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C3 GlomerulopathyPhase 3NCT03955445

Long-term Efficacy, Safety and Tolerability of Iptacopan in C3G or IC-MPGN

A Phase 3 study testing LNP023 in people with C3 Glomerulopathy and Immune-complex-membranoproliferative Glomerulonephritis, run by Novartis Pharmaceuticals at 53 sites in 17 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

This is an open-label extension study to evaluate the long-term efficacy, safety and tolerability of iptacopan in subjects with C3 glomerulopathy or idiopathic immune-complex-membranoproliferative glomerulonephritis Read more about this study

Who is it for?
Ages 12 to 100 years
What is being tested?
LNP023
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
225 participants
Where
53 sitesin 17 countries
Study dates
3 Oct 2019to 30 May 2036
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 to 100 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Patients must have completed the treatment period of the CLNP023X2202, CLNP023B12301 or CLNP023B12302 study on study drug

Who cannot join

  • Severe concurrent co-morbidities, e.g. advanced cardiac disease (NYHA class IV), severe pulmonary arterial hypertension (WHO class IV), or any illness or medical condition that in the opinion of the investigator and sponsor is likely to prevent the patient from safely tolerating LNP023 or complying with the requirements of the study
  • Participants with an active systemic bacterial, viral or fungal infection within 14 days prior to screening, or the presence of fever ≥ 38oC (100.4oF) within 7 days prior to screening.
  • History or current diagnosis of ECG abnormalities indicating significant risk of safety for subjects
  • History of HIV or any other immunodeficiency disease
  • Other protocol-defined inclusion/exclusion criteria may apply

Trial locations and contacts

53 sites in 17 countries. Contact the study team or a site near you to ask about taking part.

Novartis Pharmaceuticals contact

1-888-669-6682

novartis.email@novartis.com

Novartis Pharmaceuticals contact

+41613241111

53 sites in 17 countries

Japan7 sites, 2 recruiting
  • Novartis Investigative Site

    Asahikawa, Hokkaido, 0788510

    Active, not recruiting
  • Novartis Investigative Site

    Hachiōji, Tokyo, 193-0998

    Recruiting
  • Novartis Investigative Site

    Nagoya, Aichi-ken, 4668560

    Active, not recruiting
  • Novartis Investigative Site

    Niigata, 9518520

    Active, not recruiting
  • Novartis Investigative Site

    Ohtsu, Shiga, 5202192

    Recruiting
  • Novartis Investigative Site

    Sapporo, Hokkaido, 0608543

    Completed
  • Novartis Investigative Site

    Takatsuki, Osaka, 5691192

    Active, not recruiting

Other trials in Japan

Brazil6 sites, 6 recruiting
  • Novartis Investigative Site

    Belo Horizonte, Minas Gerais, 30150-221

    Recruiting
  • Novartis Investigative Site

    Botucatu, São Paulo, 3880-1001

    Recruiting
  • Novartis Investigative Site

    Porto Alegre, Rio Grande do Sul, 90035-074

    Recruiting
  • Novartis Investigative Site

    Recife, Pernambuco, 50740-900

    Recruiting
  • Novartis Investigative Site

    Salvador, 40323-010

    Recruiting
  • Novartis Investigative Site

    São Paulo, São Paulo, 04038-002

    Recruiting

Other trials in Brazil

United States5 sites, 5 recruiting
  • Childrens Hospital Colorado

    Aurora, Colorado, 80045

    Kati Dugan, +1 720 777 6895, Kati.Dugan@childrenscolorado.org

    Bradley Dixon

    Recruiting
  • University of Iowa Health Care

    Iowa City, Iowa, 52242-1091

    Nicole Gerot, +1 319 335 7555, nicole-gerot@uiowa.edu

    Carla Nester

    Recruiting
  • Georgia Nephrology Research Inst

    Lawrenceville, Georgia, 30046

    Lisa Franklin, +1 404 645 7850#3024, lfranklin@ganephrology.com

    James A Tumlin

    Recruiting
  • University of Minnesota

    Minneapolis, Minnesota, 55455

    Brady Wallner, walln080@umn.edu

    Nattawat Klomjit

    Recruiting
  • Col Uni Med Center New York Presby

    New York, New York, 10032

    Brianna Ortiz, +1 212 304 5684, bo2323@cumc.columbia.edu

    Andrew S Bomback

    Recruiting

Other trials in United States

Germany5 sites, 3 recruiting
  • Novartis Investigative Site

    Erlangen, 91054

    Active, not recruiting
  • Novartis Investigative Site

    Essen, 45147

    Recruiting
  • Novartis Investigative Site

    Hamburg, 20246

    Active, not recruiting
  • Novartis Investigative Site

    Heidelberg, 69120

    Recruiting
  • Novartis Investigative Site

    Mainz, 55131

    Recruiting

Other trials in Germany

Spain5 sites, 4 recruiting
  • Novartis Investigative Site

    Barcelona, 08035

    Completed
  • Novartis Investigative Site

    Madrid, 28040

    Recruiting
  • Novartis Investigative Site

    Madrid, 28041

    Recruiting
  • Novartis Investigative Site

    Pamplona, Navarre, 31008

    Recruiting
  • Novartis Investigative Site

    Seville, 41009

    Recruiting

Other trials in Spain

France4 sites, 3 recruiting
  • Novartis Investigative Site

    Montpellier, 34295

    Recruiting
  • Novartis Investigative Site

    Paris, 75015

    Active, not recruiting
  • Novartis Investigative Site

    Paris, 75015

    Recruiting
  • Novartis Investigative Site

    Toulouse, 31054

    Recruiting

Other trials in France

Italy3 sites, 3 recruiting
  • Novartis Investigative Site

    Bari, BA, 70124

    Recruiting
  • Novartis Investigative Site

    Ranica, BG, 24020

    Recruiting
  • Novartis Investigative Site

    Roma, RM, 00165

    Recruiting

Other trials in Italy

Turkey (Türkiye)3 sites, 2 recruiting
  • Novartis Investigative Site

    Ankara, Yenimahalle, 06500

    Recruiting
  • Novartis Investigative Site

    Kayseri, Melikgazi, 38039

    Active, not recruiting
  • Novartis Investigative Site

    Köseköy, Kocaeli, 41380

    Recruiting

Other trials in Turkey (Türkiye)

United Kingdom3 sites, 2 recruiting
  • Novartis Investigative Site

    Cardiff, CF14 4XW

    Recruiting
  • Novartis Investigative Site

    London, W12 0HS

    Recruiting
  • Novartis Investigative Site

    Newcastle upon Tyne, Tyne and Wear, NE7 7DN

    Active, not recruiting

Other trials in United Kingdom

Argentina2 sites, 2 recruiting
  • Novartis Investigative Site

    Buenos Aires, W3400ABH

    Recruiting
  • Novartis Investigative Site

    CABA, Buenos Aires, C1425AGC

    Recruiting

Other trials in Argentina

China2 sites, 2 recruiting
  • Novartis Investigative Site

    Beijing, 100034

    Recruiting
  • Novartis Investigative Site

    Shanghai, 200040

    Recruiting

Other trials in China

Greece2 sites
  • Novartis Investigative Site

    Heraklion Crete., 715 00

    Active, not recruiting
  • Novartis Investigative Site

    Thessaloniki, 546 42

    Active, not recruiting

Other trials in Greece

Israel2 sites, 1 recruiting
  • Novartis Investigative Site

    Petah Tikva, 4920235

    Recruiting
  • Novartis Investigative Site

    Petah Tikva, 4941492

    Active, not recruiting

Other trials in Israel

Canada1 site
  • Novartis Investigative Site

    Toronto, Ontario, M5G 2C4

    Active, not recruiting

Other trials in Canada

Czechia1 site
  • Novartis Investigative Site

    Prague, 128 08

    Active, not recruiting

Other trials in Czechia

Netherlands1 site
  • Novartis Investigative Site

    Leiden, South Holland, 2333 ZA

    Active, not recruiting

Other trials in Netherlands

Switzerland1 site, 1 recruiting
  • Novartis Investigative Site

    Bern, 3010

    Recruiting

Other trials in Switzerland

About this study

Official title: An Open-label, Non-randomized Extension Study to Evaluate the Long-term Efficacy, Safety and Tolerability of Iptacopan (LNP023) in C3 Glomerulopathy or Idiopathic Immune-complex-membranoproliferative Glomerulonephritis

This is an open-label extension study to evaluate the long-term efficacy, safety and tolerability of iptacopan in subjects with C3 glomerulopathy or idiopathic immune-complex-membranoproliferative glomerulonephritis

Read the detailed description

The primary purpose of this extension study is to collect long-term efficacy, safety and tolerability data in eligible participants receiving open-label iptacopan after completing treatment in the C3G Phase 2 proof of concept study CLNP023X2202.

The primary (at 9 months) and longer-term (>9 months) efficacy and safety data of iptacopan collected from CLNP023X2202 participants will be used to support health authority submissions.

This umbrella protocol will also allow:

  • continued access to iptacopan to patients enrolled in the ongoing Phase 3 programs (C3G and IC-MPGN)
  • C3G study (CLNP023B12301): adults and adolescents
  • IC-MPGN study (CLNP023B12302): adults and adolescents
  • provision of additional efficacy and safety information following longer-term treatment in C3G and IC-MPGN populations to support health authority submissions.

Efficacy and safety assessments at the 9 month visit of this extension study in combination with data from CLNP023X2202 (baseline plus 3 months of treatment) allowed evaluation of the effects of iptacopan on potential endpoint(s) at 12 months of iptacopan treatment in C3G participants. The enrollment of C3G and IC-MPGN participants (adults and adolescents) from Phase 3 studies, CLNP023B12301 and CLNP023B12302, permits longer-term evaluation of the persistence of effects observed after iptacopan treatment. These longer term efficacy and safety assessments may be compared to historical/concurrent control data available from relevant real world databases in C3G or IC-MPGN patients and used as supportive information for registration purposes.

This extension study is expected to continue until the drug product becomes commercially available and accessible (anticipated to be up to approximately 168 months from the first patient first visit date), or the benefit-risk profile is no longer positive, or the program is discontinued for business or strategic reasons.

"Baseline" refers to the Day 1 visit (pre-dose) of CLNP023X2202, CLNP023B12301 or CLNP023B12302, whereas the Day 1 visit for this C3G/IC-MPGN extension study (CLNP023B12001B) is identified as "Extension Day 1".

Being tested
LNP023
Keywords
C3G, C3 glomerulopathy, C3GN, C3 glomerulonephritis, DDD, dense deposit disease, iptacopan, LNP023, IC-MPGN, idiopathic immune-complex- membranoproliferative glomerulonephritis
Medical subject headings
Glomerulonephritis, Membranoproliferative

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • LNP023Drug

    Also called iptacopan

    LNP023 capsules

Study groups in NCT03955445
Study groupRoleWhat they receive
Cohort A: participants with native kidneys from CLNP023X2202ExperimentalC3G participants from study CLNP023X2202 with native kidneys receiving iptacopan capsules 200 mg b.i.d
Receives: Drug: LNP023
Cohort B: participants with transplanted kidneys and recurrent C3G from CLNP023X2202ExperimentalC3G participants from study CLNP023X2202 who have undergone kidney transplant and have recurrence of C3G receiving iptacopan capsules 200 mg b.i.d
Receives: Drug: LNP023
Cohort C: Participants with native C3G randomized to placebo in CLNP023B12301ExperimentalNative C3G Participants (adults and adolescents) from CLNP023B12301 study who were randomized to placebo in the core study receiving iptacopan capsules 200mg b.i.d
Receives: Drug: LNP023
Cohort D: particpants with native C3G randomised to iptacopan in CLNP023B12301ExperimentalNative C3G participants (adults and adolescents) from study CLNP023B12301 who were randomized to iptacopan in the core study. Receiving iptacopan capsules 200mg b.i.d
Receives: Drug: LNP023
Cohort E: participants with IC-MPGN randomized to placebo in CLNP023B12302ExperimentalIC-MPGN participants (adults and adolescents) from study CLNP023B12302 who were randomized to placebo in the core study receiving iptacopan capsules 200mg b.i.d
Receives: Drug: LNP023
Cohort F: participants with IC-MPGN randomized to ipatocan in CLNP023B12302ExperimentalIC-MPGN participants (adults and adolescents) from study CLNP023B12302 who were randomized to iptacopan in the core study receiving iptacopan capsules 200mg b.i.d
Receives: Drug: LNP023

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. CLNP023X2202 Cohort A-native C3G: Number of participants who achieve the composite renal endpoint

    A participant meets the requirements of the composite renal endpoint if they satisfy the following criteria at the 9-month visit in CLNP023B12001B: (1) a stable or improved eGFR compared to the baseline visit in CLNP023X2202 (≤10% reduction in eGFR), and (2) either ≥50% reduction compared to the baseline visit in CLNP023X2202 or a reduction to <300 mg/g in UPCR and (3) either a ≥50% increase in C3 compared to baseline or an increase to ≥90 mg/dL (i.e., ≥ the lower limit of normal (LLN)). Initiation of treatment with eculizumab or any other complement pathway modifying agent automatically designates the participant as not meeting the endpoint.

    Measured: 9-month visit

  2. CLNP023X2202 Cohort B - kidney transplant and recurrent C3G: Change from baseline in the C3 Deposit Score

    Change from baseline in the C3 Deposit Score (based on immunofluorescence microscopy) compared to baseline in the CLNP023X2202 study.

    Measured: 6 - to 9- month visit

  3. Number of AEs of special interest for participants from CLNP023X2202, CLNP023B12301 and CLNP023B12302

    Number of participants with AEs of special interest will be collected to evaluate the long-term safety and tolerability of iptacopan in participants.

    Measured: Participants are expected to continue on study for a minimum of 60 months and a maximum of 84 months

  4. Number of participants with study drug discontinuation due to an AE (or any safety issue) for participants from CLNP023X2202, CLNP023B12301 and CLNP023B12302

    Number of participants with study drug discontinuation due to an AE to evaluate the long-term safety and tolerability of iptacopan in participants.

    Measured: Participants are expected to continue on study for a minimum of 60 months and a maximum of 84 months

  5. Number of participants with abnormal clinically significant vital signs,ECGs, and safety laboratory measurements for participants from CLNP023X2202, CLNP023B12301 and CLNP023B12302

    Number of participants with abnormal clinically significant vital signs, ECGs, and safety laboratory measurements to evaluate the long-term safety and tolerability of iptacopan in participants.

    Measured: Participants are expected to continue on study for a minimum of 60 months and a maximum of 84 months

Secondary outcomes (12)
  1. CLNP023X2202: Number of participants who achieve the 2-component composite renal endpoint

    A participant is defined as achieving the composite renal endpoint if they meet the following criteria at the 9-month visit in CLNP023B12001B: (1) a stable or improved eGFR compared to the baseline visit in CLNP023X2202 (≤10% reduction in eGFR), and (2) either ≥50% reduction compared to the baseline visit in CLNP023X2202 or a reduction to <300 mg/g in UPCR.

    Initiation of treatment with eculizumab or any other complement pathway modifying agent automatically designates the participant as a not meeting the composite renal endpoint.

    Measured: 9-month visit

  2. CLNP023X2202: Change from baseline in log-transformed urine protein/creatinine ratio (UPCR)

    Long-term effect of LNP023 on renal function in C3G subjects by assessing the change from baseline in log-transformed urine protein/creatinine ratio (UPCR)

    Measured: Participants are expected to continue on study for a minimum of 60 months and a maximum of 84 months

  3. CLNP023X2202: Change from baseline in log-transformed urine albumin/creatinine ratio (UACR)

    Long-term effect of LNP023 on renal function in C3G subjects by assessing the change from baseline in log-transformed urine albumin/creatinine ratio (UACR)

    Measured: Participants are expected to continue on study for a minimum of 60 months and a maximum of 84 months

  4. CLNP023X2202: Change from baseline in serum creatinine concentration

    Long-term effect of LNP023 on renal function in C3G subjects by assessing the change in serum creatinine compared to CLNP023X2202 baseline

    Measured: Participants are expected to continue on study for a minimum of 60 months and a maximum of 84 months

  5. CLNP023X2202: Change from baseline in estimated glomerular filtration rate (eGFR)

    Long-term effect of LNP023 on renal function in C3G subjects by assessing the change in eGFR compared to CLNP023X2202 baseline

    Measured: Participants are expected to continue on study for a minimum of 60 months and a maximum of 84 months

  6. CLNP023X2202: Status of C3G disease progression

    Describe the status of C3G disease progression based on glomerular histopathology in a renal biopsy at 6 to 9 months from entry to the study compared to those obtained prior to treatment in the CLNP023X2202 study

    Measured: 6 to 9 month visit

  7. CLNP023X2202: Log-transformed ratio to baseline in serum C3

    Long-term effect of LNP023 on C3 by evaluating the Log-transformed ratio to baseline in serum C3

    Measured: Participants are expected to continue on study for a minimum of 60 months and a maximum of 84 months

  8. CLNP023X2202: Number of participants who achieve the composite renal endpoint

    A participant is defined as achieving the composite renal endpoint if they meet the following criteria at times >9 months in CLNP023B12001B: (1) a stable or improved eGFR compared to the baseline visit in CLNP023X2202 (≤10% reduction in eGFR), and (2) either ≥50% reduction compared to the baseline visit in CLNP023X2202 or a reduction to <300 mg/g in UPCR and (3) either a ≥50% increase in C3 compared to baseline or an increase to ≥90 mg/dL (i.e., LLN). Initiation of treatment with eculizumab or any other complement pathway modifying agent automatically designates the participant as a not meeting the composite renal endpoint.

    Measured: Up to 66 months

  9. CLNP023X2202: Plasma LNP023 concentration up to 12 months at trough

    Measurement of LNP023 plasma concentration to evaluate the pharmacokinetics of iptacopan in participants with prolonged treatment

    Measured: 3-months, 6-months, 9-months and 12-months visits

  10. CLNP023B12301 and CLNP023B12302: Change from initiation of iptacopan treatment in the core study in log-transformed UPCR over time.

    Change from initiation of iptacopan treatment in the core study in log-transformed UPCR will be assessed to evaluate the long-term effect of iptacopan on proteinuria

    Measured: Participants are expected to continue on study for a minimum of 60 months and a maximum of 84 months

  11. CLNP023B12301 and CLNP023B12302: Change from initiation of iptacopan treatment in the core study in eGFR over time.

    Change from initiation of iptacopan treatment in the core study in eGFR over time will be assessed to evaluate the long-term effect of iptacopan on eGFR

    Measured: Participants are expected to continue on study for a minimum of 60 months and a maximum of 84 months

  12. CLNP023B12301 and CLNP023B12302: Number of participants who achieve a 2-component composite renal endpoint

    A participant is defined as meeting the requirements of the composite renal endpoint if they satisfy the eGFR (a stable or improved eGFR, i.e., ≤15% reduction in eGFR compared to the initiation of iptacopan treatment in the core study) and UPCR (≥50% reduction in UPCR compared to the initiation of iptacopan treatment in the core study) criteria assessed at a visit. Initiation of any complement pathway modifying agent or initiation/intensification of corticosteroid or immunosuppressant therapy, or renal replacement therapy automatically designates the participant as not having met the endpoint. The rate will be evaluated over time.

    Measured: Participants are expected to continue on study for a minimum of 60 months and a maximum of 84 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 May 2019
  2. Study start3 Oct 2019
  3. Primary completion30 May 2036 (estimated)
  4. Study completion30 May 2036 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
225 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03955445
Sponsor's protocol ID
CLNP023B12001B
Other IDs
2018-004253-24, 2023-509343-27-00, 2023, 2018
Status last verified
Aug 2026
First submitted
3 May 2019
Last updated
7 Aug 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: 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.
Expanded access
Available

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing LNP023 in people with C3 Glomerulopathy and Immune-complex-membranoproliferative Glomerulonephritis, run by Novartis Pharmaceuticals at 53 sites in 17 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 1 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 7 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 53 sites in Japan, Brazil, United States, Germany, Spain and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Novartis Pharmaceuticals (1-888-669-6682, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

CLNP023X2202 Cohort A-native C3G: Number of participants who achieve the composite renal endpoint (time frame: 9-month visit). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Aug 2026.

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