Clinical trial • Phase II • Immunology|Rare Disease
IPTACOPAN for ANCA-associated vasculitis|Granulomatosis with polyangiitis (GPA)|Microscopic polyangiitis (MPA)
Phase II trial of IPTACOPAN for ANCA-associated vasculitis|Granulomatosis with polyangiitis (GPA)|Microscopic polyangiitis (MPA).
Overview
- Trial Therapeutic Area
- Immunology|Rare Disease
- Trial Disease
- ANCA-associated vasculitis|Granulomatosis with polyangiitis (GPA)|Microscopic polyangiitis (MPA)
- Trial Stage
- Phase II
- Drug Modality
- Small molecule
Key dates
- Initial CTIS Submission Date
- 07-06-2024
- First CTIS Authorization Date
- 17-09-2024
Trial design
Randomised, placebo 0 mg hard gelatin capsule size 0 (placebo to lnp023); standard of care (soc) as per investigator (e.g. rituximab and glucocorticoids as indicated).-controlled Phase II trial in Czechia, Hungary, Netherlands and others.
- Randomised
- Yes
- Comparator
- Placebo 0 mg hard gelatin capsule size 0 (placebo to LNP023); Standard of Care (SOC) as per investigator (e.g. rituximab and glucocorticoids as indicated).
- Target Sample Size
- 34
- Trial Duration For Participant
- 336
Eligibility
Recruits 34 Vulnerable populations selected. "Signed informed consent obtained prior to participation in the study." All participants are adults (≥18) and must provide signed informed consent. Country-specific subject information and consent forms are provided..
- Vulnerable Population
- Vulnerable populations selected. "Signed informed consent obtained prior to participation in the study." All participants are adults (≥18) and must provide signed informed consent. Country-specific subject information and consent forms are provided.
Inclusion criteria
- {"criterion_text":"- Signed informed consent obtained prior to participation in the study."}
- {"criterion_text":"- Newly diagnosed or relapsed GPA and MPA (according to the 2022 ACR/EULAR classification criteria for GPA and MPA) requiring treatment with RTX and GC as per investigator's judgement."}
- {"criterion_text":"- BVAS assessment with ≥1 major item, or ≥3 minor items, or ≥2 renal items at Screening."}
- {"criterion_text":"- Positive antibody test for anti-proteinase 3 (PR3) or anti-myeloperoxidase (MPO) antibodies at Screening or with history of documented evidence of a positive antibody test."}
- {"criterion_text":"- Male or female subjects ≥18 years at Screening."}
Exclusion criteria
- {"criterion_text":"- Other systemic disease which constitutes the primary illness, including but not limited to: eosinophilic granulomatosis with polyangiitis (EGPA), moderate to severe systemic lupus erythematosus, IgA vasculitis (Purpura Schönlein-Henoch), rheumatoid vasculitis, Sjögren's syndrome, anti-glomerular basement membrane (GBM) disease, cryoglobulinemic vasculitis, autoimmune hemolytic anemia, autoimmune lymphoproliferative syndrome or mixed connective tissue disease."}
- {"criterion_text":"- Alveolar hemorrhage requiring invasive pulmonary ventilation support at Screening."}
- {"criterion_text":"- Severe kidney disease defined as estimated glomerular filtration rate <15 mL/min/1.73m2, or kidney failure defined as receiving renal replacement therapy such as hemo(dia)filtration, hemo-/peritoneal dialysis, or having received a kidney transplant."}
- {"criterion_text":"- Received plasma exchange/-pheresis within 12 weeks prior to Screening."}
- {"criterion_text":"- Received any of the following immunosuppressive, cell depleting or biological therapy (any other immunosuppressive, cell depleting, or biological therapy not listed here must be discussed with the Sponsor): • Received RTX or other B-cell depleting agent within 16 weeks prior to Screening. • Received any of the following biological or alkylating immunosuppressive medications within 12 weeks before screening, including but not limited to: • cyclophosphamide (CYC) • complement inhibitor (such as eculizumab, ravulizumab, avacopan) • anti-tumor necrosis factor • abatacept • tocilizumab • intravenous immunoglobulins • Received any of the following cell depleting agents within 24 weeks before Screening, including but not limited to: • alemtuzumab • antithymocyte globulin • Received azathioprine (AZA), methotrexate (MTX), or mycophenolate (MMF), or any other immunosuppressants with similar half-life within 1 week prior to Day 1. • Received leflunomide (LEF) within 6 weeks prior to Day 1."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Sustained remission through Week 48 defined as complete remission at Week 24 without major relapse up to Week 48.","definition_or_measurement_approach":"Defined as complete remission at Week 24 without major relapse up to Week 48 (sustained remission through Week 48)."}
Secondary endpoints
- {"endpoint_text":"- Time to reach BVAS = 0","definition_or_measurement_approach":"Time from baseline to first occurrence of BVAS = 0."}
- {"endpoint_text":"- Time to major relapse through Week 48","definition_or_measurement_approach":"Time from baseline to a major relapse event up to Week 48."}
- {"endpoint_text":"- Estimated glomerular filtration rate (eGFR) using the CKD-EI formula, urinary protein excretion and hematuria over 48 weeks.","definition_or_measurement_approach":"Renal function assessed by eGFR (CKD-EPI formula), measurement of urinary protein excretion and presence/degree of hematuria over 48 weeks."}
Recruitment
- Planned Sample Size
- 34
- Recruitment Window Months
- 33
- Consent Approach
- Signed informed consent obtained prior to participation in the study. Participants are adults (≥18) and provide signed informed consent. Subject information and informed consent forms (L1_ICF Main ICF - Adult) and related documents are available in multiple country/language versions (examples in the file list: English, German, French, Hungarian, Dutch, Spanish, Czech) and country-specific ICFs.
Geography
- Total Number Of Sites
- 30
- Total Number Of Participants
- 44
Czechia
- Earliest CTIS Part Ii Submission Date
- 21-06-2024
- Latest Decision Or Authorization Date
- 13-01-2026
- Processing Time Days
- 571
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Vseobecna Fakultni Nemocnice V Praze
- Department Name
- #1601: Klinika nefrologie
- Contact Person Name
- Vladimir Tesar
- Contact Person Email
- vladimir.tesar@vfn.cz
Hungary
- Earliest CTIS Part Ii Submission Date
- 24-07-2024
- Latest Decision Or Authorization Date
- 15-01-2026
- Processing Time Days
- 540
- Number Of Sites
- 3
- Number Of Participants
- 4
Sites
- Site Name
- University Of Debrecen
- Department Name
- #7002: Reumatológiai Klinika
- Contact Person Name
- Szilvia Szamosi
- Contact Person Email
- szamosi.szilvia@med.unideb.hu
- Site Name
- University Of Szeged
- Department Name
- #7001: Reumatológiai és Immunológiai Klinika
- Contact Person Name
- László Kovács
- Contact Person Email
- kovacs.laszlo@med.u-szeged.hu
- Site Name
- Orszagos Mozgasszervi Intezet
- Department Name
- #7003: ORFI Campus
- Contact Person Name
- Anna Bazsó
- Contact Person Email
- bazsoanna@yahoo.com
Netherlands
- Earliest CTIS Part Ii Submission Date
- 21-06-2024
- Latest Decision Or Authorization Date
- 13-01-2026
- Processing Time Days
- 571
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Universitair Medisch Centrum Groningen
- Department Name
- #9001: Rheumatology
- Contact Person Name
- Bram Rutgers
- Contact Person Email
- a.rutgers@umcg.nl
Germany
- Earliest CTIS Part Ii Submission Date
- 30-08-2024
- Latest Decision Or Authorization Date
- 15-01-2026
- Processing Time Days
- 503
- Number Of Sites
- 6
- Number Of Participants
- 6
Sites
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- #6001: Medizinische Klinik mit Schwerpunkt Nephrologie und Internistische Intensivmedizin
- Contact Person Name
- Adrian Schreiber
- Contact Person Email
- adrian.schreiber@charite.de
- Site Name
- medius KLINIKEN gGmbH
- Department Name
- #6005: Klinik für Innere Medizin, Rheumatologie, Pneuomologie, Nephrologie und Diabetologie
- Contact Person Name
- Christian Löffler
- Contact Person Email
- c.loeffler2@medius-kliniken.de
- Site Name
- Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
- Department Name
- #6008: I. Medizinische Klinik und Poliklinik
- Contact Person Name
- Julia Weinmann-Menke
- Contact Person Email
- Julia.weinmann-menke@unimedizin-mainz.de
- Site Name
- Klinikum der Universitaet Muenchen AöR
- Department Name
- #6004: Medizinische Klinik und Poliklinik IV Nephrologisches Zentrum
- Contact Person Name
- Ulf Schönermarck
- Contact Person Email
- Ulf.schoenermarck@med.uni-muenchen.de
- Site Name
- Medical Center - University Of Freiburg
- Department Name
- #6002: Klinik für Rheumatologie und Klinische Immunologie / Department Innere Medizin
- Contact Person Name
- Stephanie Finzel
- Contact Person Email
- stephanie.finzel@uniklinik-freiburg.de
- Site Name
- Klinikum der Stadt Ludwigshafen am Rhein gGmbH
- Department Name
- #6006: Med A Klinik/Rheumatologie
- Contact Person Name
- Raoul Bergner
- Contact Person Email
- bergnerr@klilu.de
Belgium
- Earliest CTIS Part Ii Submission Date
- 23-08-2024
- Latest Decision Or Authorization Date
- 02-02-2026
- Processing Time Days
- 528
- Number Of Sites
- 2
- Number Of Participants
- 3
Sites
- Site Name
- Algemeen Ziekenhuis Delta
- Department Name
- #2002: Nephrology
- Contact Person Name
- Bart Maes
- Contact Person Email
- bart.maes@azdelta.be
- Site Name
- UZ Leuven
- Department Name
- #2001: Internal Medicine
- Contact Person Name
- Albrecht Betrains
- Contact Person Email
- albrecht.betrains@uzleuven.be
Denmark
- Earliest CTIS Part Ii Submission Date
- 29-08-2024
- Latest Decision Or Authorization Date
- 13-01-2026
- Processing Time Days
- 502
- Number Of Sites
- 3
- Number Of Participants
- 5
Sites
- Site Name
- Aarhus Universitetshospital
- Department Name
- #4003: Nyremedicinsk Forskning, Nyresygdomme
- Contact Person Name
- Per Ramløv Ivarsen
- Contact Person Email
- perivars@rm.dk
- Site Name
- Rigshospitalet
- Department Name
- #4001: Afdeling for Nyresygdomme
- Contact Person Name
- Morten Buus Jørgensen
- Contact Person Email
- Morten.buus.joergensen@regionh.dk
- Site Name
- Region Hovedstaden
- Department Name
- #4002: Afdeling for Nyresygdomme
- Contact Person Name
- Casper Rydahl
- Contact Person Email
- casper.rydahl.01@regionh.dk
France
- Earliest CTIS Part Ii Submission Date
- 30-07-2024
- Latest Decision Or Authorization Date
- 19-01-2026
- Processing Time Days
- 538
- Number Of Sites
- 8
- Number Of Participants
- 11
Sites
- Site Name
- Centre Hospitalier Universitaire De Nantes
- Department Name
- #5007: Service de Médecine Interne
- Contact Person Name
- Antoine NEEL
- Contact Person Email
- antoine.neel@chu-nantes.fr
- Site Name
- Centre Hospitalier Universitaire D'Angers
- Department Name
- #5002: Néphrologie
- Contact Person Name
- Jean-François AUGUSTO
- Contact Person Email
- jfaugusto@chu-angers.fr
- Site Name
- Centre Hospitalier Regional Et Universitaire De Brest
- Department Name
- #5008: Rhumatologie
- Contact Person Name
- Divi CORNEC
- Contact Person Email
- divi.cornec@chu-brest.fr
- Site Name
- Centre Hospitalier Universitaire De Dijon
- Department Name
- #5005: Département de Médecine Interne et Immunologie Clinique
- Contact Person Name
- Maxime SAMSON
- Contact Person Email
- maxime.samson@chu-dijon.fr
- Site Name
- Centre Hospitalier Regional De Marseille
- Department Name
- #5009: Néphrologie
- Contact Person Name
- Noémie JOURDE-CHICHE
- Contact Person Email
- noemie.jourde@ap-hm.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- #5004: Néphrologie
- Contact Person Name
- Alexandre KARRAS
- Contact Person Email
- alexandre.karras@aphp.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- #5001: Service de Médecine interne
- Contact Person Name
- Benjamin TERRIER
- Contact Person Email
- benjamin.terrier@aphp.fr
- Site Name
- Centre Hospitalier Universitaire De Toulouse
- Department Name
- #5006: Service de Médecine interne
- Contact Person Name
- Grégory PUGNET
- Contact Person Email
- pugnet.g@chu-toulouse.fr
Spain
- Earliest CTIS Part Ii Submission Date
- 21-08-2024
- Latest Decision Or Authorization Date
- 15-01-2026
- Processing Time Days
- 512
- Number Of Sites
- 3
- Number Of Participants
- 5
Sites
- Site Name
- Clinica Universidad De Navarra
- Department Name
- #1102: Servicio de Reumatología
- Contact Person Name
- Miguel Angel Suarez Santisteban
- Contact Person Email
- miguelangel.suarez@salud-juntaex.es
- Site Name
- Hospital Universitario La Paz
- Department Name
- #1101: Servicio de Reumatología
- Contact Person Name
- Eugenio De Miguel Mendieta
- Contact Person Email
- eugenio.miguel@salud.madrid.org
- Site Name
- Hospital Virgen Del Puerto
- Department Name
- #1103: Servicio de Nefrología
- Contact Person Name
- Enrique Ornilla Laraudogoitia
- Contact Person Email
- eornilla@unav.es
Austria
- Earliest CTIS Part Ii Submission Date
- 24-07-2024
- Latest Decision Or Authorization Date
- 16-01-2026
- Processing Time Days
- 541
- Number Of Sites
- 3
- Number Of Participants
- 4
Sites
- Site Name
- Medical University Of Vienna
- Department Name
- #1001
- Contact Person Name
- Clemens Scheinecker
- Contact Person Email
- clemens.scheinecker@meduniwien.ac.at
- Site Name
- Medical University Of Graz
- Department Name
- #1002
- Contact Person Name
- Jens Thiel
- Contact Person Email
- jens.thiel@medunigraz.at
- Site Name
- Medizinische Universitaet Innsbruck
- Department Name
- #1003: Neurologie
- Contact Person Name
- Andreas Kronbichler
- Contact Person Email
- andreas.kronbichler@i-med.ac.at
Sponsor
Primary sponsor
- Full Name
- Novartis Pharma AG
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Switzerland
Contract research organisations
- Name
- 4g Clinical LLC
- Responsibilities
- code 3
- Name
- Q Squared Solutions LLC
- Responsibilities
- code 4
- Name
- Syneos Health Inc.
- Responsibilities
- code 1
- Name
- Icon Clinical Research Limited
- Responsibilities
- code 1
- Name
- IQVIA Limited
- Responsibilities
- code 1
- Name
- Parexel International (IRL) Limited
- Responsibilities
- code 12
- Name
- Veeda Clinical Research Limited
- Responsibilities
- code 15 (PK analysis); code 4
- Name
- Mag. Andreas Raffeiner GmbH
- Responsibilities
- code 8
Third parties
- {"country":"United States","full_name":"4g Clinical LLC","duties_or_roles":"code 3","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Q Squared Solutions LLC","duties_or_roles":"code 4","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"code 1","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"code 1","organisation_type":"Pharmaceutical company"}
- {"country":"Austria","full_name":"Mag. Andreas Raffeiner GmbH","duties_or_roles":"code 8","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"code 1","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"code 12","organisation_type":"Pharmaceutical company"}
- {"country":"India","full_name":"Veeda Clinical Research Limited","duties_or_roles":"code 15 (PK analysis); code 4","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- IPTACOPAN
- Active Substance
- IPTACOPAN
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- ORAL
- Maximum Dose
- 400 mg/day
- Investigational Product Name
- Placebo 0 mg hard gelatin capsule size 0, (placebo to LNP023)
- Modality
- Other
- Routes Of Administration
- Oral (hard gelatin capsule)
- Route
- ORAL
- Starting Dose
- 0 mg
- Combination Treatment
- Yes
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