Clinical trial • Phase III|Phase IV • Immunology|Rare Disease

IPTACOPAN for C3 glomerulopathy|Idiopathic immune-complex membranoproliferative glomerulonephritis

Phase III|Phase IV trial of IPTACOPAN for C3 glomerulopathy|Idiopathic immune-complex membranoproliferative glomerulonephritis.

Overview

Trial Therapeutic Area
Immunology|Rare Disease
Trial Disease
C3 glomerulopathy|Idiopathic immune-complex membranoproliferative glomerulonephritis
Trial Stage
Phase III|Phase IV
Drug Modality
Small molecule
Paediatric Trial
Yes
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
12-07-2024
First CTIS Authorization Date
21-08-2024

Trial design

open-label, none/not specified-controlled Phase III|Phase IV trial across 34 sites in Czechia, Netherlands, Italy and others.

Open Label
Yes
Comparator
None/Not specified
Target Sample Size
148

Eligibility

Recruits 148 paediatric patients.

Vulnerable Population
Vulnerable population is selected. The trial includes paediatric participants (adolescents/children). Country-specific informed consent/assent materials are provided (e.g. Parent/Legal Guardian consent forms and Adolescent Assent forms listed for Netherlands, Italy, France, Greece, Spain, Germany and Czechia). Consent is obtained from participants or from parent/legal guardian for minors, with age-appropriate assent documents for adolescents; materials are available in local languages and English as indicated in country-specific documents.

Inclusion criteria

  • {"criterion_text":"- Participants must have completed the treatment period of the CLNP023X2202, CLNP023B12301 or CLNP023B12302 study on study drug."}

Exclusion criteria

  • {"criterion_text":"- Severe concurrent co-morbidities, e.g., advanced cardiac disease (New York Heart Association (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 iptacopan or complying with the requirements of the study."}
  • {"criterion_text":"- 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."}
  • {"criterion_text":"- History of human immunodeficiency virus (HIV) or any other immunodeficiency disease."}
  • {"criterion_text":"- History or current diagnosis of ECG abnormalities indicating significant risk of safety for participants."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Participants from study CLNP023X2202: • Composite renal endpoint met at the 9-month visit if following criteria satisfied: (1) stable or improved eGFR compared to the baseline visit in CLNP023X2202 (≤10% reduction in eGFR) and (2) either ≥50% reduction compared to the baseline visit in CLNP023X2202 or reduction to <300 mg/g in UPCR and (3) either a ≥50% increase in C3 compared to baseline or an increase to ≥90 mg/dL.","definition_or_measurement_approach":"Composite renal endpoint defined by three component criteria measured vs baseline from prior study CLNP023X2202: (1) eGFR change (≤10% reduction allowed), (2) UPCR reduction (≥50% or <300 mg/g), and (3) serum C3 increase (≥50% or ≥90 mg/dL). Assessed at 9 months."}
  • {"endpoint_text":"- Participants from study CLNP023X2202: • Change from baseline in the C3 Deposit Score (based on immunofluorescence microscopy) compared to baseline in the CLNP023X2202 study.","definition_or_measurement_approach":"C3 Deposit Score assessed by immunofluorescence microscopy comparing renal biopsy score to baseline from CLNP023X2202 (measured at 6-9 months for cohort B and at 9 months for cohort A as applicable)."}
  • {"endpoint_text":"- Participants from study CLNP023X2202: • 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 (or any safety issue).","definition_or_measurement_approach":"Safety endpoints: clinically significant changes in vital signs (msDBP, msSBP, heart rate), ECG abnormalities, safety labs, and collection/assessment of AEs, AEs of special interest, and discontinuations due to AEs across study visits."}
  • {"endpoint_text":"- Participants from studies CLNP023B12301 or CLNP023B12302: • 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 (or any safety issue).","definition_or_measurement_approach":"Safety endpoints as above for participants enrolled from CLNP023B12301/CLNP023B12302: clinical vitals, ECGs, safety labs, AEs and discontinuations."}

Secondary endpoints

  • {"endpoint_text":"- Participants from study CLNP023X2202: • Composite renal endpoint met at the 9-month visit if following criteria satisfied: (1) a stable or improved eGFR compared to the baseline visit in CLNP023X2202 (≤10% reduction in eGFR), and (2) either ≥50% reduction compared to the baseline visit in CLNP023X2202 or a reduction to <300 mg/g in UPCR.","definition_or_measurement_approach":"As defined for primary composite renal endpoint but specified as secondary in this listing; measured at 9 months vs CLNP023X2202 baseline (eGFR and UPCR)."}
  • {"endpoint_text":"- Participants from study CLNP023X2202: • Change from baseline in log-transformed urine protein/creatinine ratio (UPCR), change from baseline in log-transformed urine albumin/creatinine ratio (UACR), change from baseline in serum creatinine concentration and change from baseline in estimated glomerular filtration rate (eGFR) at the 9-month visit in CLNP023B12001B.","definition_or_measurement_approach":"Continuous renal function and proteinuria measures: log-transformed UPCR and UACR, serum creatinine, eGFR changes at 9 months vs baseline."}
  • {"endpoint_text":"- Participants from study CLNP023X2202: • Change from baseline in log-transformed urine protein/creatinine ratio (UPCR) and change from baseline in log-transformed urine albumin/creatinine ratio (UACR), at the 3-month visit in CLNP023B12001B.","definition_or_measurement_approach":"Log-transformed UPCR and UACR change at 3 months vs baseline."}
  • {"endpoint_text":"- Participants from study CLNP023X2202: • Disease activity and chronicity scores from a renal biopsy at 6 to 9 months from entry to the CLNP023B12001B study. Biopsies will be compared to those obtained in the CLNP023X2202 study (if available).","definition_or_measurement_approach":"Renal biopsy histopathology scoring for disease activity and chronicity at 6-9 months compared to prior biopsies from CLNP023X2202."}
  • {"endpoint_text":"- Participants from study CLNP023X2202: • Log-transformed ratio to baseline in serum C3 at the 9-month visit in CLNP023B12001B.","definition_or_measurement_approach":"Log-transformed ratio of serum C3 vs baseline, measured at 9 months."}
  • {"endpoint_text":"- Participants from study CLNP023X2202: • Composite renal endpoint met at times >9 months if following criteria satisfied: (1) stable or improved eGFR compared to the baseline visit in CLNP023X2202 (≤10% reduction in eGFR), (2) either ≥50% reduction compared to the baseline visit in CLNP023X2202 or a reduction to <300 mg/g in UPCR and (3) either a ≥50% increase in C3 compared to baseline or an increase to ≥90 mg/dL.","definition_or_measurement_approach":"Composite renal endpoint criteria assessed at timepoints >9 months using eGFR, UPCR and serum C3."}
  • {"endpoint_text":"- Participants from study CLNP023X2202: • Change from baseline in log-transformed UPCR, log-transformed UACR, serum creatinine concentration and estimated glomerular filtration rate (eGFR) at times >9 months in CLNP023B12001B. Change in eGFR slope.","definition_or_measurement_approach":"Longer-term changes (>9 months) in log-UPCR, log-UACR, serum creatinine and eGFR, including eGFR slope analysis."}
  • {"endpoint_text":"- Participants from study CLNP023X2202: • Log-transformed ratio to baseline in serum C3 at times >9 months in CLNP023B12001B.","definition_or_measurement_approach":"Log-transformed serum C3 ratio vs baseline at >9 months."}
  • {"endpoint_text":"- Participants from study CLNP023X2202: • Determine plasma iptacopan concentration up to 12 months at trough.","definition_or_measurement_approach":"Pharmacokinetic measurement: trough plasma iptacopan concentrations up to 12 months."}
  • {"endpoint_text":"- Participants from studies CLNP023B12301 or CLNP023B12302: • Change from initiation of iptacopan treatment in the core study in log-transformed UPCR over time.","definition_or_measurement_approach":"Change over time in log-transformed UPCR measured from initiation of iptacopan in core study."}
  • {"endpoint_text":"- Participants from studies CLNP023B12301 or CLNP023B12302: • Change from initiation of iptacopan treatment in the core study in eGFR over time.","definition_or_measurement_approach":"Longitudinal eGFR change from initiation of iptacopan in core study."}
  • {"endpoint_text":"- Participants from studies CLNP023B12301 or CLNP023B12302: • Composite renal endpoint met if following criteria satisfied (1) eGFR (a stable or improved eGFR, i.e., ≤15% reduction in eGFR compared to the initiation of iptacopan treatment in the core study) and (2) UPCR (≥50% reduction in UPCR compared to the initiation of iptacopan treatment in the core study).","definition_or_measurement_approach":"Composite renal endpoint for CLNP023B12301/12302 defined by eGFR (≤15% reduction) and UPCR (≥50% reduction) relative to iptacopan initiation in core study."}

Recruitment

Planned Sample Size
148
Recruitment Window Months
202
Consent Approach
Informed consent is collected using country-specific subject information and informed consent forms. For adult participants the Main ICF is used; for minors Parent/Legal Guardian consent forms and Adolescent Assent forms are provided (documents listed for Netherlands, Italy, France, Greece, Spain, Germany, Czechia). Documents are provided in local languages and English where indicated. Separate data protection consent and optional assessment consent forms are available; follow-up information for pregnant participants is provided.

Geography

Total Number Of Sites
34
Total Number Of Participants
50

Czechia

Earliest CTIS Part Ii Submission Date
02-08-2024
Latest Decision Or Authorization Date
22-08-2024
Processing Time Days
20
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Vseobecna Fakultni Nemocnice V Praze
Department Name
#1601: Klinika nefrologie
Principal Investigator Name
Vladimir Tesar
Principal Investigator Email
vladimir.tesar@vfn.cz
Contact Person Name
Vladimir Tesar
Contact Person Email
vladimir.tesar@vfn.cz

Netherlands

Earliest CTIS Part Ii Submission Date
02-08-2024
Latest Decision Or Authorization Date
22-08-2024
Processing Time Days
20
Number Of Sites
2
Number Of Participants
3

Sites

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
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

Italy

Earliest CTIS Part Ii Submission Date
02-08-2024
Latest Decision Or Authorization Date
24-09-2024
Processing Time Days
53
Number Of Sites
4
Number Of Participants
14

Sites

Site Name
Ospedale Pediatrico Bambino Gesu
Department Name
#3002: 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
#3001: 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
Site Name
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Department Name
#3003: S.C.Nefrologia e Dialisi Pediatrica- Trapianto di Rene
Principal Investigator Name
Giovanni Montini
Principal Investigator Email
giovanni.montini@unimi.it
Contact Person Name
Giovanni Montini
Contact Person Email
giovanni.montini@unimi.it
Site Name
Azienda Universitaria Ospedaliera Consorziale Policlinico Bari
Department Name
#3004 : U.O. Nefrologia Universitaria Dip. Emergenza e Trapianti d'Organo
Principal Investigator Name
Loreto Gesualdo
Principal Investigator Email
loreto.gesualdo@uniba.it
Contact Person Name
Loreto Gesualdo
Contact Person Email
loreto.gesualdo@uniba.it

France

Earliest CTIS Part Ii Submission Date
02-08-2024
Latest Decision Or Authorization Date
22-08-2024
Processing Time Days
20
Number Of Sites
5
Number Of Participants
8

Sites

Site Name
Centre Hospitalier Universitaire De Montpellier
Department Name
#1002: Service de Néphrologie, Dialyse et transplantation rénale
Principal Investigator Name
Moglie Le Quintrec
Principal Investigator Email
m-lequintrec-donnette@chu-montpellier.fr
Contact Person Name
Moglie Le Quintrec
Site Name
Assistance Publique Hopitaux De Paris
Department Name
#1003: Service de Néphrologie
Principal Investigator Name
Alexandre Karras
Principal Investigator Email
alexandre.karras@egp.aphp.fr
Contact Person Name
Alexandre Karras
Contact Person Email
alexandre.karras@egp.aphp.fr
Site Name
Centre Hospitalier Universitaire De Rennes
Department Name
1008: Nephrologie
Principal Investigator Name
Manon Martins
Principal Investigator Email
manon.martins@chu-rennes.fr
Contact Person Name
Manon Martins
Contact Person Email
manon.martins@chu-rennes.fr
Site Name
Centre Hospitalier Universitaire De Toulouse
Department Name
#1006: Service de Néphrologie et Transplatation d'organes
Principal Investigator Name
David Ribes
Principal Investigator Email
ribes.d@chu-toulouse.fr
Contact Person Name
David Ribes
Contact Person Email
ribes.d@chu-toulouse.fr
Site Name
Assistance Publique Hopitaux De Paris
Department Name
#1005: Service de Néphrologie et Transplantation adulte
Principal Investigator Name
Aude Servais
Principal Investigator Email
aude.servais@aphp.fr
Contact Person Name
Aude Servais
Contact Person Email
aude.servais@aphp.fr

Greece

Earliest CTIS Part Ii Submission Date
02-08-2024
Latest Decision Or Authorization Date
22-08-2024
Processing Time Days
20
Number Of Sites
8
Number Of Participants
8

Sites

Site Name
University General Hospital Attikon General Hospital Of West Attica H Agia Varvara
Department Name
1705 : Nephrology and Artificial Kidney Unit
Principal Investigator Name
Sophia Lionaki
Principal Investigator Email
sofia.lionaki@gmail.com
Contact Person Name
Sophia Lionaki
Contact Person Email
sofia.lionaki@gmail.com
Site Name
University General Hospital Of Thessaloniki Ahepa
Department Name
1704 : Internal Medicine Sector - Second Nephrology Clinic
Principal Investigator Name
Vassilios Liakopoulos
Principal Investigator Email
liakopul@otenet.gr
Contact Person Name
Vassilios Liakopoulos
Contact Person Email
liakopul@otenet.gr
Site Name
Ippokratio General Hospital Of Thessaloniki
Department Name
1702: 1st Pediatric Department
Principal Investigator Name
Styliani (Stella) Stampouli (Stabouli)
Principal Investigator Email
sstaboul@auth.gr
Contact Person Name
Styliani (Stella) Stampouli (Stabouli)
Contact Person Email
sstaboul@auth.gr
Site Name
Hippokration Hospital
Department Name
1708:Internal Medicine Sector - Department of Nephrology
Principal Investigator Name
Dimitrios Petras
Principal Investigator Email
petrasdim@hotmail.com
Contact Person Name
Dimitrios Petras
Contact Person Email
petrasdim@hotmail.com
Site Name
University General Hospital Of Ioannina
Department Name
1706: Nephrology Clinic
Principal Investigator Name
Evangelia Ntounousi
Principal Investigator Email
edounous@uoi.gr
Contact Person Name
Evangelia Ntounousi
Contact Person Email
edounous@uoi.gr
Site Name
University General Hospital Of Heraklion
Department Name
#1701: 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
Geniko Nosokomeio Thessalonikis George Papanikolaou
Department Name
1703:1st Internal Medicine Sector, Nephrology department
Principal Investigator Name
Gerasimos Bamichas
Principal Investigator Email
gbamichas@gmail.com
Contact Person Name
Gerasimos Bamichas
Contact Person Email
gbamichas@gmail.com
Site Name
General University Hospital Of Patras
Department Name
1707 : Nephrology Center
Principal Investigator Name
Evangelos Papachristou
Principal Investigator Email
epapadoct@hotmail.com
Contact Person Name
Evangelos Papachristou
Contact Person Email
epapadoct@hotmail.com

Spain

Earliest CTIS Part Ii Submission Date
02-08-2024
Latest Decision Or Authorization Date
21-08-2024
Processing Time Days
19
Number Of Sites
9
Number Of Participants
9

Sites

Site Name
Clinica Universidad De Navarra
Department Name
#4009: Nefrología
Principal Investigator Name
Jose Maria Mora Gutierrez
Principal Investigator Email
jmora@unav.es
Contact Person Name
Jose Maria Mora Gutierrez
Contact Person Email
jmora@unav.es
Site Name
Hospital Universitari Vall D Hebron
Department Name
#4010: Nefrología Pediátrica
Principal Investigator Name
Gema Ariceta Iraola
Principal Investigator Email
gema.ariceta@vallhebron.cat
Contact Person Name
Nicole van de Kar
Contact Person Email
gema.ariceta@radboudumc.nl
Site Name
Hospital Clinico San Carlos
Department Name
#4006: Nefrología
Principal Investigator Name
Antolina Rodriguez Moreno
Principal Investigator Email
antolina.rodriguez@salud.madrid.org
Contact Person Name
Antolina Rodriguez Moreno
Site Name
Hospital Universitario 12 De Octubre
Department Name
#4001: 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
Clinica Universidad De Navarra
Department Name
#4008: Nefrología
Principal Investigator Name
Jose Maria Mora Gutierrez
Principal Investigator Email
jmora@unav.es
Contact Person Name
Jose Maria Mora Gutierrez
Contact Person Email
jmora@unav.es
Site Name
Hospital Clinic De Barcelona
Department Name
#4005: Nefrología
Principal Investigator Name
Luis Fernando Quintana Porras
Principal Investigator Email
LFQUINTA@clinic.cat
Contact Person Name
Luis Fernando Quintana Porras
Contact Person Email
LFQUINTA@clinic.cat
Site Name
Hospital Universitario De Salamanca
Department Name
#4007: Nefrología
Principal Investigator Name
Maria Pilar Fraile Gomez
Principal Investigator Email
pilarfg@usal.es
Contact Person Name
Maria Pilar Fraile Gomez
Contact Person Email
pilarfg@usal.es
Site Name
Hospital Universitario Virgen De La Macarena
Department Name
#4004: Nefrología
Principal Investigator Name
Mercedes Salgueira Lazo
Contact Person Name
Mercedes Salgueira Lazo
Site Name
Hospital Universitario De Vall D Hebron
Department Name
#4003: Nefrología
Principal Investigator Name
Maria Jose Soler Romero
Principal Investigator Email
mariajose.soler@vallhebron.cat
Contact Person Name
Maria Jose Soler Romero
Contact Person Email
mariajose.soler@vallhebron.cat

Germany

Earliest CTIS Part Ii Submission Date
02-08-2024
Latest Decision Or Authorization Date
22-08-2024
Processing Time Days
20
Number Of Sites
5
Number Of Participants
6

Sites

Site Name
Universitaetsklinikum Erlangen AöR
Department Name
#2007: Medizinische Klinik 4
Principal Investigator Name
Michael Wiesener
Principal Investigator Email
Michael.Wiesener@uk-erlangen.de
Contact Person Name
Michael Wiesener
Site Name
Universitaetsklinikum Essen AöR
Department Name
#2002: Klinik für Nephrologie
Principal Investigator Name
Ute Eisenberger
Principal Investigator Email
ute.eisenberger@uk-essen.de
Contact Person Name
Ute Eisenberger
Contact Person Email
ute.eisenberger@uk-essen.de
Site Name
University Medical Center Hamburg-Eppendorf
Department Name
#2009: III. Medizinische Klinik und Poliklinik, Nephrologie
Principal Investigator Name
Malte Kluger
Principal Investigator Email
m.kluger@uke.de
Contact Person Name
Malte Kluger
Contact Person Email
m.kluger@uke.de
Site Name
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
Department Name
#2005: I. Medizinische Klinik und Poliklinik, Nephrologie
Principal Investigator Name
Julia Weinmann-Menke
Principal Investigator Email
julia.weinmann-menke@unimedizin-mainz.de
Contact Person Name
Julia Weinmann-Menke
Site Name
Universitaetsklinikum Heidelberg AöR
Department Name
#2010: Klinik Kinderheilkunde I
Principal Investigator Name
Christian Patry
Principal Investigator Email
Christian.Patry@med.uni-heidelberg.de
Contact Person Name
Christian Patry

Sponsor

Primary sponsor

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

Contract research organisations

Name
Bioclinica Inc.
Responsibilities
sponsorDuties codes: [5,6]; clinical data/operations support (contact: Tamas.Fulop@clario.com)
Name
PRA Hellas CRO A.E.
Responsibilities
sponsorDuties codes: [1]; CRO operations (contact: ICONGreekAffiliateMailbox@iconplc.com)
Name
Syneos Health Inc.
Responsibilities
sponsorDuties codes: [1]; CRO operations (contact: sm_ctis@syneoshealth.com)
Name
Icon Clinical Research Limited
Responsibilities
sponsorDuties codes: [1]; CRO operations (contact: Triona.PriceSmith1@docsglobal.com)
Name
Parexel International (IRL) Limited
Responsibilities
sponsorDuties codes: [12]; specific CRO responsibilities (contact: Clinicaltrial.Enquiries@parexel.com)
Name
IQVIA Limited / Iqvia Biotech LLC
Responsibilities
sponsorDuties codes: [1] and [3]; IRT/QC and clinical operations (contact: eu_clinical_trials_information@iqvia.com / IRT-QATeamDL@iqvia.com)
Name
Veeda Clinical Research Limited
Responsibilities
sponsorDuties codes: [4]; local clinical study operations (contact: info@veedacr.com)

Third parties

  • {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"sponsorDuties codes: [5,6]; contact email: Tamas.Fulop@clario.com","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"Greece","full_name":"PRA Hellas CRO A.E.","duties_or_roles":"sponsorDuties codes: [1]; contact email: ICONGreekAffiliateMailbox@iconplc.com","organisation_type":"Pharmaceutical company"}
  • {"country":"Italy","full_name":"Opis S.r.l.","duties_or_roles":"sponsorDuties: TMF archive","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"sponsorDuties codes: [1]; contact email: sm_ctis@syneoshealth.com","organisation_type":"Pharmaceutical company"}
  • {"country":"India","full_name":"Veeda Clinical Research Limited","duties_or_roles":"sponsorDuties codes: [4]; contact email: info@veedacr.com","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Publicis Healthcare Communications Group Limited","duties_or_roles":"sponsorDuties: Patients tools - IMP Safety Card","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"RWS Life Sciences Inc.","duties_or_roles":"sponsorDuties: CCI","organisation_type":"Pharmaceutical company"}
  • {"country":"France","full_name":"Kayentis","duties_or_roles":"sponsorDuties: CCI","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"sponsorDuties: Patient travel and accommodation","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"sponsorDuties codes: [1]; contact email: Triona.PriceSmith1@docsglobal.com","organisation_type":"Pharmaceutical company"}
  • {"country":"Denmark","full_name":"Eurofins Genomics Europe AgriGenomics Products & Services A/S","duties_or_roles":"sponsorDuties codes: [4]; contact email: Samples-dk@eurofins.dk","organisation_type":"Pharmaceutical company"}
  • {"country":"France","full_name":"SGS France","duties_or_roles":"sponsorDuties codes: [4]; contact email: fr.saint-benoit.sales@sgs.com","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"sponsorDuties codes: [6]; contact email: info@medidata.com","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Q Squared Solutions Limited","duties_or_roles":"sponsorDuties codes: [4]; contact email: q2_eu_clinical_trials_information@q2labsolutions.com","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Iqvia Biotech LLC","duties_or_roles":"sponsorDuties codes: [3]; contact email: IRT-QATeamDL@iqvia.com","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"sponsorDuties codes: [12]; contact email: Clinicaltrial.Enquiries@parexel.com","organisation_type":"Pharmaceutical company"}
  • {"country":"Cyprus","full_name":"Clinbay Limited","duties_or_roles":"sponsorDuties codes: [10]; contact email: info@clinbay.com","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"sponsorDuties codes: [1]; contact email: eu_clinical_trials_information@iqvia.com","organisation_type":"Pharmaceutical company"}
  • {"country":"Netherlands","full_name":"Eurofins Central Laboratory B.V.","duties_or_roles":"sponsorDuties codes: [4]; contact email: SH-Breda_StudyManagement_BioMarkers@bcl.eurofins.com","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
IPTACOPAN HYDROCHLORIDE
Active Substance
IPTACOPAN HYDROCHLORIDE
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
Authorised
Orphan Designation
Yes
Maximum Dose
400 mg

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