A Rollover Extension Program (REP) to Evaluate the Long-term Safety and Tolerability of Open Label Iptacopan/LNP023 in Participants With Primary IgA Nephropathy

A Multicenter Rollover Extension Program (REP) to Evaluate the Long-term Safety and Tolerability of Open Label Iptacopan in Adult Participants With Primary IgA Nephropathy Who Have Completed Study CLNP023X2203 or CLNP023A2301

The purpose of this study is to evaluate the long-term safety and tolerability, of open label iptacopan in primary IgA nephropathy participants who have completed either the CLNP023X2203 or CLNP023A2301 clinical trials. The open-label design of the current study is appropriate to provide study participants the opportunity to receive treatment with iptacopan until marketing authorizations are received and the drug product becomes commercially available while enabling collection of long-term safety and tolerability data for the investigational drug. Furthermore efficacy assessments conducted every 6 months will afford the opportunity to evaluate the clinical effects of iptacopan on long-term disease progression.

Study Overview

• Status: Active, not recruiting
• Conditions: Primary IgA Nephropathy
• Intervention / Treatment: Drug: LNP023

Detailed Description

This is an open-label, non-randomized, multicenter roll-over extension program (REP) to:

• CLNP023X2203, a Phase II trial investigating the dose ranging effects of LNP023 on efficacy, pharmacokinetics (PK), pharmacodynamics (PD), safety and tolerability in primary IgAN patients, and
• CLNP023A2301, a Phase III trial, investigating the efficacy, pharmacokinetics (PK), pharmacodynamics (PD), safety and tolerability of LNP023 in patients with primary IgAN.

Subjects completing the CLNP023X2203 and CLNP023A2301 trials on study drug, who want to continue treatment and who meet the inclusion/exclusion requirements of the roll over extension program, will have the opportunity to receive iptacopan until:

• 3 years from LPFV of this study CLNP023A2002B, or
• the participant no longer derives benefit from iptacopan according to the Investigator, or
• the benefit-risk profile of the product in IgAN is no longer positive, or
• initiation of maintenance hemodialysis, kidney transplantation or eGFR < 15 mL/min/1.73m2 , or
• the product becomes commercially available in a specific country following product launch and subsequent reimbursement for IgAN, where applicable, or
• if a marketing application or reimbursement of an investigational product is rejected/not pursued in a region/country for the indication under study or which ever is sooner

• Study Type: Interventional
• Enrollment (Actual): 448
• Phase: Phase 3

Expanded Access

Available outside the clinical trial. See expanded access record.

Contacts and Locations

Study Locations

• Argentina
  • CABA, Argentina, C1181ACH
    • Novartis Investigative Site
  • CABA, Argentina, C1426ABP
    • Novartis Investigative Site
  • Santa Fe, Argentina, S3000EPV
    • Novartis Investigative Site
  • Córdoba Province
    • Córdoba, Córdoba Province, Argentina, 5000
      • Novartis Investigative Site
    • Córdoba, Córdoba Province, Argentina, X5016KEH
      • Novartis Investigative Site
• Australia
  • Queensland
    • Woolloongabba, Queensland, Australia, 4102
      • Novartis Investigative Site
  • South Australia
    • Adelaide, South Australia, Australia, 5000
      • Novartis Investigative Site
  • Victoria
    • Parkville, Victoria, Australia, 3065
      • Novartis Investigative Site
• Belgium
  • Edegem, Belgium, 2650
    • Novartis Investigative Site
  • Leuven, Belgium, 3000
    • Novartis Investigative Site
  • West-Vlaanderen
    • Roeselare, West-Vlaanderen, Belgium, 8800
      • Novartis Investigative Site
• Brazil
  • Sao Jose Rio Preto, Brazil, 15090 000
    • Novartis Investigative Site
  • Minas Gerais
    • Belo Horizonte, Minas Gerais, Brazil, 30150-221
      • Novartis Investigative Site
  • Paraná
    • Curitiba, Paraná, Brazil, 80440-020
      • Novartis Investigative Site
  • Rio Grande do Sul
    • Porto Alegre, Rio Grande do Sul, Brazil, 90035-074
      • Novartis Investigative Site
  • São Paulo
    • São Paulo, São Paulo, Brazil, 05403 000
      • Novartis Investigative Site
    • São Paulo, São Paulo, Brazil, 04038-002
      • Novartis Investigative Site
• Chile
  • Temuco, Chile, 4781151
    • Novartis Investigative Site
• China
  • Beijing, China, 100034
    • Novartis Investigative Site
  • Guangzhou, China, 510080
    • Novartis Investigative Site
  • Ningbo, China, 315010
    • Novartis Investigative Site
  • Qingdao, China, 266000
    • Novartis Investigative Site
  • Shanghai, China, 200025
    • Novartis Investigative Site
  • Shanghai, China, 200040
    • Novartis Investigative Site
  • Shenzhen, China, 518036
    • Novartis Investigative Site
  • Beijing Municipality
    • Beijing, Beijing Municipality, China, 102218
      • Novartis Investigative Site
    • Beijing, Beijing Municipality, China, 100013
      • Novartis Investigative Site
  • Guangdong
    • Guangzhou, Guangdong, China, 510030
      • Novartis Investigative Site
  • Henan
    • Luoyang, Henan, China, 471003
      • Novartis Investigative Site
    • Zhengzhou, Henan, China, 450003
      • Novartis Investigative Site
  • Hunan
    • Changsha, Hunan, China, 410011
      • Novartis Investigative Site
  • Jilin
    • Changchun, Jilin, China, 130041
      • Novartis Investigative Site
  • Ningxia
    • Yinchuan, Ningxia, China, 750004
      • Novartis Investigative Site
  • Shanxi
    • Taiyuan, Shanxi, China, 030001
      • Novartis Investigative Site
    • Xian, Shanxi, China, 710061
      • Novartis Investigative Site
  • Xinjiang
    • Ürümqi, Xinjiang, China, 830001
      • Novartis Investigative Site
  • Zhejiang
    • Ningbo, Zhejiang, China, 315016
      • Novartis Investigative Site
    • Wenzhou, Zhejiang, China, 325000
      • Novartis Investigative Site
• Colombia
  • Antioquia
    • Medellín, Antioquia, Colombia, 050001
      • Novartis Investigative Site
  • Atlántico
    • Barranquilla, Atlántico, Colombia, 080020
      • Novartis Investigative Site
• Czechia
  • Prague, Czechia, 128 08
    • Novartis Investigative Site
• Denmark
  • Aalborg, Denmark, 9000
    • Novartis Investigative Site
  • Aarhus N, Denmark, 8200
    • Novartis Investigative Site
  • Copenhagen, Denmark, DK-2100
    • Novartis Investigative Site
  • Odense C, Denmark, 5000
    • Novartis Investigative Site
• France
  • Marseille, France, 13005
    • Novartis Investigative Site
  • Montpellier, France, 34295
    • Novartis Investigative Site
  • Paris, France, 75015
    • Novartis Investigative Site
• Germany
  • Aachen, Germany, 52074
    • Novartis Investigative Site
  • Berlin, Germany, 13353
    • Novartis Investigative Site
  • Essen, Germany, 45147
    • Novartis Investigative Site
  • Hanover, Germany, 30625
    • Novartis Investigative Site
  • Kiel, Germany, 24105
    • Novartis Investigative Site
  • Magdeburg, Germany, 39120
    • Novartis Investigative Site
  • Mainz, Germany, 55131
    • Novartis Investigative Site
  • Tübingen, Germany, 72076
    • Novartis Investigative Site
  • Ulm, Germany, 89081
    • Novartis Investigative Site
  • Baden-Wurttemberg
    • Freiburg im Breisgau, Baden-Wurttemberg, Germany, 79106
      • Novartis Investigative Site
    • Stuttgart, Baden-Wurttemberg, Germany, 70376
      • Novartis Investigative Site
• Hong Kong
  • Hong Kong
    • Hong Kong, Hong Kong, Hong Kong, 999077
      • Novartis Investigative Site
• Hungary
  • Baranya
    • Pécs, Baranya, Hungary, 7623
      • Novartis Investigative Site
  • Hajdu Bihar Megye
    • Debrecen, Hajdu Bihar Megye, Hungary, 4032
      • Novartis Investigative Site
• India
  • Karnataka
    • Bangalore, Karnataka, India, 560004
      • Novartis Investigative Site
  • National Capital Territory of Delhi
    • New Delhi, National Capital Territory of Delhi, India, 110029
      • Novartis Investigative Site
    • New Delhi, National Capital Territory of Delhi, India, 110017
      • Novartis Investigative Site
  • Telangana
    • Hyderabad, Telangana, India, 500082
      • Novartis Investigative Site
• Israel
  • Ashkelon, Israel, 7830604
    • Novartis Investigative Site
  • Jerusalem, Israel, 9112001
    • Novartis Investigative Site
  • Petah Tikva, Israel, 4941492
    • Novartis Investigative Site
• Italy
  • Naples, Italy, 80131
    • Novartis Investigative Site
  • BO
    • Bologna, BO, Italy, 40138
      • Novartis Investigative Site
• Japan
  • Chiba, Japan, 2608712
    • Novartis Investigative Site
  • Niigata, Japan, 9518520
    • Novartis Investigative Site
  • Okayama, Japan, 7008558
    • Novartis Investigative Site
  • Osaka, Japan, 5300012
    • Novartis Investigative Site
  • Osaka, Japan, 5308480
    • Novartis Investigative Site
  • Aichi-ken
    • Kasugai, Aichi-ken, Japan, 486-8510
      • Novartis Investigative Site
    • Toyoake, Aichi-ken, Japan, 4701192
      • Novartis Investigative Site
    • Toyota, Aichi-ken, Japan, 471-8513
      • Novartis Investigative Site
  • Hokkaido
    • Sapporo, Hokkaido, Japan, 0068555
      • Novartis Investigative Site
    • Sapporo, Hokkaido, Japan, 0608604
      • Novartis Investigative Site
  • Kanagawa
    • Kawasaki, Kanagawa, Japan, 213-8587
      • Novartis Investigative Site
    • Yokohama, Kanagawa, Japan, 224-8503
      • Novartis Investigative Site
    • Yokohama, Kanagawa, Japan, 236-0004
      • Novartis Investigative Site
  • Kyoto
    • Kyoto, Kyoto, Japan, 605-0981
      • Novartis Investigative Site
  • Nagano
    • Matsumoto, Nagano, Japan, 3908621
      • Novartis Investigative Site
  • Osaka
    • Osaka, Osaka, Japan, 5340021
      • Novartis Investigative Site
  • Shiga
    • Ōmihachiman, Shiga, Japan, 523-0082
      • Novartis Investigative Site
• Malaysia
  • Kuala Lumpur, Malaysia, 59100
    • Novartis Investigative Site
  • Kuala Lumpur
    • Kuala Lumpur, Kuala Lumpur, Malaysia, 50586
      • Novartis Investigative Site
• Mexico
  • Querétaro, Mexico, 76000
    • Novartis Investigative Site
  • Mexico City
    • Benito Juárez, Mexico City, Mexico, 03100
      • Novartis Investigative Site
• Netherlands
  • Provincie Groningen
    • Groningen, Provincie Groningen, Netherlands, 9713 GZ
      • Novartis Investigative Site
• Norway
  • Bergen, Norway, NO-5021
    • Novartis Investigative Site
  • Oslo
    • Nordbyhagen, Oslo, Norway, 1478
      • Novartis Investigative Site
• Russia
  • Rostov-on-Don, Russia, 344022
    • Novartis Investigative Site
  • Saint Petersburg, Russia, 197110
    • Novartis Investigative Site
• Singapore
  • Singapore, Singapore, 119074
    • Novartis Investigative Site
  • Singapore, Singapore, 169608
    • Novartis Investigative Site
• Slovakia
  • Košice, Slovakia, 040 11
    • Novartis Investigative Site
• Slovenia
  • Ljubljana, Slovenia, 1000
    • Novartis Investigative Site
• South Africa
  • Free State
    • Bloemfontein, Free State, South Africa, 9301
      • Novartis Investigative Site
• South Korea
  • Busan, South Korea, 47392
    • Novartis Investigative Site
  • Seoul, South Korea, 03722
    • Novartis Investigative Site
  • Seoul, South Korea, 06973
    • Novartis Investigative Site
  • Seoul, South Korea, 134 727
    • Novartis Investigative Site
  • Seoul, South Korea, 06591
    • Novartis Investigative Site
  • Taegu, South Korea, 41944
    • Novartis Investigative Site
  • Gyeonggi-do
    • Bundang Gu, Gyeonggi-do, South Korea, 13620
      • Novartis Investigative Site
  • Korea
    • Seoul, Korea, South Korea, 03312
      • Novartis Investigative Site
    • Seoul, Korea, South Korea, 02841
      • Novartis Investigative Site
  • North Chungcheong
    • Cheongju-si, North Chungcheong, South Korea, 28644
      • Novartis Investigative Site
  • Seoul
    • Seoul, Seoul, South Korea, 03080
      • Novartis Investigative Site
• Spain
  • Barcelona, Spain, 08036
    • Novartis Investigative Site
  • Salamanca, Spain, 37007
    • Novartis Investigative Site
  • Navarre
    • Pamplona, Navarre, Spain, 31008
      • Novartis Investigative Site
• Sweden
  • Stockholm, Sweden, 141 86
    • Novartis Investigative Site
• Taiwan
  • Kaohsiung City, Taiwan, 83301
    • Novartis Investigative Site
  • New Taipei City, Taiwan, 23561
    • Novartis Investigative Site
  • New Taipei City, Taiwan, 22060
    • Novartis Investigative Site
  • Taichung, Taiwan, 40447
    • Novartis Investigative Site
  • Taichung, Taiwan, 407219
    • Novartis Investigative Site
  • Taipei, Taiwan, 10002
    • Novartis Investigative Site
  • Taoyuan, Taiwan, 33305
    • Novartis Investigative Site
• Thailand
  • Bangkok, Thailand, 10330
    • Novartis Investigative Site
  • Bangkok, Thailand, 10700
    • Novartis Investigative Site
  • Bangkok, Thailand, 10400
    • Novartis Investigative Site
• Turkey (Türkiye)
  • Fatih
    • Istanbul, Fatih, Turkey (Türkiye), 34098
      • Novartis Investigative Site
  • Kocaeli
    • Köseköy, Kocaeli, Turkey (Türkiye), 41380
      • Novartis Investigative Site
  • Konyaalti
    • Antalya, Konyaalti, Turkey (Türkiye), 07070
      • Novartis Investigative Site
  • Melikgazi
    • Kayseri, Melikgazi, Turkey (Türkiye), 38039
      • Novartis Investigative Site
  • Sariyer
    • Istanbul, Sariyer, Turkey (Türkiye), 34396
      • Novartis Investigative Site
  • Yenimahalle
    • Ankara, Yenimahalle, Turkey (Türkiye), 06500
      • Novartis Investigative Site
  • Yenisehir
    • Mersin, Yenisehir, Turkey (Türkiye), 33110
      • Novartis Investigative Site
• United Kingdom
  • Cambridge, United Kingdom, CB2 0QQ
    • Novartis Investigative Site
  • Leicester, United Kingdom, LE5 4PW
    • Novartis Investigative Site
  • London, United Kingdom, SE5 9RS
    • Novartis Investigative Site
  • Salford, United Kingdom, M6 8HD
    • Novartis Investigative Site
  • Tyne and Wear
    • Newcastle upon Tyne, Tyne and Wear, United Kingdom, NE7 7DN
      • Novartis Investigative Site
• United States
  • Arizona
    • Glendale, Arizona, United States, 85306
      • AZ Kidney Dise and Hypertension Ctr
  • California
    • San Diego, California, United States, 92111
      • Kaiser Permanente
    • San Dimas, California, United States, 91773
      • North America Research Institute
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado Anschutz
  • Idaho
    • Chubbuck, Idaho, United States, 83202
      • CaRe Research
  • Illinois
    • Hinsdale, Illinois, United States, 60521
      • Nep Assoc of Northern Illinois
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • Johns Hopkins Hospital
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Brigham and Womens Hosp Harvard Med School
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic Rochester
  • Missouri
    • Kansas City, Missouri, United States, 64111
      • Clin Rsrch Consult a JCCT Company
  • Nevada
    • Las Vegas, Nevada, United States, 89146
      • Davita Clinical Research
  • New Jersey
    • Jersey City, New Jersey, United States, 07305
      • New Jersey Kidney Care
  • New York
    • New York, New York, United States, 10032
      • Col Uni Med Center New York Presby
  • Texas
    • Dallas, Texas, United States, 75230
      • Dallas Renal Group
• Vietnam
  • Ho Chi Minh City, Vietnam, 700000
    • Novartis Investigative Site
  • VNM
    • Ho Chi Minh City, VNM, Vietnam, 700000
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• For LNP023X2203, participants must have completed part 1 or part 2 of the trial. For LNP023A2301, participants must have completed the entire core trial defined as the full 24 month treatment period.

• eGFR* ≥ 20 ml/min/1.73m2

  *eGFR calculated using the CKD-EPI formula (or modified MDRD formula according to specific ethnic groups and local practice guidelines)

• Per investigator's clinical judgement, the participant may benefit from receiving the open-label treatment of iptacopan 200 mg b.i.d.
• Prior Vaccination against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections should be up to date (i.e. any boosters required administered according to local regulations.

• All participants must be on supportive care regimen of ACEi or ARB* as per KDIGO guidelines.

  • participants who are not taking KDIGO guideline doses because they have documented allergies or intolerance to ACEi and ARB are eligible for the study

Exclusion Criteria:

• participants who screen or baseline failed in the CLNP023X2203 Part 1 or Part 2, or CLNP023A2301 studies or who prematurely withdrew from either study for any reason.
• Evidence of severe urinary obstruction or difficulty in voiding; any urinary tract disorder other than IgAN at screening and before dosing with LNP023.
• Current (within 4 weeks of study drug administration in the REP) acute kidney injury (AKI)
• Presence of Rapidly Progressive Glomerulonephritis (RPGN) as defined by 50% decline in eGFR within the last 3 months.
• Participants treated with immunosuppressive or other immunmodulatory agents such as but not limited to cyclophosphamide, rituximab, infliximab, eculizumab, canakinumab, mycophenolate mofetil (MMF) or mycophenolate sodium (MPS), cyclosporine, tacrolimus, sirolimus, everolimus and/or systemic corticosteroids exposure (>7.5 mg/d prednisone/prednisolone equivalent) within 5 half-lives of respective medication or 90 days prior to first study drug administration, whichever is shorter. Rituximab requires 180 days wash out.
• Use of other investigational drugs at the time of enrolment, or within 5 half-lives of enrolment or within 30 days whichever is longer.
• History of recurrent invasive infections caused by encapsulated organisms, such as meningococcus and pneumococcus.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: LNP023; All participants are receiving 200 mg b.i.d	Drug: LNP023; Capsule 200 mg (b.i.d.) taken orally twice a day; Other Names: iptacopan

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number and percentage of participants with serious adverse event	Summary statistics on serious adverse events	Date of first administration of (Day 1) to 7 days after the date of the last actual administration of study treatment
Number and percentage of participants with adverse event	Summary statistics on adverse events	Date of first administration of study treatment (Day 1) to 7 days after the date of the last actual administration of study treatment
Number and percentage of participants with adverse events of special interest	Summary statistics on adverse events of special interest	Date of first administration of study treatment (Day 1) to 7 days after the date of the last actual adminstration of study treatment
Number and percentage of participants with abnormalities in vital signs	Summary statistics on abnormalities in vital sign parameters	Date of first administration of study treatment (Day 1) to 7 days after the date of the last actual administration of study treatment
Number and percentage of participants with abnormalities in ECG	Summary statistics in abnormalities in ECG parameters	Date of first administration of study treatment (Day 1) to 7 days after the date of the last actual administration of study treatment
Number and percentage of participants with abnormalities in clinical laboratory evaluations	Summary statistics on abnormalities in clinical laboratory evaluations	Date of first administration of study treatment (Day 1) to 7 days after the date of the last actual administration of study treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Annualized total eGFR slope	Annualized rate of renal disease progression as measured by mean eGFR slope at post baseline visits	Screening visit, Months 1, 3, 6, 9, 12 and every 6 months thereafter
Change from baseline in eGFR	Average change from baseline in eGFR at post-baseline visits	Screening visit, Months 1, 3, 6, 9, 12 and every 6 months thereafter
Log transformed ratio to baseline in UPCR, UACR	Log transformed ratio to baseline in UPCR, UACR at post-baseline visits. The log transformation refers to the natural log (base on e)	Screening visit, Months 1, 3, 6, 9, 12 and every 6 months thereafter

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): September 20, 2021
• Primary Completion (Estimated): December 4, 2030
• Study Completion (Estimated): December 4, 2030

Study Registration Dates

• First Submitted: September 15, 2020
• First Submitted That Met QC Criteria: September 15, 2020
• First Posted (Actual): September 21, 2020

Study Record Updates

• Last Update Posted (Actual): June 1, 2026
• Last Update Submitted That Met QC Criteria: May 28, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: chronic kidney disease; glomerulonephritis; eGFR; IgAN; UPCR; LNP023; Immunoglobulin A nephropathy; Primary IgA nephropathy; complement alternative pathway; UACR
• Additional Relevant MeSH Terms: Urogenital Diseases; Pathologic Processes; Male Urogenital Diseases; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Chronic Disease; Disease Attributes; Autoimmune Diseases; Immune System Diseases; Renal Insufficiency; Nephritis; Pathological Conditions, Signs and Symptoms; Glomerulonephritis; Renal Insufficiency, Chronic; Glomerulonephritis, IGA; iptacopan
• Other Study ID Numbers: CLNP023A2002B; 2020-002200-40 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

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.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No