Clinical trial • Phase III • Immunology|Rare Disease
iptacopan for Paroxysmal nocturnal haemoglobinuria (PNH)
Phase III trial of iptacopan for Paroxysmal nocturnal haemoglobinuria (PNH). open-label, none/not specified-controlled. 107 participants.
Overview
- Trial Therapeutic Area
- Immunology|Rare Disease
- Trial Disease
- Paroxysmal nocturnal haemoglobinuria (PNH)
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 19-04-2024
- First CTIS Authorization Date
- 05-06-2024
Trial design
open-label, none/not specified-controlled Phase III trial across 25 sites in Germany, Spain, Czechia and others.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 107
- Trial Duration For Participant
- 1095
Eligibility
Recruits 107 Vulnerable population selected (isVulnerablePopulationSelected=true). Informed consent is required from adult participants; child assent documentation is present in the dossier (document title: 'L1_ICF - Child Assent_1_FR_French_NonRed'). Additional subject information/ICF documents exist for adults and for follow-up of pregnant participants in multiple languages..
- Pregnancy Exclusion
- Female participants who are pregnant or breastfeeding, or intending to conceive during the course of the study
- Vulnerable Population
- Vulnerable population selected (isVulnerablePopulationSelected=true). Informed consent is required from adult participants; child assent documentation is present in the dossier (document title: 'L1_ICF - Child Assent_1_FR_French_NonRed'). Additional subject information/ICF documents exist for adults and for follow-up of pregnant participants in multiple languages.
Inclusion criteria
- {"criterion_text":"-Male and female participants ≥ 18 years of age with a diagnosis of PNH who have completed the treatment extension period (without tapering down) of Phase II iptacopan studies (CLNP023X2204, CLNP023X2201), Period 4 of CLFGX2201 or completed any Phase III (eg. CLNP023C12302, CLNP023C12301, CLNP023C12303) clinical study at the time point of enrollment visit in this roll over extension study"}
- {"criterion_text":"-Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections should be up to date (i.e., any boosters required administered according to local regulations)."}
- {"criterion_text":"-Per investigator’s clinical judgement, the patient may benefit from continued treatment with iptacopan and has been clinically stable on iptacopan monotherapy for at least 3 months"}
Exclusion criteria
- {"criterion_text":"-Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data"}
- {"criterion_text":"-History of recurrent invasive infections caused by encapsulated organisms, such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae"}
- {"criterion_text":"-Female participants who are pregnant or breastfeeding, or intending to conceive during the course of the study"}
- {"criterion_text":"-Women of childbearing potential, defined as all women physiologically capable of becoming pregnant from menarche until becoming post-menopausal, unless they are using effective methods of contraception during dosing of investigational drug and for 1 week after stopping investigational drug. Women are considered post-menopausal if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g., hormonal profile confirming menopause and/or age-appropriate history of vasomotor symptoms)."}
Endpoints
Primary endpoints
- {"endpoint_text":"-Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc., through End of Study visit","definition_or_measurement_approach":"Safety evaluated by monitoring adverse events and serious adverse events, safety laboratory parameters, vital signs and other standard safety assessments through the End of Study visit."}
Secondary endpoints
- {"endpoint_text":"-Response defined as maintaining sustained hemoglobin levels ≥ 12 g/dL in the absence of transfusions evaluated over yearly follow up intervals","definition_or_measurement_approach":"Response is defined as sustained hemoglobin ≥ 12 g/dL without red blood cell transfusion, evaluated at yearly follow-up intervals."}
- {"endpoint_text":"-Absence of administration of packed-red blood cell transfusions evaluated over yearly follow up intervals","definition_or_measurement_approach":"Transfusion avoidance measured as absence of packed red blood cell transfusion administration, evaluated over yearly follow-up intervals."}
- {"endpoint_text":"-Occurrences of breakthrough hemolysis and of Major Adverse Vascular Events","definition_or_measurement_approach":"Occurrences of breakthrough hemolysis (BTH) and Major Adverse Vascular Events (MAVE) assessed/reported during follow-up."}
- {"endpoint_text":"-MAVE occurring evaluated over yearly follow up intervals","definition_or_measurement_approach":"Major Adverse Vascular Events assessed and reported over yearly follow-up intervals."}
Recruitment
- Planned Sample Size
- 107
- Recruitment Window Months
- 74
- Consent Approach
- Informed consent obtained from adult participants via main ICF documents. Multiple language ICFs available (English, German, French, Spanish, Italian, Dutch, Czech, Lithuanian). A child assent form is present in the dossier (L1_ICF - Child Assent_1_FR_French_NonRed). Specific country ICF/recruitment documents are provided per Member State.
Geography
- Total Number Of Sites
- 25
- Total Number Of Participants
- 86
Germany
- Earliest CTIS Part Ii Submission Date
- 14-05-2024
- Latest Decision Or Authorization Date
- 14-04-2025
- Number Of Sites
- 6
- Number Of Participants
- 21
Sites
- Site Name
- Institut fuer Klinische Transfusionsmedizin und Immungenetik Ulm gGmbH
- Department Name
- 4203: Transfusionsmedizin und Immungenetik
- Contact Person Name
- Hubert Schrezenmeier
- Contact Person Email
- h.schrezenmeier@blutspende.de
- Site Name
- ELBLANDKLINIKEN Stiftung & Co. KG
- Department Name
- 4204: Klinik für Innere Medizin II, Hämatologie, Onkologie, Gastroenterologie und Palliativmedizin
- Contact Person Name
- Joerg Schubert
- Contact Person Email
- Joerg.Schubert@elblandkliniken.de
- Site Name
- Universitaetsklinikum Aachen AöR
- Department Name
- 4201: Onkologie, Hämatologie und Stammzellentransplantation
- Contact Person Name
- Jens-Peter Panse
- Contact Person Email
- jpanse@ukaachen.de
- Site Name
- University Medical Center Hamburg-Eppendorf
- Department Name
- 4202: II. Medizinische Klinik und Poliklinik
- Contact Person Name
- Philippe Schafhausen
- Contact Person Email
- schafhausen@uke.de
- Site Name
- Universitaetsklinikum Essen AöR
- Department Name
- 4200: Hämatologie und Stammzelltrans
- Contact Person Name
- Alexander Roeth
- Contact Person Email
- alexander.roeth@uk-essen.de
- Site Name
- Technische Universitaet Dresden
- Department Name
- 4205: Medizinische Klinik und Poliklinik 1 Klinik f. Haematologie
- Contact Person Name
- Joerg Schubert
- Contact Person Email
- Joerg.Schubert@uniklinikum-dresden.de
Spain
- Earliest CTIS Part Ii Submission Date
- 14-05-2024
- Latest Decision Or Authorization Date
- 02-02-2026
- Number Of Sites
- 3
- Number Of Participants
- 4
Sites
- Site Name
- Hospital Clinic De Barcelona
- Department Name
- 4700: Hematología
- Contact Person Name
- Anna Gaya Valls
- Contact Person Email
- AGAYAV@clinic.cat
- Site Name
- Hospital Universitario Donostia
- Department Name
- 4701: Hematología
- Contact Person Name
- Ana Arroyo Rubio
- Contact Person Email
- ana.arroyorubio@osakidetza.eus
- Site Name
- Complexo Hospitalario Universitario De Santiago
- Department Name
- 4704: Hematología
- Contact Person Name
- Ana María Fernández Villar
- Contact Person Email
- ana.maria.fernandez.villar@sergas.es
Czechia
- Earliest CTIS Part Ii Submission Date
- 14-05-2024
- Latest Decision Or Authorization Date
- 23-01-2026
- Number Of Sites
- 2
- Number Of Participants
- 4
Sites
- Site Name
- Fakultni Nemocnice Ostrava
- Department Name
- 4001: Klinika hematoonkologie
- Contact Person Name
- Jaromir Gumulec
- Contact Person Email
- jaromir.gumulec@fno.cz
- Site Name
- Fakultni Nemocnice Brno
- Department Name
- 4000: Interni hematologicka a onkologicka klinika
- Contact Person Name
- Libor Cervinek
- Contact Person Email
- cervinek.libor@fnbrno.cz
Netherlands
- Earliest CTIS Part Ii Submission Date
- 14-05-2024
- Latest Decision Or Authorization Date
- 04-02-2026
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Stichting Radboud universitair medisch centrum
- Department Name
- 4600: Hematology
- Contact Person Name
- Saskia Langemeijer
- Contact Person Email
- saskia.langemeijer@radboudumc.nl
Italy
- Earliest CTIS Part Ii Submission Date
- 14-05-2024
- Latest Decision Or Authorization Date
- 20-01-2026
- Number Of Sites
- 7
- Number Of Participants
- 32
Sites
- Site Name
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
- Department Name
- 4401: U.O.C. Ematologia
- Contact Person Name
- Bruno Fattizzo
- Contact Person Email
- bruno.fattizzo@policlinico.mi.it
- Site Name
- Regione Del Veneto Azienda ULSS N 7 Pedemontana
- Department Name
- 4402: U.O.C. Oncoematologia
- Contact Person Name
- Eros Di Bona
- Contact Person Email
- eros.dibona@aulss7.veneto.it
- Site Name
- Azienda Ospedaliero-Universitaria Policlinico Umberto I
- Department Name
- 4404: U.O.C. Ematologia
- Contact Person Name
- Maurizio Martelli
- Contact Person Email
- principalinvestigatormartelli.dmtp@uniroma1.it
- Site Name
- AORN San Giuseppe Moscati Avellino
- Department Name
- 4400: U.O.C. di Ematologia e Trapianto Emopoietico
- Contact Person Name
- Camilla Frieri
- Contact Person Email
- camillafrieri@gmail.com
- Site Name
- Careggi University Hospital
- Department Name
- 4407: S.O.D. Ematologia
- Contact Person Name
- Barbara Scappini
- Contact Person Email
- scappinib@aou-careggi.toscana.it
- Site Name
- Azienda Sanitaria Territoriale Di Ascoli Piceno
- Department Name
- 4405: U.O.C. Ematologia
- Contact Person Name
- Piero Galieni
- Contact Person Email
- piero.galieni@sanita.marche.it
- Site Name
- Careggi University Hospital (duplicate listing in dataset context)
- Department Name
- 4407: S.O.D. Ematologia
Lithuania
- Earliest CTIS Part Ii Submission Date
- 14-05-2024
- Latest Decision Or Authorization Date
- 26-01-2026
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Vilniaus Universiteto Ligonine Santaros Klinikos Vsi
- Department Name
- 4500: Hematology, Oncology and Transfusion medicine center
- Contact Person Name
- Andrius Degulys
- Contact Person Email
- Andrius.degulys@santa.lt
France
- Earliest CTIS Part Ii Submission Date
- 14-05-2024
- Latest Decision Or Authorization Date
- 26-01-2026
- Number Of Sites
- 5
- Number Of Participants
- 20
Sites
- Site Name
- Hopital Saint Louis
- Department Name
- 4101:Hematology -Transplant Department
- Contact Person Name
- Regis PEFFAULT de LATOUR
- Contact Person Email
- regis.peffaultdelatour@aphp.fr
- Site Name
- Centre Hospitalier Universitaire De Nantes
- Department Name
- 4110:Service d’Hématologie clinique
- Contact Person Name
- Nicolas BLIN
- Contact Person Email
- Nicolas.BLIN@chu-nantes.fr
- Site Name
- Institut Universitaire Du Cancer Toulouse-Oncopole
- Department Name
- 4105:Service d’Hématologie
- Contact Person Name
- Suzanne Tavitian
- Contact Person Email
- tavitian.suzanne@iuct-oncopole.fr
- Site Name
- Centre Hospitalier Universitaire De Nice
- Department Name
- 4108:Hematology Clinical Department
- Contact Person Name
- Michael LOSCHI
- Contact Person Email
- loschi.m@chu-nice.fr
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- 4102:Unité d’Immunologie clinique
- Contact Person Name
- Louis TERRIOU
- Contact Person Email
- Louis.TERRIOU@CHRU-LILLE.FR
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
- sponsorDuties code: 12
- Name
- Syneos Health Clinical Spain S.L.
- Responsibilities
- sponsorDuties code: 1
- Name
- IQVIA Limited
- Responsibilities
- sponsorDuties code: 3
- Name
- IQVIA RDS Spain S.L.
- Responsibilities
- sponsorDuties code: 1
- Name
- Rps Research Iberica S.L.
- Responsibilities
- sponsorDuties code: 1
Third parties
- {"country":"Spain","full_name":"Rps Research Iberica S.L.","duties_or_roles":"sponsorDuties code: 1","organisation_type":"Pharmaceutical company"}
- {"country":"Spain","full_name":"Syneos Health Clinical Spain S.L.","duties_or_roles":"sponsorDuties code: 1","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Publicis Healthcare Communications Group Limited","duties_or_roles":"Patient Safety Card","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"sponsorDuties code: 12","organisation_type":"Pharmaceutical company"}
- {"country":"France","full_name":"Kayentis","duties_or_roles":"Patients Reported Outcomes Assessments PRO management, data collection via tablet.","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"sponsorDuties code: 3","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Q Squared Solutions Limited","duties_or_roles":"Central laboratory","organisation_type":"Non-Pharmaceutical company"}
- {"country":"France","full_name":"SGS France","duties_or_roles":"Biomarker- SGS Cephac Europe, Analysis blood biomarkers","organisation_type":"Pharmaceutical company"}
- {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"PNH testing","organisation_type":"Pharmaceutical company"}
- {"country":"Italy","full_name":"Opis S.r.l.","duties_or_roles":"TMF archive","organisation_type":"Pharmaceutical company"}
- {"country":"Spain","full_name":"IQVIA RDS Spain S.L.","duties_or_roles":"sponsorDuties code: 1","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- IPTACOPAN
- Active Substance
- iptacopan
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- ORAL
- Authorisation Status
- Authorised
- Orphan Designation
- Yes
- Maximum Dose
- 400 mg
- Investigational Product Name
- LNP023
- Active Substance
- iptacopan
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- ORAL
- Authorisation Status
- Authorised
- Orphan Designation
- Yes
- Maximum Dose
- 400 mg
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