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

NCT ID: NCT03955445

Last Updated: 2025-12-04

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

225 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-10-03

Study Completion Date

2036-05-30

Brief Summary

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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

Detailed Description

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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".

Conditions

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C3 Glomerulopathy Immune-complex-membranoproliferative Glomerulonephritis

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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

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

Group Type EXPERIMENTAL

LNP023

Intervention Type DRUG

LNP023 capsules

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

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

Group Type EXPERIMENTAL

LNP023

Intervention Type DRUG

LNP023 capsules

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

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

Group Type EXPERIMENTAL

LNP023

Intervention Type DRUG

LNP023 capsules

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

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

Group Type EXPERIMENTAL

LNP023

Intervention Type DRUG

LNP023 capsules

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

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

Group Type EXPERIMENTAL

LNP023

Intervention Type DRUG

LNP023 capsules

Cohort A: participants with native kidneys from CLNP023X2202

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

Group Type EXPERIMENTAL

LNP023

Intervention Type DRUG

LNP023 capsules

Interventions

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LNP023

LNP023 capsules

Intervention Type DRUG

Other Intervention Names

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iptacopan

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

* 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
Minimum Eligible Age

12 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Novartis Investigative Site

Bern, , Switzerland

Site Status RECRUITING

Novartis Investigative Site

Köseköy, Kocaeli, Turkey (Türkiye)

Site Status RECRUITING

Novartis Investigative Site

Kayseri, Melikgazi, Turkey (Türkiye)

Site Status RECRUITING

Novartis Investigative Site

Ankara, Yenimahalle, Turkey (Türkiye)

Site Status RECRUITING

Novartis Investigative Site

London, London, United Kingdom

Site Status RECRUITING

Novartis Investigative Site

Newcastle upon Tyne, Tyne and Wear, United Kingdom

Site Status RECRUITING

Novartis Investigative Site

Madrid, , Spain

Site Status RECRUITING

Novartis Investigative Site

Madrid, , Spain

Site Status RECRUITING

Childrens Hospital Colorado

Aurora, Colorado, United States

Site Status RECRUITING

Georgia Nephrology Research Inst

Lawrenceville, Georgia, United States

Site Status RECRUITING

University of Iowa Health Care

Iowa City, Iowa, United States

Site Status RECRUITING

University of Minnesota

Minneapolis, Minnesota, United States

Site Status RECRUITING

Col Uni Med Center New York Presby

New York, New York, United States

Site Status RECRUITING

Novartis Investigative Site

CABA, Buenos Aires, Argentina

Site Status RECRUITING

Novartis Investigative Site

Buenos Aires, , Argentina

Site Status RECRUITING

Novartis Investigative Site

Belo Horizonte, Minas Gerais, Brazil

Site Status RECRUITING

Novartis Investigative Site

Porto Alegre, Rio Grande do Sul, Brazil

Site Status RECRUITING

Novartis Investigative Site

Botucatu, São Paulo, Brazil

Site Status RECRUITING

Novartis Investigative Site

São Paulo, São Paulo, Brazil

Site Status RECRUITING

Novartis Investigative Site

Salvador, , Brazil

Site Status RECRUITING

Novartis Investigative Site

Toronto, Ontario, Canada

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Beijing, , China

Site Status RECRUITING

Novartis Investigative Site

Shanghai, , China

Site Status RECRUITING

Novartis Investigative Site

Prague, , Czechia

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Montpellier, , France

Site Status RECRUITING

Novartis Investigative Site

Paris, , France

Site Status RECRUITING

Novartis Investigative Site

Toulouse, , France

Site Status RECRUITING

Novartis Investigative Site

Erlangen, , Germany

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Essen, , Germany

Site Status RECRUITING

Novartis Investigative Site

Hamburg, , Germany

Site Status RECRUITING

Novartis Investigative Site

Mainz, , Germany

Site Status RECRUITING

Novartis Investigative Site

Heraklion Crete., , Greece

Site Status RECRUITING

Novartis Investigative Site

Petah Tikva, , Israel

Site Status RECRUITING

Novartis Investigative Site

Petah Tikva, , Israel

Site Status RECRUITING

Novartis Investigative Site

Ranica, BG, Italy

Site Status RECRUITING

Novartis Investigative Site

Roma, RM, Italy

Site Status RECRUITING

Novartis Investigative Site

Nagoya, Aichi-ken, Japan

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Asahikawa, Hokkaido, Japan

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Sapporo, Hokkaido, Japan

Site Status COMPLETED

Novartis Investigative Site

Takatsuki, Osaka, Japan

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Ohtsu, Shiga, Japan

Site Status RECRUITING

Novartis Investigative Site

Hachiōji, Tokyo, Japan

Site Status RECRUITING

Novartis Investigative Site

Niigata, , Japan

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Leiden, South Holland, Netherlands

Site Status RECRUITING

Novartis Investigative Site

Seville, Andalusia, Spain

Site Status RECRUITING

Novartis Investigative Site

Barcelona, Catalonia, Spain

Site Status ACTIVE_NOT_RECRUITING

Novartis Investigative Site

Pamplona, Navarre, Spain

Site Status RECRUITING

Novartis Investigative Site

Cardiff, , United Kingdom

Site Status RECRUITING

Countries

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United States Argentina Brazil Canada China Czechia France Germany Greece Israel Italy Japan Netherlands Spain Switzerland Turkey (Türkiye) United Kingdom

Central Contacts

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Novartis Pharmaceuticals

Role: CONTACT

1-888-669-6682

Novartis Pharmaceuticals

Role: CONTACT

+41613241111

Facility Contacts

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Nathan Rogers

Role: primary

+1 720 777 6895

Julie Scoggin

Role: primary

+1 404 645 7850#3024

Role: primary

+1 319 335 7555

Katherine Wu

Role: primary

Brianna Ortiz

Role: primary

+1 212 304 5684

Other Identifiers

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2018-004253-24

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2023-509343-27-00

Identifier Type: OTHER

Identifier Source: secondary_id

CLNP023B12001B

Identifier Type: -

Identifier Source: org_study_id

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