

First QC Date

September 9, 2020

Last Update Submit

December 22, 2025

Conditions

Keywords

LNP023IgA nephropathyIgAN, proteinuriaUPCReGFReGFR slopekidney function decline

Outcome Measures

Primary Outcomes (2)

  • Ratio to baseline in Urine Protein to Creatinine Ratio (sampled from 24h urine collection) at 9 months

    Evaluated at interim analysis - To demonstrate superiority of LNP023 vs. placebo in the change of proteinuria at 9 months by measuring Urine Protein to Creatinine Ratio sampled from a 24h urine collection.

    Baseline and 9 months

  • Annualized total estimated Glomerular Filtration Rate (eGFR) slope over 24 months).

    Evaluated at the final analysis - to demonstrate superiority of LNP023 vs. placebo in slowing IgAN progression measured by the annualized total slope of Estimated Glomerular Filtration Rate (eGFR) change over 24 months.

    Baseline and 24 months

Secondary Outcomes (8)

  • Change from baseline in estimated glomerular filtration rate at 9 months

    Baseline and 9 months

  • Proportion of participants reaching Urine Protein To Creatinine Ratio <1g/g without receiving Corticosteroids/Immunosuppressant or other newly approved drugs or initiating new background therapy for treatment of IgAN or Kidney Replacement Therapy (KRT)

    Baseline and 9 months

  • Annualized total Estimated Glomerular Filtration Rate slope estimated over 12 months

    Baseline and 12 months

  • Change from baseline to 9 months in the fatigue scale measured by the Functional Assessment Of Chronic Illness Therapy-Fatigue questionnaire

    Baseline and 9 months

  • Time from randomization to first occurrence of composite kidney failure endpoint event

    Up to 24 months

  • +3 more secondary outcomes

Study Arms (2)

LNP023 200mg b.i.d

EXPERIMENTAL
Drug: LNP023

Placebo to LNP023 200mg b.i.d

PLACEBO COMPARATOR
Drug: Placebo

Interventions

Placebo to LNP023 200mg b.i.d

Placebo to LNP023 200mg b.i.d
LNP023DRUG

LNP023 200mg b.i.d

Also known as: iptacopan
LNP023 200mg b.i.d

Eligibility Criteria

Age18 Years - 100 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Male and female patients ≥ 18 years of age with an eGFR level and biopsy-confirmed IgA nephropathy as follows:
  • For patients eGFR\* ≥ 45ml/min/1.73m2, a qualifying biopsy performed within the last 5 years is required.
  • For patients with eGFR\* 30 to \<45ml/min/1.73m2, a qualifying biopsy performed within 2 years with \< 50% tubulointerstitial fibrosis is required.
  • For patients with eGFR\* 20 to \<30ml/min/1.73m2, a qualifying biopsy performed at any time.
  • In all cases, if a historical biopsy is not available, one may be performed during screening. \*eGFR calculated using the CKD-EPI formula (or modified MDRD formula according to specific ethnic groups and local practice guidelines)
  • Proteinuria due to primary diagnosis of IgA nephropathy as assessed at screening by UPCR ≥1 g/g (113 mg/mmol) sampled from FMV or 24h urine collection, as well as at the completion of the run-in period by UPCR ≥1 g/g (113 mg/mmol) calculated as the (geometric) mean of two 24h urine collections obtained within 14 days of each other at baseline.
  • Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection is 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, prophylactic antibiotic treatment should be initiated.
  • If not previously vaccinated, vaccination against Haemophilus influenzae infections should be given, if available and according to local regulations, at least 2 weeks prior to first study drug administration.
  • All patients must have been on supportive care including stable dose regimen of ACEi or ARB at either the locally approved maximal daily dose or the maximally tolerated dose (per investigators' judgment) for approximately 90 days before first study drug administration. In addition, if patients are taking diuretics, other antihypertensive medication, or other background medication for IgAN, the doses should also be stabilized for approximately 90 days prior to the first dosing of study treatment.

You may not qualify if:

  • Any secondary IgAN as defined by the investigator; secondary IgAN can be associated with cirrhosis, celiac disease, Human Immunodeficiency Virus (HIV) infection, dermatitis herpetiformis, seronegative arthritis, small-cell carcinoma, lymphoma, disseminated tuberculosis, bronchiolitis obliterans, and inflammatory bowel disease, familial mediterranean fever, etc.
  • Sitting office SBP \>140 mmHg or DBP \>90 mmHg at the randomization visit
  • Patients previously treated with immunosuppressive or other immunomodulatory agents such as but not limited to cyclophosphamide, rituximab, infliximab, eculizumab, canakinumab, mycophenolate mofetil (MMF) or mycophenolate sodium (MPS), cyclosporine, tacrolimus, sirolimus, everolimus, or systemic corticosteroids exposure (\>7.5 mg/d prednisone/prednisolone equivalent) within 90 days (or 180 days for rituximab) prior to first study drug administration. Participants previously or currently treated with oral budesonide. Participants treated with endothelin (receptor) antagonists within 90 days prior to first study drug administration.
  • Prior use of iptacopan (LNP023) or prior enrollment in any other LNP023 clinical trial where study drug was taken, including matching placebo
  • History of recurrent invasive infections caused by encapsulated organisms, such as meningococcus and pneumococcus.
  • Active systemic bacterial, viral (including COVID-19) or fungal infection within 14 days prior to study drug administration.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (177)

AZ Kidney Dise and Hypertension Ctr

Glendale, Arizona, 85306, United States

Location

AKDHC Medical Research ServicesLLC

Phoenix, Arizona, 85016, United States

Location

UCLA Medical Center

Los Angeles, California, 90095, United States

Location

Kaiser Permanente

San Diego, California, 92111, United States

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North America Research Institute

San Dimas, California, 91773, United States

Location

University of Colorado Anschutz

Aurora, Colorado, 80045, United States

Location

Nephrology Associates PA

Newark, Delaware, 19713, United States

Location

Boise Kidney and Hypertension

Boise, Idaho, 83706, United States

Location

CaRe Research

Chubbuck, Idaho, 83202, United States

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Nep Assoc of Northern Illinois

Hinsdale, Illinois, 60521, United States

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Renal Associates of Baton Rouge

Baton Rouge, Louisiana, 80808, United States

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Johns Hopkins Hospital

Baltimore, Maryland, 21287, United States

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Brigham and Womens Hosp Harvard Med School

Boston, Massachusetts, 02115, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Mayo Clinic Rochester

Rochester, Minnesota, 55905, United States

Location

Clin Rsrch Consult a JCCT Company

Kansas City, Missouri, 64111, United States

Location

DaVita Clinical Research

Las Vegas, Nevada, 89146, United States

Location

New Jersey Kidney Care

Jersey City, New Jersey, 07305, United States

Location

Columbia University Irving Medical

New York, New York, 10032, United States

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Dallas Renal Group

Dallas, Texas, 75230, United States

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Prolato Clinical Research Center

Houston, Texas, 77054, United States

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Univ of Washington Medi Cen

Seattle, Washington, 98104, United States

Location

Novartis Investigative Site

Córdoba, Córdoba Province, X5016JDA, Argentina

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Novartis Investigative Site

Córdoba, Córdoba Province, X5016KEH, Argentina

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Novartis Investigative Site

CABA, C1181ACH, Argentina

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Novartis Investigative Site

CABA, C1426ABP, Argentina

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Novartis Investigative Site

Santa Fe, S3000EPV, Argentina

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Westmead, New South Wales, 2145, Australia

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Woolloongabba, Queensland, 4102, Australia

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Adelaide, South Australia, 5000, Australia

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Parkville, Victoria, 3065, Australia

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St Albans, Victoria, 3021, Australia

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Roeselare, West-Vlaanderen, 8800, Belgium

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Edegem, 2650, Belgium

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Leuven, 3000, Belgium

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Belo Horizonte, Minas Gerais, 30150-221, Brazil

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Curitiba, Paraná, 80440-020, Brazil

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Porto Alegre, Rio Grande do Sul, 90035-074, Brazil

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São Paulo, São Paulo, 04038-002, Brazil

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São Paulo, São Paulo, 05403 000, Brazil

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Sao Jose Rio Preto, 15090 000, Brazil

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Oshawa, Ontario, L1G 2B9, Canada

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Temuco, 4781151, Chile

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Beijing, Beijing Municipality, 100013, China

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Beijing, Beijing Municipality, 102218, China

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Guangzhou, Guangdong, 510030, China

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Guangzhou, Guangdong, 510630, China

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Shenzhen, Guangdong, 518000, China

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Luoyang, Henan, 471003, China

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Zhengzhou, Henan, 450003, China

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Changsha, Hunan, 410011, China

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Changchun, Jilin, 130041, China

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Yinchuan, Ningxia, 750004, China

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Taiyuan, Shanxi, 030001, China

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Xian, Shanxi, 710061, China

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Ürümqi, Xinjiang, 830001, China

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Hangzhou, Zhejiang, 310003, China

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Ningbo, Zhejiang, 315016, China

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Wenzhou, Zhejiang, 325000, China

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Beijing, 100029, China

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Beijing, 100034, China

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Guangzhou, 510080, China

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Ningbo, 315010, China

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Qingdao, 266000, China

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Shanghai, 200025, China

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Shanghai, 200040, China

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Shenzhen, 518036, China

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Medellín, Antioquia, 050001, Colombia

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Barranquilla, Atlántico, 080020, Colombia

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Prague, 128 08, Czechia

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Aalborg, 9000, Denmark

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Arhus N, DK-8200, Denmark

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Copenhagen, DK-2100, Denmark

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Odense C, 5000, Denmark

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Marseille, 13005, France

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Montpellier, 34295, France

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Paris, 75015, France

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Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

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Stuttgart, Baden-Wurttemberg, 70376, Germany

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Göttingen, Lower Saxony, 37075, Germany

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Dresden, Saxony, 01307, Germany

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Aachen, 52074, Germany

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Berlin, 13353, Germany

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Essen, 45147, Germany

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Hanover, 30625, Germany

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Kiel, 24105, Germany

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Magdeburg, 39120, Germany

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Mainz, 55131, Germany

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Tübingen, 72076, Germany

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Ulm, 89081, Germany

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Pécs, Baranya, 7623, Hungary

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Debrecen, Hajdu Bihar Megye, 4032, Hungary

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Szeged, 6725, Hungary

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Bangalore, Karnataka, 560004, India

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New Delhi, National Capital Territory of Delhi, 110017, India

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New Delhi, National Capital Territory of Delhi, 110029, India

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Hyderabad, Telangana, 500082, India

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Ashkelon, 7830604, Israel

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Jerusalem, 9112001, Israel

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Petah Tikva, 4941492, Israel

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Bologna, BO, 40138, Italy

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Naples, 80131, Italy

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Kasugai, Aichi-ken, 486-8510, Japan

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Toyoake, Aichi-ken, 470 1192, Japan

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Toyota, Aichi-ken, 471-8513, Japan

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Sapporo, Hokkaido, 060-8604, Japan

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Sapporo, Hokkaido, 68555, Japan

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Kawasaki, Kanagawa, 213-8587, Japan

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Yokohama, Kanagawa, 224-8503, Japan

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Yokohama, Kanagawa-ku, 236-0004, Japan

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Kyoto, Kyoto, 605-0981, Japan

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Matsumoto, Nagano, 3908621, Japan

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Osaka, Osaka, 5340021, Japan

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Ōmihachiman, Shiga, 523-0082, Japan

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Chiba, 2608712, Japan

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Niigata, 9518520, Japan

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Okayama, 7008558, Japan

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Osaka, 5300012, Japan

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Kuala Lumpur, Kuala Lumpur, 50586, Malaysia

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Kuala Lumpur, 59100, Malaysia

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Mexicali, Baja California Norte, 21200, Mexico

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Mexico City, 03100, Mexico

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Querétaro, 76000, Mexico

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Groningen, Provincie Groningen, 9713 GZ, Netherlands

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Nordbyhagen, Oslo County, 1478, Norway

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Bergen, NO-5021, Norway

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Omsk, 644112, Russia

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Rostov-on-Don, 344022, Russia

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Saint Petersburg, 197110, Russia

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Singapore, 119074, Singapore

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Singapore, 169608, Singapore

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Košice, Slovakia, 040 11, Slovakia

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Maribor, Slovenia, 2000, Slovenia

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Ljubljana, 1000, Slovenia

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Bloemfontein, Free State, 9301, South Africa

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Bundang Gu, Gyeonggi-do, 13620, South Korea

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Seoul, Korea, 02841, South Korea

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Seoul, Korea, 03080, South Korea

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Seoul, Korea, 03312, South Korea

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Cheongju-si, North Chungcheong, 28644, South Korea

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Busan, 47392, South Korea

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Seoul, 03722, South Korea

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Seoul, 06591, South Korea

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Seoul, 06973, South Korea

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Seoul, 134 727, South Korea

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Taegu, 41944, South Korea

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Pamplona, Navarre, 31008, Spain

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Barcelona, 08036, Spain

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Salamanca, 37007, Spain

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Seville, 41013, Spain

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Danderyd, 182 88, Sweden

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Stockholm, 141 86, Sweden

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Kaohsiung City, 83301, Taiwan

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New Taipei City, 22060, Taiwan

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New Taipei City, 23561, Taiwan

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Taichung, 40447, Taiwan

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Taipei, 10002, Taiwan

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Taoyuan District, 33305, Taiwan

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Bangkok, 10330, Thailand

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Bangkok, 10400, Thailand

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Bangkok, 10700, Thailand

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Istanbul, Fatih, 34093, Turkey (Türkiye)

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Istanbul, Fatih, 34098, Turkey (Türkiye)

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Köseköy, Kocaeli, 41380, Turkey (Türkiye)

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Antalya, Konyaalti, 07070, Turkey (Türkiye)

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Kayseri, Melikgazi, 38039, Turkey (Türkiye)

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Istanbul, Sariyer, 34396, Turkey (Türkiye)

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Istanbul, Sultangazi, 34265, Turkey (Türkiye)

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Ankara, Yenimahalle, 06500, Turkey (Türkiye)

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Mersin, Yenisehir, 33110, Turkey (Türkiye)

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Newcastle upon Tyne, Tyne and Wear, NE7 7DN, United Kingdom

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Leicester, LE5 4PW, United Kingdom

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London, SE5 9RS, United Kingdom

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London, SW17 0QT, United Kingdom

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Salford, M6 8HD, United Kingdom

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Ho Chi Minh City, VNM, 700000, Vietnam

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Novartis Investigative Site

Ho Chi Minh City, 700000, Vietnam

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Related Publications (6)

  • Barratt J, Eren N, Kashihara N, Maes B, Rizk DV, Rovin B, Trimarchi H, Zhang H, Wang W, Kocyigit I, Hao C, Tesar V, Turgutalp K, Yang L, Xing G, Duro Garcia V, Han SH, Lu W, Pisani A, Weinmann-Menke J, Eitner F, Guerard N, Butylin D, Monaco L, Scosyrev E, Magirr A, Renfurm R, Hach T, Perkovic V; APPLAUSE-IgAN Study Group. Iptacopan in IgA Nephropathy - Final 24-Month Data. N Engl J Med. 2026 Mar 29. doi: 10.1056/NEJMoa2600743. Online ahead of print.

  • Perkovic V, Barratt J, Rovin B, Kashihara N, Maes B, Zhang H, Trimarchi H, Kollins D, Papachristofi O, Jacinto-Sanders S, Merkel T, Guerard N, Renfurm R, Hach T, Rizk DV; APPLAUSE-IgAN Investigators. Alternative Complement Pathway Inhibition with Iptacopan in IgA Nephropathy. N Engl J Med. 2025 Feb 6;392(6):531-543. doi: 10.1056/NEJMoa2410316. Epub 2024 Oct 25.

  • Tunnicliffe DJ, Reid S, Craig JC, Samuels JA, Molony DA, Strippoli GF. Non-immunosuppressive treatment for IgA nephropathy. Cochrane Database Syst Rev. 2024 Feb 1;2(2):CD003962. doi: 10.1002/14651858.CD003962.pub3.

  • Zhang H, Rizk DV, Perkovic V, Maes B, Kashihara N, Rovin B, Trimarchi H, Sprangers B, Meier M, Kollins D, Papachristofi O, Milojevic J, Junge G, Nidamarthy PK, Charney A, Barratt J. Results of a randomized double-blind placebo-controlled Phase 2 study propose iptacopan as an alternative complement pathway inhibitor for IgA nephropathy. Kidney Int. 2024 Jan;105(1):189-199. doi: 10.1016/j.kint.2023.09.027. Epub 2023 Oct 31.

  • El Karoui K, Fervenza FC, De Vriese AS. Treatment of IgA Nephropathy: A Rapidly Evolving Field. J Am Soc Nephrol. 2024 Jan 1;35(1):103-116. doi: 10.1681/ASN.0000000000000242. Epub 2023 Sep 29.

  • Reich HN, Floege J. How I Treat IgA Nephropathy. Clin J Am Soc Nephrol. 2022 Aug;17(8):1243-1246. doi: 10.2215/CJN.02710322. Epub 2022 Jun 8. No abstract available.

MeSH Terms

Conditions

Glomerulonephritis, IGAProteinuria

Interventions

iptacopan

Condition Hierarchy (Ancestors)

GlomerulonephritisNephritisKidney DiseasesUrologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesMale Urogenital DiseasesAutoimmune DiseasesImmune System DiseasesUrination DisordersUrological ManifestationsSigns and SymptomsPathological Conditions, Signs and Symptoms

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 9, 2020

First Posted

October 8, 2020

Study Start

January 25, 2021

Primary Completion

September 19, 2025

Study Completion

September 19, 2025

Last Updated

December 23, 2025

Record last verified: 2025-12

Data Sharing

IPD Sharing
Will share

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Locations