Clinical trial • Phase II/III • Musculoskeletal

EFGARTIGIMOD ALFA for Idiopathic inflammatory myopathy (IIM)

Phase II/III trial of EFGARTIGIMOD ALFA for Idiopathic inflammatory myopathy (IIM).

Overview

Trial Therapeutic Area
Musculoskeletal
Trial Disease
Idiopathic inflammatory myopathy (IIM)
Trial Stage
Phase II/III
Drug Modality
Peptide/protein/enzyme

Key dates

Initial CTIS Submission Date
28-06-2024
First CTIS Authorization Date
14-08-2024

Trial design

Randomised, efgartigimod ph20 sc (argx-113) subcutaneous solution for injection vs matching placebo (placebo to argx-113 solution for injection); dose and schedule not specified in the provided part i summary-controlled, adaptive Phase II/III trial in Austria, Cyprus, Czechia and others.

Randomised
Yes
Comparator
efgartigimod PH20 SC (ARGX-113) subcutaneous solution for injection vs matching placebo (Placebo to ARGX-113 solution for injection); dose and schedule not specified in the provided Part I summary
Adaptive
True, operationally seamless Phase 2/3 design with the phase 3 design and sample size to be confirmed after analysis of the phase 2 stage data
Target Sample Size
240
Trial Duration For Participant
364

Stratification factors

  • IIM subtype (IMNM, dermatomyositis (DM), polymyositis (PM))
  • Physician Global Assessment of Disease Activity (MDGA)

Eligibility

Recruits 240 Vulnerable population flag selected. Participants must be able to provide informed consent in the jurisdiction of the trial and be capable of giving signed informed consent; participants are adults (≥18 years or legal age of consent in country). Caregiver-specific information and consent forms are provided (caregiver ICFs exist). Multiple ICF addenda (COVID-19, genetic testing, pregnant partner, optional samples, etc.) and language-specific ICFs are provided as indicated in the submission documents..

Pregnancy Exclusion
Participant is pregnant or lactating or intends to become pregnant during the study.
Vulnerable Population
Vulnerable population flag selected. Participants must be able to provide informed consent in the jurisdiction of the trial and be capable of giving signed informed consent; participants are adults (≥18 years or legal age of consent in country). Caregiver-specific information and consent forms are provided (caregiver ICFs exist). Multiple ICF addenda (COVID-19, genetic testing, pregnant partner, optional samples, etc.) and language-specific ICFs are provided as indicated in the submission documents.

Inclusion criteria

  • {"criterion_text":"- Ability to consent in the jurisdiction in which the study is taking place and capable of giving signed informed consent"}
  • {"criterion_text":"- A definite or probable clinical diagnosis of idiopathic inflammatory myopathy (IIM)"}
  • {"criterion_text":"- One of the following medical histories: (a) Diagnosis of dermatomyositis (DM) or juvenile dermatomyositis (JDM), (age of disease onset <18 years of age). The diagnosis date for JDM should not be >5 years from the screening date; (b) Diagnosis of polymyositis (PM) (including antisynthetase syndrome (ASyS)); (c) Diagnosis of immune-mediated necrotizing myopathy (IMNM)"}
  • {"criterion_text":"- Diagnosed with active disease as defined by the presence of at least 1 of the following criteria: (a) Abnormal levels of at least 1 of the following enzymes: creatine kinase (CK), aldolase, lactate dehydrogenase, aspartate aminotransaminase (AST), alanine aminotransferase (ALT), based on central laboratory results; (b) Electromyography demonstrating active disease performed within the past 3 months; (c) Active dermatomyositis (DM) skin rash; (d) Muscle biopsy indicative of active idiopathic inflammatory myopathy (IIM) (in the past 3 months); (e) Magnetic resonance imaging within the past 3 months indicative of active inflammation"}
  • {"criterion_text":"- Muscle weakness"}
  • {"criterion_text":"- Receiving a permitted background treatment for IIM"}
  • {"criterion_text":"- Contraceptive use consistent with local regulations, where available, for individuals participating in clinical studies. Women of childbearing potential must have a negative serum pregnancy test during screening and a negative urine pregnancy test at baseline before receiving investigational medicinal product (IMP)"}

Exclusion criteria

  • {"criterion_text":"- A clinically significant active infection at screening"}
  • {"criterion_text":"- Clinically significant disease, recent major surgery or intends to have surgery during the study, or has any other condition in the opinion of the investigator that could confound the results of the trial or put the patient at undue risk"}
  • {"criterion_text":"- Known hypersensitivity reaction to investigational medicinal product (IMP) or 1 of its excipients"}
  • {"criterion_text":"- Received a live or live-attenuated vaccine less than 4 weeks before screening."}
  • {"criterion_text":"- Positive serum test at screening for active viral infection with any of the following conditions: a. Hepatitis B virus (HBV); b. Hepatitis C virus (HCV); c. HIV"}
  • {"criterion_text":"- Participant has previously participated in an efgartigimod clinical trial and received at least 1 dose of investigational medicinal product (IMP)."}
  • {"criterion_text":"- Participant is concurrently participating in any other clinical study, including a noninterventional study."}
  • {"criterion_text":"- Participant has a current or history (ie, within 12 months of screening) of alcohol, drug, or medication abuse."}
  • {"criterion_text":"- Participant is pregnant or lactating or intends to become pregnant during the study."}
  • {"criterion_text":"- Participant has severe renal impairment."}
  • {"criterion_text":"- Participant is institutionalized by a court or other governmental order or is in a dependent relationship with the sponsor or investigator."}
  • {"criterion_text":"- A COVID-19 polymerase chain reaction (PCR)-positive test before enrollment"}
  • {"criterion_text":"- Any other known autoimmune disease that, in the investigator's opinion, would interfere with an accurate assessment of clinical symptoms of idiopathic inflammatory myopathy (IIM) or put the patient at undue risk"}
  • {"criterion_text":"- A history of malignancy unless considered cured by adequate treatment, with no evidence of recurrence for ≥ 3 years before the first administration of the investigational medicinal product (IMP). Adequately treated participants with the following cancers can be included at any time: (a) Basal cell or squamous cell skin cancer; (b) Carcinoma in situ of the cervix; (c) Carcinoma in situ of the breast; (d) Incidental histological finding of prostate cancer"}
  • {"criterion_text":"- Severe muscle damage"}
  • {"criterion_text":"- Glucocorticoid-induced myopathy that the investigator considers the primary cause of muscle weakness or permanent weakness linked to a non-idiopathic inflammatory myopathy (IIM) cause"}
  • {"criterion_text":"- Juvenile myositis (JDM) diagnosed > 5 years from screening or juvenile myositis with extensive calcinosis or severe calcinosis."}
  • {"criterion_text":"- Uncontrolled interstitial lung disease or any other uncontrolled idiopathic inflammatory myopathy (IIM) manifestation that, in the opinion of the investigator, would be likely to require treatment with prohibited medication during the study"}
  • {"criterion_text":"- Other inflammatory and noninflammatory myopathies: inclusion body myositis, infectious myopathy, overlap myositis, metabolic myopathies, muscle dystrophies or a family history of muscle dystrophy, druginduced or endocrine-induced myositis, and juvenile myositis (other than juvenile dermatomyositis (JDM))"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Mean total improvement score (TIS) at week 52","definition_or_measurement_approach":"Mean Total Improvement Score (TIS) measured at Week 52 (TIS = Total Improvement Score)"}

Secondary endpoints

  • {"endpoint_text":"- 1. Time to reach sustained TIS ≥40 (“moderate clinical improvement”)","definition_or_measurement_approach":"Time from baseline to first sustained Total Improvement Score (TIS) ≥40"}
  • {"endpoint_text":"- 2. Percentage of participants with TIS ≥40 at week 52","definition_or_measurement_approach":"Proportion of participants achieving TIS ≥40 at Week 52"}
  • {"endpoint_text":"- 3. Percentage of participants with TIS ≥60 at week 52","definition_or_measurement_approach":"Proportion of participants achieving TIS ≥60 at Week 52"}
  • {"endpoint_text":"- 4. Change in manual muscle testing-8 (MMT8) score at week 52","definition_or_measurement_approach":"Change from baseline in MMT8 score at Week 52"}
  • {"endpoint_text":"- 5. Change in Patient Global Assessment of Disease Activity (PGA) at week 52","definition_or_measurement_approach":"Change from baseline in Patient Global Assessment at Week 52"}
  • {"endpoint_text":"- 6. Change in Physician Global Assessment of Disease Activity (MDGA) at week 52","definition_or_measurement_approach":"Change from baseline in Physician Global Assessment (MDGA) at Week 52"}
  • {"endpoint_text":"- 7. Proportion of participants who have at least moderate improvement (≥40) in TIS at week 52 and adhere to an oral prednisone dosage of ≤5 mg/day (or equivalent) from week 44 onward","definition_or_measurement_approach":"Proportion meeting TIS ≥40 at Week 52 and maintaining oral prednisone ≤5 mg/day (or equivalent) from Week 44"}
  • {"endpoint_text":"- 8. Change in CK abnormality grades at week 52","definition_or_measurement_approach":"Change from baseline in creatine kinase (CK) abnormality grades at Week 52"}
  • {"endpoint_text":"- 9. Change in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) activity score at week 52","definition_or_measurement_approach":"Change from baseline in CDASI activity score at Week 52"}

Recruitment

Digital Remote Recruitment
True, online website materials and non-PII website copies are provided (K2_Website, K2_Non PII Website) to support digital recruitment and information provision
Planned Sample Size
240
Recruitment Window Months
52
Consent Approach
Written informed consent is required from participants able to consent in the trial jurisdiction; participants are adults (≥18 years or legal age of consent). Dedicated subject information and informed consent forms are provided for Phase 2 and Phase 3, caregiver-specific ICFs, pregnant partner information, COVID-19 addenda, genetic testing addenda, optional-samples and optional home care consents. Materials are available in multiple languages and country-specific versions as listed in the submission documents.

Methods

  • Flyers (K2_Flyer, country-specific flyers) — patient-facing printed materials for awareness and recruitment
  • Patient letters (K2_Patient Letter) — direct mailed/clinic-distributed recruitment letters
  • Patient brochures / patient brochure materials (K2_Patient Brochure) — informational printed material for potential participants
  • ICF Flipbook (K2_ICF Flipbook) — informational flipbook to support informed consent discussions
  • Non-PII Website / Website recruitment copy (K2_Website, K2_Non PII Website) — online recruitment information and study website content
  • Patient brochures and recruitment materials adapted per country/language (multiple country-specific K2 materials listed)
  • Recruitment arrangements documents (K1_Recruit-ICF process and variants) — documented site-level recruitment and informed consent processes

Geography

Total Number Of Sites
63
Total Number Of Participants
186

Austria

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
20-08-2024
Processing Time Days
35
Number Of Sites
2
Number Of Participants
12

Sites

Site Name
Medizinische Universitaet Innsbruck
Department Name
Department of Neurology and Neurosurgery Focus on Neuromuscular Diseases
Principal Investigator Name
Julia Wanschitz
Principal Investigator Email
julia.wanschitz@i-med.ac.at
Contact Person Name
Julia Wanschitz
Contact Person Email
julia.wanschitz@i-med.ac.at
Site Name
Medical University Of Vienna
Department Name
Department of Internal Medicine III
Principal Investigator Name
Clemens Scheinecker
Principal Investigator Email
clemens.scheinecker@meduniwien.ac.at
Contact Person Name
Clemens Scheinecker

Cyprus

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
19-08-2024
Processing Time Days
34
Number Of Sites
1
Number Of Participants
16

Sites

Site Name
The Cyprus Foundation For Muscular Dystrophy Research
Department Name
Neuromuscular Disorders Centre
Principal Investigator Name
Kleopas Kleopa
Principal Investigator Email
kleopa@cing.ac.cy
Contact Person Name
Kleopas Kleopa
Contact Person Email
kleopa@cing.ac.cy

Czechia

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
15-08-2024
Processing Time Days
30
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
Revmatologicky Ustav
Principal Investigator Name
Jiří Vencovský
Principal Investigator Email
vencovsky@revma.cz
Contact Person Name
Jiří Vencovský
Contact Person Email
vencovsky@revma.cz

Denmark

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
14-08-2024
Processing Time Days
29
Number Of Sites
1
Number Of Participants
8

Sites

Site Name
Copenhagen University Hospital
Department Name
Center for Rheumatology and Spine Diseases
Principal Investigator Name
Louise Diederichsen
Principal Investigator Email
Louise.diederichsen@regionh.dk
Contact Person Name
Louise Diederichsen
Contact Person Email
Louise.diederichsen@regionh.dk

Lithuania

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
14-08-2024
Processing Time Days
29
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
Vilniaus Universiteto Ligonine Santaros Klinikos Vsi
Department Name
Rheumatology Center
Principal Investigator Name
Irena Butrimiene
Principal Investigator Email
Irena.Butrimiene@santa.lt
Contact Person Name
Irena Butrimiene
Contact Person Email
Irena.Butrimiene@santa.lt

Portugal

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
16-08-2024
Processing Time Days
31
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Unidade Local De Saude De Santa Maria E.P.E.
Department Name
Rheumatology
Principal Investigator Name
RAQUEL MARQUES
Principal Investigator Email
ensaiosreumatologiacaml@gmail.com
Contact Person Name
RAQUEL MARQUES
Site Name
Unidade Local De Saude De Santo Antonio E.P.E.
Department Name
Clinical Immunology
Principal Investigator Name
ANTONIO MARINHO
Principal Investigator Email
ensaiosclinicos.defi@chporto.min-saude.pt
Contact Person Name
ANTONIO MARINHO

Sweden

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
14-08-2024
Processing Time Days
29
Number Of Sites
1
Number Of Participants
5

Sites

Site Name
Karolinska University Hospital
Department Name
Klinisk Forskningsenhet GHR
Principal Investigator Name
Maryam Dastmalchi
Principal Investigator Email
Maryam.dastmalchi@regionstockholm.se
Contact Person Name
Maryam Dastmalchi

Netherlands

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
19-08-2024
Processing Time Days
34
Number Of Sites
2
Number Of Participants
3

Sites

Site Name
Amsterdam UMC Stichting
Department Name
Neurology
Principal Investigator Name
Anneke van der Kooi
Principal Investigator Email
neurocentrum@amsterdamumc.nl
Contact Person Name
Anneke van der Kooi
Contact Person Email
neurocentrum@amsterdamumc.nl
Site Name
Universitair Medisch Centrum Utrecht
Department Name
Internal Medicine
Principal Investigator Name
Helen Leavis
Principal Investigator Email
reumatologie-research@umcutrecht.nl
Contact Person Name
Helen Leavis

Slovakia

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
14-08-2024
Processing Time Days
29
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
Narodny Ustav Reumatickych Chorob
Department Name
Reumatologická ambulancia Nábrežie Ivana Krasku 4 921 12 Piešťany
Principal Investigator Name
Oľga Lukáčová
Principal Investigator Email
olga.lukacova@nurch.sk
Contact Person Name
Oľga Lukáčová
Contact Person Email
olga.lukacova@nurch.sk

Poland

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
16-08-2024
Processing Time Days
31
Number Of Sites
3
Number Of Participants
18

Sites

Site Name
Szpital Uniwersytecki Nr 2 Im Dr Jana Biziela W Bydgoszczy
Department Name
Klinika Reumatologii i Ukladowych Chorob Tkanki Lacznej
Principal Investigator Name
Tomasz Jankowski
Principal Investigator Email
tojankowski@gazeta.pl
Contact Person Name
Tomasz Jankowski
Contact Person Email
tojankowski@gazeta.pl
Site Name
Centrum Wsparcia Badań Klinicznych Pomorskiego Uniwersytetu Medycznego w Szczecinie
Department Name
Klinika Chorób Wewnętrznych, Reumatologii, Diabetologii, Geriatrii i Immunologii Klinicznej
Principal Investigator Name
Marek Brzosko
Principal Investigator Email
marek.brzosko@pum.edu.pl
Contact Person Name
Marek Brzosko
Contact Person Email
marek.brzosko@pum.edu.pl
Site Name
SPZOZ Uniwersytecki Szpital Kliniczny nr 2 Uniwersytetu Medycznego w Łodzi
Department Name
Klinika Reumatologii i Immunologii z Pododdzialem Chorob Wewnetrznych
Principal Investigator Name
Joanna Makowska
Principal Investigator Email
joanna.makowska@umed.lodz.pl
Contact Person Name
Joanna Makowska
Contact Person Email
joanna.makowska@umed.lodz.pl

Hungary

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
14-08-2024
Processing Time Days
29
Number Of Sites
2
Number Of Participants
14

Sites

Site Name
University Of Debrecen
Department Name
Belgyógyászati Klinika, Immunológia
Principal Investigator Name
Zoltán Griger
Principal Investigator Email
griger.zoltan@med.unideb.hu
Contact Person Name
Zoltán Griger
Contact Person Email
griger.zoltan@med.unideb.hu
Site Name
Semmelweis University
Department Name
Genomika Medicina és Ritka Betegségek Intézete
Principal Investigator Name
Judit Mária Molnár
Principal Investigator Email
molnar.mariajudit@med.semmelweis-univ.hu
Contact Person Name
Judit Mária Molnár

Ireland

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
14-08-2024
Processing Time Days
29
Number Of Sites
3
Number Of Participants
16

Sites

Site Name
Our Ladys Hospital Manorhamilton
Department Name
Rheumatology
Principal Investigator Name
Bryan Whelan
Principal Investigator Email
Bryan.whelan@hse.ie
Contact Person Name
Bryan Whelan
Contact Person Email
Bryan.whelan@hse.ie
Site Name
St Vincent's University Hospital
Department Name
Rheumatology
Principal Investigator Name
Eamonn Molloy
Principal Investigator Email
emolloy@svhg.ie
Contact Person Name
Eamonn Molloy
Contact Person Email
emolloy@svhg.ie
Site Name
Connolly Hospital
Department Name
Rheumatology
Principal Investigator Name
Trevor Duffy
Principal Investigator Email
tuduffy@crp.healthcare
Contact Person Name
Trevor Duffy
Contact Person Email
tuduffy@crp.healthcare

France

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
14-08-2024
Processing Time Days
29
Number Of Sites
4
Number Of Participants
14

Sites

Site Name
Assistance Publique Hopitaux De Paris
Department Name
Immunologie Clinique
Principal Investigator Name
Olivier Benveniste
Principal Investigator Email
olivier.benveniste@aphp.fr
Contact Person Name
Olivier Benveniste
Contact Person Email
olivier.benveniste@aphp.fr
Site Name
Hospital Edouard Herriot
Department Name
Service de Medicine Interne
Principal Investigator Name
Laure Gallay
Principal Investigator Email
laure.gallay01@chu-lyon.fr
Contact Person Name
Laure Gallay
Contact Person Email
laure.gallay01@chu-lyon.fr
Site Name
Les Hopitaux Universitaires De Strasbourg
Department Name
Centre de Références des Maladie Auto-Immunes Rares, Service de Rhumatologie
Principal Investigator Name
Alain Meyer
Principal Investigator Email
alain.meyer1@chru-strasbourg.fr
Contact Person Name
Alain Meyer
Site Name
Centre Hospitalier Universitaire Rouen
Department Name
Département de médecine interne, Hôpital Charles Nicolle
Principal Investigator Name
Maëlle Le Besnerais
Principal Investigator Email
maelle.le-besnerais@chu-rouen.fr
Contact Person Name
Maëlle Le Besnerais

Bulgaria

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
15-08-2024
Processing Time Days
30
Number Of Sites
2
Number Of Participants
12

Sites

Site Name
Medical Center Artmed Ltd.
Principal Investigator Name
Mariela Geneva-Popova
Principal Investigator Email
artmedcenter@yahoo.com
Contact Person Name
Mariela Geneva-Popova
Contact Person Email
artmedcenter@yahoo.com
Site Name
University Multiprofile Hospital For Active Treatment Kaspela EOOD
Department Name
Clinic of Rheumatology
Principal Investigator Name
Anastas Batalov
Principal Investigator Email
abatalov@hotmail.com
Contact Person Name
Anastas Batalov
Contact Person Email
abatalov@hotmail.com

Belgium

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
19-08-2024
Processing Time Days
34
Number Of Sites
5
Number Of Participants
5

Sites

Site Name
UZ Leuven
Department Name
Department of Neuroscience
Principal Investigator Name
Kristl Claeys
Principal Investigator Email
Kristl.Claeys@uzleuven.be
Contact Person Name
Kristl Claeys
Contact Person Email
Kristl.Claeys@uzleuven.be
Site Name
Centre hospitalier universitaire de Liege
Department Name
Rheumatology Department
Principal Investigator Name
Christian Von Frenckell
Principal Investigator Email
cvonfrenckell@chuliege.be
Contact Person Name
Christian Von Frenckell
Contact Person Email
cvonfrenckell@chuliege.be
Site Name
Universitair Ziekenhuis Gent
Department Name
Rheumatology Department
Principal Investigator Name
Vanessa Smitt
Principal Investigator Email
vanessa.smith@ugent.be
Contact Person Name
Vanessa Smitt
Contact Person Email
vanessa.smith@ugent.be
Site Name
UZ Leuven
Department Name
Department of general internal medicine
Principal Investigator Name
Ellen De Langhe
Principal Investigator Email
ellen.delanghe@uzleuven.be
Contact Person Name
Ellen De Langhe
Contact Person Email
ellen.delanghe@uzleuven.be
Site Name
AZ Sint-Lucas & Volkskliniek
Department Name
Neurology department
Principal Investigator Name
Jan De Bleecker
Principal Investigator Email
jan.debleecker@azstlucas.be
Contact Person Name
Jan De Bleecker
Contact Person Email
jan.debleecker@azstlucas.be

Germany

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
15-08-2024
Processing Time Days
30
Number Of Sites
7
Number Of Participants
10

Sites

Site Name
St. Elisabeth Gruppe GmbH Katholische Kliniken Rhein-Ruhr
Department Name
Rheumatism center Ruhrgebiet
Principal Investigator Name
Ioana Andreica
Principal Investigator Email
ioana.andreica@elisabethgruppe.de
Contact Person Name
Ioana Andreica
Site Name
Universitaetsklinikum Ulm AöR
Department Name
Department of Neurogeriatry and Neurological Rehabilitation
Principal Investigator Name
Angela Rosenbohm
Principal Investigator Email
angela.rosenbohm@uni-ulm.de
Contact Person Name
Angela Rosenbohm
Contact Person Email
angela.rosenbohm@uni-ulm.de
Site Name
Universitaetsklinikum Tuebingen AöR
Department Name
Clinic for Neurology
Principal Investigator Name
Alexander Grimm
Principal Investigator Email
alexander.grimm@med.uni-tuebingen.de
Contact Person Name
Alexander Grimm
Site Name
Universitaetsklinikum Mannheim GmbH
Department Name
Neuromuskuläres Zentrum
Principal Investigator Name
Hans-Werner Rausch
Principal Investigator Email
Hans-Werner.Rausch@umm.de
Contact Person Name
Hans-Werner Rausch
Contact Person Email
Hans-Werner.Rausch@umm.de
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Medizinische Klinik mit Schwerpunkt Rheumatologie und Klinische Immunologie
Principal Investigator Name
Fredrik Albach
Principal Investigator Email
fredrik.albach@charite.de
Contact Person Name
Fredrik Albach
Contact Person Email
fredrik.albach@charite.de
Site Name
University Medical Center Hamburg-Eppendorf
Department Name
Medizinische Klinik und Poliklinik III Nephrologie/Rheumatologie/ Endokrinologie
Principal Investigator Name
Martin Krusche
Principal Investigator Email
m.krusche@uke.de
Contact Person Name
Martin Krusche
Contact Person Email
m.krusche@uke.de
Site Name
Universitaetsmedizin Goettingen
Department Name
Klinik für Neurologie
Principal Investigator Name
Jana Zschüntzsch
Principal Investigator Email
j.zschuentzsch@med.uni-goettingen.de
Contact Person Name
Jana Zschüntzsch

Greece

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
22-08-2024
Processing Time Days
37
Number Of Sites
5
Number Of Participants
12

Sites

Site Name
Athens Medical Center S.A.
Department Name
Neurology Department
Principal Investigator Name
Michalis Kosmidis
Principal Investigator Email
kosmidism@yahoo.gr
Contact Person Name
Michalis Kosmidis
Contact Person Email
kosmidism@yahoo.gr
Site Name
Laiko General Hospital Of Athens
Department Name
Department of Pathophysiology
Principal Investigator Name
Menelaos Manousakis
Principal Investigator Email
menman@med.uoa.gr
Contact Person Name
Menelaos Manousakis
Contact Person Email
menman@med.uoa.gr
Site Name
Eginitio Hospital
Department Name
A’ Neurology Department
Principal Investigator Name
George Papadimas
Principal Investigator Email
gkpapad@yahoo.gr
Contact Person Name
George Papadimas
Contact Person Email
gkpapad@yahoo.gr
Site Name
University General Hospital Attikon
Department Name
B’ Neurology Department
Principal Investigator Name
George Tsivgoulis
Principal Investigator Email
tsivgoulisgiorg@yahoo.gr
Contact Person Name
George Tsivgoulis
Contact Person Email
tsivgoulisgiorg@yahoo.gr
Site Name
University General Hospital Of Ioannina
Department Name
Clinic of Neurology
Principal Investigator Name
Spyridon Konitsiotis
Principal Investigator Email
skonitso@gmail.com
Contact Person Name
Spyridon Konitsiotis
Contact Person Email
skonitso@gmail.com

Italy

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
20-08-2024
Processing Time Days
35
Number Of Sites
11
Number Of Participants
17

Sites

Site Name
Azienda Sanitaria Universitaria Friuli Centrale
Department Name
SOC Clinica di Reumatologia
Principal Investigator Name
Luca Quartuccio
Principal Investigator Email
Luca.quartuccio@asufc.sanita.fvg.it
Contact Person Name
Luca Quartuccio
Site Name
Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
Department Name
SC Reumatologia
Principal Investigator Name
Simone Parisi
Principal Investigator Email
sparisi@cittadellasalute.to.it
Contact Person Name
Simone Parisi
Contact Person Email
sparisi@cittadellasalute.to.it
Site Name
Fondazione IRCCS Policlinico San Matteo
Department Name
UOC Reumatologia
Principal Investigator Name
Lorenzo Cavagna
Principal Investigator Email
lorenzo.cavagna@unipv.it
Contact Person Name
Lorenzo Cavagna
Contact Person Email
lorenzo.cavagna@unipv.it
Site Name
Universita Cattolica Del Sacro Cuore
Department Name
UOC Neurologia
Principal Investigator Name
Massimiliano Mirabella
Contact Person Name
Massimiliano Mirabella
Site Name
Azienda Ospedaliera Universitaria Pisana
Department Name
UO Reumatologia
Principal Investigator Name
Marta Mosca
Principal Investigator Email
marta.mosca@med.unipi.it
Contact Person Name
Marta Mosca
Contact Person Email
marta.mosca@med.unipi.it
Site Name
Azienda Ospedaliera Universitaria Integrata Verona
Department Name
Clinica Neurologica
Principal Investigator Name
Gaetano Nicola Alfio Vattemi
Principal Investigator Email
Gaetano.vattemi@univr.it
Contact Person Name
Gaetano Nicola Alfio Vattemi
Contact Person Email
Gaetano.vattemi@univr.it
Site Name
Careggi University Hospital
Department Name
SODc Reumatologia
Principal Investigator Name
Serena Guiducci
Principal Investigator Email
serena.guiducci@unifi.it
Contact Person Name
Serena Guiducci
Contact Person Email
serena.guiducci@unifi.it
Site Name
Azienda Universitaria Ospedaliera Consorziale Policlinico Bari
Department Name
UOC Reumatologia Universitaria
Principal Investigator Name
Marco Fornaro
Principal Investigator Email
marco.fornaro@uniba.it
Contact Person Name
Marco Fornaro
Contact Person Email
marco.fornaro@uniba.it
Site Name
Ospedale San Raffaele S.r.l.
Department Name
Unità di Neurologia
Principal Investigator Name
Stefano Previtali
Principal Investigator Email
previtali.stefano@hsr.it
Contact Person Name
Stefano Previtali
Contact Person Email
previtali.stefano@hsr.it
Site Name
Azienda USL IRCCS Di Reggio Emilia
Department Name
Dipartimento di Medicina Interna e Specialità Mediche
Principal Investigator Name
Nicolo Pipitone
Principal Investigator Email
pipitone.nicolo@ausl.re.it
Contact Person Name
Nicolo Pipitone
Contact Person Email
pipitone.nicolo@ausl.re.it
Site Name
Humanitas Mirasole S.p.A.
Department Name
UO Reumatologia e Immunologia Clinica
Principal Investigator Name
Carlo Selmi
Principal Investigator Email
carlo.selmi@hunimed.eu
Contact Person Name
Carlo Selmi
Contact Person Email
carlo.selmi@hunimed.eu

Spain

Earliest CTIS Part Ii Submission Date
16-07-2024
Latest Decision Or Authorization Date
19-08-2024
Processing Time Days
34
Number Of Sites
9
Number Of Participants
11

Sites

Site Name
Hospital General Universitario Dr. Balmis
Department Name
Neurology
Principal Investigator Name
Carmen Diaz Marin
Principal Investigator Email
carmina.diaz.marin@gmail.com
Contact Person Name
Carmen Diaz Marin
Contact Person Email
carmina.diaz.marin@gmail.com
Site Name
Hospital Universitario Ramon Y Cajal
Department Name
Rheumatology
Principal Investigator Name
Jesus Loarce Martos
Principal Investigator Email
jesus.loarce@gmail.com
Contact Person Name
Jesus Loarce Martos
Contact Person Email
jesus.loarce@gmail.com
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Neurology
Principal Investigator Name
Nuria Muelas Gomez
Principal Investigator Email
muelas_nur@gva.es
Contact Person Name
Nuria Muelas Gomez
Contact Person Email
muelas_nur@gva.es
Site Name
Complexo Hospitalario Universitario De Vigo
Department Name
Rheumatology – Hospital Do Meixoeiro
Principal Investigator Name
Jose Maria Pego Reigosa
Principal Investigator Email
jose.maria.pego.reigosa@sergas.es
Contact Person Name
Jose Maria Pego Reigosa
Site Name
Hospital Universitario 12 De Octubre
Department Name
Rheumatology
Principal Investigator Name
Patricia Esmeralda Carreira Delgado
Principal Investigator Email
carreira@h12o.es
Contact Person Name
Patricia Esmeralda Carreira Delgado
Contact Person Email
carreira@h12o.es
Site Name
Hospital Clinic De Barcelona
Department Name
Neurology
Principal Investigator Name
Jose Cesar Milisenda
Principal Investigator Email
jcmilise@clinic.cat
Contact Person Name
Jose Cesar Milisenda
Contact Person Email
jcmilise@clinic.cat
Site Name
Hospital Universitario Virgen De Valme
Department Name
Rheumatology
Principal Investigator Name
Sergio Rodriguez Montero
Principal Investigator Email
sergio.mont@gmail.com
Contact Person Name
Sergio Rodriguez Montero
Contact Person Email
sergio.mont@gmail.com
Site Name
Hospital Universitario La Paz
Department Name
Rheumatology
Principal Investigator Name
Maria Gema Bonilla Herman
Principal Investigator Email
gemabonilla@ser.es
Contact Person Name
Maria Gema Bonilla Herman
Contact Person Email
gemabonilla@ser.es
Site Name
Hospital De La Santa Creu I Sant Pau
Department Name
Rheumatology
Principal Investigator Name
Ivan Castellvi Barranco
Principal Investigator Email
icastellvi@santpau.cat
Contact Person Name
Ivan Castellvi Barranco
Contact Person Email
icastellvi@santpau.cat

Sponsor

Primary sponsor

Full Name
Argenx
Organisation Type
Pharmaceutical company
Country Of Registered Address
Belgium

Contract research organisations

Name
Icon Clinical Research Limited
Responsibilities
Site management, regulatory support (sponsor duties codes listed)
Name
ICON Clinical Research Limited Ireland Filial
Responsibilities
On site monitoring in Denmark
Name
IQVIA Limited
Responsibilities
Pharmacovigilance and clinical trial support
Name
Suvoda LLC
Responsibilities
eClinical / trial technology support

Third parties

  • {"country":"United States","full_name":"Actigraph LLC","duties_or_roles":"code 1","organisation_type":"Pharmaceutical company"}
  • {"country":"Netherlands","full_name":"Universitair Medisch Centrum Utrecht","duties_or_roles":"code 4","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"code 3","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"Total Improvement Score, Muscle Enzyme analysis; code 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Resolian Bioanalytics","duties_or_roles":"code 4","organisation_type":"Industry"}
  • {"country":"Germany","full_name":"Azenta Germany GmbH","duties_or_roles":"Biobanking/ Storage","organisation_type":"Pharmaceutical company"}
  • {"country":"Sweden","full_name":"ICON Clinical Research Limited Ireland Filial","duties_or_roles":"On site monitoring in Denmark","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Medical Equipment Supplies And Management Limited","duties_or_roles":"Provision of required equipment to sites","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Cytel Inc.","duties_or_roles":"code 6; code 7","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Pharmacovigilance; code 8","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"code 1; code 12; code 5","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Central Cardiology, Central eCOA/ePRO provision","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"The Doctors Laboratory Limited","duties_or_roles":"code 4","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Mayo Collaborative Services LLC","duties_or_roles":"code 4","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Gray Consulting Inc.","duties_or_roles":"Travel and accommodation, Concierge support to patients and caregivers","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Icon Development Solutions LLC","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"The Doctors Laboratory Limited (additional listing)","duties_or_roles":"code 4","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United Kingdom","full_name":"Imperial Clinical Research Services International Limited","duties_or_roles":"Printing, production and shipping of patient materials","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United States","full_name":"Icon Laboratory Services Inc.","duties_or_roles":"Biopsy Slides Scanning","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"National Jewish Health","duties_or_roles":"code 4","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"Ireland","full_name":"Accellacare Limited","duties_or_roles":"In-home health care services, nursing","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"CellCarta","duties_or_roles":"IFN score testing","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"Greece","full_name":"Pharmaceutical Research Associates Greece A.E","duties_or_roles":"Regulatory submissions in Greece and Cyprus","organisation_type":"Industry"}
  • {"country":"United States","full_name":"Icon Laboratory Services Inc. (additional listing)","duties_or_roles":"Sample management (Whitesboro)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Vanderbilt University Medical Center","duties_or_roles":"code 4","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"Ireland","full_name":"Accellacare Limited (additional listing)","duties_or_roles":"In-home health care services, nursing","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"CellCarta (additional listing)","duties_or_roles":"IFN score testing","organisation_type":"Laboratory/Research/Testing facility"}

Investigational products

Investigational Product Name
ARGX-113
Active Substance
EFGARTIGIMOD ALFA
Modality
Peptide/protein/enzyme
Routes Of Administration
SUBCUTANEOUS
Route
SUBCUTANEOUS
Authorisation Status
Investigational (MIA DE_BW_01_MIA_2023_0054)
Maximum Dose
max daily 143 mg; max total 52000 mg
Investigational Product Name
Placebo to ARGX-113 solution for injection
Modality
Other
Combination Treatment
Yes

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