Clinical trial • Phase III • Neurology

IPTACOPAN for Generalized myasthenia gravis

Phase III trial of IPTACOPAN for Generalized myasthenia gravis.

Overview

Trial Therapeutic Area
Neurology
Trial Disease
Generalized myasthenia gravis
Trial Stage
Phase III
Drug Modality
Small molecule

Key dates

Initial CTIS Submission Date
29-02-2024
First CTIS Authorization Date
26-06-2024

Trial design

Randomised, open-label, placebo (placebo 0 mg hard gelatin capsule size 0, matching placebo) compared to iptacopan (iptacopan, hard gelatin capsules, oral). dose and schedule not specified in the provided record.-controlled Phase III trial in Portugal, France, Netherlands and others.

Randomised
Yes
Open Label
Yes
Comparator
Placebo (Placebo 0 mg hard gelatin capsule size 0, matching placebo) compared to IPTACOPAN (IPTACOPAN, hard gelatin capsules, oral). Dose and schedule not specified in the provided record.
Target Sample Size
81

Eligibility

Recruits 81 Vulnerable population flag selected. Consent handled via subject information and informed consent forms; specific ICFs for pregnant participants and newborn follow-up are provided (documents titled e.g. "Pregnant-Participant-ICF", "Newborn-FUp-ICF"), and optional genetic research consent is available. All main informed consent forms (Main ICF, OG-ICF, pregnancy ICF, optional genetic ICF) are provided for participating adults; languages and country-specific ICFs are available in the submitted document set..

Pregnancy Exclusion
Female participants who are pregnant or lactating, or are intending to become pregnant
Vulnerable Population
Vulnerable population flag selected. Consent handled via subject information and informed consent forms; specific ICFs for pregnant participants and newborn follow-up are provided (documents titled e.g. "Pregnant-Participant-ICF", "Newborn-FUp-ICF"), and optional genetic research consent is available. All main informed consent forms (Main ICF, OG-ICF, pregnancy ICF, optional genetic ICF) are provided for participating adults; languages and country-specific ICFs are available in the submitted document set.

Inclusion criteria

  • {"criterion_text":"- Adult patients with gMG (age 18-85 years) at screening\n- Positive serology testing for AChR+ antibody at screening\n- Myasthenia Gravis Foundation of America (MGFA) Class II-IV gMG at screening and likely not in need of a respirator for the duration of the study, as judged by the Investigator\n- The confirmation of the diagnosis of gMG should be documented and supported by ≥1 of the following 3 tests: • History of abnormal neuromuscular transmission demonstrated by single-fiber electromyography or repetitive nerve stimulation. • History of positive test with short-acting acetylcholinesterase inhibitors (e.g. neostigmine or edrophonium chloride) • Patient has demonstrated improvement in MG signs on oral acetylcholinesterase inhibitors as assessed by the treating physician.\n- Baseline MG-ADL score ≥6, with ≥50% of the total score due to non-ocular symptoms\n- Participants receiving at least one of the following treatments for gMG for ≥ 6 months prior to baseline • One or more NSISTs; or • plasmapheresis, plasma exchange, or intravenous immunoglobulin (at least quarterly) to control symptoms despite treatment with steroids and NSISTs; or • an approved FcRN antagonist; or • rituximab; or • other approved gMG disease modifying therapies excluding complement inhibitors\n- Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection is required prior to the start of study treatment. If the participant has not been previously vaccinated, or if a booster was required, the vaccine should be given according to local guidelines at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post-vaccination, prophylactic antibiotic treatment should be initiated at the start of the study treatment and continued until at least 2 weeks after vaccination or booster was completed.. Note: for US sites participating in Study CLNP023Q12301, the completion of the meningococcal vaccination or booster is required for patients with gMG prior to initiating study treatment, irrespective of prophylactic antibiotic use.\n- If not previously vaccinated, vaccination against Haemophilus influenzae infections should be given, if available and according to local guidelines, at least 2 weeks prior to first study drug administration."}

Exclusion criteria

  • {"criterion_text":"- Have been treated with intravenous immunoglobulin (IVIG)/plasma exchange (PLEX) in the past month, with rituximab in the past 6 months, eculizumab in the past 2 months, ravulizumab or other complement inhibitors in the past 3 months, efgartigimod or other anti-FcRn therapies in the past 3 months, or had a thymectomy in the past 6 months or a planned thymectomy during the trial period.\n- Participants with clinically significant active or chronic uncontrolled bacterial, viral, or fungal infection at screening, including patients who test positive for an active viral infection at screening with: Active Hepatitis B Virus (HBV); Active Hepatitis C Virus (HCV); Human Immunodeficiency Virus (HIV) positive serology associated with an Acquired Immune Deficiency Syndrome (AIDS)-defining condition or with a cluster of differentiation 4 (CD4) count ≤200 cells/mm3\n- Female participants who are pregnant or lactating, or are intending to become pregnant\n- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of study treatment and an additional one week following cessation of study treatment.\n- Active systemic bacterial, viral (including COVID-19) or fungal infection or any major episode of infection that required hospitalization or injectable antimicrobial therapy within 14 days prior to study drug administration\n- History of recurrent invasive infections caused by encapsulated organisms, e.g., N. meningitidis and S. pneumoniae\n- Presence of fever ≥ 38 °C (100.4 °F) within 7 days prior to study drug administration"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Change from baseline to Month 6 in Myasthenia Gravis Activity of Daily Living (MG-ADL) total score","definition_or_measurement_approach":"Change from baseline to Month 6 (Day 180) in the total score of the Myasthenia Gravis Activity of Daily Living (MG-ADL) scale."}

Secondary endpoints

  • {"endpoint_text":"- Change from baseline to Month 6 in Quantitative MG (QMG) total score","definition_or_measurement_approach":"Change from baseline to Month 6 in QMG total score."}
  • {"endpoint_text":"- Proportion of participants with ≥5 points reduction from baseline to Month 6 of QMG total score without rescue medication and/or strongly confounding prohibited medication","definition_or_measurement_approach":"Proportion achieving ≥5-point reduction from baseline to Month 6 in QMG total score, assessed without rescue medication and/or strongly confounding prohibited medication."}
  • {"endpoint_text":"- Proportion of participants with ≥3 points reduction from baseline to Month 6 of MG-ADL total score without rescue medication and/or strongly confounding prohibited medication","definition_or_measurement_approach":"Proportion achieving ≥3-point reduction from baseline to Month 6 in MG-ADL total score, assessed without rescue medication and/or strongly confounding prohibited medication."}
  • {"endpoint_text":"- Change from baseline to Month 6 in Myasthenia Gravis Composite (MGC) total score","definition_or_measurement_approach":"Change from baseline to Month 6 in MGC total score."}
  • {"endpoint_text":"- Change from baseline to Month 6 in revised MG Quality of Life Questionnaire (MG-QOL15r) survey score","definition_or_measurement_approach":"Change from baseline to Month 6 in MG-QOL15r score."}
  • {"endpoint_text":"- Incidence of adverse events and changes in clinical laboratory values, vital signs, and electrocardiograms, Columbia Suicide Severity Rating","definition_or_measurement_approach":"Incidence and type of adverse events; changes in clinical laboratory values, vital signs and ECGs; and assessment using the Columbia Suicide Severity Rating Scale."}
  • {"endpoint_text":"- Proportion of time during which participants showed a reduction of ≥ 2 points in MG-ADL total score that was maintained up to Month 6","definition_or_measurement_approach":"Proportion of time that a participant maintained a ≥2-point reduction in MG-ADL total score up to Month 6."}
  • {"endpoint_text":"- Proportion of early MG-ADL responders during treatment (early responders with first MG-ADL improvement from baseline of ≥2 points occurring by week 4）","definition_or_measurement_approach":"Proportion of participants with earliest MG-ADL improvement ≥2 points from baseline occurring by Week 4."}
  • {"endpoint_text":"- Change from baseline to Month 6 in EuroQol-5 Dimensions-5 Level (EQ-5D-5L)","definition_or_measurement_approach":"Change from baseline to Month 6 in EQ-5D-5L."}
  • {"endpoint_text":"- Change from baseline in MG-ADL total score","definition_or_measurement_approach":"Change from baseline in MG-ADL total score (various timepoints including Month 6)."}
  • {"endpoint_text":"- Proportion of participants achieving a reduction in oral corticosteroids (OCS) dose compared to Core Part that was maintained up to end of Extension Part","definition_or_measurement_approach":"Proportion achieving a reduction in oral corticosteroid dose relative to the Core Part that was maintained through the end of the Extension Part."}
  • {"endpoint_text":"- Incidence of adverse events and changes in clinical laboratory values, vital signs, and electrocardiograms, Columbia Suicide Severity Rating","definition_or_measurement_approach":"As above (safety assessments): incidence of AEs, laboratory, vitals, ECGs, and C-SSRS."}
  • {"endpoint_text":"- Proportion of participants achieving MSE at Month 6, defined as MG-ADL score of 0 or 1 at Month 6 without rescue therapy and/or strongly confounding prohibited medication","definition_or_measurement_approach":"Proportion achieving Minimal Symptom Expression (MG-ADL = 0 or 1) at Month 6 without rescue therapy and/or strongly confounding prohibited medication."}
  • {"endpoint_text":"- Proportion of participants with reduction of ≥ 3 points from baseline to Month 6 in MGC total score","definition_or_measurement_approach":"Proportion with ≥3-point reduction from baseline to Month 6 in MGC total score."}

Recruitment

Planned Sample Size
81
Recruitment Window Months
92
Consent Approach
Informed consent is obtained using subject information and informed consent forms (Main ICFs) provided for each country/language. Additional ICFs include pregnancy-specific ICFs, newborn follow-up ICFs, OG-ICF and optional genetic research ICFs. ICFs are available in multiple languages as submitted (examples in Portuguese, French, Dutch, Polish, Spanish, Greek, Italian, German, Danish, English). Consent is provided by the adult participant (study population is adults 18–85).

Methods

  • Country-specific recruitment arrangements documents (e.g. documents titled 'K1_CLNP023Q12301_Recruitment-Arrangements_*' and 'K1_CLNP023Q12301_Addendum-to-Recruitment-Arrangements_DE_Public')
  • GP referral / GP-letter documents (titles include 'K2_CLNP023Q12301_GP_Referral_Letter_PT_Portuguese_Public' and 'K2_CLNP023Q12301_GP-Referral_IT_Italian_Public')
  • Patient-facing information: Fact sheets and Welcome Booklet documents (titles include 'Myasthenia Gravis Fact Sheet', 'Welcome Booklet' in multiple languages)
  • Dr-to-Patient letter (Denmark: 'K2_CLNP023Q12301_Dr-to-Patient-letter_DNK_Danish_Public')
  • Patient diaries and patient-facing PRO documents (e.g. 'K2_CLNP023Q12301_Diary_FR_French_Public', PRO patient-facing documents in protocol)

Geography

Total Number Of Sites
55
Total Number Of Participants
65

Portugal

Earliest CTIS Part Ii Submission Date
05-06-2024
Latest Decision Or Authorization Date
26-06-2024
Processing Time Days
21
Number Of Sites
4
Number Of Participants
6

Sites

Site Name
Centro Hospitalar De Lisboa Ocidental E.P.E.
Department Name
Department of Neurology
Principal Investigator Name
Marco Fernandes
Principal Investigator Email
marcoa.fernandes91@gmail.com
Contact Person Name
Marco Fernandes
Contact Person Email
marcoa.fernandes91@gmail.com
Site Name
Unidade Local De Saude De Santa Maria E.P.E.
Department Name
Department of Neurology
Principal Investigator Name
Mamede de Carvalho
Principal Investigator Email
mamedealves@medicina.ulisboa.pt
Contact Person Name
Mamede de Carvalho
Site Name
Centro Hospitalar De Vila Nova De Gaia/Espinho E.P.E.
Department Name
Department of Neurology
Principal Investigator Name
Henrique Moniz Costa
Principal Investigator Email
henrique.costa@ulsge.min-saude.pt
Contact Person Name
Henrique Moniz Costa
Site Name
Centro Hospitalar Universitario De Santo Antonio E.P.E.
Department Name
Department of Neurology
Principal Investigator Name
Ernestina Santos
Principal Investigator Email
ernestina.santos@gmail.com
Contact Person Name
Ernestina Santos
Contact Person Email
ernestina.santos@gmail.com

France

Earliest CTIS Part Ii Submission Date
15-09-2025
Latest Decision Or Authorization Date
19-09-2025
Processing Time Days
4
Number Of Sites
5
Number Of Participants
3

Sites

Site Name
Centre Hospitalier Et Universitaire De Limoges
Department Name
Dupuytren 1
Principal Investigator Name
Laurent Magy
Principal Investigator Email
Laurent.magy@unilim.fr
Contact Person Name
Laurent Magy
Contact Person Email
Laurent.magy@unilim.fr
Site Name
Centre Hospitalier Universitaire De Nice
Department Name
Hôpital Pasteur 2
Principal Investigator Name
Saskia Bresch
Principal Investigator Email
bresch.s@chu-nice.fr
Contact Person Name
Saskia Bresch
Contact Person Email
bresch.s@chu-nice.fr
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Hopital Raymond Poincaré
Principal Investigator Name
Djilali Annane
Principal Investigator Email
djilali.annane@aphp.fr
Contact Person Name
Djilali Annane
Contact Person Email
djilali.annane@aphp.fr
Site Name
Fondation A De Rothschild
Principal Investigator Name
Antoine Gueguen
Principal Investigator Email
agueguen@for.paris
Contact Person Name
Antoine Gueguen
Contact Person Email
agueguen@for.paris
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Institut de Myologie
Principal Investigator Name
Anthony Behin
Principal Investigator Email
Anthony.behin@aphp.fr
Contact Person Name
Anthony Behin
Contact Person Email
Anthony.behin@aphp.fr

Netherlands

Earliest CTIS Part Ii Submission Date
03-07-2025
Latest Decision Or Authorization Date
10-07-2025
Processing Time Days
7
Number Of Sites
1
Number Of Participants
1

Sites

Site Name
Amsterdam UMC Stichting
Department Name
Neurology
Principal Investigator Name
Anneke van der Kooi
Principal Investigator Email
a.j.kooi@amsterdamumc.nl
Contact Person Name
Anneke van der Kooi
Contact Person Email
a.j.kooi@amsterdamumc.nl

Poland

Earliest CTIS Part Ii Submission Date
21-03-2024
Latest Decision Or Authorization Date
01-07-2024
Processing Time Days
102
Number Of Sites
11
Number Of Participants
17

Sites

Site Name
Krakowska Akademia Neurologii Sp. z o.o.
Principal Investigator Name
Andrzej Szczudlik
Principal Investigator Email
andrzej.szczudlik@neurologia.org.pl
Contact Person Name
Andrzej Szczudlik
Site Name
Centrum Medyczne Neuroprotect
Principal Investigator Name
Mariusz Grudniak
Principal Investigator Email
mariusz.grudniak@neuroprotect.pl
Contact Person Name
Mariusz Grudniak
Site Name
Neurologia Śląska Centrum Medyczne
Principal Investigator Name
Marek Śmiłowski
Principal Investigator Email
marek.smilowski@neurologiaslaska.pl
Contact Person Name
Marek Śmiłowski
Site Name
Clinical Research Center Sp. z o.o. Medic-R sp.k.
Principal Investigator Name
Artur Drużdż
Principal Investigator Email
artur.druzdz@cr-center.pl
Contact Person Name
Artur Drużdż
Contact Person Email
artur.druzdz@cr-center.pl
Site Name
Neurocor Banaszkiewicz Tomaszewski Lekarze sp.p.
Principal Investigator Name
Krzysztof Banaszkiewicz
Principal Investigator Email
krzysztof@banaszkiewicz.net
Contact Person Name
Krzysztof Banaszkiewicz
Contact Person Email
krzysztof@banaszkiewicz.net
Site Name
Medicover Integrated Clinical Services Sp. z o.o.
Principal Investigator Name
Łukasz Rzepiński
Principal Investigator Email
luk.rzepinski@gmail.com
Contact Person Name
Łukasz Rzepiński
Contact Person Email
luk.rzepinski@gmail.com
Site Name
Galen Clinic
Principal Investigator Name
Urszula Chyrchel-Paszkiewicz
Principal Investigator Email
pgluchyrchel@gmail.com
Contact Person Name
Urszula Chyrchel-Paszkiewicz
Contact Person Email
pgluchyrchel@gmail.com
Site Name
LUXMED Sp. z o.o. - Warszawa
Principal Investigator Name
Anna Kostera-Pruszczyk
Principal Investigator Email
akosterapruszczyk@gmail.com
Contact Person Name
Anna Kostera-Pruszczyk
Contact Person Email
akosterapruszczyk@gmail.com
Site Name
Indywidualna Praktyka Lekarska Konrad Rejdak
Principal Investigator Name
Konrad Rejdak
Principal Investigator Email
krejdak@europe.com
Contact Person Name
Konrad Rejdak
Contact Person Email
krejdak@europe.com
Site Name
Centrum Medyczne Medyk Sp. z o.o. S.K.
Principal Investigator Name
Halina Bartosik-Psujek
Principal Investigator Email
bartosikpsujek@op.pl
Contact Person Name
Halina Bartosik-Psujek
Contact Person Email
bartosikpsujek@op.pl
Site Name
Novo-Med Zielinski I Wspolnicy Sp. j.
Principal Investigator Name
Barbara Kściuk
Principal Investigator Email
ksciuk@interia.pl
Contact Person Name
Barbara Kściuk
Contact Person Email
ksciuk@interia.pl

Spain

Earliest CTIS Part Ii Submission Date
07-05-2024
Latest Decision Or Authorization Date
26-06-2024
Processing Time Days
50
Number Of Sites
8
Number Of Participants
13

Sites

Site Name
Hospital General Universitario Dr. Balmis
Department Name
Neurology
Principal Investigator Name
Raquel Hernandez Lorido
Principal Investigator Email
rakel_her@hotmail.com
Contact Person Name
Raquel Hernandez Lorido
Contact Person Email
rakel_her@hotmail.com
Site Name
Bellvitge University Hospital
Department Name
Neurology
Principal Investigator Name
Carlos Casasnovas Pons
Principal Investigator Email
carloscasasnovas@bellvitgehospital.cat
Contact Person Name
Carlos Casasnovas Pons
Site Name
Hospital Universitario Regional De Malaga
Department Name
Neurology
Principal Investigator Name
Guillermina García Martin
Principal Investigator Email
guille.garcia.eecc@gmail.com
Contact Person Name
Guillermina García Martin
Contact Person Email
guille.garcia.eecc@gmail.com
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Neurology
Principal Investigator Name
Teresa Sevilla Mantecón
Principal Investigator Email
sevilla_ter@gva.es
Contact Person Name
Teresa Sevilla Mantecón
Contact Person Email
sevilla_ter@gva.es
Site Name
Hospital Ruber Juan Bravo
Department Name
Neurology
Principal Investigator Name
Rafael Arroyo Gonzalez
Principal Investigator Email
rafaelarroyo09@gmail.com
Contact Person Name
Rafael Arroyo Gonzalez
Contact Person Email
rafaelarroyo09@gmail.com
Site Name
Hospital Universitari Arnau De Vilanova De La Gerencia Territorial De Lleida
Department Name
Neurology
Principal Investigator Name
Cristina Gonzalez Mingot
Principal Investigator Email
crismingot@hotmail.com
Contact Person Name
Cristina Gonzalez Mingot
Contact Person Email
crismingot@hotmail.com
Site Name
Hospital Clinic De Barcelona
Department Name
Neurology
Principal Investigator Name
Eugenia Martinez-Hernandez
Principal Investigator Email
emmartinez@clinic.cat
Contact Person Name
Eugenia Martinez-Hernandez
Contact Person Email
emmartinez@clinic.cat
Site Name
Complexo Hospitalario Universitario De Santiago
Department Name
Neurology
Principal Investigator Name
Tania Garcia Sobrino
Principal Investigator Email
ainat.tania@hotmail.com
Contact Person Name
Tania Garcia Sobrino
Contact Person Email
ainat.tania@hotmail.com

Greece

Earliest CTIS Part Ii Submission Date
21-03-2024
Latest Decision Or Authorization Date
28-06-2024
Processing Time Days
99
Number Of Sites
5
Number Of Participants
9

Sites

Site Name
University General Hospital Attikon General Hospital Of West Attica H Agia Varvara
Department Name
Second Department of Neurology Clinic
Principal Investigator Name
Sotirios Giannopoulos
Principal Investigator Email
sgiannop@uoi.gr
Contact Person Name
Sotirios Giannopoulos
Contact Person Email
sgiannop@uoi.gr
Site Name
University General Hospital Of Thessaloniki Ahepa
Department Name
1st Department of Neurology
Principal Investigator Name
Vasilios Kimiskidis
Principal Investigator Email
kimiskid@auth.gr
Contact Person Name
Vasilios Kimiskidis
Contact Person Email
kimiskid@auth.gr
Site Name
Eginitio Hospital
Department Name
1st Neurology Department of National and Kapodistrian University of Athens
Principal Investigator Name
Vasiliki Zouvelou
Principal Investigator Email
vzouvelu@med.uoa.gr
Contact Person Name
Vasiliki Zouvelou
Contact Person Email
vzouvelu@med.uoa.gr
Site Name
General University Hospital Of Patras
Department Name
Neurology Department, Neuromuscular Unit
Principal Investigator Name
Elizabeth Chroni
Principal Investigator Email
echroni@yahoo.com
Contact Person Name
Elizabeth Chroni
Contact Person Email
echroni@yahoo.com
Site Name
General University Hospital Of Larissa
Department Name
Neurology Department
Principal Investigator Name
Efthimios Dardiotis
Principal Investigator Email
ebsdar@gmail.com
Contact Person Name
Efthimios Dardiotis
Contact Person Email
ebsdar@gmail.com

Germany

Earliest CTIS Part Ii Submission Date
08-08-2025
Latest Decision Or Authorization Date
18-09-2025
Processing Time Days
41
Number Of Sites
9
Number Of Participants
4

Sites

Site Name
Katholisches Klinikum Bochum gGmbH
Department Name
St. Josef Hospital
Principal Investigator Name
Christiane Schneider-Gold
Principal Investigator Email
christiane.schneider-gold@rub.de
Contact Person Name
Christiane Schneider-Gold
Site Name
Universitaetsklinikum Regensburg AöR
Department Name
Klinik und Poliklinik für Neurologie
Principal Investigator Name
De-Hyung Lee
Principal Investigator Email
De-Hyung.Lee@medbo.de
Contact Person Name
De-Hyung Lee
Contact Person Email
De-Hyung.Lee@medbo.de
Site Name
Goethe University Frankfurt
Department Name
Klinik für Neurologie
Principal Investigator Name
Yavor Yalachkov
Principal Investigator Email
Yalachkov@med.uni-frankfurt.de
Contact Person Name
Yavor Yalachkov
Contact Person Email
Yalachkov@med.uni-frankfurt.de
Site Name
Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)
Department Name
Klinik und Poliklinik für Neurologie
Principal Investigator Name
Achim Berthele
Principal Investigator Email
achim.berthele@tum.de
Contact Person Name
Achim Berthele
Contact Person Email
achim.berthele@tum.de
Site Name
Philipps-Universitaet Marburg
Department Name
Klinik für Neurologie
Principal Investigator Name
Clemens Warnke
Principal Investigator Email
clemens.warnke@uni-marburg.de
Contact Person Name
Clemens Warnke
Contact Person Email
clemens.warnke@uni-marburg.de
Site Name
Universitaetsklinikum Essen AöR
Department Name
Klinik für Neurologie
Principal Investigator Name
Tim Hagenacker
Principal Investigator Email
tim.hagenacker@uk-essen.de
Contact Person Name
Tim Hagenacker
Contact Person Email
tim.hagenacker@uk-essen.de
Site Name
Universitaetsklinikum Schleswig-Holstein AöR
Department Name
Klinik für Neurologie
Principal Investigator Name
Julian Großkreutz
Principal Investigator Email
Julian.Grosskreutz@uksh.de
Contact Person Name
Julian Großkreutz
Contact Person Email
Julian.Grosskreutz@uksh.de
Site Name
Berufsgenossenschaftliches Universitaetsklinikum Bergmannsheil gGmbH
Department Name
Neurologische Klinik und Poliklinik
Principal Investigator Name
Felix Kleefeld
Principal Investigator Email
felix.kleefeld@bergmannsheil.de
Contact Person Name
Felix Kleefeld
Site Name
Klinikum Wuerzburg Mitte gGmbH
Principal Investigator Name
Mathias Mäurer
Principal Investigator Email
mathias.maeurer@kwm-klinikum.de
Contact Person Name
Mathias Mäurer

Italy

Earliest CTIS Part Ii Submission Date
21-03-2024
Latest Decision Or Authorization Date
26-06-2024
Processing Time Days
97
Number Of Sites
11
Number Of Participants
11

Sites

Site Name
Azienda Sanitaria Locale Roma 1
Department Name
Centro Malattie Rare Neurologiche e Neuromuscolari
Principal Investigator Name
Elena Maria Pennisi
Principal Investigator Email
elenamaria.pennisi@aslroma1.it
Contact Person Name
Elena Maria Pennisi
Contact Person Email
elenamaria.pennisi@aslroma1.it
Site Name
Universita' Degli Studi Di Roma Tor Vergata
Department Name
UOC Neurologia
Principal Investigator Name
Roberto Massa
Principal Investigator Email
massa@uniroma2.it
Contact Person Name
Roberto Massa
Contact Person Email
massa@uniroma2.it
Site Name
ARNAS Civico Di Cristina Benfratelli
Department Name
Neurology e Stroke Unit
Principal Investigator Name
Salvatore Cottone
Principal Investigator Email
salvatore.cottone1@arnascivico.it
Contact Person Name
Salvatore Cottone
Site Name
Azienda Ospedaliero-Universitaria San Luigi Gonzaga
Department Name
Neurology
Principal Investigator Name
Marinella Clerico
Principal Investigator Email
marinella.clerico@unito.it
Contact Person Name
Marinella Clerico
Contact Person Email
marinella.clerico@unito.it
Site Name
Azienda Ospedaliero-Universitaria Sant Andre
Department Name
Neuromuscolar Disease Center
Principal Investigator Name
Sterfania Morino
Principal Investigator Email
s.morino@ospedalesantandrea.it
Contact Person Name
Sterfania Morino
Contact Person Email
s.morino@ospedalesantandrea.it
Site Name
Azienda Unita Sanitaria Locale Di Bologna
Department Name
UOC Neurologia
Principal Investigator Name
Rocco Liguori
Principal Investigator Email
rocco.liguori@unibo.it
Contact Person Name
Rocco Liguori
Contact Person Email
rocco.liguori@unibo.it
Site Name
Azienda Ospedaliera Universitaria Federico II Di Napoli
Department Name
Dipartimento di Scienze Neurologiche
Principal Investigator Name
Francesco Saccà
Principal Investigator Email
francesco.sacca@unina.it
Contact Person Name
Francesco Saccà
Contact Person Email
francesco.sacca@unina.it
Site Name
IRCCS Foundation Istituto Neurologico Carlo Besta
Department Name
Neurology
Principal Investigator Name
Carlo Giuseppe Antozzi
Principal Investigator Email
carlo.antozzi@istituto-besta.it
Contact Person Name
Carlo Giuseppe Antozzi
Site Name
Careggi University Hospital
Department Name
SOD Neurologia 2
Principal Investigator Name
Valentina Damato
Principal Investigator Email
valentina.damato@unifi.it
Contact Person Name
Valentina Damato
Contact Person Email
valentina.damato@unifi.it
Site Name
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
Department Name
Neurofisiopatologia
Principal Investigator Name
Filippo Brighina
Principal Investigator Email
Filippo.brighina@unipa.it
Contact Person Name
Filippo Brighina
Contact Person Email
Filippo.brighina@unipa.it
Site Name
IRCCS Ospedale Policlinico San Martino
Department Name
Neurology
Principal Investigator Name
Martina Garnero
Principal Investigator Email
martina.garnero@hsanmartino.it
Contact Person Name
Martina Garnero
Contact Person Email
martina.garnero@hsanmartino.it

Denmark

Earliest CTIS Part Ii Submission Date
12-06-2025
Latest Decision Or Authorization Date
27-06-2025
Processing Time Days
15
Number Of Sites
1
Number Of Participants
1

Sites

Site Name
Rigshospitalet
Department Name
Copenhagen Neuromuscular Center
Principal Investigator Name
Nicolai Preisler
Principal Investigator Email
nicolai.rasmus.preisler@regionh.dk
Contact Person Name
Nicolai Preisler

Sponsor

Primary sponsor

Full Name
Novartis Pharma AG
Organisation Type
Pharmaceutical company
Country Of Registered Address
Switzerland

Contract research organisations

Name
IQVIA Limited
Responsibilities
Randomization of Patients and Management of drug supply logistics, dispensing and unblinding
Name
PPD Global Limited
Responsibilities
On site interim monitoring visits, In-House monitoring, Site Management, Develop and/or distribute Newsletter(s) for investigators, perform close-out visit(s), distribution of ancillaries devices.
Name
Parexel International (IRL) Limited
Name
Eresearchtechnology Inc.
Responsibilities
PRO management, PRO licensing and translations, data collection via tablet

Third parties

  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Cardiac Assessment","organisation_type":"Pharmaceutical company"}
  • {"country":"Germany","full_name":"DATAMAP-Gesellschaft fuer Datenmanagement Datenanalyse und Datenpraesentation mbH","duties_or_roles":"Independent statistical group for Data Monitoring Committee (DMC)","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"Safety tests per protocol","organisation_type":"Pharmaceutical company"}
  • {"country":"Greece","full_name":"PPD Global Ltd.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"RWS Life Sciences Inc.","duties_or_roles":"PRO formatting and translations","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"PPD Global Limited","duties_or_roles":"On site interim monitoring visits, In-House monitoring, Site Management, Develop and/or distribute Newsletter(s) for investigators, perform close-out visit(s), distribution of ancillaries devices.","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Randomization of Patients and Management of drug supply logistics, dispensing and unblinding","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Somalogic Operating Co. Inc.","duties_or_roles":"Exploratory biomarkers (protein profiling on SomaScan Platform)","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"Safety tests per protocol","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Travel patient reimbursement","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"PRO management, PRO licensing and translations, data collection via tablet","organisation_type":"Pharmaceutical company"}
  • {"country":"India","full_name":"Veeda Clinical Research Limited","duties_or_roles":"Pharmacokinetics (PK) LNP023 analysis","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
IPTACOPAN
Active Substance
IPTACOPAN
Modality
Small molecule
Routes Of Administration
ORAL
Route
Oral
Investigational Product Name
Placebo 0 mg hard gelatin capsule size 0, (placebo to LNP023)
Modality
Other
Combination Treatment
Yes

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