Clinical trial • Phase III • Immunology|Rare Disease
IPTACOPAN for C3 glomerulopathy
Phase III trial of IPTACOPAN for C3 glomerulopathy.
Overview
- Trial Therapeutic Area
- Immunology|Rare Disease
- Trial Disease
- C3 glomerulopathy
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 29-05-2024
- First CTIS Authorization Date
- 08-07-2024
Trial design
Randomised, placebo matching iptacopan (placebo 0 mg hard gelatin capsule size 1, placebo to lnp023 100 mg; placebo 0 mg hard gelatin capsule size 0, placebo to lnp023 200 mg).-controlled Phase III trial across 22 sites in France, Germany, Greece and others.
- Randomised
- Yes
- Comparator
- Placebo matching iptacopan (Placebo 0 mg hard gelatin capsule size 1, placebo to LNP023 100 mg; Placebo 0 mg hard gelatin capsule size 0, placebo to LNP023 200 mg).
- Target Sample Size
- 74
- Trial Duration For Participant
- 365
Eligibility
Recruits 74 paediatric patients.
- Vulnerable Population
- Adolescent participants (age ≥12 and ≤17 years) are included; the trial includes provisions for assent and parental/legal guardian consent. Subject information and informed consent/assent documents are provided for adolescents (Adolescent Assent forms) and for Parent/Legal Guardian consent (Parent Legal Guardian forms). There are also 'Adolescent Becoming Adult' and specific follow-up documents (e.g., follow up for pregnant participant). Country-specific consent/assent materials exist (documents in Italian, Greek, German, French, Spanish, Dutch and English are present). Germany has an additional requirement for adolescent participants to have reached Tanner stage IV or V at screening.
Inclusion criteria
- {"criterion_text":"- Male and female participants age ≥ 18 and ≤ 60 years (adult cohort) or age ≥ 12 and ≤ 17 years (adolescent cohort) at screening.\n- Diagnosis of C3G as confirmed by renal biopsy within 12 months prior to screening in adults and within 3 years of screening in adolescents (a biopsy report, review and confirmation by the Investigator is required). If such a biopsy is not available, confirmation may be obtained using tissue from the Day -45 biopsy (adults only) for local assessment (tissue may be processed, evaluated, and reported by Arkana Laboratories but eligibility is determined by the Investigator).\n- Prior to randomization, all participants must have been on a maximally recommended or tolerated dose of renin-angiotensin system inhibitors (RASi), e.g., an angiotensin converting enzyme inhibitor (ACEi) or an angiotensin receptor blocker (ARB) for at least 90 days. The doses of other antiproteinuric medications including mycophenolic acids, corticosteroids, and mineralocorticoid receptor antagonists should be stable for at least 90 days prior to randomization.\n- Reduced serum C3 (defined as less than 0.85 x lower limit of the central laboratory normal range) at Screening.\n- UPCR ≥ 1.0 g/g sampled from the first morning void urine sample at Day -75 and Day -15.\n- Estimated GFR (using the CKD-EPI formula for adults and modified Schwartz formula for adolescents) or measured GFR ≥ 30 ml/min/1.73m2 at Screening and Day -15.\n- Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection prior to the start of study treatment. If the patient has not been previously vaccinated, or if a booster is required, the vaccine should be given according to local guidelines at least 2 weeks prior to the first administration of study treatment. If study treatment has to start earlier than 2 weeks post vaccination, prophylactic antibiotic treatment should be initiated.\n- Vaccination against Haemophilus influenzae infections is recommended according to local guidelines, at least 2 weeks prior to the first study treatment administration.\n- In Germany only, adolescent participants must also have reached Tanner stage IV or V at the Screening visit to be enrolled in the study."}
Exclusion criteria
- {"criterion_text":"- Participants who have received any cell or organ transplantation, including kidney transplantation.\n- Rapidly progressive crescentic glomerulonephritis defined as a 50% decline in the eGFR within 3 months with renal biopsy findings of glomerular crescent formation seen in at least 50% of glomeruli.\n- Renal biopsy showing interstitial fibrosis/tubular atrophy (IF/TA) of more than 50%.\n- Monoclonal gammopathy of undetermined significance (MGUS) confirmed by the measurement of serum free light chains or other investigation as per local standard of care.\n- Participants with an active systemic bacterial, viral or fungal infection within 14 days prior to study treatment administration or the presence of fever ≥ 38°C (100.4°F) within 7 days prior to study treatment administration.\n- A history of recurrent invasive infections caused by encapsulated organisms, e.g., meningococcus or pneumococcus.\n- The use of inhibitors of complement factors (e.g., Factor B, Factor D, C3 inhibitors, anti Complement component 5 (C5) antibodies, C5a receptor antagonists) within 6 months prior to the Screening visit.\n- The use of immunosuppressants (except mycophenolic acids), cyclophosphamide or systemic prednisone at a dose >7.5 mg/day (or equivalent for a similar medication) within 90 days of study drug administration. The use of mycophenolic acids is not permitted within 90 days prior to randomization in India.\n- Acute post-infectious glomerulonephritis at screening based upon the opinion of the investigator."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Primary endpoint for adult participants: - Log-transformed ratio to baseline in UPCR (sampled from a 24-hour urine collection) Primary endpoint for adolescent participants: - Log-transformed ratio to baseline in UPCR (sampled from a 24-hour urine collection).","definition_or_measurement_approach":"UPCR (urine protein-to-creatinine ratio) sampled from a 24-hour urine collection; endpoint is the log-transformed ratio to baseline in UPCR."}
Secondary endpoints
- {"endpoint_text":"- Secondary endpoint for adult participants: - Change from baseline in eGFR.\n- Secondary endpoint for adult participants: - A participant meets the requirements of the composite renal endpoint if he/she satisfies: (1) a stable or improved eGFR compared to the baseline visit (≤15% reduction in eGFR), and (2) a ≥50% reduction in UPCR compared to the baseline visit.\n- Secondary endpoint for adult participants: - Change from baseline in disease total activity score in a renal biopsy.\n- Secondary endpoint for adult participants: - Change in the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT Fatigue) score.\n- Secondary endpoint for adult participants: - Occurrence of clinically significant vital signs (msDBP, msSBP, heart rate), ECGs, and safety laboratory measurements, as well as adverse events (AEs), AEs of special interest, and study drug discontinuation due to an AE.\n- Secondary endpoint for adolescent participants: - Change from baseline in eGFR.\n- Secondary endpoint for adolescent participants: - A participant meets the requirements of the composite renal endpoint if he/she satisfies the following criteria at the 6-month time point: (1) a stable or improved eGFR compared to the baseline visit (≤15% reduction in eGFR), and (2) a ≥50% reduction in UPCR compared to the baseline visit.\n- Secondary endpoint for adolescent participants: - Change from baseline to 6 months in the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) score.\n- Secondary endpoint for adolescent participants: - Occurrence of clinically significant vital signs, ECGs, and safety laboratory measurements, as well as adverse events (AEs), AEs of special interest, and study drug discontinuation due to an AE (or any safety issue) during the double-blind period of the study.","definition_or_measurement_approach":"eGFR change from baseline (adults: CKD-EPI; adolescents: modified Schwartz formula as used in eligibility), composite renal endpoint defined as (1) stable or improved eGFR vs baseline (≤15% reduction) AND (2) ≥50% reduction in UPCR vs baseline; renal biopsy total activity score change from baseline; patient-reported fatigue measured by FACIT-Fatigue; safety endpoints include vital signs (msDBP, msSBP, heart rate), ECGs, safety laboratory measurements, adverse events, AEs of special interest, and discontinuation due to AE."}
Recruitment
- Digital Remote Recruitment
- Yes
- Planned Sample Size
- 74
- Recruitment Window Months
- 66
- Consent Approach
- Adults provide written informed consent using country-specific Main ICF documents. Adolescents (age 12–17) provide assent using 'Adolescent Assent' forms while Parent/Legal Guardian consent is required (Parent Legal Guardian forms are provided). There are also 'Adolescent Becoming Adult' and specific follow-up consent documents (including pregnancy follow-up). Consent/assent materials are available in multiple languages (English and country languages shown in the submission: Italian, Greek, German, French, Spanish, Dutch, etc.). Separate data protection consent and optional assessment consents are provided where applicable.
Methods
- Country-specific recruitment advertisements/documents (K2_Advertisements / K1_Recruitment Arrangements) present for multiple countries (documents labeled for Italy, Germany, Greece, France, Netherlands, etc.).
- Use of third-party vendors for patient recruitment and retention and patient engagement: Greenphire LLC (Patient Recruitment & Retention - patient travel and accommodation), Myonex LLC (Patient Recruitment & Retention Materials), Publicis Healthcare Communications Group Limited (Patient Recruitment & Retention Material: Participant Safety Card, Participant Workbook & Study Logo), and other vendors supporting patient recruitment/engagement (e.g., Jumo Health USA Inc. - patient education and engagement).
- Provision of ePRO materials and translations (Kayentis, RWS Life Sciences, IQVIA-related services) to support patient-reported outcomes collection and participant communications.
Geography
- Total Number Of Sites
- 22
- Total Number Of Participants
- 37
France
- Latest Decision Or Authorization Date
- 17-02-2026
- Number Of Sites
- 7
- Number Of Participants
- 7
Sites
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- #3205: Service de néphrologie pédiatrique
- Principal Investigator Name
- François Provot
- Principal Investigator Email
- provotf@gmail.com
- Contact Person Name
- François Provot
- Contact Person Email
- provotf@gmail.com
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- #3206: - Service de Néphrologie et Transplantation Adulte - Service de Néphrologie Pédiatrique
- Principal Investigator Name
- Aude Servais
- Principal Investigator Email
- aude.servais@nck.aphp.fr
- Contact Person Name
- Aude Servais
- Contact Person Email
- aude.servais@nck.aphp.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- #3207: Département de Néphrologie Pédiatrique
- Principal Investigator Name
- Julien Hogan
- Principal Investigator Email
- Julien.hogan2@aphp.fr
- Contact Person Name
- Julien Hogan
- Contact Person Email
- Julien.hogan2@aphp.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- #3202: Service de Néphrologie et de dialyse
- Principal Investigator Name
- Sophie Chauvet
- Principal Investigator Email
- sophie.chauvet@aphp.fr
- Contact Person Name
- Sophie Chauvet
- Contact Person Email
- sophie.chauvet@aphp.fr
- Site Name
- Centre Hospitalier Universitaire De Montpellier
- Department Name
- #3201: Département: Néphrologie/Transplantation
- Principal Investigator Name
- Moglie Le Quintrec-Donnette
- Principal Investigator Email
- m-lequintrec-donnette@chu-montpellier.fr
- Contact Person Name
- Moglie Le Quintrec-Donnette
- Contact Person Email
- m-lequintrec-donnette@chu-montpellier.fr
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- #3205: Service de Néphrologie Adulte
- Principal Investigator Name
- François Provot
- Principal Investigator Email
- provotf@gmail.com
- Contact Person Name
- François Provot
- Contact Person Email
- provotf@gmail.com
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- Avenue Eugene Avinee (site address listed)
- Principal Investigator Name
- François Provot
- Principal Investigator Email
- provotf@gmail.com
- Contact Person Name
- François Provot
- Contact Person Email
- provotf@gmail.com
Germany
- Latest Decision Or Authorization Date
- 13-05-2026
- Number Of Sites
- 2
- Number Of Participants
- 8
Sites
- Site Name
- Universitaetsklinikum Heidelberg AöR
- Department Name
- #3311: Klinik Kinderheilkunde I
- Principal Investigator Name
- Christian Patry
- Principal Investigator Email
- Christian.Patry@med.uni-heidelberg.de
- Contact Person Name
- Christian Patry
- Contact Person Email
- Christian.Patry@med.uni-heidelberg.de
- Site Name
- University Hospital Cologne AöR
- Department Name
- #3312 : Klinik und Poliklinik für Kinder- und Jugendmedizin Pädiatrische Nephrologie
- Principal Investigator Name
- Sandra Habbig
- Principal Investigator Email
- sandra.habbig@uk-koeln.de
- Contact Person Name
- Sandra Habbig
- Contact Person Email
- sandra.habbig@uk-koeln.de
Greece
- Latest Decision Or Authorization Date
- 13-02-2026
- Number Of Sites
- 3
- Number Of Participants
- 2
Sites
- Site Name
- Laiko General Hospital Of Athens
- Department Name
- 3401: Kidney Transplant And Nephrology Clinic
- Principal Investigator Name
- Ioannis Boletis
- Principal Investigator Email
- laikneph@laiko.gr
- Contact Person Name
- Ioannis Boletis
- Contact Person Email
- laikneph@laiko.gr
- Site Name
- University General Hospital Of Heraklion
- Department Name
- 3403: Nephrology Department
- Principal Investigator Name
- Konstantinos Stylianou
- Principal Investigator Email
- kstylianu@gmail.com
- Contact Person Name
- Konstantinos Stylianou
- Contact Person Email
- kstylianu@gmail.com
- Site Name
- Ippokratio General Hospital Of Thessaloniki
- Department Name
- 3404: 1st dpt. of Pediatrics
- Principal Investigator Name
- Stella Stabouli
- Principal Investigator Email
- sstaboul@auth.gr
- Contact Person Name
- Stella Stabouli
- Contact Person Email
- sstaboul@auth.gr
Italy
- Latest Decision Or Authorization Date
- 23-03-2026
- Number Of Sites
- 3
- Number Of Participants
- 11
Sites
- Site Name
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
- Department Name
- 3603: S.C. Nefrologia e Dialisi Pediatrica Trapianti di Rene Padiglione De Marchi
- Principal Investigator Name
- Gianluigi Ardissino
- Principal Investigator Email
- ardissino@centroseu.org
- Contact Person Name
- Gianluigi Ardissino
- Contact Person Email
- ardissino@centroseu.org
- Site Name
- Bambino Gesu Childrens Hospital
- Department Name
- 3602: U.O. Nefrologia e Dialisi
- Principal Investigator Name
- Marina Vivarelli
- Principal Investigator Email
- marina.vivarelli@opbg.net
- Contact Person Name
- Marina Vivarelli
- Contact Person Email
- marina.vivarelli@opbg.net
- Site Name
- Istituto Di Ricerche Farmacologiche Mario Negri
- Department Name
- 3601: Centro Ricerche Cliniche per Malattie Rare Aldo e Cele Daccò
- Principal Investigator Name
- Giuseppe Remuzzi
- Principal Investigator Email
- giuseppe.remuzzi@marionegri.it
- Contact Person Name
- Giuseppe Remuzzi
- Contact Person Email
- giuseppe.remuzzi@marionegri.it
Spain
- Latest Decision Or Authorization Date
- 24-02-2026
- Number Of Sites
- 4
- Number Of Participants
- 4
Sites
- Site Name
- Hospital Universitari Vall D Hebron
- Department Name
- #3807: Nefrología pediátrica
- Principal Investigator Name
- Gema Ariceta Iraola
- Principal Investigator Email
- gema.ariceta@vallhebron.cat
- Contact Person Name
- Gema Ariceta Iraola
- Contact Person Email
- gema.ariceta@vallhebron.cat
- Site Name
- Hospital Universitario 12 De Octubre
- Department Name
- #3801: Nefrología
- Principal Investigator Name
- Teresa Cavero Escribano
- Principal Investigator Email
- tcaveroescribano@gmail.com
- Contact Person Name
- Teresa Cavero Escribano
- Contact Person Email
- tcaveroescribano@gmail.com
- Site Name
- Hospital Universitario Virgen De La Victoria
- Department Name
- #3805: Nefrología
- Principal Investigator Name
- Monica Martin Velazquez
- Principal Investigator Email
- monica.martin.velazquez@gmail.com
- Contact Person Name
- Monica Martin Velazquez
- Contact Person Email
- monica.martin.velazquez@gmail.com
- Site Name
- Hospital Universitario Virgen De La Macarena
- Department Name
- #3804: Nefrología
- Principal Investigator Name
- Mercedes Salgueira Lazo
- Principal Investigator Email
- mercedes.salgueira.sspa@juntadeandalucia.es
- Contact Person Name
- Mercedes Salgueira Lazo
- Contact Person Email
- mercedes.salgueira.sspa@juntadeandalucia.es
Netherlands
- Latest Decision Or Authorization Date
- 16-02-2026
- Number Of Sites
- 2
- Number Of Participants
- 4
Sites
- Site Name
- Leids Universitair Medisch Centrum (LUMC)
- Department Name
- 3701: Nephrology
- Principal Investigator Name
- Aiko De Vries
- Principal Investigator Email
- a.p.j.de_vries@lumc.nl
- Contact Person Name
- Aiko De Vries
- Contact Person Email
- a.p.j.de_vries@lumc.nl
- Site Name
- Radboud universitair medisch centrum / RADBOUDUMC
- Department Name
- 3702: Paediatrics
- Principal Investigator Name
- Nicole van de Kar
- Principal Investigator Email
- nicole.vandekar@radboudumc.nl
- Contact Person Name
- Nicole van de Kar
- Contact Person Email
- nicole.vandekar@radboudumc.nl
Sponsor
Primary sponsor
- Full Name
- Novartis Pharma AG
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Switzerland
Contract research organisations
- Name
- Parexel International (IRL) Limited
- Responsibilities
- Regulatory and trial support (duty code 12; clinical trial support)
- Name
- Icon Clinical Research Limited
- Responsibilities
- DMC support and clinical trial management support
- Name
- Syneos Health Inc.
- Responsibilities
- Clinical trial support (operational duties)
- Name
- IQVIA Limited
- Responsibilities
- Operational support including cardiac monitoring devices and other trial services
Third parties
- {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"Imaging Assessment","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"RWS Life Sciences Inc.","duties_or_roles":"ePRO Translations","organisation_type":"Pharmaceutical company"}
- {"country":"Greece","full_name":"IQVIA RDS Hellas Single Member S.A.","duties_or_roles":"unspecified (product role code 1 listed)","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Q Squared Solutions Limited","duties_or_roles":"unspecified (product role code 4 listed)","organisation_type":"Non-Pharmaceutical company"}
- {"country":"France","full_name":"Kayentis","duties_or_roles":"Providing materials for ePROs to sites, ePRO data transfer","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient Recruitment & Retention (Patient travel and accommodation)","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Myonex LLC","duties_or_roles":"Patient Recruitment & Retention Materials (Cooler Bags & Tote Bags)","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United Kingdom","full_name":"Adelphi Values Limited","duties_or_roles":"CCI","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"DMC support and other trial support","organisation_type":"Pharmaceutical company"}
- {"country":"Netherlands","full_name":"Eurofins Central Laboratory B.V.","duties_or_roles":"Central laboratory / investigator services","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"Data capture / Rave/ EDC (product role codes 6 and 7)","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Jumo Health USA Inc.","duties_or_roles":"Patient education and engagement","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"Clinical trial support (product role code 1)","organisation_type":"Pharmaceutical company"}
- {"country":"India","full_name":"Veeda Clinical Research Limited","duties_or_roles":"Local clinical research support","organisation_type":"Pharmaceutical company"}
- {"country":"France","full_name":"SGS France","duties_or_roles":"Local support / services","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"Regulatory / trial support (code 12 duties listed)","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Multiple trial support roles including cardiac monitoring devices and other services","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Publicis Healthcare Communications Group Limited","duties_or_roles":"Patient Recruitment & Retention Material (Participant Safety Card, Participant Workbook & Study Logo)","organisation_type":"Non-Pharmaceutical company"}
Investigational products
- Investigational Product Name
- IPTACOPAN
- Active Substance
- IPTACOPAN
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- ORAL
- Authorisation Status
- euMpNumber PRD10338043 (product record present in submission)
- Orphan Designation
- Yes
- Maximum Dose
- 400 mg
- Investigational Product Name
- IPTACOPAN HYDROCHLORIDE
- Active Substance
- IPTACOPAN HYDROCHLORIDE
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- ORAL
- Authorisation Status
- euMpNumber PRD5330958 (product record present in submission)
- Orphan Designation
- Yes
- Maximum Dose
- 400 mg
- Investigational Product Name
- Placebo 0 mg hard gelatin capsule size 1 (placebo to LNP023 100 mg)
- Modality
- Other
- Starting Dose
- 0 mg (placebo)
- Investigational Product Name
- Placebo 0 mg hard gelatin capsule size 0 (placebo to LNP023 200 mg)
- Modality
- Other
- Starting Dose
- 0 mg (placebo)
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