NCT03955445

Brief 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

Trial Health

88
On Track

Trial Health Score

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

Enrollment
225

participants targeted

Target at P25-P50 for phase_3

Timeline
123mo left

Started Oct 2019

Longer than P75 for phase_3

Geographic Reach
17 countries

50 active sites

Status
recruiting

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

Study Progress40%
Oct 2019May 2036

First Submitted

Initial submission to the registry

May 3, 2019

Completed
17 days until next milestone

First Posted

Study publicly available on registry

May 20, 2019

Completed
5 months until next milestone

Study Start

First participant enrolled

October 3, 2019

Completed
16.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 30, 2036

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 30, 2036

Last Updated

April 14, 2026

Status Verified

April 1, 2026

Enrollment Period

16.7 years

First QC Date

May 3, 2019

Last Update Submit

April 9, 2026

Conditions

Keywords

C3GC3 glomerulopathyC3GNC3 glomerulonephritisDDDdense deposit diseaseiptacopanLNP023IC-MPGNidiopathic immune-complex- membranoproliferative glomerulonephritis

Outcome Measures

Primary Outcomes (5)

  • 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.

    9-month visit

  • 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.

    6 - to 9- month visit

  • 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.

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

  • 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.

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

  • 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.

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

Secondary Outcomes (12)

  • CLNP023X2202: Number of participants who achieve the 2-component composite renal endpoint

    9-month visit

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

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

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

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

  • CLNP023X2202: Change from baseline in serum creatinine concentration

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

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

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

  • +7 more secondary outcomes

Study Arms (6)

Cohort A: participants with native kidneys from CLNP023X2202

EXPERIMENTAL

C3G participants from study CLNP023X2202 with native kidneys receiving iptacopan capsules 200 mg b.i.d

Drug: LNP023

Cohort B: participants with transplanted kidneys and recurrent C3G from CLNP023X2202

EXPERIMENTAL

C3G participants from study CLNP023X2202 who have undergone kidney transplant and have recurrence of C3G receiving iptacopan capsules 200 mg b.i.d

Drug: LNP023

Cohort C: Participants with native C3G randomized to placebo in CLNP023B12301

EXPERIMENTAL

Native C3G Participants (adults and adolescents) from CLNP023B12301 study who were randomized to placebo in the core study receiving iptacopan capsules 200mg b.i.d

Drug: LNP023

Cohort D: particpants with native C3G randomised to iptacopan in CLNP023B12301

EXPERIMENTAL

Native C3G participants (adults and adolescents) from study CLNP023B12301 who were randomized to iptacopan in the core study. Receiving iptacopan capsules 200mg b.i.d

Drug: LNP023

Cohort E: participants with IC-MPGN randomized to placebo in CLNP023B12302

EXPERIMENTAL

IC-MPGN participants (adults and adolescents) from study CLNP023B12302 who were randomized to placebo in the core study receiving iptacopan capsules 200mg b.i.d

Drug: LNP023

Cohort F: participants with IC-MPGN randomized to ipatocan in CLNP023B12302

EXPERIMENTAL

IC-MPGN participants (adults and adolescents) from study CLNP023B12302 who were randomized to iptacopan in the core study receiving iptacopan capsules 200mg b.i.d

Drug: LNP023

Interventions

LNP023DRUG

LNP023 capsules

Also known as: iptacopan
Cohort A: participants with native kidneys from CLNP023X2202Cohort B: participants with transplanted kidneys and recurrent C3G from CLNP023X2202Cohort C: Participants with native C3G randomized to placebo in CLNP023B12301Cohort D: particpants with native C3G randomised to iptacopan in CLNP023B12301Cohort E: participants with IC-MPGN randomized to placebo in CLNP023B12302Cohort F: participants with IC-MPGN randomized to ipatocan in CLNP023B12302

Eligibility Criteria

Age12 Years - 100 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (50)

Childrens Hospital Colorado

Aurora, Colorado, 80045, United States

RECRUITING

Georgia Nephrology Research Inst

Lawrenceville, Georgia, 30046, United States

RECRUITING

University of Iowa Health Care

Iowa City, Iowa, 52242-1091, United States

RECRUITING

University of Minnesota

Minneapolis, Minnesota, 55455, United States

RECRUITING

Col Uni Med Center New York Presby

New York, New York, 10032, United States

RECRUITING

Novartis Investigative Site

CABA, Buenos Aires, C1425AGC, Argentina

RECRUITING

Novartis Investigative Site

Buenos Aires, W3400ABH, Argentina

RECRUITING

Novartis Investigative Site

Belo Horizonte, Minas Gerais, 30150-221, Brazil

RECRUITING

Novartis Investigative Site

Recife, Pernambuco, 50740-900, Brazil

RECRUITING

Novartis Investigative Site

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

RECRUITING

Novartis Investigative Site

Botucatu, São Paulo, 3880-1001, Brazil

RECRUITING

Novartis Investigative Site

São Paulo, São Paulo, 04038-002, Brazil

RECRUITING

Novartis Investigative Site

Salvador, 40323-010, Brazil

RECRUITING

Novartis Investigative Site

Toronto, Ontario, M5G 2C4, Canada

ACTIVE NOT RECRUITING

Novartis Investigative Site

Beijing, 100034, China

RECRUITING

Novartis Investigative Site

Shanghai, 200040, China

RECRUITING

Novartis Investigative Site

Prague, 128 08, Czechia

ACTIVE NOT RECRUITING

Novartis Investigative Site

Montpellier, 34295, France

RECRUITING

Novartis Investigative Site

Paris, 75015, France

RECRUITING

Novartis Investigative Site

Toulouse, 31054, France

RECRUITING

Novartis Investigative Site

Erlangen, 91054, Germany

ACTIVE NOT RECRUITING

Novartis Investigative Site

Essen, 45147, Germany

RECRUITING

Novartis Investigative Site

Hamburg, 20246, Germany

RECRUITING

Novartis Investigative Site

Mainz, 55131, Germany

RECRUITING

Novartis Investigative Site

Heraklion Crete., 715 00, Greece

RECRUITING

Novartis Investigative Site

Thessaloniki, 546 42, Greece

RECRUITING

Novartis Investigative Site

Petah Tikva, 4920235, Israel

RECRUITING

Novartis Investigative Site

Petah Tikva, 4941492, Israel

RECRUITING

Novartis Investigative Site

Ranica, BG, 24020, Italy

RECRUITING

Novartis Investigative Site

Roma, 165, Italy

RECRUITING

Novartis Investigative Site

Nagoya, Aichi-ken, 4668560, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Asahikawa, Hokkaido, 0788510, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Sapporo, Hokkaido, 0608543, Japan

COMPLETED

Novartis Investigative Site

Takatsuki, Osaka, 5691192, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Ohtsu, Shiga, 5202192, Japan

RECRUITING

Novartis Investigative Site

Hachiōji, Tokyo, 193-0998, Japan

RECRUITING

Novartis Investigative Site

Niigata, 9518520, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Leiden, South Holland, 2333 ZA, Netherlands

RECRUITING

Novartis Investigative Site

Pamplona, Navarre, 31008, Spain

RECRUITING

Novartis Investigative Site

Barcelona, 08035, Spain

ACTIVE NOT RECRUITING

Novartis Investigative Site

Madrid, 28040, Spain

RECRUITING

Novartis Investigative Site

Madrid, 28041, Spain

RECRUITING

Novartis Investigative Site

Seville, 41009, Spain

RECRUITING

Novartis Investigative Site

Bern, 3010, Switzerland

RECRUITING

Novartis Investigative Site

Köseköy, Kocaeli, 41380, Turkey (Türkiye)

RECRUITING

Novartis Investigative Site

Kayseri, Melikgazi, 38039, Turkey (Türkiye)

RECRUITING

Novartis Investigative Site

Ankara, Yenimahalle, 06500, Turkey (Türkiye)

RECRUITING

Novartis Investigative Site

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

RECRUITING

Novartis Investigative Site

Cardiff, CF14 4XW, United Kingdom

RECRUITING

Novartis Investigative Site

London, W12 0HS, United Kingdom

RECRUITING

MeSH Terms

Conditions

Glomerulonephritis, Membranoproliferative

Interventions

iptacopan

Condition Hierarchy (Ancestors)

GlomerulonephritisNephritisKidney DiseasesUrologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesMale Urogenital DiseasesImmune System Diseases

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

Novartis Pharmaceuticals

CONTACT

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR
Expanded Access
Yes

Study Record Dates

First Submitted

May 3, 2019

First Posted

May 20, 2019

Study Start

October 3, 2019

Primary Completion (Estimated)

May 30, 2036

Study Completion (Estimated)

May 30, 2036

Last Updated

April 14, 2026

Record last verified: 2026-04

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